title: A Prospective, Multicentre European Registry for Newly Diagnosed Patients With Myelodysplastic Syndromes (MDS), Including Acute Myeloid Leukaemia With 20-<30 Percent Marrow Blasts (Former RAEB-t), and Chronic Myelomonocytic Leukaemia (CMML)

acronym: EUMDS

summary:
Study Objectives:

To collect and describe demographics, disease-management, and treatment outcomes of Myelodysplastic Syndromes (MDS) patients who are newly diagnosed and classified according to the World Health Organization (WHO) criteria.

To perform observational studies concerning relevant scientific research questions in MDS using clinical data and biological samples, and to present relevant research outcomes in the fields of diagnosis and prognostication, health related quality of life issues, health economics, and risk stratification for newly developed classes of drugs.

To disseminate results of the studies to all stakeholders involved.

ReferenceCitation: de Swart L, Crouch S, Hoeks M, Smith A, Langemeijer S, Fenaux P, Symeonidis A, Cermâk J, Hellström-Lindberg E, Stauder R, Sanz G, Mittelman M, Holm MS, Malcovati L, Mądry K, Germing U, Tatic A, Savic A, Almeida AM, Gredelj-Simec N, Guerci-Bresler A, Beyne-Rauzy O, Culligan D, Kotsianidis I, Itzykson R, van Marrewijk C, Blijlevens N, Bowen D, de Witte T; EUMDS Registry Participants. Impact of red blood cell transfusion dose density on progression-free survival in patients with lower-risk myelodysplastic syndromes. Haematologica. 2020 Mar;105(3):632-639. doi: 10.3324/haematol.2018.212217. Epub 2019 Jun 6.

ReferenceCitation: Itzykson R, Crouch S, Travaglino E, Smith A, Symeonidis A, Hellström-Lindberg E, Sanz G, Čermák J, Stauder R, Elena C, Germing U, Mittelman M, Langemeijer S, Mądry K, Tatic A, Holm MS, Almeida AM, Savic A, Šimec NG, Luño E, Culligan D, Guerci-Bresler A, Malcovati L, van Marrewijk C, Bowen D, de Witte T, Fenaux P; European MDS Registry members. Early platelet count kinetics has prognostic value in lower-risk myelodysplastic syndromes. Blood Adv. 2018 Aug 28;2(16):2079-2089. doi: 10.1182/bloodadvances.2018020495.

ReferenceCitation: de Swart L, Reiniers C, Bagguley T, van Marrewijk C, Bowen D, Hellström-Lindberg E, Tatic A, Symeonidis A, Huls G, Cermak J, van de Loosdrecht AA, Garelius H, Culligan D, Macheta M, Spanoudakis M, Panagiotidis P, Krejci M, Blijlevens N, Langemeijer S, Droste J, Swinkels DW, Smith A, de Witte T; EUMDS Steering Committee. Labile plasma iron levels predict survival in patients with lower-risk myelodysplastic syndromes. Haematologica. 2018 Jan;103(1):69-79. doi: 10.3324/haematol.2017.171884. Epub 2017 Nov 9.

ReferenceCitation: Garelius HK, Johnston WT, Smith AG, Park S, de Swart L, Fenaux P, Symeonidis A, Sanz G, Čermák J, Stauder R, Malcovati L, Mittelman M, van de Loosdrecht AA, van Marrewijk CJ, Bowen D, Crouch S, de Witte TJ, Hellström-Lindberg E. Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome. J Intern Med. 2017 Mar;281(3):284-299. doi: 10.1111/joim.12579. Epub 2016 Dec 7.

ipd_sharing_description: The study results (aggregated data) will be published in an academic journal and presented at scientific meetings. We are working on an update of the website: www.eumds.org. EUMDS related activities and results are also shared via www.mds-europe.eu.

lead_sponsor: Radboud University Medical Center

location_country:
Denmark
Croatia
Poland
Portugal
Serbia
Spain
Greece
Austria
Italy
Netherlands
United Kingdom
Switzerland
Sweden
France
Israel
Romania
Germany
Czechia

location_facility: Radboud University Nijmegen Medical Centre, dept of Hematology

location_facility: Instituto Português de Oncologia de Lisboa,

location_facility: University of Pavia Medical SChool, Dept of Hematology

location_facility: Fundeni CLinical Institute, Clinic of Hematology

location_facility: University of Innsbruck Fachartz fur Innere Medizin, Dept of Hematology

location_facility: Institute of Haematology and Blood Transfusion U nemocnice

location_facility: Karolinsky Institute Huddinge University Hospital, Dept of Medicine Division HAematology

location_facility: Hopital Avicenne Universite Paris, Dept of Hematology

location_facility: Tel-Aviv Sourasky Medical Center, Dept. of Medicine A

location_facility: Medical University, Dept of Hematology, Oncology and Internal Medicine

location_facility: Medizinische Klinik und Poliklinik fur Haematologie, Onkologie und Klinische Immunologie

location_facility: University of Patras Medical School, Haematology Division, Dept of Internal Medicine

location_facility: Leeds General Infirmary Dept of Hematology


status: Completed

primary_purpose: Treatment

intervention_type: Drug

title: International Randomized Study to Evaluate the Addition of Docetaxel to the Combination of Cisplatin-5-fluorouracil (TCF) vs. Cisplatin-5-fluorouracil (CF) in the Induction Treatment of Nasopharyngeal Carcinoma (NPC) in Children and Adolescents

study_completion_date_type: Actual

lead_sponsor: Sanofi

type: EudraCT Number

source: euctr

design_allocation: Randomized

study_type: Interventional

enrollment_type: Actual

location_country:
Brazil
Italy
Thailand
Indonesia
Tunisia
Algeria
India
France
China
Korea, Republic of
Mexico
Philippines
Turkey
Morocco

location_facility:
Investigational Site Number 156005
Investigational Site Number 764001
Investigational Site Number 410002
Investigational Site Number 608002
Investigational Site Number 380001
Investigational Site Number 410001
Investigational Site Number 504002
Investigational Site Number 360001
Investigational Site Number 076002
Investigational Site Number 788002
Investigational Site Number 484001
Investigational Site Number 764002
Investigational Site Number 504003
Investigational Site Number 792002
Investigational Site Number 788003
Investigational Site Number 410003
Investigational Site Number 356004
Investigational Site Number 792001
Investigational Site Number 356003
Investigational Site Number 356002
Investigational Site Number 012001
Investigational Site Number 076001
Investigational Site Number 792003
Investigational Site Number 250001
Investigational Site Number 504001
Investigational Site Number 356001

summary:
The primary objective is to estimate the Complete Response rate of docetaxel to the combination of cisplatin-5-fluorouracil (TCF) compared to cisplatin-5-fluorouracil (CF) in the Induction treatment of Nasopharyngeal Carcinoma (NPC).

The secondary objectives are to determine:

the safety of TCF in comparison to CF after induction treatment of NPC,
the pharmacokinetics of docetaxel when added to CF,
the Overall Response rate of TCF and CF on completion of induction and consolidation (chemo-radiotherapy) treatment of NPC, and to compare overall survival between TCF and CF.

lead_sponsor_type: industriel

status_simplified: Completed


acronym: ALFACHIN

title: 12-week, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel-group Study to Investigate the Efficacy, Pharmacodynamic and Safety of 2 Doses of Alfuzosin (0.1 mg/kg/Day, 0.2 mg/kg/Day) in the Treatment of Children and Adolescents 2-16 Years With Elevated Detrusor Leak Point Pressure of Neuropathic Etiology Followed by a 40-week Open-label Extension

location_country:
Poland
Taiwan
Portugal
Serbia
Spain
Russian Federation
Estonia
Canada
United States
France
India
Malaysia
Singapore
Philippines
Germany
Turkey
Bulgaria
Slovakia

location_facility:
Sanofi-Aventis Administrative Office
Sanofi-Aventis Aministrative Office

summary:
The primary objective of the study was to evaluate the efficacy of Alfuzosin in comparison to Placebo on the detrusor Leak Point Pressure (LPP) in children and adolescents 2-16 years of age with elevated detrusor LPP of neuropathic etiology and detrusor LPP ≥ 40 cm H2O.

Secondary objectives were:

To investigate the safety and tolerability of two doses of Alfuzosin in comparison to Placebo in children and adolescents,

To evaluate the effects of the two doses of Alfuzosin in comparison to Placebo on:

Detrusor compliance,
Urinary tract infection,
To investigate the pharmacokinetics of Alfuzosin (population kinetics),
To evaluate the 12-month long-term safety of Alfuzosin 0.1 mg/kg/day and 0.2 mg/kg/day.

The study consisted of 2 periods:

a 12-week double blind treatment period where patients were to receive either Alfuzosin 0.1 mg/kg/day or Alfuzosin 0.2 mg/kg/day or placebo then,
a 40-week open label extension treatment period where patients were to receive either Alfuzosin 0.1 mg/kg/day or Alfuzosin 0.2 mg/kg/day.


title: Efficacy and Safety of 2 mg/Day M100907 on Sleep Maintenance Insomnia: a 6-week, Multicenter, Randomized, Double-blind, Placebo-controlled Polysomnographic Study

summary: The purpose of this study is to assess efficacy and safety of volinanserin in the population of patients complaining of sleep maintenance insomnia. The patients suffering from that condition frequently wake up during the night, their sleep is non restorative and they suffer from a significant distress or impairment in their daily activities consecutive to insomnia.

location_country:
Austria
Australia
Russian Federation
Canada
United States
France
Germany


title: Treatment Protocol for Relapsed Anaplastic Large Cell Lymphoma of Childhood and Adolescence

summary:
RATIONALE: Giving combination chemotherapy and total-body irradiation before a peripheral stem cell transplant that uses the patient's or a donor's stem cells, helps stop both the growth of cancer cells and the patient's immune system from rejecting the stem cells. When the stem cells are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Giving combination chemotherapy and total-body irradiation followed by a stem cell transplant may be an effective treatment for anaplastic large cell lymphoma.

PURPOSE: This clinical trial is studying how well combination chemotherapy followed by stem cell transplant works in treating young patients with progressive or relapsed anaplastic large cell lymphoma.

status: Unknown status

design_allocation: Non-Randomized

enrollment_type: Anticipated

ReferenceType: derived

ReferenceCitation: Knörr F, Brugières L, Pillon M, Zimmermann M, Ruf S, Attarbaschi A, Mellgren K, Burke GAA, Uyttebroeck A, Wróbel G, Beishuizen A, Aladjidi N, Reiter A, Woessmann W; European Inter-Group for Childhood Non-Hodgkin Lymphoma. Stem Cell Transplantation and Vinblastine Monotherapy for Relapsed Pediatric Anaplastic Large Cell Lymphoma: Results of the International, Prospective ALCL-Relapse Trial. J Clin Oncol. 2020 Dec 1;38(34):3999-4009. doi: 10.1200/JCO.20.00157. Epub 2020 Jul 30.

lead_sponsor: European Inter-group Cooperation on Childhood and Adolescent Non Hodgkin Lymphoma

location_country:
Poland
United Kingdom
Ireland
Austria
Italy
Netherlands
Czech Republic
Switzerland
Sweden
France
Belgium
Germany

location_facility: Great Ormond Street Hospital for Children

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Universitaetskinderklinik - Universitaetsklinikum Freiburg

location_facility: Helios Kliniken Wuppertal University Hospital

location_facility: Charite University Hospital - Campus Virchow Klinikum

location_facility: Our Lady's Hospital for Sick Children Crumlin

location_facility: Comprehensive Cancer Center Ulm at Universitaetsklinikum Ulm

location_facility: University Hospital Schleswig-Holstein - Kiel Campus

location_facility: Klinik und Poliklinik fuer Kinder und Jugendmedizin - Universitaetsklinikum Muenster

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Sir James Spence Institute of Child Health at Royal Victoria Infirmary

location_facility: Kinderklinik - Universitaetsklinikum Aachen

location_facility: Kinderklinik - Universitaetsklinikum Rostock

location_facility: Kliniken der Stadt Koeln gGmbH - Kinderkrankenhaus Riehl

location_facility: Klinik und Poliklinik Fuer Kinder-und Jugendmedizin - Universitaetsklinikum Greifswald

location_facility: University Medical Center Hamburg - Eppendorf

location_facility: Kinderklink Siegen Deutsches Rotes Kreuz

location_facility: Krankenanstalt Mutterhaus der Borromaerinnen

location_facility: Institute of Child Health at University of Bristol

location_facility: Dr. von Haunersches Kinderspital der Universitaet Muenchen

location_facility: Leeds Cancer Centre at St. James's University Hospital

intervention_type: Biological

status_simplified: Unknown


title: A Randomized, Double Blind, Two-arm Placebo Controlled, 12-Month Study of the Effects of Rimonabant 20mg Once Daily on the Amount and the Activity of Visceral Fat in Abdominally Obese Patients With Metabolic Syndrome.

acronym: VICTORIA

summary:
Primary objective:

To assess the effect of rimonabant on visceral fat area over a period of 12 months when prescribed with a mild hypocaloric diet in abdominally obese patients with metabolic syndrome

Secondary objectives:

To assess the effect of rimonabant over a period of 12 months on:

Liver fat content using CT scan (Computed Tomography scan)
Anthropometric measures (weight, waist circumference, body composition using Dual Energy X-ray Absorptiometry (DEXA))
Lipid, lipoprotein profile
Glycemia, insulinemia and HbA1c
Adipokines, inflammatory and hemostatic markers
To evaluate the percentage of patients with metabolic syndrome at 12 months
To evaluate the safety and tolerability of rimonabant in these patients

In four selected US sites the effect of rimonabant at 12 months will be also assessed on:

Basal lipolysis and insulin suppressed lipolysis (euglycemic hyperinsulinemic clamp).
Resting metabolic rate and substrate oxidation at rest using indirect calorimetry.
Adipose tissue histology and expression of genes involved in glucose and lipid metabolism (superficial adipose tissue biopsy).

ReferenceType: result

ReferenceCitation: Triay J, Mundi M, Klein S, Toledo FG, Smith SR, Abu-Lebdeh H, Jensen M. Does rimonabant independently affect free fatty acid and glucose metabolism? J Clin Endocrinol Metab. 2012 Mar;97(3):819-27. doi: 10.1210/jc.2011-2486. Epub 2011 Dec 14.

location_country:
Denmark
Spain
Italy
United Kingdom
Finland
Canada
United States
France
Sweden

intervention_type: Other


title: A Double Blind, Randomized, Phase II-III Maintenance Study of SR48692 Versus Placebo in Patients With Extensive Stage Small Cell Lung Cancer Following a First Line Chemotherapy With Cisplatin + Etoposide

acronym: SESAME

summary:
To demonstrate an increase in overall survival for patients with newly diagnosed extended stage small cell lung cancer when treated with SR48692 versus placebo, after an initial response (complete or partial response or stable) to first line cisplatin plus etoposide.

Primary objective: comparison of overall survival between patients in the control arm and the meclinertant arm.

Secondary objectives: comparison of the progression free survival, the time to progression, the clinical benefit, the quality of life, the toxicity and safety between patients in the control arm and the meclinertant arm.

location_country:
Poland
Brazil
Spain
Italy
Russian Federation
Australia
Mexico
Argentina
Netherlands
United Kingdom
Hungary
France
Belgium
Germany

location_facility:
Sanofi-Aventis Administrative Office
Sanofi-aventis adminsitrative office


summary: The purpose of this study was to determine the safety and effectiveness of different doses of botulinum toxin Type A in treating lower urinary tract symptoms due to benign prostatic hyperplasia.

ReferenceCitation: Marberger M, Chartier-Kastler E, Egerdie B, Lee KS, Grosse J, Bugarin D, Zhou J, Patel A, Haag-Molkenteller C. A randomized double-blind placebo-controlled phase 2 dose-ranging study of onabotulinumtoxinA in men with benign prostatic hyperplasia. Eur Urol. 2013 Mar;63(3):496-503. doi: 10.1016/j.eururo.2012.10.005. Epub 2012 Oct 12.

lead_sponsor: Allergan

location_country:
Taiwan
United Kingdom
Italy
Austria
Australia
Canada
Czech Republic
Korea, Republic of
France
Germany
Slovakia


title: Cooperative Multicenter Study for Children and Adolescents With Low Grade Glioma

summary:
RATIONALE: Radiation therapy uses high-energy x-rays to kill tumor cells. Drugs used in chemotherapy, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This clinical trial is studying giving radiation therapy or combination chemotherapy to see how well it works in treating patients with clinically or radiologically progressive low-grade gliomas.

study_start_date_type: Actual

ReferenceCitation: Gnekow AK, Walker DA, Kandels D, Picton S, Giorgio Perilongo, Grill J, Stokland T, Sandstrom PE, Warmuth-Metz M, Pietsch T, Giangaspero F, Schmidt R, Faldum A, Kilmartin D, De Paoli A, De Salvo GL; of the Low Grade Glioma Consortium and the participating centers. A European randomised controlled trial of the addition of etoposide to standard vincristine and carboplatin induction as part of an 18-month treatment programme for childhood (≤16 years) low grade glioma - A final report. Eur J Cancer. 2017 Aug;81:206-225. doi: 10.1016/j.ejca.2017.04.019. Epub 2017 Jun 22. Erratum in: Eur J Cancer. 2017 Dec 13;:.

ReferenceCitation: Kandels D, Pietsch T, Bison B, Warmuth-Metz M, Thomale UW, Kortmann RD, Timmermann B, Hernáiz Driever P, Witt O, Schmidt R, Gnekow AK. Loss of efficacy of subsequent nonsurgical therapy after primary treatment failure in pediatric low-grade glioma patients-Report from the German SIOP-LGG 2004 cohort. Int J Cancer. 2020 Dec 15;147(12):3471-3489. doi: 10.1002/ijc.33170. Epub 2020 Jul 11. Erratum in: Int J Cancer. 2021 Jul 15;149(2):E3-E4.

lead_sponsor: Societe Internationale d'Oncologie Pediatrique

location_country:
Denmark
Croatia
Poland
Portugal
United Kingdom
Italy
Austria
Switzerland
France
Belgium
Norway
Germany

location_facility: Allg. Krankenhaus der Stadt Wien Universitaets-Kinderklinik

intervention_type: Radiation


title: Multicenter International Study of Oxaliplatin/ 5FU-LV in the Adjuvant Treatment of Colon Cancer

summary:
To evaluate the FOLFOX regimen versus LV5FU2 in the adjuvant treatment of stage II and III colon cancer.

Primary objective: Disease Free Survival (DFS) Secondary objective: Overall Survival (OS), safety (including long term toxicity)

ReferenceCitation: André T, Boni C, Mounedji-Boudiaf L, Navarro M, Tabernero J, Hickish T, Topham C, Zaninelli M, Clingan P, Bridgewater J, Tabah-Fisch I, de Gramont A; Multicenter International Study of Oxaliplatin/5-Fluorouracil/Leucovorin in the Adjuvant Treatment of Colon Cancer (MOSAIC) Investigators. Oxaliplatin, fluorouracil, and leucovorin as adjuvant treatment for colon cancer. N Engl J Med. 2004 Jun 3;350(23):2343-51.

ReferenceCitation: Gallois C, Shi Q, Meyers JP, Iveson T, Alberts SR, de Gramont A, Sobrero AF, Haller DG, Oki E, Shields AF, Goldberg RM, Kerr R, Lonardi S, Yothers G, Kelly C, Boukovinas I, Labianca R, Sinicrope FA, Souglakos I, Yoshino T, Meyerhardt JA, Andre T, Papamichael D, Taieb J. Prognostic Impact of Early Treatment and Oxaliplatin Discontinuation in Patients With Stage III Colon Cancer: An ACCENT/IDEA Pooled Analysis of 11 Adjuvant Trials. J Clin Oncol. 2022 Oct 28:JCO2102726. doi: 10.1200/JCO.21.02726. Online ahead of print.

ReferenceCitation: Webster-Clark M, Keil AP, Robert N, Frytak JR, Boyd M, Stürmer T, Sanoff H, Westreich D, Lund JL. Comparing Trial and Real-world Adjuvant Oxaliplatin Delivery in Patients With Stage III Colon Cancer Using a Longitudinal Cumulative Dose. JAMA Oncol. 2022 Oct 13. doi: 10.1001/jamaoncol.2022.4445. [Epub ahead of print]

ReferenceCitation: Cohen R, Taieb J, Fiskum J, Yothers G, Goldberg R, Yoshino T, Alberts S, Allegra C, de Gramont A, Seitz JF, O'Connell M, Haller D, Wolmark N, Erlichman C, Zaniboni A, Lonardi S, Kerr R, Grothey A, Sinicrope FA, André T, Shi Q. Microsatellite Instability in Patients With Stage III Colon Cancer Receiving Fluoropyrimidine With or Without Oxaliplatin: An ACCENT Pooled Analysis of 12 Adjuvant Trials. J Clin Oncol. 2021 Feb 20;39(6):642-651. doi: 10.1200/JCO.20.01600. Epub 2020 Dec 23.

ReferenceCitation: Salem ME, Yin J, Goldberg RM, Pederson LD, Wolmark N, Alberts SR, Taieb J, Marshall JL, Lonardi S, Yoshino T, Kerr RS, Yothers G, Grothey A, Andre T, De Gramont A, Shi Q. Evaluation of the change of outcomes over a 10-year period in patients with stage III colon cancer: pooled analysis of 6501 patients treated with fluorouracil, leucovorin, and oxaliplatin in the ACCENT database. Ann Oncol. 2020 Apr;31(4):480-486. doi: 10.1016/j.annonc.2019.12.007. Epub 2020 Jan 16.

ReferenceCitation: Auclin E, André T, Taieb J, Banzi M, Van Laethem JL, Tabernero J, Hickish T, de Gramont A, Vernerey D. Association of post-operative CEA with survival and oxaliplatin benefit in patients with stage II colon cancer: a post hoc analysis of the MOSAIC trial. Br J Cancer. 2019 Aug;121(4):312-317. doi: 10.1038/s41416-019-0521-7. Epub 2019 Jul 12.

ReferenceCitation: André T, de Gramont A, Vernerey D, Chibaudel B, Bonnetain F, Tijeras-Raballand A, Scriva A, Hickish T, Tabernero J, Van Laethem JL, Banzi M, Maartense E, Shmueli E, Carlsson GU, Scheithauer W, Papamichael D, Möehler M, Landolfi S, Demetter P, Colote S, Tournigand C, Louvet C, Duval A, Fléjou JF, de Gramont A. Adjuvant Fluorouracil, Leucovorin, and Oxaliplatin in Stage II to III Colon Cancer: Updated 10-Year Survival and Outcomes According to BRAF Mutation and Mismatch Repair Status of the MOSAIC Study. J Clin Oncol. 2015 Dec 10;33(35):4176-87. doi: 10.1200/JCO.2015.63.4238. Epub 2015 Nov 2.

ReferenceCitation: Tournigand C, André T, Bonnetain F, Chibaudel B, Lledo G, Hickish T, Tabernero J, Boni C, Bachet JB, Teixeira L, de Gramont A. Adjuvant therapy with fluorouracil and oxaliplatin in stage II and elderly patients (between ages 70 and 75 years) with colon cancer: subgroup analyses of the Multicenter International Study of Oxaliplatin, Fluorouracil, and Leucovorin in the Adjuvant Treatment of Colon Cancer trial. J Clin Oncol. 2012 Sep 20;30(27):3353-60. doi: 10.1200/JCO.2012.42.5645. Epub 2012 Aug 20.

location_country:
Denmark
Poland
Portugal
Spain
Greece
Austria
Australia
Italy
Netherlands
United Kingdom
Switzerland
Sweden
Hungary
France
Israel
Singapore
Belgium
Norway
Germany

location_facility: sanofi-aventis Australia & New Zealand administrative office


title: EURopean Trial In Atrial Fibrillation or Flutter Patients Receiving Dronedarone for the maIntenance of Sinus Rhythm (EURIDIS)

acronym: EURIDIS

summary:
To assess the efficacy of dronedarone versus placebo for the maintenance of normal sinus rhythm after electrical, pharmacological or spontaneous conversion of atrial fibrillation/atrial flutter (AF/AFL).

To assess the efficacy of dronedarone versus placebo on AF/AFL-related symptoms.

To assess the efficacy of dronedarone versus placebo on ventricular rate control in case of AF/AFL recurrence.

ReferenceCitation: Singh BN, Connolly SJ, Crijns HJ, Roy D, Kowey PR, Capucci A, Radzik D, Aliot EM, Hohnloser SH; EURIDIS and ADONIS Investigators. Dronedarone for maintenance of sinus rhythm in atrial fibrillation or flutter. N Engl J Med. 2007 Sep 6;357(10):987-99.

ReferenceCitation: Handelsman Y, Bunch TJ, Rodbard HW, Steinberg BA, Thind M, Bigot G, Konigsberg L, Wieloch M, Kowey PR. Impact of dronedarone on patients with atrial fibrillation and diabetes: A sub-analysis of the ATHENA and EURIDIS/ADONIS studies. J Diabetes Complications. 2022 Jul;36(7):108227. doi: 10.1016/j.jdiacomp.2022.108227. Epub 2022 Jun 8.

location_country:
Denmark
Poland
Spain
Italy
United Kingdom
Netherlands
Finland
Czech Republic
Hungary
France
Belgium
Germany

location_facility:
Sanofi-Aventis Administrative Office
Sanofi- Aventis Administrative Office


title: A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children Treated With SAIZEN®

summary: The study aims at identifying the predictive markers after one month of Saizen therapy in Growth Hormone Deficiency (GHD) and Turner Syndrome children.

ReferenceCitation: Stevens A, Clayton P, Tatò L, Yoo HW, Rodriguez-Arnao MD, Skorodok J, Ambler GR, Zignani M, Zieschang J, Della Corte G, Destenaves B, Champigneulle A, Raelson J, Chatelain P. Pharmacogenomics of insulin-like growth factor-I generation during GH treatment in children with GH deficiency or Turner syndrome. Pharmacogenomics J. 2014 Feb;14(1):54-62. doi: 10.1038/tpj.2013.14. Epub 2013 Apr 9.

ReferenceCitation: Stevens A, Murray P, De Leonibus C, Garner T, Koledova E, Ambler G, Kapelari K, Binder G, Maghnie M, Zucchini S, Bashnina E, Skorodok J, Yeste D, Belgorosky A, Siguero JL, Coutant R, Vangsøy-Hansen E, Hagenäs L, Dahlgren J, Deal C, Chatelain P, Clayton P. Gene expression signatures predict response to therapy with growth hormone. Pharmacogenomics J. 2021 Oct;21(5):594-607. doi: 10.1038/s41397-021-00237-5. Epub 2021 May 27.

ReferenceCitation: Murray PG, Stevens A, De Leonibus C, Koledova E, Chatelain P, Clayton PE. Transcriptomics and machine learning predict diagnosis and severity of growth hormone deficiency. JCI Insight. 2018 Apr 5;3(7). pii: 93247. doi: 10.1172/jci.insight.93247. eCollection 2018 Apr 5.

ReferenceCitation: Valsesia A, Chatelain P, Stevens A, Peterkova VA, Belgorosky A, Maghnie M, Antoniazzi F, Koledova E, Wojcik J, Farmer P, Destenaves B, Clayton P; PREDICT Investigator group. GH deficiency status combined with GH receptor polymorphism affects response to GH in children. Eur J Endocrinol. 2015 Dec;173(6):777-89. doi: 10.1530/EJE-15-0474. Epub 2015 Sep 4.

lead_sponsor: Merck KGaA, Darmstadt, Germany

location_country:
Spain
Italy
Austria
Australia
Russian Federation
Argentina
Canada
United Kingdom
Sweden
France
Singapore
Norway
Germany

location_facility:
Local Medical Information Office
Local Medical InformationOffice


title: An Eight-Week, Multicenter, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy, Safety, and Tolerability of One Fixed 100 mg Dose of Saredutant in Patients With Major Depressive Disorder

summary:
The purpose of the study is to evaluate the efficacy and safety of saredutant (or SR48968C) in patients with depression.

The primary objective is to evaluate the efficacy of a 100 mg dose of saredutant compared to placebo in patients with depression.

The secondary objectives are to evaluate the safety of saredutant, to evaluate the efficacy of saredutant on disability and quality of life in patients with depression, and to evaluate blood levels of saredutant.

location_country:
Croatia
Portugal
Canada
Czech Republic
France
Chile
Mexico
Germany


title: An Open Label, Randomized, Parallel Design Study to Investigate the Efficacy and Safety of Sevelamer Hydrochloride (Renagel®) Compared With Calcium Acetate in Peritoneal Dialysis Patients

summary: The purpose of this study is to demonstrate that sevelamer hydrochloride is non-inferior to calcium acetate for the treatment of hyperphosphataemia in patients receiving peritoneal dialysis.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Denmark
Spain
Italy
United Kingdom
France
Belgium
Netherlands

location_facility: The Churchill Hospital, Oxford Radcliffe Hospitals NHS Trust

location_facility: University Hospital ,Queen Elizabeth Hospital


title: A Double-blind, Randomized Study of Clopidogrel 75 mg/d vs Placebo, on a Background of ASA 75-100 mg/d,in Peripheral Arterial Disease (PAD) Patients Receiving a Unilateral Below Knee Bypass Graft.

summary:
Primary objective:

To evaluate whether clopidogrel 75 mg o.d. versus placebo (on a background of ASA 75-100 mg/d) will lead to an increased rate of primary patency, limb salvage and survival, in patients receiving a below knee bypass graft for the treatment of PAD.

Secondary objectives:

Comparison, between the two treatment groups, of :

Primary patency,
Assisted primary patency,
Cardiovascular death / myocardial infarction / stroke / any amputation above the ankle.
Ankle Brachial Pressure Index (ABPI) changes from baseline

ReferenceCitation: Belch JJ, Dormandy J; CASPAR Writing Committee, Biasi GM, Cairols M, Diehm C, Eikelboom B, Golledge J, Jawien A, Lepäntalo M, Norgren L, Hiatt WR, Becquemin JP, Bergqvist D, Clement D, Baumgartner I, Minar E, Stonebridge P, Vermassen F, Matyas L, Leizorovicz A. Results of the randomized, placebo-controlled clopidogrel and acetylsalicylic acid in bypass surgery for peripheral arterial disease (CASPAR) trial. J Vasc Surg. 2010 Oct;52(4):825-33, 833.e1-2. doi: 10.1016/j.jvs.2010.04.027. Epub 2010 Aug 1. Erratum in: J Vasc Surg. 2011 Feb;53(2):564. Biasi, B M [corrected to Biasi, G M].

location_country:
Poland
Spain
Italy
Austria
Australia
Netherlands
Finland
United Kingdom
Switzerland
Sweden
Hungary
France
Belgium
Germany


summary: This study will evaluate the safety and efficacy of an intravitreal implant of dexamethasone for the treatment of diabetic macular edema.

ReferenceCitation: Rittiphairoj T, Mir TA, Li T, Virgili G. Intravitreal steroids for macular edema in diabetes. Cochrane Database Syst Rev. 2020 Nov 17;11:CD005656. doi: 10.1002/14651858.CD005656.pub3.

ReferenceCitation: Yoon YH, Boyer DS, Maturi RK, Bandello F, Belfort R Jr, Augustin AJ, Li XY, Bai Z, Hashad Y; Ozurdex MEAD Study Group. Natural history of diabetic macular edema and factors predicting outcomes in sham-treated patients (MEAD study). Graefes Arch Clin Exp Ophthalmol. 2019 Dec;257(12):2639-2653. doi: 10.1007/s00417-019-04464-2. Epub 2019 Oct 25.

ReferenceCitation: Danis RP, Sadda S, Li XY, Cui H, Hashad Y, Whitcup SM. Anatomical effects of dexamethasone intravitreal implant in diabetic macular oedema: a pooled analysis of 3-year phase III trials. Br J Ophthalmol. 2016 Jun;100(6):796-801. doi: 10.1136/bjophthalmol-2015-306823. Epub 2015 Nov 18.

ReferenceCitation: Augustin AJ, Kuppermann BD, Lanzetta P, Loewenstein A, Li XY, Cui H, Hashad Y, Whitcup SM; Ozurdex MEAD Study Group. Dexamethasone intravitreal implant in previously treated patients with diabetic macular edema: subgroup analysis of the MEAD study. BMC Ophthalmol. 2015 Oct 30;15:150. doi: 10.1186/s12886-015-0148-2.

location_country:
Poland
Taiwan
Brazil
United Kingdom
Italy
New Zealand
Canada
Hungary
Korea, Republic of
France
United States
India
Colombia
Singapore


title: An Open-label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe Disease

summary: Pompe disease (also known as glycogen storage disease type II, "GSD-II") is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied.

ReferenceCitation: Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, Bali D, Smith SA, Li JS, Mandel H, Koeberl D, Rosenberg A, Chen YT. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab. 2010 Jan;99(1):26-33. doi: 10.1016/j.ymgme.2009.08.003.

location_country:
Taiwan
United Kingdom
United States
Israel
France

location_facility: University of Florida College of Medicine


type: Registry Identifier

title: A Randomized Trial of Adjuvant Treatment With Radiation Plus Chemotherapy Versus Radiation Alone in High Risk Endometrial Carcinoma

summary:
RATIONALE: Radiation therapy uses high-energy x-rays to damage tumor cells. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known whether radiation therapy with chemotherapy is more effective than radiation therapy alone in treating high-risk endometrial cancer.

PURPOSE: This randomized phase III trial is studying radiation therapy and chemotherapy to see how well they work compared to radiation therapy alone in treating patients with high-risk endometrial cancer.

ReferenceType: background

ReferenceCitation: Hogberg T, Signorelli M, de Oliveira CF, Fossati R, Lissoni AA, Sorbe B, Andersson H, Grenman S, Lundgren C, Rosenberg P, Boman K, Tholander B, Scambia G, Reed N, Cormio G, Tognon G, Clarke J, Sawicki T, Zola P, Kristensen G. Sequential adjuvant chemotherapy and radiotherapy in endometrial cancer--results from two randomised studies. Eur J Cancer. 2010 Sep;46(13):2422-31. doi: 10.1016/j.ejca.2010.06.002. Epub 2010 Jul 7.

ReferenceCitation: Hogberg T, Rosenberg P, Kristensen G, et al.: A randomized phase-III study on adjuvant treatment with radiation (RT) ± chemotherapy (CT) in early-stage high-risk endometrial cancer (NSGO-EC-9501/EORTC 55991). [Abstract] J Clin Oncol 25 (Suppl 18): A-5503, 274s, 2007.

lead_sponsor: Nordic Society of Gynaecological Oncology - Clinical Trials Unit

location_country:
Poland
Portugal
Spain
Ireland
Italy
United Kingdom
France
South Africa
Belgium
Norway
Netherlands

location_facility: Centre for Cancer Research and Cell Biology at Belfast City Hospital

location_facility: Hospitais da Universidade de Coimbra (HUC)

location_facility: Universitair Medisch Centrum St. Radboud - Nijmegen

location_facility: Hospital Universitario Central de Asturias

location_facility: Fondazione I.R.C.C.S. Policlinico San Matteo

location_facility: Cazk Groeninghe - Campus Maria's Voorzienigheid

location_facility: Istituto Nazionale per lo Studio e la Cura dei Tumori


title: A Randomised Trial in Relapsed Ovarian Cancer: Early Treatment Based on CA 125 Levels Alone Vs. Delayed Treatment Based On Conventional Clinical Indicators

summary:
RATIONALE: It is not yet known if treatment for recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer is more effective if it is begun when blood levels of CA 125 become elevated rather than waiting for other indicators of disease recurrence.

PURPOSE: This randomized phase III trial is studying early chemotherapy based on blood levels of CA 125 alone to see how well it works compared to chemotherapy based on conventional clinical indicators in patients with recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer.

ReferenceCitation: Markman M, Petersen J, Belland A, Burg K. CA-125 monitoring in ovarian cancer: patient survey responses to the results of the MRC/EORTC CA-125 Surveillance Trial. Oncology. 2010;78(1):1-2. doi: 10.1159/000292352. Epub 2010 Mar 6.

ReferenceCitation: Rustin GJ, van der Burg ME, Griffin CL, Guthrie D, Lamont A, Jayson GC, Kristensen G, Mediola C, Coens C, Qian W, Parmar MK, Swart AM; MRC OV05; EORTC 55955 investigators. Early versus delayed treatment of relapsed ovarian cancer (MRC OV05/EORTC 55955): a randomised trial. Lancet. 2010 Oct 2;376(9747):1155-63. doi: 10.1016/S0140-6736(10)61268-8.

ReferenceCitation: Rustin GJ, van der Burg ME: A randomized trial in ovarian cancer (OC) of early treatment of relapse based on CA125 level alone versus delayed treatment based on conventional clinical indicators (MRC OV05/EORTC 55955 trials). [Abstract] J Clin Oncol 27 (Suppl 18): A-1, 2009.

lead_sponsor: Medical Research Council

location_country:
Portugal
Spain
Ireland
Austria
Italy
United Kingdom
France
South Africa
Belgium
Netherlands

location_facility: Academisch Medisch Centrum at University of Amsterdam

location_facility: Daniel Den Hoed Cancer Center at Erasmus Medical Center

location_facility: Nijmegen Cancer Center at Radboud University Medical Center

location_facility: Mount Vernon Cancer Centre at Mount Vernon Hospital

location_facility: Academisch Ziekenhuis der Vrije Universiteit Brussel


title: A Multicentric Phase 1/2 Trial to Evaluate the Safety and Efficacy of SOT102 as Monotherapy and in Combination With Standard of Care Treatment in Patients With Gastric and Pancreatic Adenocarcinoma

summary: This trial will assess the MTD and RP2D of SOT102 administered as monotherapy (Part A) and in combination with first-line SoC treatment (mFOLFOX6 with nivolumab and nab-paclitaxel/ gemcitabine; Part B) and efficacy of SOT102 administered as monotherapy (Part C) and in combination with first-line SoC treatment (Part D) in patients with advanced inoperable or metastatic gastric/GEJ adenocarcinoma or inoperable or metastatic pancreatic adenocarcinoma.

status: Recruiting

study_completion_date_type: Anticipated

ipd_sharing: No

lead_sponsor: SOTIO Biotech

location_country:
Spain
France
United States
Belgium
Czechia

location_facility: Washington University School of Medicine in St. Louis

location_facility: VHIO - Vall d'Hebron Institut d'Oncologia

location_facility: Universitair Ziekenhuis Leuven - Campus Gasthuisberg

status_simplified: Ongoing


type: Other Identifier

title: A Randomized, Placebo-controlled, Double-blind Study to Assess the Effect of TOTUM-854 on Blood Pressure in Subjects With Moderately Elevated Blood Pressure

acronym: INSIGHT

summary: This clinical study aims to assess the efficacy of TOTUM-854, a mix of 6 plant extracts, consumed twice a day on automated office blood pressure in subjects with moderately elevated blood pressure. The hypothesis is that TOTUM-854 is superior to placebo for decrease of automated office blood pressure after 12 weeks of consumption.

primary_purpose: Prevention

lead_sponsor: Valbiotis

location_country: Poland Bulgaria France

location_facility: Multiprofile Hospital for Active Treatment Sveta Karidad EAD, Cardiology Department

location_facility: Multiprofile Hospital for Active Treatment - Uni Hospital OOD, Cardiology Department

location_facility: CHU Clermont Ferrand PIC/CIC Inserm 1405

location_facility: Diagnostic Consultative Center XX- Sofia EOOD

location_facility: Przychodnia Zespołu Lekarza Rodzinnego "Medyk"

location_facility: Diagnostic Consultative Center VII-Plovdiv South district EOOD, Cabinet of Endocrinology

location_facility: Multiprofile Hospital for Active Treatment Cardiology Department

intervention_type: Dietary Supplement


title: A Multi-center, Non-interventional Phase 0 Study for the Collection of Biospecimens From Patients With Parkinson's Disease.

summary: This study aims to collect, analyze and preserve biospecimens from patients with or without LRRK2-associated Parkinson's Disease for the purpose of discovering and developing new treatments and novel biomarkers.

study_type: Observational

time_perspective: Prospective

lead_sponsor: Neuron23 Inc.

location_country:
United States
Israel
Canada
France

location_facility: Assistance Publique - Hospitaux de Paris

location_facility: Rocky Mountain Movement Disorders Center


title: Real-World Outcomes Study on Subjects Treated With Radiofrequency Ablation

acronym: ROSTRA

summary:
ROSTRA is an international, prospective, non-randomized, single-arm, multi-center, and post-market study to collect real-world safety and effectiveness data on Abbott's IonicRF™Generator and compatible RFA accessories. This post-market study is intended to satisfy EU MDR requirements.

The study will enroll up to 180 subjects at up to 10 sites in Europe and the United States.

The total duration of the study is expected to be 19 months, including enrollment, data collection from all subjects, and study closeout.

status: Active, not recruiting

lead_sponsor: Abbott Medical Devices

location_country:
Spain
Netherlands
United States
France
Belgium
Germany

location_facility: Hospital Clínico Universitario de Valencia

location_facility: Krankenhaus Neuwerk Maria von den Aposteln

location_facility: The Spine & Nerve Center of St Francis Hospital

location_facility: Universitätsmedizin Berlin - Charité Campus Mitte

intervention_type: Device


title: A Long Term Prospective Post Market Clinical Follow-up (PMCF) of the GYNECARE TVT EXACT® Continence System for the Treatment of Stress Urinary Incontinence

summary: The objective of this single arm, post market, clinical study is to evaluate the performance of Gynecare TVT EXACT® Continence System in women undergoing retropubic mid-urethral sling surgery for Stress Urinary Incontinence (SUI).

ipd_sharing: Yes

ipd_sharing_description: Johnson & Johnson Medical Device Companies have an agreement with the Yale Open Data Access (YODA) Project to serve as the independent review panel for evaluation of requests for clinical study reports and participant level data from investigators and physicians for scientific research that will advance medical knowledge and public health. Requests for access to the study data can be submitted through the YODA Project site at http://yoda.yale.edu.

lead_sponsor: Ethicon, Inc.

location_country:
Denmark
Austria
United States
France
Sweden

location_facility:
UPMC
Institute for Female Pelvic Medicine
Hopital Jeanne de Flandres
Karolinska Institute
Duke University
Herlev Hospital
Ordensklinikum Linz


title: Psychosocial Care During the COVID-19 Pandemic in Acute Hospitals - an International Online Survey of Psychosomatic, Psychiatric and Psychological Consultation and Liaison (C&L) Services

summary: Heads of all psychosomatic, psychiatric and psychological consultation and liaison (C&L) services in acute care hospitals across all participating countries will be contacted and asked to participate in this international cross-sectional online survey. The survey consists of questions about care structures and their utilisation that psychosomatic, psychiatric and psychological C&L services have established in acute hospitals in the context of the COVID 19 pandemic.

time_perspective: Cross-Sectional

ReferenceCitation: Schaefert R, Stein B, Meinlschmidt G, Roemmel N, Huber CG, Hepp U, Saillant S, Fazekas C, Vitinius F. COVID-19-Related Psychosocial Care in General Hospitals: Results of an Online Survey of Psychosomatic, Psychiatric, and Psychological Consultation and Liaison Services in Germany, Austria, and Switzerland. Front Psychiatry. 2022 Jun 24;13:870984. doi: 10.3389/fpsyt.2022.870984. eCollection 2022.

lead_sponsor: University Hospital, Basel, Switzerland

location_country:
Poland
Portugal
Spain
Greece
Austria
Iran, Islamic Republic of
Ireland
Finland
Canada
Italy
Switzerland
United Kingdom
France
Belgium
Norway
Germany

location_facility: Université de Paris, Service de Psychiatrie de l'adulte

location_facility: Ghent University Hospital, Department of Psychiatry

location_facility: University Hospital Cologne, Department of Psychosomatics and Psychotherapy

location_facility: Medical University of Graz, University Hospital Graz

location_facility: University of Montreal, Department of Psychosomatic Medicine

location_facility: University of Medical Sciences, Department of Psychiatry

location_facility: TorontoSinai Health, University of Toronto, Department of Psychiatry

location_facility: Oulu University Hospital and University of Oulu

location_facility: Nuremberg Hospital, Department of Psychosomatic Medicine and Psychotherapy

location_facility: University Hospital Basel, Department of Psychosomatic Medicine

location_facility: University of Basel, Department of Psychiatry (UPK)

lead_sponsor_type: academique


title: An Observational Post-marketing Study for Evaluation of Ongoing Safety and Effectiveness of Catheter Mapping and Ablation Using Commercially Approved BWI Medical Devices for the Treatment of Patients With Cardiac Arrhythmias

summary: The purpose of this study is to prospectively collect clinical data evaluating the ongoing safety and performance during routine-use standard cardiac arrhythmia mapping and/or ablation procedures while using commercial Biosense Webster Inc. (BWI) medical devices. Data generated from the study will be used to confirm safety and performance of BWI medical devices in the marketed phase and to expand the body of evidence on the use of these devices and techniques in treatment of cardiac arrhythmias.

ipd_sharing_description:
Johnson & Johnson Medical Device Companies have an agreement with the Yale Open Data Access (YODA) Project to serve as the independent review panel

for evaluation of requests for clinical study reports and participant level data from investigators and physicians for scientific research that will advance medical knowledge and public health. Requests for access to the study data can be submitted through the YODA Project site at http://yoda.yale.edu

lead_sponsor: Biosense Webster, Inc.

location_country:
United Kingdom
Italy
Austria
France
Israel
Belgium
Germany

location_facility: Cardioangiologisches Centrum Bethanien (CCB)

location_facility: Centre Hospitalier Universitaire de Bordeaux


title: A Randomised, Double-blind, Placebo Controlled, Parallel Group, Multi-centre Trial in Adult Subjects With Newly Diagnosed Type 1 Diabetes Mellitus Investigating the Effect of Verapamil SR on Preservation of Beta-cell Function (Ver-A-T1D)

lead_sponsor: Medical University of Graz

ReferenceCitation: Atkinson MA, Eisenbarth GS, Michels AW. Type 1 diabetes. Lancet. 2014 Jan 4;383(9911):69-82. doi: 10.1016/S0140-6736(13)60591-7. Epub 2013 Jul 26. Review.

ReferenceCitation: Lachin JM, McGee P, Palmer JP; DCCT/EDIC Research Group. Impact of C-peptide preservation on metabolic and clinical outcomes in the Diabetes Control and Complications Trial. Diabetes. 2014 Feb;63(2):739-48. doi: 10.2337/db13-0881. Epub 2013 Oct 2.

ReferenceCitation: Sørensen JS, Johannesen J, Pociot F, Kristensen K, Thomsen J, Hertel NT, Kjaersgaard P, Brorsson C, Birkebaek NH; Danish Society for Diabetes in Childhood and Adolescence. Residual β-Cell function 3-6 years after onset of type 1 diabetes reduces risk of severe hypoglycemia in children and adolescents. Diabetes Care. 2013 Nov;36(11):3454-9. doi: 10.2337/dc13-0418. Epub 2013 Aug 29.

ReferenceCitation: Ovalle F, Grimes T, Xu G, Patel AJ, Grayson TB, Thielen LA, Li P, Shalev A. Verapamil and beta cell function in adults with recent-onset type 1 diabetes. Nat Med. 2018 Aug;24(8):1108-1112. doi: 10.1038/s41591-018-0089-4. Epub 2018 Jul 9.

ReferenceCitation: Yin T, Kuo SC, Chang YY, Chen YT, Wang KK. Verapamil Use Is Associated With Reduction of Newly Diagnosed Diabetes Mellitus. J Clin Endocrinol Metab. 2017 Jul 1;102(7):2604-2610. doi: 10.1210/jc.2016-3778.

ReferenceCitation: Cooper-Dehoff R, Cohen JD, Bakris GL, Messerli FH, Erdine S, Hewkin AC, Kupfer S, Pepine CJ; INVEST Investigators. Predictors of development of diabetes mellitus in patients with coronary artery disease taking antihypertensive medications (findings from the INternational VErapamil SR-Trandolapril STudy [INVEST]). Am J Cardiol. 2006 Oct 1;98(7):890-4. Epub 2006 Aug 7.

ReferenceCitation: Chen J, Saxena G, Mungrue IN, Lusis AJ, Shalev A. Thioredoxin-interacting protein: a critical link between glucose toxicity and beta-cell apoptosis. Diabetes. 2008 Apr;57(4):938-44. doi: 10.2337/db07-0715. Epub 2008 Jan 2.

ReferenceCitation: Xu G, Chen J, Jing G, Shalev A. Preventing β-cell loss and diabetes with calcium channel blockers. Diabetes. 2012 Apr;61(4):848-56. doi: 10.2337/db11-0955.

ReferenceCitation: Seaquist ER, Anderson J, Childs B, Cryer P, Dagogo-Jack S, Fish L, Heller SR, Rodriguez H, Rosenzweig J, Vigersky R. Hypoglycemia and diabetes: a report of a workgroup of the American Diabetes Association and the Endocrine Society. Diabetes Care. 2013 May;36(5):1384-95. doi: 10.2337/dc12-2480. Epub 2013 Apr 15.

ReferenceCitation: Workgroup on Hypoglycemia, American Diabetes Association. Defining and reporting hypoglycemia in diabetes: a report from the American Diabetes Association Workgroup on Hypoglycemia. Diabetes Care. 2005 May;28(5):1245-9. Review.

location_country:
Poland
United Kingdom
Italy
Austria
Sweden
France
Belgium
Germany

location_facility: Université Libre de Bruxelles/ Hôpital Erasme

location_facility: Institut National de la Santé et de la Recherche Médicale

location_facility: Medical University of Graz, Department of Internal Medicine Division of Endocrinology and Metabolism

location_facility: Slaski Uniwersytet Medyczny w Katowicach

summary:
This study has been set up within the framework of the INNODIA network. INNODIA is a global partnership between 31 academic institutions, 6 industrial partners, a small sized enterprise and 2 patient organizations, bringing their knowledge and experience together with one common goal: "To fight type 1 diabetes". (www.innodia.eu) The overall aim of INNODIA is to advance in a decisive way how to predict, stage, evaluate and prevent the onset and progression of type 1 diabetes (T1D).

For this, INNODIA has established a comprehensive and interdisciplinary network of clinical and basic scientists, who are leading experts in the field of T1D research in Europe and UK (United Kingdom), with complementary expertise from the areas of immunology, Beta-cell biology, biomarker research and T1D therapy, joining forces in a coordinated fashion with industry partners and two foundations, as well as with all major stakeholders in the process, including regulatory bodies and patients with T1D and their families.


title: A Natural History Study of Patients With Genetically Confirmed Primary Hyperoxaluria Type 3 and, as Applicable Per Age, a History of Stone Events

acronym: PHYOX-OBX

summary: This is a natural history study of adults, adolescents, and children (starting at birth) with genetically confirmed primary hyperoxaluria type 3 (PH3) who have a history of stone events during the last 3 years and/or the presence of pre existing stones detected by renal ultrasound at screening.

lead_sponsor: Dicerna Pharmaceuticals, Inc.

location_country:
Poland
United Kingdom
Lebanon
Canada
United States
France
Germany


title: Outcome pRognostication of Acute Brain Injury With the NeuroloGical Pupil

acronym: ORANGE

summary:
The use of quantitative, automated, infrared technology for pupillary examination has long been used in ophthalmology and anesthesiology research. Its interest in neurocritical care has progressively grown, in parallel with the advancements in device technology. In this regard, the use of the noninvasive NPi®-200 pupillometer (Neuroptics, Laguna Hills, California, USA) allows the measurement of a series of dynamic pupillary variables (including the percentage pupillary constriction, latency, constriction velocity, and dilation velocity), which can be integrated into an algorithm, to compute the Neurological Pupil index (NPi). The NPi is a proprietary scalar index with values between 0 and 5 (with a 0.1 decimal precision), an NPi value < 3 indicating an abnormal pupillary reactivity. Importantly, the NPi is not influenced by sedation-analgesia, at the doses used in neurocritical care practice, and by mild hypothermia.

Preliminary single-center data recently demonstrated that abnormal NPi is associated with worse outcome in patients with traumatic and hemorrhagic ABI, and can be a useful adjunct for ICP monitoring and therapy. There is currently a great need for quantitative tools to predict early prognostication in ABI patients, and the NPi appears of potential great value.

We hypothesize that:

Abnormal NPi (defined as NPi <3) are strongly predictive of poor GOS-E (1-4) at 6 months after the acute event.
NPi=0 is strongly predictive of mortality (GOS 1).
Abnormal NPi is predictive of a higher ICP 20 index (number of end-hourly measures of ICP >20 mm Hg divided by the total number of measurements, multiplied by 100) and a greater burden of interventions needed to control ICP (measured by the Therapy Intensity Level scale for ICP management, Therapy Intensity Level (TIL) 4).

Methods This international multicentre prospective observational study aims to recruit >400 patients admitted to intensive care units.

Duration of the study 18 months, including 12-month of recruitment based on 60 patients/centre plus 6 months GOS-E follow-up.

ReferenceCitation: Sharshar T, Citerio G, Andrews PJ, Chieregato A, Latronico N, Menon DK, Puybasset L, Sandroni C, Stevens RD. Neurological examination of critically ill patients: a pragmatic approach. Report of an ESICM expert panel. Intensive Care Med. 2014 Apr;40(4):484-95. doi: 10.1007/s00134-014-3214-y. Epub 2014 Feb 13.

ReferenceCitation: Chen JW, Gombart ZJ, Rogers S, Gardiner SK, Cecil S, Bullock RM. Pupillary reactivity as an early indicator of increased intracranial pressure: The introduction of the Neurological Pupil index. Surg Neurol Int. 2011;2:82. doi: 10.4103/2152-7806.82248. Epub 2011 Jun 21.

ReferenceCitation: Chen JW, Vakil-Gilani K, Williamson KL, Cecil S. Infrared pupillometry, the Neurological Pupil index and unilateral pupillary dilation after traumatic brain injury: implications for treatment paradigms. Springerplus. 2014 Sep 23;3:548. doi: 10.1186/2193-1801-3-548. eCollection 2014.

ReferenceCitation: Larson MD, Behrends M. Portable infrared pupillometry: a review. Anesth Analg. 2015 Jun;120(6):1242-53. doi: 10.1213/ANE.0000000000000314. Review.

ReferenceCitation: Larson MD, Singh V. Portable infrared pupillometry in critical care. Crit Care. 2016 Jun 22;20(1):161. doi: 10.1186/s13054-016-1349-7.

ReferenceCitation: Volpi PC, Robba C, Rota M, Vargiolu A, Citerio G. Trajectories of early secondary insults correlate to outcomes of traumatic brain injury: results from a large, single centre, observational study. BMC Emerg Med. 2018 Dec 5;18(1):52. doi: 10.1186/s12873-018-0197-y.

ReferenceCitation: Han J, King NK, Neilson SJ, Gandhi MP, Ng I. External validation of the CRASH and IMPACT prognostic models in severe traumatic brain injury. J Neurotrauma. 2014 Jul 1;31(13):1146-52. doi: 10.1089/neu.2013.3003. Epub 2014 May 12.

ReferenceCitation: Couret D, Boumaza D, Grisotto C, Triglia T, Pellegrini L, Ocquidant P, Bruder NJ, Velly LJ. Reliability of standard pupillometry practice in neurocritical care: an observational, double-blinded study. Crit Care. 2016 Mar 13;20:99. doi: 10.1186/s13054-016-1239-z.

ReferenceCitation: Couret D, Simeone P, Freppel S, Velly L. The effect of ambient-light conditions on quantitative pupillometry: a history of rubber cup. Neurocrit Care. 2019 Apr;30(2):492-493. doi: 10.1007/s12028-018-0664-z.

ReferenceCitation: Olson DM, Stutzman S, Saju C, Wilson M, Zhao W, Aiyagari V. Interrater Reliability of Pupillary Assessments. Neurocrit Care. 2016 Apr;24(2):251-7. doi: 10.1007/s12028-015-0182-1.

ReferenceCitation: Hall CA, Chilcott RP. Eyeing up the Future of the Pupillary Light Reflex in Neurodiagnostics. Diagnostics (Basel). 2018 Mar 13;8(1). pii: E19. doi: 10.3390/diagnostics8010019. Review.

ReferenceCitation: Larson MD, Sessler DI. Pupillometry to guide postoperative analgesia. Anesthesiology. 2012 May;116(5):980-2. doi: 10.1097/ALN.0b013e318251d21b.

ReferenceCitation: Morelli P, Oddo M, Ben-Hamouda N. Role of automated pupillometry in critically ill patients. Minerva Anestesiol. 2019 Sep;85(9):995-1002. doi: 10.23736/S0375-9393.19.13437-2. Epub 2019 Mar 29. Review.

ReferenceCitation: Shoyombo I, Aiyagari V, Stutzman SE, Atem F, Hill M, Figueroa SA, Miller C, Howard A, Olson DM. Understanding the Relationship Between the Neurologic Pupil Index and Constriction Velocity Values. Sci Rep. 2018 May 3;8(1):6992. doi: 10.1038/s41598-018-25477-7.

ReferenceCitation: Jahns FP, Miroz JP, Messerer M, Daniel RT, Taccone FS, Eckert P, Oddo M. Quantitative pupillometry for the monitoring of intracranial hypertension in patients with severe traumatic brain injury. Crit Care. 2019 May 2;23(1):155. doi: 10.1186/s13054-019-2436-3.

ReferenceCitation: Aoun SG, Stutzman SE, Vo PN, El Ahmadieh TY, Osman M, Neeley O, Plitt A, Caruso JP, Aiyagari V, Atem F, Welch BG, White JA, Batjer HH, Olson DM. Detection of delayed cerebral ischemia using objective pupillometry in patients with aneurysmal subarachnoid hemorrhage. J Neurosurg. 2019 Jan 11;132(1):27-32. doi: 10.3171/2018.9.JNS181928.

ReferenceCitation: Osman M, Stutzman SE, Atem F, Olson D, Hicks AD, Ortega-Perez S, Aoun SG, Salem A, Aiyagari V. Correlation of Objective Pupillometry to Midline Shift in Acute Stroke Patients. J Stroke Cerebrovasc Dis. 2019 Jul;28(7):1902-1910. doi: 10.1016/j.jstrokecerebrovasdis.2019.03.055. Epub 2019 Apr 25.

ReferenceCitation: Ong C, Hutch M, Barra M, Kim A, Zafar S, Smirnakis S. Effects of Osmotic Therapy on Pupil Reactivity: Quantification Using Pupillometry in Critically Ill Neurologic Patients. Neurocrit Care. 2019 Apr;30(2):307-315. doi: 10.1007/s12028-018-0620-y.

ReferenceCitation: Phillips SS, Mueller CM, Nogueira RG, Khalifa YM. A Systematic Review Assessing the Current State of Automated Pupillometry in the NeuroICU. Neurocrit Care. 2019 Aug;31(1):142-161. doi: 10.1007/s12028-018-0645-2.

ReferenceCitation: Oddo M, Sandroni C, Citerio G, Miroz JP, Horn J, Rundgren M, Cariou A, Payen JF, Storm C, Stammet P, Taccone FS. Quantitative versus standard pupillary light reflex for early prognostication in comatose cardiac arrest patients: an international prospective multicenter double-blinded study. Intensive Care Med. 2018 Dec;44(12):2102-2111. doi: 10.1007/s00134-018-5448-6. Epub 2018 Nov 26.

ReferenceCitation: Zuercher P, Groen JL, Aries MJ, Steyerberg EW, Maas AI, Ercole A, Menon DK. Reliability and Validity of the Therapy Intensity Level Scale: Analysis of Clinimetric Properties of a Novel Approach to Assess Management of Intracranial Pressure in Traumatic Brain Injury. J Neurotrauma. 2016 Oct 1;33(19):1768-1774. Epub 2016 Feb 11.

ReferenceCitation: Wilson L, von Steinbuechel N, Menon DK, Maas AIR. Outcome assessment after traumatic brain injury - Authors' reply. Lancet Neurol. 2018 Apr;17(4):299-300. doi: 10.1016/S1474-4422(18)30045-0. Epub 2018 Mar 13.

ReferenceCitation: Oddo M, Taccone F, Galimberti S, Rebora P, Citerio G; Orange Study Group. Outcome Prognostication of Acute Brain Injury using the Neurological Pupil Index (ORANGE) study: protocol for a prospective, observational, multicentre, international cohort study. BMJ Open. 2021 May 11;11(5):e046948. doi: 10.1136/bmjopen-2020-046948.

ipd_sharing_description: Access to data The University of Milano-Bicocca has the property of all the data collected. The data resides at the University Milano-Bicocca as study Sponsor; all procedures will comply with the European Union (EU) regulation on data protection 2016/679 on the protection of natural persons regarding personal data processing and movement. Local site data will be co-owned by each participating centre, and they will be given access to local data for any scientific purpose upon request. By entering data into the ORANGE study database, each centre agrees that the chief can use these data for scientific purposes. Any requests for the use of the data set for subsequent studies will be made to the ORANGE study chief investigators. Any requests for the use of the data set for subsequent studies will be made to the ORANGE study chief investigators.

lead_sponsor: University of Milano Bicocca

location_country:
Spain
Italy
Switzerland
United States
France
Belgium
Norway
Germany

location_facility: Department Anesthesia and Critical Care, University Hospital

location_facility: Hospital Clinic Universitari de València, University of Valencia

location_facility: Centre Hospitalier Universitaire Vaudois (CHUV), University Hospital and University of Lausanne

location_facility: Department of Neurology, University of California

location_facility: Erasme Hospital, Université Libre de Bruxelles


title: Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

acronym: RETRIEVE

summary:
This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2). Information is planned to be gathered on at least 180 patients with GM1 gangliosidosis, GM2 gangliosidoses, and Gaucher Disease type 2. Retrospective data collection is planned for at least 150 deceased patients (Group A). Group B is for patients alive at the time of enrollment. In Group B it is planned to prospectively collect more comprehensive data from at least 30 patients. The purpose of this study is to collect relevant information for a adequate design of a potential subsequent research program in these diseases.

In this study no therapy is being offered.

time_perspective: Other

lead_sponsor: Idorsia Pharmaceuticals Ltd.

location_country:
Brazil
Portugal
Spain
Italy
United Kingdom
Switzerland
France
United States
Belgium
Germany

location_facility: AP-HP - Hôpitaux Universitaires Est Parisien

location_facility: Lysosomal and Rare Disorders Research and Treatment Center

location_facility: Centro Hospitalar Universitario Lisboa Norte, EPE

location_facility: Centro Universitario Hospitalar de São João, EPE

location_facility: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago

location_facility: Hospital de Clínicas de Porto Alegre - HCPA

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Great Ormond Street Hospital for Children NHS Found. Trust


title: The RESPOND Outcomes Study - A Study in the RESPOND Consortium (RESPOND: International Cohort Consortium of Infectious Diseases)

summary: The RESPOND Outcomes study is a research study around use of antiretroviral and other relevant drugs and long-term clinical outcomes in patients living with HIV. Data collected in this study will be used to answer key unanswered questions regarding treatment of people living with HIV.

ReferenceCitation: Obel N, Omland LH, Kronborg G, Larsen CS, Pedersen C, Pedersen G, Sørensen HT, Gerstoft J. Impact of non-HIV and HIV risk factors on survival in HIV-infected patients on HAART: a population-based nationwide cohort study. PLoS One. 2011;6(7):e22698. doi: 10.1371/journal.pone.0022698. Epub 2011 Jul 25.

ReferenceCitation: Smith CJ, Ryom L, Weber R, Morlat P, Pradier C, Reiss P, Kowalska JD, de Wit S, Law M, el Sadr W, Kirk O, Friis-Moller N, Monforte Ad, Phillips AN, Sabin CA, Lundgren JD; D:A:D Study Group. Trends in underlying causes of death in people with HIV from 1999 to 2011 (D:A:D): a multicohort collaboration. Lancet. 2014 Jul 19;384(9939):241-8. doi: 10.1016/S0140-6736(14)60604-8.

ReferenceCitation: Schouten J, Wit FW, Stolte IG, Kootstra NA, van der Valk M, Geerlings SE, Prins M, Reiss P; AGEhIV Cohort Study Group. Cross-sectional comparison of the prevalence of age-associated comorbidities and their risk factors between HIV-infected and uninfected individuals: the AGEhIV cohort study. Clin Infect Dis. 2014 Dec 15;59(12):1787-97. doi: 10.1093/cid/ciu701. Epub 2014 Sep 2.

ReferenceCitation: Chary A, Nguyen NN, Maiton K, Holodniy M. A review of drug-drug interactions in older HIV-infected patients. Expert Rev Clin Pharmacol. 2017 Dec;10(12):1329-1352. doi: 10.1080/17512433.2017.1377610. Epub 2017 Sep 19. Review.

ReferenceCitation: Weber R, Sabin CA, Friis-Møller N, Reiss P, El-Sadr WM, Kirk O, Dabis F, Law MG, Pradier C, De Wit S, Akerlund B, Calvo G, Monforte Ad, Rickenbach M, Ledergerber B, Phillips AN, Lundgren JD. Liver-related deaths in persons infected with the human immunodeficiency virus: the D:A:D study. Arch Intern Med. 2006 Aug 14-28;166(15):1632-41.

ReferenceCitation: Kattakuzhy S, Gross C, Emmanuel B, Teferi G, Jenkins V, Silk R, Akoth E, Thomas A, Ahmed C, Espinosa M, Price A, Rosenthal E, Tang L, Wilson E, Bentzen S, Masur H, Kottilil S; ASCEND Providers. Expansion of Treatment for Hepatitis C Virus Infection by Task Shifting to Community-Based Nonspecialist Providers: A Nonrandomized Clinical Trial. Ann Intern Med. 2017 Sep 5;167(5):311-318. doi: 10.7326/M17-0118. Epub 2017 Aug 8.

ReferenceCitation: Falade-Nwulia O, Suarez-Cuervo C, Nelson DR, Fried MW, Segal JB, Sulkowski MS. Oral Direct-Acting Agent Therapy for Hepatitis C Virus Infection: A Systematic Review. Ann Intern Med. 2017 May 2;166(9):637-648. doi: 10.7326/M16-2575. Epub 2017 Mar 21. Review.

ReferenceCitation: van der Meer AJ, Veldt BJ, Feld JJ, Wedemeyer H, Dufour JF, Lammert F, Duarte-Rojo A, Heathcote EJ, Manns MP, Kuske L, Zeuzem S, Hofmann WP, de Knegt RJ, Hansen BE, Janssen HL. Association between sustained virological response and all-cause mortality among patients with chronic hepatitis C and advanced hepatic fibrosis. JAMA. 2012 Dec 26;308(24):2584-93. doi: 10.1001/jama.2012.144878.

ReferenceCitation: Friis-Møller N, Ryom L, Smith C, Weber R, Reiss P, Dabis F, De Wit S, Monforte AD, Kirk O, Fontas E, Sabin C, Phillips A, Lundgren J, Law M; D:A:D study group. An updated prediction model of the global risk of cardiovascular disease in HIV-positive persons: The Data-collection on Adverse Effects of Anti-HIV Drugs (D:A:D) study. Eur J Prev Cardiol. 2016 Jan;23(2):214-23. doi: 10.1177/2047487315579291. Epub 2015 Apr 16.

ReferenceCitation: Mocroft A, Lundgren JD, Ross M, Fux CA, Reiss P, Moranne O, Morlat P, Monforte Ad, Kirk O, Ryom L; Data Collection on Adverse events of Anti-HIV Drugs (D:A:D) Study. Cumulative and current exposure to potentially nephrotoxic antiretrovirals and development of chronic kidney disease in HIV-positive individuals with a normal baseline estimated glomerular filtration rate: a prospective international cohort study. Lancet HIV. 2016 Jan;3(1):e23-32. doi: 10.1016/S2352-3018(15)00211-8. Epub 2015 Nov 17.

ReferenceCitation: Bruyand M, Ryom L, Shepherd L, Fatkenheuer G, Grulich A, Reiss P, de Wit S, D Arminio Monforte A, Furrer H, Pradier C, Lundgren J, Sabin C; D:A:D study group. Cancer risk and use of protease inhibitor or nonnucleoside reverse transcriptase inhibitor-based combination antiretroviral therapy: the D: A: D study. J Acquir Immune Defic Syndr. 2015 Apr 15;68(5):568-77. doi: 10.1097/QAI.0000000000000523.

ReferenceCitation: Neesgaard B, Greenberg L, Miró JM, Grabmeier-Pfistershammer K, Wandeler G, Smith C, De Wit S, Wit F, Pelchen-Matthews A, Mussini C, Castagna A, Pradier C, d'Arminio Monforte A, Vehreschild JJ, Sönnerborg A, Anne AV, Carr A, Bansi-Matharu L, Lundgren JD, Garges H, Rogatto F, Zangerle R, Günthard HF, Rasmussen LD, Necsoi C, van der Valk M, Menozzi M, Muccini C, Peters L, Mocroft A, Ryom L. Associations between integrase strand-transfer inhibitors and cardiovascular disease in people living with HIV: a multicentre prospective study from the RESPOND cohort consortium. Lancet HIV. 2022 Jul;9(7):e474-e485. doi: 10.1016/S2352-3018(22)00094-7. Epub 2022 Jun 7.

lead_sponsor: Rigshospitalet, Denmark

location_country:
Denmark
Spain
Italy
Austria
Australia
Georgia
Netherlands
United Kingdom
Switzerland
Sweden
France
Belgium
Germany

location_facility: The Australian HIV Observational Database (AHOD)

location_facility: Austrian HIV Cohort Study (AHIVCOS), Medizinische Universität Innsbruch

location_facility: Modena HIV Cohort, Università degli Studi di Modena

location_facility: Royal Free HIV Cohort Study, Royal Free Hospital

location_facility: Frankfurt HIV Cohort Study, Goethe-University Frankfurt

location_facility: The ATHENA (AIDS Therapy Evaluation in the Netherlands) national observational HIV cohort, Stichting HIV Monitorin, AMC, University of Amsterdam

location_facility: PISCIS Cohort Study, Germans Trias i Pujol University Hospital

location_facility: The EuroSIDA Study, CHIP, Rigshospitalet

location_facility: Georgian National AIDS Health Information System (AIDS HIS), IDACIRC

location_facility: Italian Cohort Naive Antiretrovirals (ICONA)

location_facility: Swedish InfCare HIV Cohort, Karolinska University Hospital

location_facility: Swiss HIV Cohort Study (SHCS), University Hospital Zurich

location_facility: Nice HIV Cohort, Centre Hospitalier Universitaire de Nice

location_facility: San Raffaele Scientific Institute, Ospedale San Raffaele


title: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

summary: This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will be conducted for the selection of a high dose (100 mg/kg versus 200 mg/kg) and its comparison with the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.

lead_sponsor: Sarepta Therapeutics, Inc.

location_country:
Poland
Taiwan
Spain
Greece
Ireland
New Zealand
United Kingdom
Canada
Korea, Republic of
United States
France
Turkey
Mexico
Norway
Czechia

location_facility: New Zealand Clinical Research - Auckland

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: Children's Health Ireland (CHI) at Temple Street

location_facility: Oslo Universitetssykehus HF Rikshospitalet

location_facility: UCL Institute of Child Health Great Ormond Street

location_facility: Instituto de Investigaciones Clinicas para la Salud A.C

location_facility: Children's Hospital - London Health Sciences Centre

location_facility: Pusan National University Yangsan Hospital

location_facility: Kyungpook National University Chilgok Hospital


title: Vascular Access Outcome Measure for Function: a vaLidation Study In haemoDialysis (VALID)

acronym: VALID

summary: A functioning vascular access provides a lifeline for patients requiring haemodialysis but vascular access dysfunction remains one of the leading causes of excessive morbidity, mortality and healthcare costs in this group. Despite increasing numbers of vascular access trials, successful interventions to improve vascular access function have been sparse and compromised by highly variable, often selectively reported outcome measures of limited relevance to patients and health professionals. Through engagement of all relevant stakeholders including patients and caregivers, vascular access function, defined by the need for interventions to enable and maintain the use of a vascular access for haemodialysis, has been identified as one of the most critically important outcome measures for trials in haemodialysis. This prospective, multi-centre, multinational validation study aims to assess the accuracy and feasibility of measuring vascular access function part of routine clinical practice and across different clinical settings to ensure successful global implementation of this core outcome measure in future trials in haemodialysis.

ReferenceCitation: Viecelli AK, Teixeira-Pinto A, Valks A, Baer R, Cherian R, Cippà PE, Craig JC, DeSilva R, Jaure A, Johnson DW, Kiriwandeniya C, Kopperschmidt P, Liu WJ, Lee T, Lok C, Madhan K, Mallard AR, Oliver V, Polkinghorne KR, Quinn RR, Reidlinger D, Roberts M, Sautenet B, Hooi LS, Smith R, Snoeijs M, Tordoir J, Vachharajani TJ, Vanholder R, Vergara LA, Wilkie M, Yang B, Yuo TH, Zou L, Hawley CM; VALID Investigator Team. Study protocol for Vascular Access outcome measure for function: a vaLidation study In hemoDialysis (VALID) : A multi-center, multinational validation study to assess the accuracy and feasibility of measuring vascular access function in clinical practice. BMC Nephrol. 2022 Nov 19;23(1):372. doi: 10.1186/s12882-022-02987-1.

ipd_sharing_description: Individual participant data that underlie the results reported in the primary publication, after deidentification (text, tables, figures and appendices) will be available for individual participant data meta-analysis.

lead_sponsor: The University of Queensland

location_country:
United Kingdom
Australia
Netherlands
Canada
Switzerland
France
Malaysia

location_facility: Centre Hospitalier Régional Universitaire de Tours


title: A Real World Evidence Prospective Cohort Study in the Management of Metastatic Colorectal Cancer: A Clinical and Patient Perspective

acronym: PROMETCO

ReferenceCitation: Koopman M, Pinto C, Bodoky G, Garcia-Carbonero R, Marti FM, Bachet JB. Rationale and design of the PROMETCO study: a real-world, prospective, longitudinal cohort on the continuum of care of metastatic colorectal cancer from a clinical and patient perspective. Future Oncol. 2022 Apr;18(11):1313-1320. doi: 10.2217/fon-2021-1333. Epub 2022 Jan 19. Review.

lead_sponsor: Servier Affaires Médicales

location_country:
Croatia
Portugal
Slovenia
Greece
Austria
Ireland
Italy
Argentina
Netherlands
Spain
Switzerland
Sweden
Hungary
France
United Kingdom
Belgium
Germany
Czechia

location_facility: University Hospital in Hradec Kralove Clinic of Oncology and Radiotherapy

location_facility: The Adelaide & Meath Hospital-Dublin Incorporating The National Children's Hospital

location_facility: Centro Des Estudos Egas Moniz - Hospital Santa Maria

location_facility: Cancer Research UK, Department of Medical Oncology-The Christie NHS Foundation Trust

location_facility: Del-Pesti Centrum Korhaz OHII, Szent Laszlo Korhaz Telephely

location_facility: UniversitaetsSpital Zuerich (University Hospital Zurich)


acronym: INFORM2 NivEnt

title: INFORM2 Exploratory Multinational Phase I/II Combination Study of Nivolumab and Entinostat in Children and Adolescents With Refractory High-risk Malignancies

lead_sponsor: University Hospital Heidelberg

ReferenceCitation: van Tilburg CM, Witt R, Heiss M, Pajtler KW, Plass C, Poschke I, Platten M, Harting I, Sedlaczek O, Freitag A, Meyrath D, Taylor L, Balasubramanian GP, Jäger N, Pfaff E, Jones BC, Milde T, Pfister SM, Jones DTW, Kopp-Schneider A, Witt O. INFORM2 NivEnt: The first trial of the INFORM2 biomarker driven phase I/II trial series: the combination of nivolumab and entinostat in children and adolescents with refractory high-risk malignancies. BMC Cancer. 2020 Jun 5;20(1):523. doi: 10.1186/s12885-020-07008-8.

ipd_sharing_description:
The pseudonymized data will be shared for transparency reasons in the context of publications and after publication with other physicians and scientists (national and international academia) to promote and accelerate research on causes and treatment development of oncological diseases.

Requests for access to pseudonymized patient data for non-INFORM-related scientific purposes will be reviewed by a Data Access Committee consisting of the INFORM Board, and the participating investigators. A positive statement of the respective ethic committee and a signed data protection commitment are requested. Requests should be addressed to the coordinating investigator Prof. Olaf Witt. Results of scientific research based on the INFORM2 NivEnt data may be used for academic teaching, research and scientific publications or presentations at scientific meetings.

location_country: Austria Australia Netherlands Switzerland Sweden France Germany

location_facility: Hopp Children's Cancer Center Heidelberg (KiTZ)

summary: The aim of this trial is to determine preliminary activity of the combination treatment with nivolumab and entinostat in children and adolescents with high risk refractory/relapsed/progressive tumors harboring a high mutational load, high PD-L1 mRNA expression or focal MYC(N) amplification and explore activity in biomarker low tumors (low mutational load, low PD-L1 mRNA expression and non-MYC(N) amplified).


title: E²-RADIatE: EORTC-ESTRO RADiotherapy InfrAstrucTure for Europe

summary: The primary objective is the collection of real-world data of cancer patients treated with radiotherapy, to support radiotherapy research and to provide evidence of the role of radiation oncology in a multidisciplinary approach. This is an open ended prospective non-interventional non-therapeutic multi-cohort study.

ipd_sharing: Undecided

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country: Italy Switzerland France Belgium Netherlands

location_facility: AZ Groeninge Kortrijk - Campus Kennedylaan

location_facility: Radboudumc - Radboud University Medical Center Nijmegen

location_facility: Hôpitaux universitaires de Genève - HUG - site de Cluse-Roseraie


title: A Survivorship Project to UnerstAnd and to impRove Long-Term Outcomes for Acute Myeloid Leukemia Patients (SPARTA): the Sparta Platform

summary: This international observational study aims at examining the patterns of health-related quality of life differences between long-term acute myeloid leukemia patients and their healthy peers from the general population.

lead_sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

location_country:
Croatia
Slovenia
Italy
North Macedonia
France
Turkey
Belgium
Slovakia

location_facility: U.O. Ematologia con trapianto - Azienda Ospedaliero-Universitaria Policlinico di Bari

location_facility: Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova

location_facility: S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro

location_facility: Torino Dipartimento di Oncologia ed Ematologia S.C. Ematologia 2 A.O. Città della Salute e della Scienza di Torino San Giovanni Battista

location_facility: Azienda Ospedaliera Universitaria - Università degli Studi di Napoli "Federico II" - Facoltà di Medicina e Chirurgia

location_facility: Milano Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano

location_facility: U.O. di Ematologia - Casa Sollievo della Sofferenza

location_facility: Orbassano Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2

location_facility: UOC di Ematologia-Centro Emofilia e Trombosi - Ospedale Ascalesi - ASL Napoli 1

location_facility: Divisione di Ematologia Ospedale A. Perrino

location_facility: Università degli Studi "Sapienza" - Dip Medicina di Precisione e Ricerca Traslazionale UOC Ematologia

location_facility: Ankara University School Of Medicine Cebeci Campus Adult Hematology Department and BMT Unit

location_facility: Az. Ospedaliera "Sant' Andrea"-Università la Sapienza Seconda Facoltà di Medicina e Chirurgia

location_facility: Divisione clinicizzata di Ematologia - Dipartimento di Scienze Mediche - Osp. Ferrarotto

location_facility: Struttura Complessa di Ematologia Ospedali Riuniti Foggia - Azienda Ospedaliero-Universitaria

location_facility: ASL N.8 - Ospedale "A. Businco" - Struttura Complessa di Ematologia e CTMO

location_facility: Divisione di Ematologia - Ospedale S.Eugenio

location_facility: Les Hôpitaux Universitaires de Strasbourg

location_facility: Azienda Ospedaliero Universitaria Arcispedale Sant'Anna Dipartimento di Scienze Mediche Sezione di Ematologia e Fisiopatologia dell'Emostasi

location_facility: IRCCS_AOU San Martino-IST.Clinica Ematologica

location_facility: Università degli Studi di Padova - Ematologia ed Immunologia Clinica

location_facility: Istituto di Semeiotica Medica - Ematologia - Università Cattolica del Sacro Cuore - Policlinico A. Gemelli

location_facility: U.O. di Ematologia con trapianto - A.U. Policlinico "Paolo Giaccone"

location_facility: Roma Divisione Ematologia - Università Campus Bio-Medico

location_facility: Ematologia Policlinico - Università degli Studi di Roma Tor Vergata (PTV)

location_facility: Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale G. da Saliceto

location_facility: UOC Ematologia Ospedale " Monsignor Raffaele Dimiccoli"

location_facility: Azienda Ospedaliero - Universitaria Ospedali Riuniti Umberto I - G.M. LANCISI - G. SALESI


acronym: MATINS

title: A Phase I/II Open-Label, Three-Part, Dose-Finding and Separate Cohort Expansion Trial to Assess the Safety, Tolerability and Preliminary Efficacy of Repeated Doses of CLEVER-1 Antibody FP-1305, in Subjects With Advanced Solid Tumours

lead_sponsor: Faron Pharmaceuticals Ltd

ReferenceCitation: Hollmén M, Maksimow M, Rannikko JH, Karvonen MK, Vainio M, Jalkanen S, Jalkanen M, Mandelin J. Nonclinical Characterization of Bexmarilimab, a Clever-1-Targeting Antibody for Supporting Immune Defense Against Cancers. Mol Cancer Ther. 2022 Jul 5;21(7):1207-1218. doi: 10.1158/1535-7163.MCT-21-0840.

ReferenceCitation: Virtakoivu R, Rannikko JH, Viitala M, Vaura F, Takeda A, Lönnberg T, Koivunen J, Jaakkola P, Pasanen A, Shetty S, de Jonge MJA, Robbrecht D, Ma YT, Skyttä T, Minchom A, Jalkanen S, Karvonen MK, Mandelin J, Bono P, Hollmén M. Systemic Blockade of Clever-1 Elicits Lymphocyte Activation Alongside Checkpoint Molecule Downregulation in Patients with Solid Tumors: Results from a Phase I/II Clinical Trial. Clin Cancer Res. 2021 Aug 1;27(15):4205-4220. doi: 10.1158/1078-0432.CCR-20-4862. Epub 2021 Jun 2.

location_country:
Spain
United Kingdom
Finland
United States
France
Netherlands

location_facility: The University of Texas Health Science Center at San Antonio

location_facility: Erasmus University Medical Center Rotterdam

location_facility: START Madrid - CIOCC Hospital HM Sanchinarro

summary:
This is a first in human study to identify whether FP-1305 is suitable to use in humans. The previous pre-clinical studies have demonstrated that FP-1305 binds to a receptor known as CLEVER-1. CLEVER-1 has been shown to support tumour growth. No significant adverse events were witnessed in primates and the dose used will be 300 fold lower than the dose provided to primates which showed no toxicity.

The patients with advanced melanoma, uveal melanoma, cholangiocarcinoma, gallbladder cancer, ER+ breast, gastric, ovarian, pancreatic, colorectal, liver or anaplastic thyroid cancer who have exhausted all licenced therapeutic options will die due to their disease. Based on the investigator's existing data CLEVER-1 is expressed in these tumour types. Inhibition of CLEVER-1 with FP-1305 may have an anti-tumour effect in these patients.


title: A Two-Part, Randomized, Open-label, Multicenter, Phase 2a/2b Study of the Efficacy, Safety, and Pharmacokinetics of KRT-232 Compared to Ruxolitinib in Patients With Phlebotomy-Dependent Polycythemia Vera

summary:
This study evaluates KRT-232, a novel oral small molecule inhibitor of MDM2, for the treatment of patients with phlebotomy-dependent polycythemia vera (PV). Inhibition of MDM2 in PV is a new mechanism of action in PV. In Part A, patients must be resistant or intolerant to hydroxyurea or have undergone treatment with interferon. In Part B, patients must be resistant or intolerant to hydroxyurea.

This study is a global, open-label Phase 2a/2b study to determine the efficacy and safety of KRT-232. In Part A of the study, patients will be randomly assigned to 5 arms with 2 different doses and 3 different dosing schedules of KRT 232. In Part B of the study, patients will be randomized either to treatment with KRT-232 administered at the recommended dose and schedule from Part A or to treatment with ruxolitinib.

lead_sponsor: Kartos Therapeutics, Inc.

location_country:
Poland
Spain
Hungary
United States
France
Germany

location_facility: Dolnośląskie Centrum Transplantacji Komórkowych z Krajowym Bankiem Dawców Szpiku

location_facility: Békés Megyei Központi Kórház Pándy Kálmán Tagkórház

location_facility: Washington University School of Medicine

location_facility: The Ohio State University Comprehensive Cancer Center

location_facility: University of Southern California Norris Comprehensive Cancer Center

location_facility: Hospital Universitario de Gran Canaria Doctor Negrin

location_facility: Gemeinschaftspraxis Haematologie - Onkologie - Hauptstelle

location_facility: Center Hospitalier Universitaire d'Angers

location_facility: Hospital Universitario Virgen de la Victoria


acronym: CAPtivate

title: A Long-Term Follow-up Study to Evaluate Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
United Kingdom
Spain
Canada
United States
France

location_facility: University Hospital Cleveland Medical Center/Case Western Reserve University

location_facility: Hospital Universitario de Cruces. Servicio de Pediatria

location_facility: Hospital Clinico Universitario de Santiago

location_facility: Queen Elizabeth Hospital, Department of Endocrinology

summary: Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.


title: A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy

lead_sponsor: Wave Life Sciences Ltd.

location_country:
United Kingdom
Italy
Canada
United States
France
Belgium
Netherlands

location_facility: London Health Sciences Centre - Hospital

location_facility: University Hospitals Bristol NHS Foundation Trust

location_facility: Radbound University Nijmegen Medical Care

location_facility: UCL Institute of Child Health & Great Ormond Street Hospital for Children

location_facility: U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud

summary: This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.


title: A Prospective, Randomized, Active-Controlled, Single-blind, Parallel Group Clinical Trial to Evaluate the Safety and Efficacy of Fibrin Sealant Grifols (FS Grifols) as an Adjunct to Haemostasis During Surgery in Paediatric Subjects

summary:
This is a prospective, randomized, active-controlled, single-blind, parallel group clinical trial to evaluate the safety and efficacy of FS Grifols as an adjunct to hemostasis during surgery in pediatric subjects.

Pediatric subjects (<18 years of age) requiring an elective (non-emergent), open (non-laparoscopic), pelvic, abdominal, or thoracic (non-cardiac) surgical procedure, wherein a target bleeding site (TBS) is identified, and a topical hemostatic agent is indicated, will be eligible to participate in the clinical trial.

The study treatments will be applied on the cut parenchymous surface of a solid organ (ie, liver) and in soft tissue (ie, fat, muscle, or connective tissue).

lead_sponsor: Grifols Therapeutics LLC

location_country:
Serbia
United Kingdom
Hungary
United States
France
Romania
Bulgaria

location_facility: Birmingham Children's Hospital NHS Foundation Trust

location_facility: Semmelweis University, 2nd Dpt of Paediatrics

location_facility: Clinical Centre Nis, Clinic for Pediatric Surgery and Orthopedics

location_facility: Insititute for Health Protection of Children and Youth

location_facility: Mother and Child Healthcare Institute of Serbia "Dr Vukan Cupic"

location_facility: Spitalul Clinic de Urgenta pentru Copii "Grigore Alexandrescu"

location_facility: University of Pécs Clinical Centre, Pediatric Clinic


status: Terminated

title: A Phase 1/2, Open Label, Multicenter Study to Investigate the Safety, Pharmacokinetics, and Efficacy of TAS0728, an Oral Covalent Binding Inhibitor of HER2, in Subjects With Advanced Solid Tumors With HER2 or HER3 Abnormalities

lead_sponsor: Taiho Oncology, Inc.

ReferenceCitation: Piha-Paul SA, Azaro A, Arkenau HT, Oh DY, Galsky MD, Pal SK, Hamada K, He Y, Yamamiya I, Benhadji KA, Hollebecque A. A first-in-human phase I study of TAS0728, an oral covalent binding inhibitor of HER2, in patients with advanced solid tumors with HER2 or HER3 aberrations. Invest New Drugs. 2021 Oct;39(5):1324-1334. doi: 10.1007/s10637-021-01104-7. Epub 2021 Mar 27.

ReferenceCitation: Irie H, Ito K, Fujioka Y, Oguchi K, Fujioka A, Hashimoto A, Ohsawa H, Tanaka K, Funabashi K, Araki H, Kawai Y, Shimamura T, Wadhwa R, Ohkubo S, Matsuo K. TAS0728, A Covalent-binding, HER2-selective Kinase Inhibitor Shows Potent Antitumor Activity in Preclinical Models. Mol Cancer Ther. 2019 Apr;18(4):733-742. doi: 10.1158/1535-7163.MCT-18-1085. Epub 2019 Feb 20.

location_country:
United Kingdom
United States
Spain
France

location_facility: City of Hope Comprehensive Cancer Center

summary: This is a First-in-Human (FIH), 2-part, Phase 1/2, open-label, multicenter study design to evaluate the safety, tolerability, PK, pharmacodynamics, PGx, and efficacy of TAS0728. This study consists of Phase 1 and Phase 2 components in subjects with advanced solid tumors with HER2 or HER3 overexpression, amplification, or mutation who have progressed despite standard therapy or for which no standard therapy exists, particularly urothelial cancer, biliary tract cancer, metastatic breast cancer, non-small cell lung cancer and colorectal cancer.


title: A Multicenter Study to Explore the Mechanism of Acquired Immune Escape In Patients With Metastatic Cancer Progressing on Checkpoint Inhibitor Therapy

summary: The purpose of this study is to identify mechanisms associated with acquired immune escape by comparing baseline and at-progression tissue samples from participants who derive clinical benefit from CPI treatment of metastatic cancer or hematologic malignancies.

primary_purpose: Basic Science

lead_sponsor: Hoffmann-La Roche

location_country:
United Kingdom
Spain
Korea, Republic of
United States
France

location_facility: Seoul National University Hospital; Department of Oncology

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: Washington University School of Medicine; Dept of Medicine/Div of Medical Oncology

location_facility: Asan Medical Center, Uni Ulsan Collegemedicine; Dept.Internal Medicine / Divisionhematology/Oncology

location_facility: Institut Universitaire du Cancer - Oncopole Toulouse (IUCT-O)

location_facility: Clinica Universitaria de Navarra; Servicio de oncología

location_facility: Herbert Irving Comprehensive Cancer Center; Herbert Irving Pavillion

intervention_type: Procedure


title: Clinical Trial Evaluation of the Percutaneous 4Tech TriCinch Coil Tricuspid Valve Repair System

summary:
The objective of the study is to generate safety and performance data for the 4Tech TriCinch Coil System in symptomatic patients suffering from significant functional tricuspid regurgitation with annular dilatation.

The TriCinch Coil System is a percutaneous catheter-based medical device for tricuspid valve repair.

lead_sponsor: 4Tech Cardio Ltd.

location_country:
Denmark
United Kingdom
Australia
Netherlands
France
Belgium
Germany

location_facility: Brighton & Sussex University Hospitals - Sussex County Hospital


title: Phase 1b, Multi-arm, Open-label Study of PDR001 and/or MBG453 in Combination With Decitabine in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

summary: To characterize the safety and tolerability of 1) MBG453 as a single agent or in combination with PDR001 or 2) PDR001 and/or MBG453 in combination with decitabine or azacitidine in AML and intermediate or high- risk MDS patients, and to identify recommended doses for future studies.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Spain
United Kingdom
Australia
Netherlands
Finland
United States
France
Germany

location_facility:
Novartis Investigative Site
Oregon Health and Science University
MD Anderson Cancer Center
Massachusetts General Hospital


title: A Disease Registry for Patients With Tenosynovial Giant Cell Tumors (TGCT), Also Known as Pigmented Villonodular Synovitis (PVNS) and Giant Cell Tumor of the Tendon Sheath (GCT-TS)

acronym: TOP

summary:
TGCT is a rare disease that is difficult to manage, surgical resection is the primary treatment currently available. To date no disease registry exists and there is little data available detailing the management of patients with diffuse TGCT, the burden of diffuse TGCT for patients (including pain, joint stiffness, swelling, reduced mobility and quality of life) or the economic impact of diffuse TGCT.

This study aims to collect data by an observational disease registry involving no intervention to the patient or changes to investigators treatment decisions.

ReferenceCitation: Bernthal NM, Spierenburg G, Healey JH, Palmerini E, Bauer S; TOPP Study Group, Gelderblom H, Staals EL, Lopez-Bastida J, Fronk EM, Ye X, Laeis P, van de Sande MAJ. The diffuse-type tenosynovial giant cell tumor (dt-TGCT) patient journey: a prospective multicenter study. Orphanet J Rare Dis. 2021 Apr 29;16(1):191. doi: 10.1186/s13023-021-01820-6.

ipd_sharing_description: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/

lead_sponsor: Daiichi Sankyo, Inc.

location_country:
Spain
Italy
Austria
Netherlands
United Kingdom
United States
France
Germany

location_facility: Istituto Nazionale Tumori-Foundazione IRCCS

location_facility: University of Texas MD Anderson Cancer Center


title: The RELIEF Trial: REduction or eLimination of mItral rEgurgitation in Degenerative or Functional Mitral Regurgitation With the CardiAQ-Edwards™ Transcatheter Mitral Valve

acronym: RELIEF

summary: Clinical study to evaluate the safety and performance of the CardiAQ-Edwards™ Transcatheter Mitral Valve

status: Withdrawn

lead_sponsor: Edwards Lifesciences

location_country: Denmark Italy Netherlands Canada Switzerland France Germany

location_facility: St. Paul's Hospital, University of British Columbia

location_facility: Centre Hospitalier Régional Universitaire de Lille

location_facility: Centre de recherche Institut Universitaire de Cardiologie et de Pneumologie de Québec (CRIUCPQ)


title: INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger

acronym: INCEPTUS

summary: This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger. Many of these clinically relevant measures have not yet been routinely assessed in this population and may provide important insight on the natural history of XLMTM and for future evaluation of potential therapies.

lead_sponsor: Astellas Gene Therapies

location_country:
United Kingdom
Canada
United States
France
Germany

location_facility: National Institute of Neurological Disorders and Stroke/NIH Porter

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago


title: A Randomised, Double-blind, Placebo-controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients

acronym: IMPALA

summary: This study evaluates inhaled molgramostim (recombinant human granulocyte macrophage-colony stimulating factor [rhGM-CSF]) in the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) patients. A third of the patients will receive inhaled molgramostim once daily for 24 weeks, a third will receive inhaled molgramostim intermittently (7 days on, 7 days off) for 24 weeks and a third will receive inhaled matching placebo for 24 weeks.

ReferenceCitation: Trapnell BC, Inoue Y, Bonella F, Morgan C, Jouneau S, Bendstrup E, Campo I, Papiris SA, Yamaguchi E, Cetinkaya E, Ilkovich MM, Kramer MR, Veltkamp M, Kreuter M, Baba T, Ganslandt C, Tarnow I, Waterer G, Jouhikainen T; IMPALA Trial Investigators. Inhaled Molgramostim Therapy in Autoimmune Pulmonary Alveolar Proteinosis. N Engl J Med. 2020 Oct 22;383(17):1635-1644. doi: 10.1056/NEJMoa1913590. Epub 2020 Sep 7.

lead_sponsor: Savara Inc.

location_country:
Denmark
Portugal
Spain
Greece
Italy
Australia
Netherlands
Russian Federation
United Kingdom
Switzerland
Korea, Republic of
United States
France
Israel
Turkey
Japan
Germany
Slovakia

location_facility: Niigata University Medical and Dental Hospital

location_facility: Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: National Hospital Organization Kinki-Chuo

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Samsung Medical Center, Division of Pulmonary and Critical Care Medicine

location_facility: Universitätsklinikum Schleswig-Holstein Zentralklinikum, Lübeck Medizinische Klinik III, Pneumologie

location_facility: Asklepios Fachkliniken München - Gauting

location_facility: II. Pulmonary Department National Institut for TB, Lung Diseases and Chest Surgery

location_facility: Westdeutsches Lungenzentrum am Universitätsklinikum Essen

location_facility: Asan Medical Center, Division of Pulmonary and Critical Care Medicine

location_facility: Kanagawa Cardiovascular and Respiratory Center

location_facility: Thoraxklinik am Universitätsklinikum Heidelberg