title: A Prospective, Single Arm Clinical Investigation Evaluating Safety and Performance of the Colibri Transcatheter Aortic Heart Valve System for the Treatment of Symptomatic Severe Aortic Stenosis Via Transfemoral Access in High Surgical Risk Patients

summary:
The Colibri Heart Valve clinical investigation ("COL-01") is a prospective, multicenter, exploratory single arm and controlled clinical investigation compared to recent historical results. This study will be conducted in about 7 European sites. The study will evaluate the safety and performance of the Colibri Transcatheter Aortic Heart Valve System for the treatment of symptomatic severe aortic stenosis via transfemoral access in high surgical risk patients.

30 subjects suffering from symptomatic severe aortic tricuspid valve stenosis and who are at high surgical risk (logistic EuroSCORE I > 20% or other risk factors not included in this score such as frailty, porcelain aorta, sequelae of chest radiation or logistic EuroSCORE I < 20% but considered at high risk by local Heart Team evaluation) will be enrolled in this clinical investigation.

The primary objective of this clinical investigation is to evaluate the all-cause mortality at 30 days post implantation. The secondary objectives are to evaluate the safety and performance of the investigational device at 30 days, 6 and 12 months, and 2, 3, 4, 5 years post implantation.

lead_sponsor: Colibri Heart Valve LLC

location_country:
United Kingdom
France

location_facility: Brighton & Sussex University Hospital, Roayl Sussex County Hospital

location_facility: Barts Heart Centre / St Bartholomew's Hospital


title: A Phase 1 Study to Evaluate the Safety and Pharmacokinetics of ADU-1604, an Anti-CTLA-4 Antibody, in Adults With Metastatic Melanoma

summary: This study is a first-in-human, open-label, multicenter, dose-escalation study designed to evaluate the safety, PK, and PD of ADU-1604 and explore initial clinical activity in adults with metastatic melanoma.

study_start_date_type: Actual

status: Terminated

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Actual

ipd_sharing: No

lead_sponsor: Aduro Biotech, Inc.

location_country: Spain France

location_facility:
Hospital Saint Louis
Hospital Universitario Virgen Macarena
Gustave- Roussy Institute
Hôpital de la Timone
Hospital Universitario Ramon y Cajal

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Unknown


title: In Situ Immune Parameters and Their Prognostic Role on the Survival of Patients With Glioblastoma

acronym: IMMUNOGLIO

summary:
Glioblastoma (GBM) is the most frequent brain tumor. Currently survival is poor and few treatments are available. Recent data show that there is no immune privilege of the central nervous system (CNS) and that GBM are invaded by effector CD8 T cells, letting us hypothesis that GBM growth is dependent of immunosurveillance.

The aim of this study is to better understand the antitumor immune response against GBM to unravel new effectors and immunosuppressive pathways important for the regulation of anticancer immunity and to discover new immune activating strategies with the objectives to isolate subgroups of GBMs that could benefit from an immunotherapy approach. To achieve this goal, GBM tumor samples and a blood sample will be collected during the initial tumor resection.

The sites involved in the recruitment of the patients will be the neurosurgical teams in Brussel, Dijon, Nantes and Padova.

status: Active, not recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

lead_sponsor: Centre Georges Francois Leclerc

location_country: Italy France

location_facility: Azienda Ospedaliera Università di Padova

status_simplified: Ongoing


type: EudraCT Number

title: An Open Label, Phase I, Dose Escalation Study to Characterize the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Doses of BI 836909 in Relapsed and/or Refractory Multiple Myeloma Patients

summary: The primary objective of this trial is to determine the maximum tolerated dose (MTD) of BI 836909 administered by continuous i.v. infusion in patients with relapsed and/or refractory multiple myeloma. If the MTD is not reached based on safety findings, a recommended dose for further development will be determined. This will depend on the safety data, pharmacokinetic/pharmacodynamics data and potentially preliminary efficacy data. Secondary objectives are to document the safety and tolerability of BI 836909, to perform pharmacokinetic and pharmacodynamic analyses and to evaluate relevant biological effects in terms of parameters of efficacy.

status: Completed

design_allocation: Non-Randomized

ReferenceType: derived

ReferenceCitation: Topp MS, Duell J, Zugmaier G, Attal M, Moreau P, Langer C, Kronke J, Facon T, Salnikov AV, Lesley R, Beutner K, Kalabus J, Rasmussen E, Riemann K, Minella AC, Munzert G, Einsele H. Anti-B-Cell Maturation Antigen BiTE Molecule AMG 420 Induces Responses in Multiple Myeloma. J Clin Oncol. 2020 Mar 10;38(8):775-783. doi: 10.1200/JCO.19.02657. Epub 2020 Jan 2.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: American Society of Clinical Oncology (ASCO)

ipd_sharing_description:
Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization).

For more details refer to: https://www.mystudywindow.com/msw/datatransparency

lead_sponsor: Boehringer Ingelheim

location_country: Germany France

location_facility:
HOP Claude Huriez
Universitätsklinikum Würzburg
HOP Hôtel-Dieu
Universitätsklinikum Ulm
INS Universitaire du Cancer

status_simplified: Completed


title: Impact of a Neuro-cardiac Rehabilitation Program on the Quality of Life of Children, Adolescents, and Young Adults With Congenital Heart Disease: the Multicentre Randomized Controlled QUALINEUROREHAB Trial

summary: Remarkable progress in paediatric cardiology and surgery have led to the substantial increase of congenital heart disease (CHD) survivors. Long-term outcomes in rare and complex CHD have become a critical priority as three major sources of morbidity have been identified in this population: neurodevelopmental sequelae, mental health issues and reduced exercise capacity. These challenges adversely affect their quality of life and constitute a major public health issue. We seek to evaluate the efficacy of the first integrative and holistic program in Neuro-Cardiac Health associating physical and psychological rehabilitation for children with rare CHD compared to the standard of care. Children randomly assigned to the intervention will undergo a 12-week neuro-cardiac intervention including home-based adaptive physical exercise, telehealth parent and child psycho-education and child computerized cognitive training, as well in-person individual sessions of intervention reinforcement. Parents will be actively involved and will receive personalized feedback and educational resources. Children assigned to the control group will receive the standard of care in congenital cardiology. Post-intervention effects will be measured after 12-months on several outcomes including health-related quality of life (HRQoL), trained and untrained cognitive skills, mental health outcomes and cardiovascular/physical variables.

study_start_date_type: Anticipated

status: Not yet recruiting

design_allocation: Randomized

primary_purpose: Prevention

enrollment_type: Anticipated

ReferenceType: background

ReferenceCitation: Amedro P, Dorka R, Moniotte S, Guillaumont S, Fraisse A, Kreitmann B, Borm B, Bertet H, Barrea C, Ovaert C, Sluysmans T, De La Villeon G, Vincenti M, Voisin M, Auquier P, Picot MC. Quality of Life of Children with Congenital Heart Diseases: A Multicenter Controlled Cross-Sectional Study. Pediatr Cardiol. 2015 Dec;36(8):1588-601. doi: 10.1007/s00246-015-1201-x. Epub 2015 May 31.

oa_host_type: repository

evidence: oa repository (via OAI-PMH doi match)

host_type: repository

license_normalized: no license

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Paris Descartes

repository_normalized: HAL

version: publishedVersion

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Archive ouverte HAL

repositories_concat: HAL

unpaywall_oa_status: green

publisher_dissemination: Springer-Nature

ReferenceCitation: Muller J, Christov F, Schreiber C, Hess J, Hager A. Exercise capacity, quality of life, and daily activity in the long-term follow-up of patients with univentricular heart and total cavopulmonary connection. Eur Heart J. 2009 Dec;30(23):2915-20. doi: 10.1093/eurheartj/ehp305. Epub 2009 Aug 18.

ReferenceCitation: Amedro P, Picot MC, Moniotte S, Dorka R, Bertet H, Guillaumont S, Barrea C, Vincenti M, De La Villeon G, Bredy C, Soulatges C, Voisin M, Matecki S, Auquier P. Correlation between cardio-pulmonary exercise test variables and health-related quality of life among children with congenital heart diseases. Int J Cardiol. 2016 Jan 15;203:1052-60. doi: 10.1016/j.ijcard.2015.11.028. Epub 2015 Nov 10.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - CEA - Commissariat à l’énergie atomique

version: submittedVersion

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Artxiker

publisher_dissemination: Elsevier

ReferenceCitation: Amedro P, Gavotto A, Guillaumont S, Bertet H, Vincenti M, De La Villeon G, Bredy C, Acar P, Ovaert C, Picot MC, Matecki S. Cardiopulmonary fitness in children with congenital heart diseases versus healthy children. Heart. 2018 Jun;104(12):1026-1036. doi: 10.1136/heartjnl-2017-312339. Epub 2017 Nov 23.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - ParisTech

publisher_dissemination: British Medical Journal

ReferenceCitation: Muller J, Bohm B, Semsch S, Oberhoffer R, Hess J, Hager A. Currently, children with congenital heart disease are not limited in their submaximal exercise performance. Eur J Cardiothorac Surg. 2013 Jun;43(6):1096-100. doi: 10.1093/ejcts/ezs712. Epub 2013 Jan 22.

ReferenceCitation: Calderon J, Bellinger DC, Hartigan C, Lord A, Stopp C, Wypij D, Newburger JW. Improving neurodevelopmental outcomes in children with congenital heart disease: protocol for a randomised controlled trial of working memory training. BMJ Open. 2019 Feb 19;9(2):e023304. doi: 10.1136/bmjopen-2018-023304.

ReferenceCitation: Calderon J, Angeard N, Moutier S, Plumet MH, Jambaque I, Bonnet D. Impact of prenatal diagnosis on neurocognitive outcomes in children with transposition of the great arteries. J Pediatr. 2012 Jul;161(1):94-8.e1. doi: 10.1016/j.jpeds.2011.12.036. Epub 2012 Jan 28.

ReferenceCitation: Calderon J, Stopp C, Wypij D, DeMaso DR, Rivkin M, Newburger JW, Bellinger DC. Early-Term Birth in Single-Ventricle Congenital Heart Disease After the Fontan Procedure: Neurodevelopmental and Psychiatric Outcomes. J Pediatr. 2016 Dec;179:96-103. doi: 10.1016/j.jpeds.2016.08.084. Epub 2016 Sep 28.

ReferenceCitation: Pelliccia A, Sharma S, Gati S, Back M, Borjesson M, Caselli S, Collet JP, Corrado D, Drezner JA, Halle M, Hansen D, Heidbuchel H, Myers J, Niebauer J, Papadakis M, Piepoli MF, Prescott E, Roos-Hesselink JW, Graham Stuart A, Taylor RS, Thompson PD, Tiberi M, Vanhees L, Wilhelm M; ESC Scientific Document Group. 2020 ESC Guidelines on sports cardiology and exercise in patients with cardiovascular disease. Eur Heart J. 2021 Jan 1;42(1):17-96. doi: 10.1093/eurheartj/ehaa605. No abstract available. Erratum In: Eur Heart J. 2021 Feb 1;42(5):548-549.

ReferenceCitation: Amedro P, Gavotto A, Legendre A, Lavastre K, Bredy C, De La Villeon G, Matecki S, Vandenberghe D, Ladeveze M, Bajolle F, Bosser G, Bouvaist H, Brosset P, Cohen L, Cohen S, Corone S, Dauphin C, Dulac Y, Hascoet S, Iriart X, Ladouceur M, Mace L, Neagu OA, Ovaert C, Picot MC, Poirette L, Sidney F, Soullier C, Thambo JB, Combes N, Bonnet D, Guillaumont S. Impact of a centre and home-based cardiac rehabilitation program on the quality of life of teenagers and young adults with congenital heart disease: The QUALI-REHAB study rationale, design and methods. Int J Cardiol. 2019 May 15;283:112-118. doi: 10.1016/j.ijcard.2018.12.050. Epub 2018 Dec 20.

ReferenceCitation: Calderon J, Wypij D, Rofeberg V, Stopp C, Roseman A, Albers D, Newburger JW, Bellinger DC. Randomized Controlled Trial of Working Memory Intervention in Congenital Heart Disease. J Pediatr. 2020 Dec;227:191-198.e3. doi: 10.1016/j.jpeds.2020.08.038. Epub 2020 Aug 19.

ReferenceCitation: Meyer M, Brudy L, Fuertes-Moure A, Hager A, Oberhoffer-Fritz R, Ewert P, Muller J. E-Health Exercise Intervention for Pediatric Patients with Congenital Heart Disease: A Randomized Controlled Trial. J Pediatr. 2021 Jun;233:163-168. doi: 10.1016/j.jpeds.2021.01.058. Epub 2021 Jan 29.

ReferenceCitation: Abassi H, Gavotto A, Picot MC, Bertet H, Matecki S, Guillaumont S, Moniotte S, Auquier P, Moreau J, Amedro P. Impaired pulmonary function and its association with clinical outcomes, exercise capacity and quality of life in children with congenital heart disease. Int J Cardiol. 2019 Jun 15;285:86-92. doi: 10.1016/j.ijcard.2019.02.069. Epub 2019 Mar 1.

oa_colors: green other

oa_host_type: publisher;repository

evidence: open (via crossref license, author manuscript)

host_type: publisher

license: publisher-specific-oa

license_normalized: publisher-specific

version: acceptedVersion

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Diderot

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Aix-Marseille Université

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université de Nantes

unpaywall_oa_status: hybrid

ReferenceCitation: Morales Mestre N, Reychler G, Goubau C, Moniotte S. Correlation Between Cardiopulmonary Exercise Test, Spirometry, and Congenital Heart Disease Severity in Pediatric Population. Pediatr Cardiol. 2019 Apr;40(4):871-877. doi: 10.1007/s00246-019-02084-5. Epub 2019 Mar 8.

ReferenceCitation: Hager A, Hess J. Comparison of health related quality of life with cardiopulmonary exercise testing in adolescents and adults with congenital heart disease. Heart. 2005 Apr;91(4):517-20. doi: 10.1136/hrt.2003.032722.

ReferenceCitation: Muller J, Berner A, Ewert P, Hager A. Reduced health-related quality of life in older patients with congenital heart disease: a cross sectional study in 2360 patients. Int J Cardiol. 2014 Aug 1;175(2):358-62. doi: 10.1016/j.ijcard.2014.06.008. Epub 2014 Jun 18.

ReferenceCitation: Amedro P, Bajolle F, Bertet H, Cheurfi R, Lasne D, Nogue E, Auquier P, Picot MC, Bonnet D. Quality of life in children participating in a non-selective INR self-monitoring VKA-education programme. Arch Cardiovasc Dis. 2018 Mar;111(3):180-188. doi: 10.1016/j.acvd.2017.05.013. Epub 2017 Nov 1.

ReferenceCitation: Werner O, Abassi H, Lavastre K, Guillaumont S, Picot MC, Serrand C, Dulac Y, Souletie N, Acar P, Bredy C, Amedro P. Factors influencing the participation of adolescents and young adults with a congenital heart disease in a transition education program: A prospective multicentre controlled study. Patient Educ Couns. 2019 Dec;102(12):2223-2230. doi: 10.1016/j.pec.2019.06.023. Epub 2019 Jun 26.

ReferenceCitation: Abassi H, Bajolle F, Werner O, Auer A, Marquina A, Mura T, Lavastre K, Guillaumont S, Manna F, Auquier P, Bonnet D, Amedro P. Health-related quality of life correlates with time in therapeutic range in children on anticoagulants with International Normalised Ratio self-monitoring. Arch Cardiovasc Dis. 2020 Dec;113(12):811-820. doi: 10.1016/j.acvd.2020.05.022. Epub 2020 Oct 14.

ReferenceCitation: Abassi H, Huguet H, Picot MC, Vincenti M, Guillaumont S, Auer A, Werner O, De La Villeon G, Lavastre K, Gavotto A, Auquier P, Amedro P. Health-related quality of life in children with congenital heart disease aged 5 to 7 years: a multicentre controlled cross-sectional study. Health Qual Life Outcomes. 2020 Nov 12;18(1):366. doi: 10.1186/s12955-020-01615-6.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - memSIC

evidence: oa repository (via OAI-PMH title and first author match)

ReferenceCitation: Amedro P, Gavotto A, Bredy C, Guillaumont S. [Cardiac rehabilitation for children and adults with congenital heart disease]. Presse Med. 2017 May;46(5):530-537. doi: 10.1016/j.lpm.2016.12.001. Epub 2017 Jan 23. French.

ReferenceCitation: Calderon J, Newburger JW. Working Memory Training: A Promising Intervention? Crit Care Med. 2018 Jul;46(7):1199-1201. doi: 10.1097/CCM.0000000000003172. No abstract available.

ReferenceCitation: Moreau J, Lavastre K, Romieu H, Charbonnier F, Guillaumont S, Bredy C, Abassi H, Werner O, De La Villeon G, Requirand A, Auer A, Matecki S, Karsenty C, Guitarte A, Hadeed K, Dulac Y, Souletie N, Acar P, Bajolle F, Bonnet D, Negre-Pages L, Mura T, Mounier M, Seguela PE, Thomas J, Iriart X, Jean-Benoit-Thambo, Amedro P. Impact of Sophrology on cardiopulmonary fitness in teenagers and young adults with a congenital heart disease: The SOPHROCARE study rationale, design and methods. Int J Cardiol Heart Vasc. 2020 Mar 3;27:100489. doi: 10.1016/j.ijcha.2020.100489. eCollection 2020 Apr.

ReferenceCitation: Amedro P, Werner O, Abassi H, Boisson A, Souilla L, Guillaumont S, Calderon J, Requirand A, Vincenti M, Pommier V, Matecki S, De La Villeon G, Lavastre K, Lacampagne A, Picot MC, Beyler C, Delclaux C, Dulac Y, Guitarte A, Charron P, Denjoy-Urbain I, Probst V, Baruteau AE, Chevalier P, Di Filippo S, Thambo JB, Bonnet D, Pasquie JL. Health-related quality of life and physical activity in children with inherited cardiac arrhythmia or inherited cardiomyopathy: the prospective multicentre controlled QUALIMYORYTHM study rationale, design and methods. Health Qual Life Outcomes. 2021 Jul 28;19(1):187. doi: 10.1186/s12955-021-01825-6.

ReferenceCitation: Gavotto A, Huguet H, Picot MC, Guillaumont S, Matecki S, Amedro P. The V̇e/V̇co2 slope: a useful tool to evaluate the physiological status of children with congenital heart disease. J Appl Physiol (1985). 2020 Nov 1;129(5):1102-1110. doi: 10.1152/japplphysiol.00520.2020. Epub 2020 Sep 10.

ReferenceCitation: Werner O, Bredy C, Lavastre K, Guillaumont S, De La Villeon G, Vincenti M, Gerl C, Dulac Y, Souletie N, Acar P, Pages L, Picot MC, Bourrel G, Oude Engberink A, Million E, Abassi H, Amedro P. Impact of a transition education program on health-related quality of life in pediatric patients with congenital heart disease: study design for a randomised controlled trial. Health Qual Life Outcomes. 2021 Jan 19;19(1):23. doi: 10.1186/s12955-021-01668-1.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inria

location_facility: University Hospital of Bordeaux - Haut-Levêque Hospital

location_facility: University Hospital of Montpellier - Arnaud de Villeneuve Hospital


title: A Global Multicenter Phase 1/2 Trial of EO4010, a Novel Microbial Derived Peptide Therapeutic Vaccine, in Combination With Nivolumab, for Treatment of Patients With Previously Treated Metastatic Colorectal Carcinoma

acronym: AUDREY

summary: Open-label multicenter study

status: Recruiting

lead_sponsor: Enterome

location_country:
United States
Spain
France

location_facility:
ICM Val d'Aurelle
Hôpital Jean Minjoz
Hospital Universitari Vall d'Hebron
Saint Antoine hospital
MD Anderson


title: LISA Post Market Clinical Follow-Up Study:Post Marketing Prospective Documentation of Clinical Outcomes (Post-operative, Safety and Performance) After Lumbar Dynamic Stabilization Surgery With LISA Implant

acronym: LISA-PMCF

summary:
The study is a post-market surveillance study of the Lumbar Implant for Stiffness Augmentation (LISA), a medical device, which is used to treat low-back pain that accompanies degenerative lesions of grades II, III, and IV (Pfirrmann MRI classification). "Post-market" means the device (i.e. the LISA implant) being used in this study has already obtained CE certification and is commercially available for use in the European market.

The LISA device consists of 3 components: A PEEK interspinous spacer, a polyester band, and a titanium blocker. The spacer is positioned between two adjacent spinous processes, the band is belted around the spinous processes and through the spacer, and the blocker is used to lock the band inside the spacer.

Medical Device manufacturers conduct "post-market" clinical studies in order to continuously evaluate the product scientifically and to comply with legal and ethical obligations. With these studies, the long-term safety as well as performance of their medical devices are assessed.

This study, which is initiated and sponsored by BACKBONE (LISA designer, developer, manufacturer and marketer), aims to evaluate the long-term safety and performance of the LISA implant for the treatment of lumbar degenerative disease and to evaluate the treatment.

lead_sponsor: BACKBONE

location_country: Germany Denmark France

location_facility: Pitié-Salpêtrière University Hospital Center

location_facility: Elective Surgery Center, Silkeborg Regional Hospital

intervention_type: Device


title: A First in Human Study to Assess Safety and Performance of the DaVingi™ System in the Treatment of Patients With Functional Tricuspid Regurgitation

summary: The DaVingi™ System is a percutaneous trans-catheter device delivered using right heart catheterization through the right internal jugular vein. The DaVingi™ System is designed for performing ring annuloplasty by using a Ring Delivery System to place a complete, flexible fabric ring around the annulus of the atrial side of the tricuspid valve. Fluoroscopy and echocardiography are used to monitor the ring placement procedure.

status: Unknown status

primary_purpose: Device Feasibility

ipd_sharing: Undecided

lead_sponsor: Cardiac Implants LLC

location_country: Czechia Israel France

location_facility:
Rambam Health Care Campus
Na Homolce Hospital
Institut Mutualiste Montsouris
Hopital Bichat
Clinique Pasteur


title: A Multicenter Double-blind Placebo-controlled Trial of Non-cultured Epidermal Cellular Grafting Versus Hyaluronic Acid for Repigmenting Stable Leukoderma (Vitiligo and Piebaldism)

summary:
The purpose of this study is to assess the efficacy of autologous epidermal cells suspension grafting using a newly developed device named VITICELL® compared to placebo.

The secondary objective is to evaluate safety of VITICELL® and global satisfaction of patient and investigator.

lead_sponsor: Laboratoires Genévrier

location_country: Italy Belgium France

location_facility: Department of Dermatology - Ghent University Hospital


title: A Phase I Dose Escalation Study To Determine The Optimal Biological Dose Of BN83495 - An Oral Steroid Sulphatase Inhibitor - In Postmenopausal Women With Oestrogen-Receptor Positive Breast Cancer Whose Disease Progressed After Prior Therapy For Locally Advanced/Metastatic Disease

summary:
The purpose of this study is to determine the optimal biological dose (OBD) of Irosustat (BN83495) in postmenopausal women with oestrogen receptor (ER) positive locally advanced or metastatic breast cancer with disease progression after prior hormonal therapy.

This study is designed to provide necessary information on safety and dose response of BN83495, when given by repeated once daily oral administration, while achieving a maximal STS inhibition and a maximal reduction in plasma oestradiol (E2) and adiol levels. The data obtained will be used to plan further clinical studies.

lead_sponsor: Ipsen

location_country:
United Kingdom
Belgium
France

location_facility:
Institut Jules Bordet
Institut Georges François Leclerc
Centre Paul Papin
Centre Eugène Marquis
Imperial College Healthcare NHS Trust


type: Other Grant/Funding Number

title: Preimplantation Factor (PIF): Biomarker Detection in Maternal Blood - Correlation With Live Birth.

summary:
PIF: biomarker of pregnancy, miscarriage, premature birth, preeclampsia, placenta accreta.

Except for serum hCG, no pregnancy-complication markers are widely employed to predict the need for medical intervention. Since circulating PIF is present from very early and throughout viable pregnancy, it may represent a specific biomarker candidate. PIF levels will be analyzed in serum of pregnant women in a range of settings: a) following IVF; b) index pregnancy of women with history of recurrent pregnancy loss, c) index pregnancy of women with history of placenta mediated complications such as: intrauterine growth restriction, spontaneous idiopathic preterm delivery, and preeclampsia; and d) index pregnancy in women with evidence of abnormal placentation, namely placenta accreta and related conditions.

lead_sponsor: BioIncept LLC

sponsor_collaborators:
BioIncept LLC
Merck KGaA, Darmstadt, Germany

location_country:
United States
Greece
France

location_facility: Yale Women and Children's Center for Blood Disorders & Yale Fertility Center

location_facility: Lab Clement - Seine St Denis Hospital, Le Blanc Mesnil


title: Preimplantation Factor (PIF): Prognostic Value of a Non-invasive Embryo Viability Biomarker Detection in Culture Media Followed by Single Embryo Transfer - Correlation With Live Birth.

summary: PIF: biomarker of successful implantation To overcome the poor reproductive potential of embryos generated during in vitro fertilization cycles and the lack of markers enabling the identification of the most competent ones, it is common to transfer multiple embryos. However this practice is associated with the risks of multi-fetal pregnancies and high morbidity/mortality. Ideally, the availability of a marker specifically produced by viable embryos would permit the transfer of a single embryo (SET) without affecting the chances of pregnancy and, most importantly, capable to drastically reduce multiple pregnancies after IVF. In preliminary work, we demonstrated that no pregnancy resulted following the transfer of embryos where PIF was undetectable in culture media.(Keramitsoglou, T et al. ASRI Meeting, Hamburg, 2012) Using a non-invasive method of detection of PIF in the media surrounding the embryo will be correlated to live birth following single embryo transfer. By selecting only viable embryos, it will reduce the need for multiple IVF cycles, increase the rate of pregnancy outcome associated with SET, and will minimize multi-fetal pregnancy that has very high medical and societal costs both in pregnancy and after delivery.

location_facility: Lab Clement - Seine St. Denis Hospital, Le Blanc Mesnil


title: Short Term Follow-up of Patient Implanted With the Proximal Interphalangeal

summary:
Hammertoe and ClawToe are ones of the most common foot deformities. The second toe is the most frequently affected, but the other small toes may also be involved. When nonoperative treatment fails, surgery often is recommended. One surgical option is the arthrodesis of the Proximal Interphalangeal (PIP) joint. This arthrodesis is technically demanding, its needs:

Internal fixation;
Interphalangeal Compression;
Anatomic PIP joint plantar flexion angle. The implant Ipp-On has been developed for this specific arthrodesis. The objective of this study is to evaluate the efficacy and safety of the Proximal Interphalangeal Implant: Ipp-On at short term follow up.

The safety of the Ipp-On will be described in terms of rate of device related complications.

The efficacy of the Ipp-On will be described in terms of fusion rate at 6 months.

lead_sponsor: Integra LifeSciences Services

location_country: Switzerland Belgium France

location_facility: Ospedale Regionale di Bellinzona e Valli


title: A Multi-centred, Non-comparative, Non-randomised, Open, Post Marketing Investigation to Look at the Long-term Clinical Efficacy of the Delta Xtend Reverse Total Shoulder Used in the Treatment of Shoulders With Rotator Cuff Deficiencies.

summary: This post-marketing study is designed to collect data relating to the use of the Delta Xtend™ Total Shoulder Replacement System in shoulder with rotator cuff deficiencies. This allows for continued monitoring of the performance and safety of the prosthesis in a broader range of clinical situations.

lead_sponsor: DePuy International

location_country: Germany Belgium France

location_facility:
Universitair Ziekenhuis
Clinique Generale D'Annecy
Krankenhaus Munchen Bogenhausen
CHRU Reims Hôpital Maison Blanche
Polyclinique de l'Atlantique


title: International Collaborative Treatment Protocol for Infants Under One Year With Acute Lymphoblastic or Biphenotypic Leukemia

summary:
RATIONALE: Giving chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine, methotrexate, leucovorin, and antithymocyte globulin before and after transplant may stop this from happening. It is not yet known which treatment regimen is most effective in treating acute leukemia.

PURPOSE: This randomized clinical trial is studying how well different therapies work in treating infants with newly diagnosed acute leukemia.

ReferenceCitation: Stutterheim J, van der Sluis IM, de Lorenzo P, Alten J, Ancliffe P, Attarbaschi A, Brethon B, Biondi A, Campbell M, Cazzaniga G, Escherich G, Ferster A, Kotecha RS, Lausen B, Li CK, Lo Nigro L, Locatelli F, Marschalek R, Meyer C, Schrappe M, Stary J, Vora A, Zuna J, van der Velden VHJ, Szczepanski T, Valsecchi MG, Pieters R. Clinical Implications of Minimal Residual Disease Detection in Infants With KMT2A-Rearranged Acute Lymphoblastic Leukemia Treated on the Interfant-06 Protocol. J Clin Oncol. 2021 Feb 20;39(6):652-662. doi: 10.1200/JCO.20.02333. Epub 2021 Jan 6. Erratum In: J Clin Oncol. 2023 Oct 20;41(30):4825.

oa_colors: green hybrid

evidence: open (via page says license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repository_institution: Erasmus University Rotterdam - EUR Research Repository

evidence: oa repository (via pmcid lookup)

repository_normalized: PubMed Central

repositories_concat: PubMed Central;pure.eur.nl

ReferenceCitation: Pieters R, De Lorenzo P, Ancliffe P, Aversa LA, Brethon B, Biondi A, Campbell M, Escherich G, Ferster A, Gardner RA, Kotecha RS, Lausen B, Li CK, Locatelli F, Attarbaschi A, Peters C, Rubnitz JE, Silverman LB, Stary J, Szczepanski T, Vora A, Schrappe M, Valsecchi MG. Outcome of Infants Younger Than 1 Year With Acute Lymphoblastic Leukemia Treated With the Interfant-06 Protocol: Results From an International Phase III Randomized Study. J Clin Oncol. 2019 Sep 1;37(25):2246-2256. doi: 10.1200/JCO.19.00261. Epub 2019 Jul 8.

lead_sponsor: Dutch Childhood Oncology Group

collaborators: Associazione Italiana Ematologia Oncologia Pediatrica

collaborators: Australian and New Zealand Children's Oncology Group

location_country:
Germany
Italy
United Kingdom
Netherlands
Austria
Belgium
United States
Czechia
France

location_facility: M. D. Anderson Cancer Center at University of Texas

location_facility: Children's Hospital and Regional Medical Center - Seattle

location_facility: Great Ormond Street Hospital for Children

location_facility: University Medical Center Hamburg - Eppendorf

location_facility: Nuovo Ospedale San Gerardo at University of Milano-Bicocca

location_facility: Hopital Universitaire Des Enfants Reine Fabiola


title: The Development and Validation of the SWADOC Tool: A Study Protocol for a Multicentric Prospective Cohort Study

summary:
Background: After a period of coma, patients with severe brain injury may present disorders of consciousness (DOC). A wide proportion of these patients also suffer from severe dysphagia. Assessment and therapy of swallowing disabilities of DOC patients are essential because dysphagia has major functional consequences and comorbidities. Dysphagia evaluation in patients with DOC is hampered by the lack of adapted tools. The first aim of the study was to develop a new tool, the SWallowing Assessment in Disorders Of Consciousness (SWADOC), and propose a validation protocol. The SWADOC tool has been developed to help therapists to apprehend components related to swallowing in patients with DOC. The second aim is to appreciate the relationship between patients' level of consciousness and SWADOC items and scores.

Method/design: In this multicentric prospective cohort, 104 patients with DOC will be tested three times during two consecutive days with the SWADOC tool. Statistical analyses will focus on the reliability and validity of the SWADOC tool, especially the intra and inter-rater reliability, internal consistency, measures of dispersion and concurrent validity with the FOTT Swallowing Assessment of Saliva (FOTT-SAS). The level of consciousness will be assessed with the Simplified Evaluation of CONsciousness Disorders (SECONDs) and the Coma Recovery Scale-Revised (CRS-R)

Discussion: The assessment of swallowing abilities among patients with DOC is the first necessary step towards the development of an individualized dysphagia care plan. A validated scoring tool will be essential for clinicians to better apprehend dysphagia in DOC patients and to document the evolution of their disorders.

ReferenceCitation: Melotte E, Maudoux A, Delhalle S, Lagier A, Thibaut A, Aubinet C, Kaux JF, Vanhaudenhuyse A, Ledoux D, Laureys S, Gosseries O. Swallowing in individuals with disorders of consciousness: A cohort study. Ann Phys Rehabil Med. 2021 Jul;64(4):101403. doi: 10.1016/j.rehab.2020.04.008. Epub 2020 Jul 18.

ReferenceCitation: Sanz LRD, Aubinet C, Cassol H, Bodart O, Wannez S, Bonin EAC, Barra A, Lejeune N, Martial C, Chatelle C, Ledoux D, Laureys S, Thibaut A, Gosseries O. SECONDs Administration Guidelines: A Fast Tool to Assess Consciousness in Brain-injured Patients. J Vis Exp. 2021 Feb 6;(168). doi: 10.3791/61968.

ReferenceCitation: Mortensen J, Jensen D, Kjaersgaard A. A validation study of the Facial-Oral Tract Therapy Swallowing Assessment of Saliva. Clin Rehabil. 2016 Apr;30(4):410-5. doi: 10.1177/0269215515584381. Epub 2015 Apr 28.

ReferenceCitation: Bicego A, Lejoly K, Maudoux A, Lefebvre P, Laureys S, Schweizer V, Diserens K, Faymonville ME, Vanhaudenhuyse A. [Swallowing in disorders of consciousness]. Rev Neurol (Paris). 2014 Oct;170(10):630-41. doi: 10.1016/j.neurol.2014.04.004. Epub 2014 Jun 18. French.

ReferenceCitation: Wannez S, Gosseries O, Azzolini D, Martial C, Cassol H, Aubinet C, Annen J, Martens G, Bodart O, Heine L, Charland-Verville V, Thibaut A, Chatelle C, Vanhaudenhuyse A, Demertzi A, Schnakers C, Donneau AF, Laureys S. Prevalence of coma-recovery scale-revised signs of consciousness in patients in minimally conscious state. Neuropsychol Rehabil. 2018 Dec;28(8):1350-1359. doi: 10.1080/09602011.2017.1310656. Epub 2017 Apr 11.

ReferenceCitation: Melotte E, Belorgeot M, Herr R, Simon J, Kaux JF, Laureys S, Sanz LRD, Lagier A, Morsomme D, Pellas F, Gosseries O. The Development and Validation of the SWADOC: A Study Protocol for a Multicenter Prospective Cohort Study. Front Neurol. 2021 Apr 29;12:662634. doi: 10.3389/fneur.2021.662634. eCollection 2021.

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

repository_institution: University of Liège - Open Repository and Bibliography

repositories_concat: PubMed Central;orbi.ulg.ac.be

unpaywall_oa_status: gold

publisher_dissemination: Frontiers

lead_sponsor: University of Liege

collaborators: Centre du Cerveau², Centre hospitalier universitaire de Liège

collaborators: Centre de Traumatologie et de Réadaptation de Jette (Erasme, ULB)

collaborators: Cliniques de Soins Spécialisés Valdor-Pèrî

collaborators: Centre Hospitalier Neurologique William Lennox (UCL)

collaborators: Centre Hospitalier Universitaire de Liege

collaborators: Centre Hospitalier Universitaire de Nīmes

collaborators: Fontfroide functional re-education centre, Montpellier, France

location_country: Belgium France

location_facility: Centre neurologique et de réadaptation fonctionnelle de Fraiture-en-Condroz

location_facility: Centre du Cerveau², University Hospital of Liège

intervention_type: Behavioral

lead_sponsor_type: academique


title: A Phase I, Open Label, Study to Assess Safety, Tolerability, Biodistribution, Radiation Dosimetry and SPECT/CT Imaging Characteristics of Intravenous 111In-IPN01087 in Patients With Locally Advanced or Metastatic Pancreatic Ductal Adenocarcinoma or Colorectal Cancer.

summary: 111Indium-labelled IPN01087 (111In-IPN01087) is developed as a radioactive diagnostic imaging agent in patients with colorectal or pancreatic cancer. It is used with single-photon emission computed tomography (SPECT) for the identification of tumours that overexpress the neurotensin receptor-1 (NTSR1). The purpose of this study is to assess how well 111In-IPN01087 is tolerated and what the most suitable amount to be injected is to obtain good quality images. The study will also look at how 111In-IPN01087 is distributed throughout the body and what the optimal time for doing the scans will be after it has been given as a single intravenous injection.

status: Withdrawn

primary_purpose: Diagnostic

location_facility:
Institut Jules Bordet
Centre Léon Bérard
UZ Leuven
CHU de Nantes - Hôpital Hôtel Dieu
CHRU de Nancy - Hôpital de Brabois


lead_sponsor: Medicines Development for Global Health

summary: The effective dose of moxidectin to treat human scabies is not known. This study aims to provide proof of concept that a single dose of moxidectin is effective in eliminating the scabies parasite in humans and to enable the determination of an optimal dose of moxidectin for treatment of scabies for further clinical studies.

location_facility:
Royal Darwin Hospital
Hopital Henri Mondor AP-HP
CHU Saint-Etienne Hopital Nord
Medizinischen Universität Wien
CHU Nice Hopital Archet 2

title: A Phase II, Randomized, Double-blind, Parallel Group Dose Finding Study of Single Oral Doses of Moxidectin in Adults With Scabies

source: euctr

location_country: Australia Austria France


title: An Open-label, Dose-escalation Phase I/Ib Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of AsiDNA, a DNA Repair Inhibitor Administered Intravenously, as Single Agent and to Assess the Safety and the Efficacy of AsiDNA in Combination With Carboplatin With or Without Paclitaxel, in Patients With Advanced Solid Tumors

summary:
The aim of the study is to assess:

Part A: the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of AsiDNA in patients with advanced solid tumors.
Part B: the safety and preliminary efficacy of AsiDNA in combination with Carboplatin with or without Paclitaxel in patients with Advanced solid tumors.

ReferenceCitation: Le Tourneau C, Delord JP, Kotecki N, Borcoman E, Gomez-Roca C, Hescot S, Jungels C, Vincent-Salomon A, Cockenpot V, Eberst L, Mole A, Jdey W, Bono F, Trochon-Joseph V, Toussaint H, Zandanel C, Adamiec O, de Beaumont O, Cassier PA. A Phase 1 dose-escalation study to evaluate safety, pharmacokinetics and pharmacodynamics of AsiDNA, a first-in-class DNA repair inhibitor, administered intravenously in patients with advanced solid tumours. Br J Cancer. 2020 Nov;123(10):1481-1489. doi: 10.1038/s41416-020-01028-8. Epub 2020 Aug 25.

evidence: open (via crossref license)

repositories_concat: PubMed Central

lead_sponsor: Valerio Therapeutics

location_facility:
Institut Jules Bordet
Grand Hôpital de Charleroi
Institut Claudius Regaud
Centre Leon Berard
Institut Curie


title: Phase 1/2a Study to Evaluate the Safety, Activity, and Pharmacokinetics of Escalating Doses of GNS561 in Patients With Primary and Secondary Liver Cancer

summary: This is a first in human, open-label dose escalation study to investigate the safety, tolerability and pharmacokinetics of GNS561 in patients Primary and Secondary liver cancer

ReferenceCitation: Harding JJ, Awada A, Roth G, Decaens T, Merle P, Kotecki N, Dreyer C, Ansaldi C, Rachid M, Mezouar S, Menut A, Bestion EN, Paradis V, Halfon P, Abou-Alfa GK, Raymond E. First-In-Human Effects of PPT1 Inhibition Using the Oral Treatment with GNS561/Ezurpimtrostat in Patients with Primary and Secondary Liver Cancers. Liver Cancer. 2022 Feb 15;11(3):268-277. doi: 10.1159/000522418. eCollection 2022 Jun.

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: Université Libre de Bruxelles - Dépôt institutionnel de l'Université libre de Bruxelles

repositories_concat: PubMed Central;dipot.ulb.ac.be

publisher_dissemination: S. Karger AG

lead_sponsor: Genoscience Pharma

location_country:
United States
Belgium
France

location_facility:
Croix-Rousse Hospital
CHU Grenoble
Memorial Sloan Kettering
Saint-Joseph Hospital
Jules Bordet Institute


type: Registry Identifier

title: Phase I Dose-escalation Study of the Orally Administered Selective Bcl-2 Inhibitor S 055746 as Monotherapy for the Treatment of Patients With Acute Myeloid Leukaemia (AML) or High or Very High Risk Myelodysplastic Syndrome (MDS)

summary: The purpose of this study is to determine the safety profile and tolerability of S 055746 in patients with AML, and high or very high risk MDS, in terms of Dose-Limiting Toxicities (DLTs), Maximum Tolerated Dose (MTD) and determine the Recommended Phase 2 Dose (RP2D) through safety profile (DLT, MTD), PK profile, PD profile and preliminary efficacy.

ipd_sharing: Yes

ipd_sharing_description:
Researchers can ask for a study protocol, patient-level and/or study-level clinical trial data including clinical study reports (CSRs).

They can ask all interventional clinical studies:

submitted for new medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
Where Servier or an affiliate are the Marketing Authorization Holders (MAH). The date of the first Marketing Authorization of the new medicine (or the new indication) in one of the EEA Member States will be considered within this scope.

lead_sponsor: Institut de Recherches Internationales Servier

location_country: Australia France

location_facility:
Hôpital Saint Louis
Royal Melbourne Hospital
The Alfred Hospital
Institut Paoli Calmettes
Centre Hospitalier Lyon Sud


intervention_type: Biological

lead_sponsor: Alexion Pharmaceuticals, Inc.

summary: This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study is to provide access to treatment in a disease where no approved treatment exists. This is an experimental treatment provided under specific treatment guidelines in which safety endpoints will be collected.

location_facility:
Hôpital des Enfants
Children's Hospital of Pittsburgh
Hôpital Necker - Enfants Malades
Hôpital Bicêtre
Colorado Center for Bone Research

status: Approved for marketing

title: An Open-label, Multicenter, Expanded Access Program for Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)

location_country:
United States
France

study_type: Expanded Access


type: Other Identifier

title: Empirical Treatment Against Cytomegalovirus and Tuberculosis in HIV-infected Infants With Severe Pneumonia: a Multicenter, Open-label Randomized Controlled Clinical Trial

acronym: EMPIRICAL

summary: This trial will evaluate whether empirical treatment against cytomegalovirus and tuberculosis improves survival of HIV-infected infants with severe pneumonia.

ReferenceCitation: Rojo P, Moraleda C, Tagarro A, Dominguez-Rodriguez S, Castillo LM, Tato LMP, Lopez AS, Manukyan L, Marcy O, Leroy V, Nardone A, Burger D, Bassat Q, Bates M, Moh R, Iroh Tam PY, Mvalo T, Magallhaes J, Buck WC, Sacarlal J, Musiime V, Chabala C, Mujuru HA. Empirical treatment against cytomegalovirus and tuberculosis in HIV-infected infants with severe pneumonia: study protocol for a multicenter, open-label randomized controlled clinical trial. Trials. 2022 Jun 27;23(1):531. doi: 10.1186/s13063-022-06203-1.

repository_institution: Universite de Bordeaux - Oskar-Bordeaux

repository_institution: Radboud University - Radboud Repository

repository_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

repository_institution: California Digital Library - eScholarship

repository_institution: Liverpool School of Tropical Medicine - LSTM Online Archive

repository_institution: Research Square - Research Square

repository_normalized: Research Square

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inria

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Paris Descartes

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - memSIC

ipd_sharing_description: After principal results, addressing primary and secondary objectives will be published. A final repository will be chosen for anonymized data sharing, and transparency after the trial is closed, according to the funder (EDCTP) rules and recommendations, unless national laws impede it.

lead_sponsor: Hospital Universitario 12 de Octubre

collaborators: Institut National de la Santé Et de la Recherche Médicale, France

collaborators: Malawi-Liverpool-Wellcome Trust Clinical Research Programme

collaborators: Centro de Investigação em Saúde de Manhiça

collaborators: University Teaching Hospital, Lusaka, Zambia

location_country:
Malawi
Italy
Uganda
United Kingdom
Zimbabwe
Côte D'Ivoire
Netherlands
Spain
Mozambique
Zambia
France

location_facility: Programme PACCI. Centre Hospitalier Cocody.

location_facility: Cemtro de Investigaçao em Saúde da Manhiça

location_facility: Fundación para la Investigación Biomédica del Hospital 12 de Octubre

location_facility: University of Zimbabwe Clinical Research Centre

location_facility: Malawi Liverpool Welcome Trust. Queen Elizabeth Central Hospital College of Medicine

location_facility: Stichting Katholieke Universiteit Radboudumc


title: The European NAFLD Registry

summary: The European NAFLD Registry is a prospectively recruited, observational study supporting the study of the clinical phenotype, natural history, disease outcomes and pathophysiology of Non-Alcoholic Fatty Liver Disease and Non-Alcoholic Steatohepatitis. The ultimate goals are to better understand the drivers of interpatient variation in disease pathophysiology and severity and to utilise this information to develop and validate biomarkers that, singly or in combination, enable detection and monitoring of disease progression and/or from NAFL through NASH to fibrosis and cirrhosis.

ReferenceCitation: McGlinchey AJ, Govaere O, Geng D, Ratziu V, Allison M, Bousier J, Petta S, de Oliviera C, Bugianesi E, Schattenberg JM, Daly AK, Hyotylainen T, Anstee QM, Oresic M. Metabolic signatures across the full spectrum of non-alcoholic fatty liver disease. JHEP Rep. 2022 Mar 26;4(5):100477. doi: 10.1016/j.jhepr.2022.100477. eCollection 2022 May.

repository_institution: Newcastle Univesity - Newcastle University ePrints

repository_institution: Nova Science Publishers, Inc. - Nova Science Publishers

repository_institution: KU Leuven - Lirias

repository_normalized: LIRIAS (KU Leuven)

repository_institution: University of Cambridge - Apollo

repositories_concat: LIRIAS (KU Leuven);PubMed Central;eprints.ncl.ac.uk;iris.unipa.it;www.repository.cam.ac.uk

lead_sponsor: Newcastle University

collaborators: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

collaborators: Institute of Cardiometabolism and Nutrition, France

collaborators: National and Kapodistrian University of Athens

location_country:
Finland
Germany
Italy
Portugal
United Kingdom
Sweden
Netherlands
Switzerland
Belgium
Spain
Greece
France

location_facility: Institute of Biomedicine of Sevilla (IBiS), Virgen del Rocío University Hospital

location_facility: Department of Medical Sciences University of Torino

location_facility: The Newcastle Upon Tyne Hospitals Nhs Foundation Trust

location_facility: Royal London Hospital, Barts Health NHS Trust

location_facility: Le Centre de Recherche Clinique (CRC) du CHU d'Angers

location_facility: UNIVERSITÄTSMEDIZIN der Johannes Gutenberg Universität Mainz

location_facility: Institut ICAN - Institute of Cardiometabolism And Nutrition Hôpital de la Pitié Salpêtrière

location_facility: University Hospitals Birmingham Nhs Foundation Trust

location_facility: Oxford University Hospitals Nhs Foundation Trust

location_facility: Marqués de Valdecilla University Hospital


title: Vitamin D3 - Omega3 - Home Exercise - Healthy Ageing and Longevity Trial (Acronym: DO-HEALTH)

acronym: DO-HEALTH

summary:
The European population is aging rapidly which poses a challenge on the individual, the European societies, and health care systems. Among the most promising public health interventions that may extend healthy life expectancy at older age are vitamin D, marine omega-3 fatty acids and physical exercise. However, their individual and combined effects have yet to be confirmed in a clinical trial.

The broad aim of DO-HEALTH is to prolong healthy life expectancy in European seniors. The specific aim is to establish whether vitamin D, omega-3 fatty acids, and a simple home exercise program will prevent disease at older age.

To achieve these aims, DO-HEALTH will enroll 2152 community-dwelling men and women who are 70 years and older, an age when chronic diseases increase substantially. The DO-HEALTH seniors will be recruited from 7 European cities (Zurich, Basel, Geneva, Toulouse, Berlin, Innsbruck and Coimbra) and will be randomized in a 2x2x2 factorial design trial to a simple home exercise program and/or vitamin D, and/or omega-3 fatty acids, over a 3 year period. This will allow to test the individual and the combined benefit of the interventions in the prevention of 5 primary endpoints: incident non-vertebral fractures; functional decline; systolic and diastolic blood pressure change; cognitive decline; and the rate of any infection. Key secondary endpoints include incidence of hip fractures, rate of falls, severity of pain in symptomatic knee osteoarthritis, gastro-intestinal symptoms, mental and oral health, quality of life, and mortality.

All clinical endpoints will be supported by a large DO-HEALTH biomarker study to evaluate the effect of the interventions at the cellular level of multi-organ function. DO-HEALTH will further evaluate reasons why or why not seniors adhere to the 3 interventions, and will assess their cost-benefit in a health economic model based on documented health care utilization and observed incidence of chronic disease.

website DO-HEALTH: http://do-health.eu/wordpress/

ReferenceCitation: Bischoff-Ferrari HA, Willett WC, Manson JE, Dawson-Hughes B, Manz MG, Theiler R, Braendle K, Vellas B, Rizzoli R, Kressig RW, Staehelin HB, Da Silva JAP, Armbrecht G, Egli A, Kanis JA, Orav EJ, Gaengler S. Combined Vitamin D, Omega-3 Fatty Acids, and a Simple Home Exercise Program May Reduce Cancer Risk Among Active Adults Aged 70 and Older: A Randomized Clinical Trial. Front Aging. 2022 Apr 25;3:852643. doi: 10.3389/fragi.2022.852643. eCollection 2022.

repository_institution: University of Zurich - Zurich Open Repository and Archive

repository_institution: University of Geneva - Archive ouverte UNIGE

repositories_concat: PubMed Central;unige.ch;www.zora.uzh.ch

ReferenceCitation: de Godoi Rezende Costa Molino C, Chocano-Bedoya PO, Sadlon A, Theiler R, Orav JE, Vellas B, Rizzoli R, Kressig RW, Kanis JA, Guyonnet S, Lang W, Egli A, Bischoff-Ferrari HA; DO-HEALTH Research Group. Prevalence of polypharmacy in community-dwelling older adults from seven centres in five European countries: a cross-sectional study of DO-HEALTH. BMJ Open. 2022 Apr 29;12(4):e051881. doi: 10.1136/bmjopen-2021-051881.

repository_institution: University Library Bern - BORIS - Bern Open Repository and Information System

ReferenceCitation: Schietzel S, Chocano-Bedoya PO, Sadlon A, Gagesch M, Willett WC, Orav EJ, Kressig RW, Vellas B, Rizzoli R, da Silva JAP, Blauth M, Kanis JA, Egli A, Bischoff-Ferrari HA. Prevalence of healthy aging among community dwelling adults age 70 and older from five European countries. BMC Geriatr. 2022 Mar 2;22(1):174. doi: 10.1186/s12877-022-02755-8.

repository_institution: Universidad de Coimbra - Estudo Geral

license: cc-by-nc-sa

license_normalized: cc-by-nc-sa

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inserm

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Paris Descartes

ReferenceCitation: Bischoff-Ferrari HA, Vellas B, Rizzoli R, Kressig RW, da Silva JAP, Blauth M, Felson DT, McCloskey EV, Watzl B, Hofbauer LC, Felsenberg D, Willett WC, Dawson-Hughes B, Manson JE, Siebert U, Theiler R, Staehelin HB, de Godoi Rezende Costa Molino C, Chocano-Bedoya PO, Abderhalden LA, Egli A, Kanis JA, Orav EJ; DO-HEALTH Research Group. Effect of Vitamin D Supplementation, Omega-3 Fatty Acid Supplementation, or a Strength-Training Exercise Program on Clinical Outcomes in Older Adults: The DO-HEALTH Randomized Clinical Trial. JAMA. 2020 Nov 10;324(18):1855-1868. doi: 10.1001/jama.2020.16909.

collaborators: University Hospital, Toulouse, France (Prof. Bruno Vellas, MD)

collaborators: University of Coimbra, Portugal (Prof. José daSilva, MD)

collaborators: University of Manchester, UK (Prof. David Felson, MD MPH)

collaborators: Max Rubner University, Germany (Prof. Bernhard Watzl, PhD)

collaborators: International Osteoporosis Foundation (IOF; Prof. John Kanis, MD)

collaborators: Gut Pictures, Switzerland (Benno Gut; animated exercise video)

collaborators: NOVAMEN, France (Sandrine Rival; logistic management partner)

collaborators: Pharmalys, UK (Marieme Ba; monitoring partner)

collaborators: DSM Nutritional Products (Dr. Elisabeth Stöcklin PhD, Dr. Manfred Eggersdorfer PhD)

collaborators: Nestlé (Michaela Höhne PhD, Irène Corthesy PhD)

collaborators: Funding within Framework 7 research program of the European Commission (project 278588)

collaborators: Further collaborators and advisors at website do-health.eu

location_country: Germany Portugal Switzerland Austria France

location_facility: University of Coimbra - Clínica Universitária de Reumatologia

location_facility: Centre on Aging and Mobility, University of Zurich and City Hospital Waid

location_facility: University of Toulouse - Centre de Recherche - Gérontopôle Hôpital La Grave


title: An Open Label, Phase 2 Study to Evaluate the Effect of A3907 on Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics in Adults With Primary Sclerosing Cholangitis (PSC)

summary: This study will test a drug called A3907 to see how safe and tolerated it is for treating people with Primary Sclerosing Cholangitis (PSC).

lead_sponsor: Albireo

location_country: Italy Spain France

location_facility: ASST di Monza - Azienda Ospedaliera San Gerardo


title: Innovative plAnt Protein Fibre and Physical Activity Solutions to Address Poor appEtite and prevenT undernutrITion in oldEr Adults (APPETITE)

summary: The APPETITE Trial aims to investigate the efficacy of innovative plant protein fibre (PPF) products (developed in a previous workpackage) as part of a personalised diet with/out physical activity on appetite and incidence of undernutrition in older persons from three European countries at high risk of undernutrition.

ReferenceCitation: Volkert D, et al. Innovative plAnt Protein fibre and Physical activity solutions to address poor appEtite and prevenT undernutrITion in oldEr adults - APPETITE. Nutrition Bulletin. 2021; 46: 486-496

lead_sponsor: University College Dublin

collaborators: Institut National de Recherche pour l'Agriculture, l'Alimentation et l'Environnement

location_country: Germany Ireland Italy France

location_facility: School of Public Health, Physiotherapy and Sports Science


title: Robotic Minimally Invasive Gynecological, Urological, and General Surgery With Dexter

summary: The purpose of this study is to confirm the perioperative and early postoperative safety and clinical performance of the Dexter Robotic System (Dexter Surgical System, Model DM-L6).

lead_sponsor: Distalmotion SA

location_country: Germany Switzerland France

location_facility:
UKSH
CHUV
Inselspital
Centre Hospitalier de Saintes


title: Standard Craniectomy Against Laparotomy for the Treatment of Traumatic Rise in Intracranial Pressure and the Effect on Long-term Outcome

acronym: SCALPEL

summary: Intractable high intracranial pressures (ICP) are associated with poor functional outcomes and mortality, so the SCALPEL trials aims to evaluate the effect of decompressive craniectomy against decompressive laparotomy to lower those pressures in diffuse TBI. The primary outcome measure for that evaluation is functional outcome after 12 months on the extended Glasgow Outcome Scale (GOS-E).

lead_sponsor: Technical University of Munich

sponsor_collaborators:
Technical University of Munich
Federal Ministry of Defence (Germany)
Bundesministerium der Verteidigung

location_country: Germany France

location_facility: Department of Neurosurgery - Klinikum rechts der Isar

intervention_type: Procedure


title: Influencia de la Ansiedad en el Aprendizaje Motor de Una Tarea Manual Precisa y en la Capacidad de Imaginación Motora

summary:
In motor learning, to consider that movements are produced by the cooperation and combination of many brain structures and are influenced by the emotions to which individuals are subjected is essential. Several neural circuits have been identified that closely link the emotional system and the motion control system.

Anxiety is defined by persistent and excessive worries that do not disappear even in the absence of the stressor. Anxiety has been found to produce inefficiencies in information processing, which can result in performance deficits, as well as self-reported anxiety has been linked to poorer working retrieval performance. In these stress and anxiety contexts, relaxation techniques have been widely used to reduce psychophysiological arousal levels.

Understanding how movement, emotions and interactions are regulated is significant because of the large number of movements humans perform. Of these, manual tasks represent precise movements that require the integration of many elements by the nervous system to perform these tasks successfully. How anxiety influence the way manual tasks are learned is still unknown.

On the other hand, motor imagery (MI) is a cognitive process that is an important contributor to how movements are planned and executed. The use of MI has been recommended to improve movement learning and task execution. Knowing MI capacity is essential for creating effective and individualized MI programs. However, how a relax intervention can affect the motor imagery ability in anxiety people is still unknown.

The aim of our study was to find out whether a relaxation intervention prior to MI practice in subjects with anxiety can influence the learning of a precise manual task not previously trained on four parameters of fine motor control: time, error, speed, and accuracy. On the other hand, the aim is to determine if the ability of internal visual, external visual, and kinaesthetic imagery varies when the anxiety participants are subjected to relaxation.

The investigators expect that participants with anxiety, to whom relaxation is induced, will show better motor performance on the fine motor task and better motor imagery ability. In contrast, the investigators expect that participants with anxiety, to whom relaxation is not induced, will show poorer motor performance on the fine motor task and poorer motor imagery ability.

primary_purpose: Basic Science

ReferenceCitation: Coombes SA, Cauraugh JH, Janelle CM. Emotion and movement: activation of defensive circuitry alters the magnitude of a sustained muscle contraction. Neurosci Lett. 2006 Apr 3;396(3):192-6. doi: 10.1016/j.neulet.2005.11.048. Epub 2005 Dec 20.

ReferenceCitation: Moran TP. Anxiety and working memory capacity: A meta-analysis and narrative review. Psychol Bull. 2016 Aug;142(8):831-864. doi: 10.1037/bul0000051. Epub 2016 Mar 10.

ReferenceCitation: Doumas M, Morsanyi K, Young WR. Cognitively and socially induced stress affects postural control. Exp Brain Res. 2018 Jan;236(1):305-314. doi: 10.1007/s00221-017-5128-8. Epub 2017 Nov 14.

ReferenceCitation: MacIntyre TE, Madan CR, Moran AP, Collet C, Guillot A. Motor imagery, performance and motor rehabilitation. Prog Brain Res. 2018;240:141-159. doi: 10.1016/bs.pbr.2018.09.010. Epub 2018 Oct 24.

ReferenceCitation: Schuster C, Hilfiker R, Amft O, Scheidhauer A, Andrews B, Butler J, Kischka U, Ettlin T. Best practice for motor imagery: a systematic literature review on motor imagery training elements in five different disciplines. BMC Med. 2011 Jun 17;9:75. doi: 10.1186/1741-7015-9-75.

ReferenceCitation: Trapero-Asenjo S, Gallego-Izquierdo T, Pecos-Martin D, Nunez-Nagy S. Translation, cultural adaptation, and validation of the Spanish version of the Movement Imagery Questionnaire-3 (MIQ-3). Musculoskelet Sci Pract. 2021 Feb;51:102313. doi: 10.1016/j.msksp.2020.102313. Epub 2020 Dec 9.

ReferenceCitation: Oldfield RC. The assessment and analysis of handedness: the Edinburgh inventory. Neuropsychologia. 1971 Mar;9(1):97-113. doi: 10.1016/0028-3932(71)90067-4. No abstract available.

lead_sponsor: Susana Nunez Nagy

sponsor_collaborators:
Susana Nunez Nagy
University of Alcala
Universidad Complutense de Madrid
European University of Madrid
Claude Bernard University

location_country: Spain France

location_facility:
University of Alcalá
Complutense University of Madrid
Claude Bernard University Lyon 1
European University of Madrid

intervention_type: Other


title: An Observational European Multi-database Linkage Study to Quantify Malignancy Rates in Crohn's Disease Patients With Complex Perianal Fistula Treated With Darvadstrocel

summary:
The main aim is to learn about the risk of cancer after treatment with darvadstrocel compared to other standards of care in people with Crohn's Disease.

In this study, the study doctors will review each participant's past medical records. This study is about collecting existing information only; participants will not receive treatment or need to visit a study doctor during this study.

time_perspective: Retrospective

ipd_sharing_description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

lead_sponsor: Takeda

location_country: Germany Spain Netherlands France

location_facility: 1. Système National des Données de Santé (SNDS)

location_facility: Estudio Nacional en Enfermedad Inflamatoria intestinal sobre Determinantes genéticos y Ambientales (ENEIDA)

location_facility: Institut für angewandte Gesundheitsforschung Berlin (InGef)


title: Influencia Del Estrés Agudo en el Aprendizaje Motor de Una Tarea Manual Precisa y en la Capacidad de Imaginación Motora

summary:
In motor learning, it is essential to consider that movements are produced by the cooperation and combination of many brain structures and are influenced by the emotions to which individuals are subjected. Several neural circuits have been identified that closely link the emotional system and the motion control system.

Stress is a physiological or psychological response to internal or external stressors. In principle, it has an adaptive role. However, the neuroendocrine and autonomic response generated by stress can affect cognitive processes such as memory. In addition, it has been shown to influence motor learning, especially the execution of skills in the early stages of learning.

Understanding how movement, emotions and interactions are regulated is significant because of the large number of movements humans perform. Of these, manual tasks represent precise movements that require the integration of many elements by the nervous system to perform these tasks successfully. It is still unknown how acute stress influence the way manual tasks are learned.

On the other hand, motor imagery (MI) is a cognitive process that is an important contributor to how movements are planned and executed. Its use has been recommended to improve movement learning and task execution. For an MI program to be effective and individualized, it is imperative to know this ability. However, it is also still unknown how acute stress can affect our motor imagery ability.

The main objective of this study is to determine and quantify the effects of acute stress in the learning of a precise manual task not previously trained on four parameters of fine motor control: trajectory error, timing error, timing accuracy, and task accuracy. On the other hand, the aim is to determine if the capacity of internal visual, external visual, and kinesthetic imagery, and the temporal congruence between movement execution and imagery varies when we are subjected to acute stress.

It is expected that non-anxious, non-stressed participants who are not induced with acute stress will show better motor performance on the fine motor task and better motor imagery ability and temporal congruence. In contrast, it is expected that participants without anxiety and stress who are induced with acute stress will show poorer motor performance on the fine motor task, and poorer motor imagery ability and temporal congruence.

ReferenceCitation: Ahn SN, Lee J. Effects of sensory awareness, imagery and observation on electroencephalography in adult with psychological stress. J Phys Ther Sci. 2019 Jan;31(1):17-19. doi: 10.1589/jpts.31.17. Epub 2019 Jan 10.

ReferenceCitation: Smeets T, Cornelisse S, Quaedflieg CW, Meyer T, Jelicic M, Merckelbach H. Introducing the Maastricht Acute Stress Test (MAST): a quick and non-invasive approach to elicit robust autonomic and glucocorticoid stress responses. Psychoneuroendocrinology. 2012 Dec;37(12):1998-2008. doi: 10.1016/j.psyneuen.2012.04.012. Epub 2012 May 18.

ReferenceCitation: Bali A, Jaggi AS. Clinical experimental stress studies: methods and assessment. Rev Neurosci. 2015;26(5):555-79. doi: 10.1515/revneuro-2015-0004.

collaborators:
University of Alcala
Universidad Complutense de Madrid
European University of Madrid
Claude Bernard University

location_facility:
University of Alcala
Complutense University of Madrid
Claude Bernard University Lyon 1
European University of Madrid


title: Influencia de Los Niveles de Alerta en el Aprendizaje Motor de Una Tarea Manual Precisa, en la Memoria a Corto y Largo Plazo y en la Capacidad de Imaginación Motora

summary:
In motor learning is essential to consider that movements are produced by the cooperation and combination of many brain structures and are influenced by the emotions to which individuals are subjected. Several neural circuits have been identified that closely link the emotional system and the motion control system.

Arousal is associated with many emotional responses and has effects on the nervous and motor system. In line with the "Inverted 'U' Hypothesis", all levels of both high and low arousal do not allow optimal task performance, yet moderate levels lead to excellent performance. Arousal also plays a vital role in movement learning, where a critical element is memory. There is evidence that a minimum level of arousal is required to encode or record information and that that moderate levels of arousal improve memory.

Understanding how movement, emotions and interactions are regulated is significant because of the large number of movements humans perform. Of these, manual tasks represent precise movements that require the integration of many elements by the nervous system to perform these tasks successfully. How different levels of arousal influence the way manual tasks are learned is still unknown.

On the other hand, motor imagery (MI) is a cognitive process that is an important contributor to how movements are planned and executed. The use of MI has been recommended to improve movement learning and task execution. For an effective and individualize MI program is imperative to know this capacity. However, how different levels of arousal can affect our motor imagery ability is also still unknown.

The main objective of this study is to determine and quantify the effects of arousal levels in the learning of a precise manual task not previously trained on four parameters of fine motor control: time, error, speed, and accuracy. On the other hand, the aim is to determine if the ability of internal visual, external visual, and kinaesthetic imagery varies when participants are subjected to different levels of arousal.

Researches expect that non-anxious, non-stressed participants who are shown images that elicit an optimal level of arousal will show better motor performance on the fine motor task and better motor imagery ability. In contrast, researches expect that participants without anxiety and stress who are shown pictures that elicit a sub-optimal level of arousal will show poorer motor performance on the fine motor task and poorer motor imagery ability.

ReferenceCitation: Stins JF, Beek PJ. Effects of affective picture viewing on postural control. BMC Neurosci. 2007 Oct 4;8:83. doi: 10.1186/1471-2202-8-83.


title: A Multimodal Approach to Personalized Tracking of Evolving State-Of-Consciousness in Brain- Injured Patients

acronym: PerBrain

summary: Improved treatment of severe brain injuries has resulted in increased survival rates. While some of these patients regain consciousness after a transient state of coma, others may develop a disorder of consciousness (DoC). Diagnosis of DoC currently relies on standardized behavioral assessment. The importance of accuracy in such diagnosis cannot be overstated, as it guides critical decisions on treatment (including pain management), and could underlie end-of-life decisions. Despite this importance, current behavioral diagnosis often fails, if because of the major sensory and motor deficits associated with DoC, or because of the heterogeneous etiology and pathophysiology associated with the condition. Finally, the need for accurate diagnosis and prognosis transcends the needs of the patients alone: caregiving of these patients is very stressful, principally for the large uncertainty associated with them. Thus, more accurate diagnosis and prognosis provide major relief for caregivers, and paradoxically, even if the news is not "good". For all these reasons it is critical to developing personalized diagnosis and prognosis prediction tools that permit a stratified analysis at the single-patient level. The PerBrain Project will benefit from the multidisciplinary partners' expertise, and the unique opportunity to perform longitudinal assessments in four clinical sites through both established and novel electrophysiological, neuroimaging, and physiological techniques. Based on the collected data, the investigators will develop a multimodal personalized diagnostic tool for DoC patients using state-of-the-art computational tools, such as machine learning, in order to better determine the current state (diagnosis) and future outcome (prognosis). The overall aim of this project will provide for a better understanding of the pathophysiological mechanisms in DoC, which will, in turn, allow personalized rehabilitation strategies, and improved single-patient predictions of state and prognosis.

ReferenceCitation: Giacino JT, Kalmar K. Diagnostic and prognostic guidelines for the vegetative and minimally conscious states. Neuropsychol Rehabil. 2005 Jul-Sep;15(3-4):166-74. doi: 10.1080/09602010443000498.

ReferenceCitation: Raimondo F, Rohaut B, Demertzi A, Valente M, Engemann DA, Salti M, Fernandez Slezak D, Naccache L, Sitt JD. Brain-heart interactions reveal consciousness in noncommunicating patients. Ann Neurol. 2017 Oct;82(4):578-591. doi: 10.1002/ana.25045. Epub 2017 Oct 11.

ReferenceCitation: Arzi A, Rozenkrantz L, Gorodisky L, Rozenkrantz D, Holtzman Y, Ravia A, Bekinschtein TA, Galperin T, Krimchansky BZ, Cohen G, Oksamitni A, Aidinoff E, Sacher Y, Sobel N. Olfactory sniffing signals consciousness in unresponsive patients with brain injuries. Nature. 2020 May;581(7809):428-433. doi: 10.1038/s41586-020-2245-5. Epub 2020 Apr 29.

ReferenceCitation: Casali AG, Gosseries O, Rosanova M, Boly M, Sarasso S, Casali KR, Casarotto S, Bruno MA, Laureys S, Tononi G, Massimini M. A theoretically based index of consciousness independent of sensory processing and behavior. Sci Transl Med. 2013 Aug 14;5(198):198ra105. doi: 10.1126/scitranslmed.3006294.

ReferenceCitation: Engemann DA, Raimondo F, King JR, Rohaut B, Louppe G, Faugeras F, Annen J, Cassol H, Gosseries O, Fernandez-Slezak D, Laureys S, Naccache L, Dehaene S, Sitt JD. Robust EEG-based cross-site and cross-protocol classification of states of consciousness. Brain. 2018 Nov 1;141(11):3179-3192. doi: 10.1093/brain/awy251.

ReferenceCitation: Vogler J, Klein AM, Bender A. Long-term health-related quality-of-life in patients with acquired brain injury and their caregivers. Brain Inj. 2014;28(11):1381-8. doi: 10.3109/02699052.2014.919536. Epub 2014 Jun 19.

lead_sponsor: Brain & Spine Institute (ICM)

collaborators: Ludwig-Maximilians - University of Munich

location_country: Germany Israel Italy France

location_facility: University Hospital of the Ludwig-Maximilians-University of Munich

location_facility: Dipartimento di Cura e Riabilitazione delle Gravi Cerebrolesioni Acquisite (GCA)

intervention_type: Diagnostic Test


title: Exhaled Breath Analysis Using eNose Technology as a Biomarker for Diagnosis and Disease Progression in Fibrotic Interstitial Lung Disease

acronym: ILDnose

summary: The ILDnose study a multinational, multicenter, prospective, longitudinal study in outpatients with pulmonary fibrosis. The aim is to assess the accuracy of eNose technology as diagnostic tool for diagnosis and differentiation between the most prevalent fibrotic interstitial lung diseases. The value of eNose as biomarker for disease progression and response to treatment is also assessed. Besides, validity of several questionnaires for pulmonary fibrosis is investigated.

lead_sponsor: Erasmus Medical Center

location_country:
Germany
United Kingdom
Netherlands
France

location_facility: University Lyon 1, Louis Pradel hospital, Lyon. FranceService de pneumologie, hôpital Louis Pradel


title: Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

summary: This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM) or wild type cardiomyopathy (ATTRwt-CM)

ReferenceCitation: Gillmore JD, Gane E, Taubel J, Kao J, Fontana M, Maitland ML, Seitzer J, O'Connell D, Walsh KR, Wood K, Phillips J, Xu Y, Amaral A, Boyd AP, Cehelsky JE, McKee MD, Schiermeier A, Harari O, Murphy A, Kyratsous CA, Zambrowicz B, Soltys R, Gutstein DE, Leonard J, Sepp-Lorenzino L, Lebwohl D. CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. N Engl J Med. 2021 Aug 5;385(6):493-502. doi: 10.1056/NEJMoa2107454. Epub 2021 Jun 26.

evidence: open (via free article)

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

repositories_concat: UCL Discovery

unpaywall_oa_status: bronze

publisher_dissemination: Massachusetts Medical Society

ReferenceCitation: Stephenson AA, Flanigan KM. Gene editing and modulation for Duchenne muscular dystrophy. Prog Mol Biol Transl Sci. 2021;182:225-255. doi: 10.1016/bs.pmbts.2021.01.029. Epub 2021 Mar 3.

lead_sponsor: Intellia Therapeutics

location_country:
United Kingdom
Sweden
New Zealand
France


title: Comparison of Pre- and Post-dilution Hemodiafiltration Modalities - Effects on Biocompatibility and Solute Clearance in Children

summary: Cardiovascular morbidity and mortality are increased in children on conventional haemodialysis. Haemodiafiltration (HDF) is a newer type of dialysis which has two main types (post-dilution and predilution HDF). Post-dilution HDF is associated with better vascular health, blood pressure and growth in children. Furthermore, pre-dilution HDF is shown to remove a wider spectrum of uremic toxin compared to post-dilution HDF in adults. The investigators need more data to define the optimum dialysis modality for children.

lead_sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

collaborators:
Istanbul University
Heidelberg University
Hospices Civils de Lyon

location_country:
Germany
United Kingdom
Turkey
France

location_facility: Istanbul University-Cerrahpasa, Cerrahpasa Medical Faculty

location_facility: Division of Pediatric Nephrology Center for Pediatrics and Adolescent Medicine

location_facility: Division of Pediatric Nephrology Hôpital Femme Mère Enfant, Hospices Civils de Lyon


title: An Integrated European Platform to Conduct Translational Studies in Myelodysplastic Syndromes Based on the EuroBloodNet Infrastructure

summary:
Rationale Myelodysplastic syndromes (MDS) are rare cancers with unmet medical needs. Study of MDS has been rapidly transformed by genome characterization.

The investigators hypothesize that comprehensive analyses of large patient population will allow to correctly estimate the effect of each mutation on clinical outcomes, and that niche factors and immune dysfunctions may influence the development of MDS, clonal evolution and response to treatments

Aims

1- Investigate gene mutations, niche factors and immune dysfunctions influencing the development of MDS, and define biomarkers for early identification of individuals at risk; 2- Develop prognostic models for MDS patients through integration of comprehensive genomic/clinical information; 3- Define biomarkers to better stratify the individual probability of response to specific treatments

Methods EuroBloodNet, the European Reference Network in rare hematological diseases, will provide a basis for research activities. Study of genomic features of clonal dominance in elderly subjects enrolled in large population-based studies and description of the dynamics of clonal establishment and evolution; study of bone marrow microenvironment to identify immune dysfunctions influencing MDS development. Development of inclusive statistical models to accurately predict clinical outcome at individual level, based on large MDS populations with comprehensive genomic/clinical data. Finally, analysis of mutational screening and immune profiles from patients enrolled in prospective trials, to provide evidence on genetic/immunologic profiles associated with probability of response to specific compounds

Expected results To characterize how clonal hematopoiesis relates to the induction of MDS clinical phenotype, and to test the utility of gene sequencing to detect subjects at risk of developing MDS. To define effective prognostic systems and biomarkers to stratify the individual probability of response to treatment

time_perspective: Other

ReferenceCitation: Malcovati L, Hellstrom-Lindberg E, Bowen D, Ades L, Cermak J, Del Canizo C, Della Porta MG, Fenaux P, Gattermann N, Germing U, Jansen JH, Mittelman M, Mufti G, Platzbecker U, Sanz GF, Selleslag D, Skov-Holm M, Stauder R, Symeonidis A, van de Loosdrecht AA, de Witte T, Cazzola M; European Leukemia Net. Diagnosis and treatment of primary myelodysplastic syndromes in adults: recommendations from the European LeukemiaNet. Blood. 2013 Oct 24;122(17):2943-64. doi: 10.1182/blood-2013-03-492884. Epub 2013 Aug 26.

ReferenceCitation: Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, Sole F, Bennett JM, Bowen D, Fenaux P, Dreyfus F, Kantarjian H, Kuendgen A, Levis A, Malcovati L, Cazzola M, Cermak J, Fonatsch C, Le Beau MM, Slovak ML, Krieger O, Luebbert M, Maciejewski J, Magalhaes SM, Miyazaki Y, Pfeilstocker M, Sekeres M, Sperr WR, Stauder R, Tauro S, Valent P, Vallespi T, van de Loosdrecht AA, Germing U, Haase D. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012 Sep 20;120(12):2454-65. doi: 10.1182/blood-2012-03-420489. Epub 2012 Jun 27.

ReferenceCitation: Schanz J, Tuchler H, Sole F, Mallo M, Luno E, Cervera J, Granada I, Hildebrandt B, Slovak ML, Ohyashiki K, Steidl C, Fonatsch C, Pfeilstocker M, Nosslinger T, Valent P, Giagounidis A, Aul C, Lubbert M, Stauder R, Krieger O, Garcia-Manero G, Faderl S, Pierce S, Le Beau MM, Bennett JM, Greenberg P, Germing U, Haase D. New comprehensive cytogenetic scoring system for primary myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia after MDS derived from an international database merge. J Clin Oncol. 2012 Mar 10;30(8):820-9. doi: 10.1200/JCO.2011.35.6394. Epub 2012 Feb 13.

ReferenceCitation: Nomdedeu M, Calvo X, Pereira A, Carrio A, Sole F, Luno E, Cervera J, Vallespi T, Munoz C, Gomez C, Arias A, Such E, Sanz G, Grau J, Insunza A, Calasanz MJ, Ardanaz MT, Hernandez-Rivas JM, Azaceta G, Alvarez S, Sanchez J, Martin ML, Bargay J, Gomez V, Cervero CJ, Allegue MJ, Collado R, Campo E, Esteve J, Nomdedeu B, Costa D; Spanish Group of Myelodysplastic Syndromes. Prognostic impact of chromosomal translocations in myelodysplastic syndromes and chronic myelomonocytic leukemia patients. A study by the spanish group of myelodysplastic syndromes. Genes Chromosomes Cancer. 2016 Apr;55(4):322-7. doi: 10.1002/gcc.22333. Epub 2015 Dec 22.

ReferenceCitation: Schanz J, Tuchler H, Sole F, Mallo M, Luno E, Cervera J, Grau J, Hildebrandt B, Slovak ML, Ohyashiki K, Steidl C, Fonatsch C, Pfeilstocker M, Nosslinger T, Valent P, Giagounidis A, Aul C, Lubbert M, Stauder R, Krieger O, Le Beau MM, Bennett JM, Greenberg P, Germing U, Haase D. Monosomal karyotype in MDS: explaining the poor prognosis? Leukemia. 2013 Oct;27(10):1988-95. doi: 10.1038/leu.2013.187. Epub 2013 Jun 21.

ReferenceCitation: Valcarcel D, Adema V, Sole F, Ortega M, Nomdedeu B, Sanz G, Luno E, Canizo C, de la Serna J, Ardanaz M, Marco V, Collado R, Grau J, Montoro J, Mallo M, Vallespi T. Complex, not monosomal, karyotype is the cytogenetic marker of poorest prognosis in patients with primary myelodysplastic syndrome. J Clin Oncol. 2013 Mar 1;31(7):916-22. doi: 10.1200/JCO.2012.41.6073. Epub 2013 Jan 14.

ReferenceCitation: Della Porta MG, Alessandrino EP, Bacigalupo A, van Lint MT, Malcovati L, Pascutto C, Falda M, Bernardi M, Onida F, Guidi S, Iori AP, Cerretti R, Marenco P, Pioltelli P, Angelucci E, Oneto R, Ripamonti F, Bernasconi P, Bosi A, Cazzola M, Rambaldi A; Gruppo Italiano Trapianto di Midollo Osseo. Predictive factors for the outcome of allogeneic transplantation in patients with MDS stratified according to the revised IPSS-R. Blood. 2014 Apr 10;123(15):2333-42. doi: 10.1182/blood-2013-12-542720. Epub 2014 Feb 20.

ReferenceCitation: Papaemmanuil E, Gerstung M, Malcovati L, Tauro S, Gundem G, Van Loo P, Yoon CJ, Ellis P, Wedge DC, Pellagatti A, Shlien A, Groves MJ, Forbes SA, Raine K, Hinton J, Mudie LJ, McLaren S, Hardy C, Latimer C, Della Porta MG, O'Meara S, Ambaglio I, Galli A, Butler AP, Walldin G, Teague JW, Quek L, Sternberg A, Gambacorti-Passerini C, Cross NC, Green AR, Boultwood J, Vyas P, Hellstrom-Lindberg E, Bowen D, Cazzola M, Stratton MR, Campbell PJ; Chronic Myeloid Disorders Working Group of the International Cancer Genome Consortium. Clinical and biological implications of driver mutations in myelodysplastic syndromes. Blood. 2013 Nov 21;122(22):3616-27; quiz 3699. doi: 10.1182/blood-2013-08-518886. Epub 2013 Sep 12.

ReferenceCitation: Papaemmanuil E, Cazzola M, Boultwood J, Malcovati L, Vyas P, Bowen D, Pellagatti A, Wainscoat JS, Hellstrom-Lindberg E, Gambacorti-Passerini C, Godfrey AL, Rapado I, Cvejic A, Rance R, McGee C, Ellis P, Mudie LJ, Stephens PJ, McLaren S, Massie CE, Tarpey PS, Varela I, Nik-Zainal S, Davies HR, Shlien A, Jones D, Raine K, Hinton J, Butler AP, Teague JW, Baxter EJ, Score J, Galli A, Della Porta MG, Travaglino E, Groves M, Tauro S, Munshi NC, Anderson KC, El-Naggar A, Fischer A, Mustonen V, Warren AJ, Cross NC, Green AR, Futreal PA, Stratton MR, Campbell PJ; Chronic Myeloid Disorders Working Group of the International Cancer Genome Consortium. Somatic SF3B1 mutation in myelodysplasia with ring sideroblasts. N Engl J Med. 2011 Oct 13;365(15):1384-95. doi: 10.1056/NEJMoa1103283. Epub 2011 Sep 26.

ReferenceCitation: Della Porta MG, Tuechler H, Malcovati L, Schanz J, Sanz G, Garcia-Manero G, Sole F, Bennett JM, Bowen D, Fenaux P, Dreyfus F, Kantarjian H, Kuendgen A, Levis A, Cermak J, Fonatsch C, Le Beau MM, Slovak ML, Krieger O, Luebbert M, Maciejewski J, Magalhaes SM, Miyazaki Y, Pfeilstocker M, Sekeres MA, Sperr WR, Stauder R, Tauro S, Valent P, Vallespi T, van de Loosdrecht AA, Germing U, Haase D, Greenberg PL, Cazzola M. Validation of WHO classification-based Prognostic Scoring System (WPSS) for myelodysplastic syndromes and comparison with the revised International Prognostic Scoring System (IPSS-R). A study of the International Working Group for Prognosis in Myelodysplasia (IWG-PM). Leukemia. 2015 Jul;29(7):1502-13. doi: 10.1038/leu.2015.55. Epub 2015 Feb 27.

ReferenceCitation: Della Porta MG, Travaglino E, Boveri E, Ponzoni M, Malcovati L, Papaemmanuil E, Rigolin GM, Pascutto C, Croci G, Gianelli U, Milani R, Ambaglio I, Elena C, Ubezio M, Da Via' MC, Bono E, Pietra D, Quaglia F, Bastia R, Ferretti V, Cuneo A, Morra E, Campbell PJ, Orazi A, Invernizzi R, Cazzola M; Rete Ematologica Lombarda (REL) Clinical Network. Minimal morphological criteria for defining bone marrow dysplasia: a basis for clinical implementation of WHO classification of myelodysplastic syndromes. Leukemia. 2015 Jan;29(1):66-75. doi: 10.1038/leu.2014.161. Epub 2014 May 20.

ReferenceCitation: Della Porta MG, Jackson CH, Alessandrino EP, Rossi M, Bacigalupo A, van Lint MT, Bernardi M, Allione B, Bosi A, Guidi S, Santini V, Malcovati L, Ubezio M, Milanesi C, Todisco E, Voso MT, Musto P, Onida F, Iori AP, Cerretti R, Grillo G, Molteni A, Pioltelli P, Borin L, Angelucci E, Oldani E, Sica S, Pascutto C, Ferretti V, Santoro A, Bonifazi F, Cazzola M, Rambaldi A. Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised International Prognostic Scoring System. Leukemia. 2017 Nov;31(11):2449-2457. doi: 10.1038/leu.2017.88. Epub 2017 Mar 21.

ReferenceCitation: Jaiswal S, Fontanillas P, Flannick J, Manning A, Grauman PV, Mar BG, Lindsley RC, Mermel CH, Burtt N, Chavez A, Higgins JM, Moltchanov V, Kuo FC, Kluk MJ, Henderson B, Kinnunen L, Koistinen HA, Ladenvall C, Getz G, Correa A, Banahan BF, Gabriel S, Kathiresan S, Stringham HM, McCarthy MI, Boehnke M, Tuomilehto J, Haiman C, Groop L, Atzmon G, Wilson JG, Neuberg D, Altshuler D, Ebert BL. Age-related clonal hematopoiesis associated with adverse outcomes. N Engl J Med. 2014 Dec 25;371(26):2488-98. doi: 10.1056/NEJMoa1408617. Epub 2014 Nov 26.

ReferenceCitation: Weidner H, Rauner M, Trautmann F, Schmitt J, Balaian E, Mies A, Helas S, Baschant U, Khandanpour C, Bornhauser M, Hofbauer LC, Platzbecker U. Myelodysplastic syndromes and bone loss in mice and men. Leukemia. 2017 Apr;31(4):1003-1007. doi: 10.1038/leu.2017.7. Epub 2017 Jan 11. No abstract available.

ReferenceCitation: Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Epub 2009 Feb 21.

ReferenceCitation: Fenaux P, Giagounidis A, Selleslag D, Beyne-Rauzy O, Mufti G, Mittelman M, Muus P, Te Boekhorst P, Sanz G, Del Canizo C, Guerci-Bresler A, Nilsson L, Platzbecker U, Lubbert M, Quesnel B, Cazzola M, Ganser A, Bowen D, Schlegelberger B, Aul C, Knight R, Francis J, Fu T, Hellstrom-Lindberg E; MDS-004 Lenalidomide del5q Study Group. A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q. Blood. 2011 Oct 6;118(14):3765-76. doi: 10.1182/blood-2011-01-330126. Epub 2011 Jul 13.

ReferenceCitation: Mallo M, Luno E, Sanzo C, Cervera J, Haase D, Schanz J, Garcia-Manero G, del Canizo C, Sanz GF, Sole F. Clinical impact of the clone size in MDS cases with monosomy 7 or 7q deletion, trisomy 8, 20q deletion and loss of Y chromosome. Leuk Res. 2011 Jun;35(6):834-6. doi: 10.1016/j.leukres.2011.01.003. Epub 2011 Jan 26.

ReferenceCitation: Della Porta MG, Galli A, Bacigalupo A, Zibellini S, Bernardi M, Rizzo E, Allione B, van Lint MT, Pioltelli P, Marenco P, Bosi A, Voso MT, Sica S, Cuzzola M, Angelucci E, Rossi M, Ubezio M, Malovini A, Limongelli I, Ferretti VV, Spinelli O, Tresoldi C, Pozzi S, Luchetti S, Pezzetti L, Catricala S, Milanesi C, Riva A, Bruno B, Ciceri F, Bonifazi F, Bellazzi R, Papaemmanuil E, Santoro A, Alessandrino EP, Rambaldi A, Cazzola M. Clinical Effects of Driver Somatic Mutations on the Outcomes of Patients With Myelodysplastic Syndromes Treated With Allogeneic Hematopoietic Stem-Cell Transplantation. J Clin Oncol. 2016 Oct 20;34(30):3627-3637. doi: 10.1200/JCO.2016.67.3616.

ReferenceCitation: Gerstung M, Pellagatti A, Malcovati L, Giagounidis A, Porta MG, Jadersten M, Dolatshad H, Verma A, Cross NC, Vyas P, Killick S, Hellstrom-Lindberg E, Cazzola M, Papaemmanuil E, Campbell PJ, Boultwood J. Combining gene mutation with gene expression data improves outcome prediction in myelodysplastic syndromes. Nat Commun. 2015 Jan 9;6:5901. doi: 10.1038/ncomms6901.

ReferenceCitation: Gerstung M, Papaemmanuil E, Martincorena I, Bullinger L, Gaidzik VI, Paschka P, Heuser M, Thol F, Bolli N, Ganly P, Ganser A, McDermott U, Dohner K, Schlenk RF, Dohner H, Campbell PJ. Precision oncology for acute myeloid leukemia using a knowledge bank approach. Nat Genet. 2017 Mar;49(3):332-340. doi: 10.1038/ng.3756. Epub 2017 Jan 16.

ReferenceCitation: Schneider RK, Adema V, Heckl D, Jaras M, Mallo M, Lord AM, Chu LP, McConkey ME, Kramann R, Mullally A, Bejar R, Sole F, Ebert BL. Role of casein kinase 1A1 in the biology and targeted therapy of del(5q) MDS. Cancer Cell. 2014 Oct 13;26(4):509-20. doi: 10.1016/j.ccr.2014.08.001. Epub 2014 Sep 18.

ReferenceCitation: Platzbecker U, Germing U, Gotze KS, Kiewe P, Mayer K, Chromik J, Radsak M, Wolff T, Zhang X, Laadem A, Sherman ML, Attie KM, Giagounidis A. Luspatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes (PACE-MDS): a multicentre, open-label phase 2 dose-finding study with long-term extension study. Lancet Oncol. 2017 Oct;18(10):1338-1347. doi: 10.1016/S1470-2045(17)30615-0. Epub 2017 Sep 1. Erratum In: Lancet Oncol. 2017 Oct;18(10):e562.

ReferenceCitation: Platzbecker U, Hofbauer LC, Ehninger G, Holig K. The clinical, quality of life, and economic consequences of chronic anemia and transfusion support in patients with myelodysplastic syndromes. Leuk Res. 2012 May;36(5):525-36. doi: 10.1016/j.leukres.2012.01.006. Epub 2012 Feb 1.

ReferenceCitation: Platzbecker U, Schetelig J, Finke J, Trenschel R, Scott BL, Kobbe G, Schaefer-Eckart K, Bornhauser M, Itzykson R, Germing U, Beelen D, Ehninger G, Fenaux P, Deeg HJ, Ades L; German MDS Study; Cooperative Transplant Study Group; Fred Hutchinson Cancer Research Center; Groupe Francophone des Myelodysplasies. Allogeneic hematopoietic cell transplantation in patients age 60-70 years with de novo high-risk myelodysplastic syndrome or secondary acute myelogenous leukemia: comparison with patients lacking donors who received azacitidine. Biol Blood Marrow Transplant. 2012 Sep;18(9):1415-21. doi: 10.1016/j.bbmt.2012.05.003. Epub 2012 May 11.

ReferenceCitation: Park S, Hamel JF, Toma A, Kelaidi C, Thepot S, Campelo MD, Santini V, Sekeres MA, Balleari E, Kaivers J, Sapena R, Gotze K, Muller-Thomas C, Beyne-Rauzy O, Stamatoullas A, Kotsianidis I, Komrokji R, Steensma DP, Fensterl J, Roboz GJ, Bernal T, Ramos F, Calabuig M, Guerci-Bresler A, Bordessoule D, Cony-Makhoul P, Cheze S, Wattel E, Rose C, Vey N, Gioia D, Ferrero D, Gaidano G, Cametti G, Pane F, Sanna A, Germing U, Sanz GF, Dreyfus F, Fenaux P. Outcome of Lower-Risk Patients With Myelodysplastic Syndromes Without 5q Deletion After Failure of Erythropoiesis-Stimulating Agents. J Clin Oncol. 2017 May 10;35(14):1591-1597. doi: 10.1200/JCO.2016.71.3271. Epub 2017 Mar 28.

ReferenceCitation: Lamarque M, Raynaud S, Itzykson R, Thepot S, Quesnel B, Dreyfus F, Rauzy OB, Turlure P, Vey N, Recher C, Dartigeas C, Legros L, Delaunay J, Visanica S, Stamatoullas A, Fenaux P, Ades L. The revised IPSS is a powerful tool to evaluate the outcome of MDS patients treated with azacitidine: the GFM experience. Blood. 2012 Dec 13;120(25):5084-5. doi: 10.1182/blood-2012-09-453555. No abstract available. Erratum In: Blood. 2014 Jun 26;123(26):4152.

ReferenceCitation: Itzykson R, Thepot S, Quesnel B, Dreyfus F, Beyne-Rauzy O, Turlure P, Vey N, Recher C, Dartigeas C, Legros L, Delaunay J, Salanoubat C, Visanica S, Stamatoullas A, Isnard F, Marfaing-Koka A, de Botton S, Chelghoum Y, Taksin AL, Plantier I, Ame S, Boehrer S, Gardin C, Beach CL, Ades L, Fenaux P; Groupe Francophone des Myelodysplasies(GFM). Prognostic factors for response and overall survival in 282 patients with higher-risk myelodysplastic syndromes treated with azacitidine. Blood. 2011 Jan 13;117(2):403-11. doi: 10.1182/blood-2010-06-289280. Epub 2010 Oct 12.

ReferenceCitation: Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Gattermann N, Germing U, Sanz G, List AF, Gore S, Seymour JF, Dombret H, Backstrom J, Zimmerman L, McKenzie D, Beach CL, Silverman LR. Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia. J Clin Oncol. 2010 Feb 1;28(4):562-9. doi: 10.1200/JCO.2009.23.8329. Epub 2009 Dec 21.

ReferenceCitation: Mallo M, Del Rey M, Ibanez M, Calasanz MJ, Arenillas L, Larrayoz MJ, Pedro C, Jerez A, Maciejewski J, Costa D, Nomdedeu M, Diez-Campelo M, Lumbreras E, Gonzalez-Martinez T, Marugan I, Such E, Cervera J, Cigudosa JC, Alvarez S, Florensa L, Hernandez JM, Sole F. Response to lenalidomide in myelodysplastic syndromes with del(5q): influence of cytogenetics and mutations. Br J Haematol. 2013 Jul;162(1):74-86. doi: 10.1111/bjh.12354. Epub 2013 Apr 25.

ReferenceCitation: Mekinian A, Grignano E, Braun T, Decaux O, Liozon E, Costedoat-Chalumeau N, Kahn JE, Hamidou M, Park S, Puechal X, Toussirot E, Falgarone G, Launay D, Morel N, Trouiller S, Mathian A, Gombert B, Schoindre Y, Lioger B, De Wazieres B, Amoura Z, Buchdaul AL, Georgin-Lavialle S, Dion J, Madaule S, Raffray L, Cathebras P, Piette JC, Rose C, Ziza JM, Lortholary O, Montestruc F, Omouri M, Denis G, Rossignol J, Nimubona S, Ades L, Gardin C, Fenaux P, Fain O. Systemic inflammatory and autoimmune manifestations associated with myelodysplastic syndromes and chronic myelomonocytic leukaemia: a French multicentre retrospective study. Rheumatology (Oxford). 2016 Feb;55(2):291-300. doi: 10.1093/rheumatology/kev294. Epub 2015 Sep 8.

ReferenceCitation: Fraison JB, Mekinian A, Grignano E, Kahn JE, Arlet JB, Decaux O, Denis G, Buchdahl AL, Omouri M, Maigne G, Aouba A, Leon N, Berthier S, Liozon E, Park S, Gardin C, Lortholary O, Rossignol J, Fenaux P, Fain O, Braun T. Efficacy of Azacitidine in autoimmune and inflammatory disorders associated with myelodysplastic syndromes and chronic myelomonocytic leukemia. Leuk Res. 2016 Apr;43:13-7. doi: 10.1016/j.leukres.2016.02.005. Epub 2016 Feb 20.

ReferenceCitation: Khaznadar Z, Boissel N, Agaugue S, Henry G, Cheok M, Vignon M, Geromin D, Cayuela JM, Castaigne S, Pautas C, Raffoux E, Lachuer J, Sigaux F, Preudhomme C, Dombret H, Dulphy N, Toubert A. Defective NK Cells in Acute Myeloid Leukemia Patients at Diagnosis Are Associated with Blast Transcriptional Signatures of Immune Evasion. J Immunol. 2015 Sep 15;195(6):2580-90. doi: 10.4049/jimmunol.1500262. Epub 2015 Aug 5.

lead_sponsor: Istituto Clinico Humanitas

location_country: Germany Italy Spain France

location_facility: Josep Carreras Leukaemia Research Institute (IJC)

location_facility: Service d'hématologie séniors - Hôpital St Louis / Université Paris 7


title: A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study in Parkinson's Disease Patients With Moderate to Severe Dyskinesia to Assess the Efficacy and Safety/Tolerability of Two Dose Combinations of JM-010

acronym: ASTORIA

summary: The current study will explore the efficacy, safety and tolerability of 2 dose combinations of JM-010 to determine the optimal doses of each component to be studied in confirmatory clinical trials.

ReferenceCitation: McFarthing K, Prakash N, Simuni T. CLINICAL TRIAL HIGHLIGHTS - DYSKINESIA. J Parkinsons Dis. 2019;9(3):449-465. doi: 10.3233/JPD-199002. No abstract available.

evidence: open (via page says Open Access)

license: unspecified-oa

license_normalized: unspecified-oa

repository_institution: PubMed Central - Europe PMC

publisher_dissemination: IOS Press

lead_sponsor: Contera Pharma

sponsor_collaborators:
Contera Pharma
Bukwang Pharmaceutical

location_facility:
Contera Investigational site_DE
Contera Investigational site_IT
Contera Investigational site_FR
Contera Investigational site_ES