title: European Network-Paediatric Hodgkin Lymphoma Study Group (EuroNet-PHL) Second International Inter-Group Study for Classical Hodgkin Lymphoma in Children and Adolescents

summary: The EuroNet-PHL-C2 trial is an international, multicentre, randomised controlled trial with the aims to reduce the indication for radiotherapy in newly diagnosed patients with classical Hodgkin lymphoma without compromising cure rates and to investigate a chemotherapy intensification randomisation in intermediate and advanced classical Hodgkin lymphoma to compensate for reduction in radiotherapy.

lead_sponsor: University of Giessen

location_country:
Denmark
Poland
Spain
Ireland
Austria
Australia
Italy
Netherlands
New Zealand
Switzerland
United Kingdom
Sweden
France
Israel
Belgium
Norway
Germany
Slovakia
Czechia

location_facility: Royal Children's Hospital and Monash Medical Centre Royal Children's Hospital

location_facility: Paediatric haemato-oncology, University Hospitals of Leuven

location_facility: Pediatric Radiotherapy and Youth Area Unit C.R.O. - Centro di Riferimento Oncologico IRCCS

location_facility: Princess Máxima Center for pediatric oncology

location_facility: Tel Aviv University Schneider Children's Medical Center of Israel The Rina Zaizov Pediatric Hematology Oncology Division

location_facility: Clinic of Pediatric Oncology University Children's Hospital

location_facility: Service d'Oncohématologie, Hopital d'Ènfants Armand Trousseau

location_facility: Dpt. of Pediatric Hematology and Oncology, Faculty Hospital Motol

location_facility: Starship Blood and Cancer Centre, Starship Children's Hospital

location_facility: Department of Medical Oncology Oslo University Hospital

location_facility: Pediatric Hematology & Oncology Children´s University Hospital

location_facility: CHUV - Centre Hospitalier Universitaire Vaudois = LS, Départment femme - meré - enfant, Service de pédiatrie, Unité d'hématologie-oncologie pédiatrique

location_facility: Head of Department of Pediatric Oncology and Hematology, Polish-American Pediatric Institute, Jagiellonian University Medical Faculty

location_facility: Sección de Onco-Hematología Pediátrica Hospital Universitario Virgen Macarena y Virgen del Rocío

location_facility: Department of Pediatric Hematology/Oncology (5054) The Child and Youth Clinic, University Hospital of Copenhagen


status: Completed

primary_purpose: Treatment

intervention_type: Drug

title: A Multicenter, Open-label, Follow-up Study to Assess the Long-term Use of Lacosamide (Flexible Dose From 200 to 600 mg/Day) Used as Monotherapy in Subjects Who Completed SP0994 and Received Lacosamide Monotherapy Treatment

study_completion_date_type: Actual

lead_sponsor: UCB Biopharma S.P.R.L.

type: EudraCT Number

source: euctr

study_start_date_type: Actual

study_type: Interventional

ReferenceType: result

ReferenceCitation: Ben-Menachem E, Dominguez J, Szász J, Beller C, Howerton C, Jensen L, McClung C, Roebling R, Steiniger-Brach B. Long-term safety and tolerability of lacosamide monotherapy in patients with epilepsy: Results from a multicenter, open-label trial. Epilepsia Open. 2021 Sep;6(3):618-623. doi: 10.1002/epi4.12522. Epub 2021 Aug 2.

enrollment_type: Actual

location_country:
Poland
Romania
Latvia
Russian Federation
Finland
Switzerland
Ukraine
Sweden
Philippines
Korea, Republic of
France
Mexico
Japan
Germany
Bulgaria

summary: Study is conducted to evaluate the long-term safety and tolerability of lacosamide (LCM) in patients receiving LCM in SP0994 [NCT01465997]. The study will enable collection of additional monotherapy safety data, and will facilitate access to treatment until commercial availability for monotherapy use.

lead_sponsor_type: industriel

status_simplified: Completed


title: The Nutrition Researcher Cohort 2014 Study; New Standardized Self-quantification Methodologies Serving Both Research and Personal Health Maintenance

summary:
The primary objective of the planned Nutrition Research Cohort (NRC) n250 study is to develop and evaluate the open access Nutrition Researcher Cohort for gathering personal health data from nutrition researchers, including analytical methods, standards and operation procedures, data infrastructure, ethical and privacy aspects, and governance.

Besides, the study aims to exploit and analyse data on food, nutrient and bioactive compound intake and exposure, biomarkers for food, health and/or disease and health and/or disease related measurements to study the relation between nutrition, health and development of disease (on individual level).

In addition, data will be used to develop applications that visualise personal health risks based for example on (validated) recommendations and applications that predict individual health risks.

The study is designed as an open, one-group, exploratory cohort study. The total NRC cohort will be composed of about 250 life sciences employees and students from different, predominantly European, countries. This allows optimal involvement of participants in shaping all aspects of the cohort and the ownership of data. We aim to recruit about in total 20 male and female scientists per participating country.

status: Active, not recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

ipd_sharing: Yes

ipd_sharing_description:
An internal review board, consisting of Principal Investigators from all participating countries, will review all data requests.

A data request format is available that should be used for filing such a request. The format requires stating the research questions that should be answered with the requested data, type of data, populations requirements (exclusion criteria), data analysis and public disclosure.

All study participants have the ability to withdraw their data from use for answering specific research questions at any time.

lead_sponsor: TNO

location_country:
Denmark
Spain
Ireland
Austria
Italy
United Kingdom
Finland
Switzerland
France
Belgium
Netherlands
Czechia

location_facility: INRA - L'Institut Nationel de la Recherche Agronomique

location_facility: CRA-NUT - Centro di Ricerca per gli Alimenti e la Nutrizione

intervention_type: Other

status_simplified: Ongoing


title: A Phase 1 First-in-Human Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AMG 228 in Subjects With Selected Advanced Solid Tumors

summary: The purpose of this study is to evaluate the safety, pharmacokinetics, anti-tumor activity, and identify a tolerable dose of AMG 228 in subjects with advanced solid tumors.

status: Terminated

ReferenceType: background

ReferenceCitation: Tran B, Carvajal RD, Marabelle A, Patel SP, LoRusso PM, Rasmussen E, Juan G, Upreti VV, Beers C, Ngarmchamnanrith G, Schöffski P. Dose escalation results from a first-in-human, phase 1 study of glucocorticoid-induced TNF receptor-related protein agonist AMG 228 in patients with advanced solid tumors. J Immunother Cancer. 2018 Sep 25;6(1):93. doi: 10.1186/s40425-018-0407-x.

lead_sponsor: Amgen

location_country:
Australia
United States
France
Belgium
Germany

status_simplified: Unknown


title: Aortic Valve Replacement Versus Conservative Treatment in Asymptomatic Severe Aortic Stenosis: (AVATAR Trial): A Multicentre Randomized Controlled Trial

acronym: AVATAR

summary: Whether to intervene in asymptomatic patients with severe aortic stenosis and normal left ventricular ejection fraction remains controversial. The investigators therefore try to compare clinical outcomes of elective aortic valve replacement to conventional treatment and watchful waiting strategy in a prospective randomized trial.

design_allocation: Randomized

ReferenceCitation: Banovic M, Iung B, Bartunek J, Asanin M, Beleslin B, Biocina B, Casselman F, da Costa M, Deja M, Gasparovic H, Kala P, Labrousse L, Loncar Z, Marinkovic J, Nedeljkovic I, Nedeljkovic M, Nemec P, Nikolic SD, Pencina M, Penicka M, Ristic A, Sharif F, Van Camp G, Vanderheyden M, Wojakowski W, Putnik S. Rationale and design of the Aortic Valve replAcemenT versus conservative treatment in Asymptomatic seveRe aortic stenosis (AVATAR trial): A randomized multicenter controlled event-driven trial. Am Heart J. 2016 Apr;174:147-53. doi: 10.1016/j.ahj.2016.02.001. Epub 2016 Feb 9.

ReferenceCitation: Banovic M, Iung B, Bartunek J, Penicka M, Vanderheyden M, Casselman F, van Camp G, Nikolic S, Putnik S. The Aortic Valve replAcemenT versus conservative treatment in Asymptomatic seveRe aortic stenosis (AVATAR trial): A protocol update. Am Heart J. 2018 Jan;195:153-154. doi: 10.1016/j.ahj.2017.10.005. Epub 2017 Oct 14.

ReferenceCitation: Banovic M, Putnik S, Penicka M, Doros G, Deja MA, Kockova R, Kotrc M, Glaveckaite S, Gasparovic H, Pavlovic N, Velicki L, Salizzoni S, Wojakowski W, Van Camp G, Nikolic SD, Iung B, Bartunek J; AVATAR Trial Investigators*. Aortic Valve Replacement Versus Conservative Treatment in Asymptomatic Severe Aortic Stenosis: The AVATAR Trial. Circulation. 2022 Mar;145(9):648-658. doi: 10.1161/CIRCULATIONAHA.121.057639. Epub 2021 Nov 13. Erratum in: Circulation. 2022 Mar;145(9):e761.

ReferenceType: derived

ReferenceCitation: Zelis JM, Tonino PAL, Pijls NHJ, De Bruyne B, Kirkeeide RL, Gould KL, Johnson NP. Coronary Microcirculation in Aortic Stenosis: Pathophysiology, Invasive Assessment, and Future Directions. J Interv Cardiol. 2020 Jul 22;2020:4603169. doi: 10.1155/2020/4603169. eCollection 2020. Review.

lead_sponsor: Clinical Centre of Serbia

location_country: Croatia Poland Serbia Ireland Italy Lithuania France Belgium Czechia

location_facility: Institute for Clinical and Experimental Medicine (IKEM)

location_facility: Insitute for Cardiovascular Diseases "Sremska Kamenica"

location_facility: Città della Salute e della Scienza di Torino

location_facility: University Clinical Center ''Sestre milosrdnice''

location_facility: Vilnius University Hospital Santariskiu klinikos

intervention_type: Procedure


title: Prospective Multicenter Evaluation of a New Predictive Model for the Progression of Adolescent Idiopathic Scoliosis

summary:
Scoliosis is a three-dimensional deformity affecting the orientation and position of the spine. Locally, the shape of the vertebra is also affected. The most common form is adolescent idiopathic scoliosis (AIS) with a prevalence of 1-3% affecting primarily young adolescent females. AIS can either be treated using a brace and in some cases necessitate surgical correction to prevent progressive deformity. Risk factors for progression include female gender, curve magnitude and location, skeletal maturity and growth velocity. However, these risk factors have been shown to be inconsistent in predicting curve progression. Over the past 6 years, the investigators have developed a predictive model of the final Cobb angle in AIS based on 3D spinal parameters. This analysis was based on a prospective cohort of 195 patients that were enrolled upon their initial visit and followed until maturity. This predictive model has a determination coefficient of 0.702. The proposed new study aims at refining and testing the external validity of this model in a larger cohort. The next step towards using the new model in the clinical setting is to redesign the model and to externally validate the model by measuring the agreement between the new method and the traditional Cobb angle at maturity in a larger multicenter study. The objective of this study is to characterize the risk of scoliosis progression based on local three-dimensional vertebral and pelvic measurements present on initial evaluation. Three-dimensional reconstructions will be derived from stereo-radiographs acquired with a new biplanar low-dose radiographic system installed in all 8 clinical sites (EOS system, EOS-Imaging, Paris). These calibrated radiographs will then be used to reconstruct the vertebrae and intervertebral disks at each level as well as the geometry of the pelvis. A series of local and regional parameters will then be calculated from these 3D reconstructions. Correlation analysis will help determine if intervertebral disk wedging, vertebral wedging, transverse plane rotation or pelvic geometry can be used as early predictors of curve progression in AIS.

Identifying a new 3D measure of scoliosis associated with rapid curve progression could help predict which curves need early treatment to prevent further progression. The ultimate goal of this research project will be to validate this new predictive model and finally transfer this new predictive tool in the hands of clinicians treating AIS.

status: Recruiting

enrollment_type: Anticipated

lead_sponsor: St. Justine's Hospital

location_country:
Hong Kong
Canada
United States
France
Japan
Singapore

location_facility:
Rady Children's Hospital
Mayo Clinic
Robert Debré Hospital
National University
Queen Mary Hospital
Alfred I. duPont Hospital for Children
Miami Children's Hospital
The Meijo Hospital
St Justine's Hospital

lead_sponsor_type: academique


title: An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment

lead_sponsor: Shire

ReferenceCitation: Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D; SHP609-302 study group. Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II. Mol Genet Metab. 2022 Sep-Oct;137(1-2):92-103. doi: 10.1016/j.ymgme.2022.07.016. Epub 2022 Aug 2.

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

location_country:
Spain
United Kingdom
Australia
Canada
United States
France
Mexico

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

location_facility: Children's Hospital and Research Center at Oakland

location_facility: Hospital Infantil Universitario Niño Jesus

summary: This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT treatment for those that did not receive idursulfase IT treatment in Study HGT-HIT-094.


title: A Phase 2, Open-Label Extension, Efficacy and Safety Study of a Retinoic Acid Receptor Gamma (RARγ) Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)

summary:
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by heterotopic ossification (HO), i.e., abnormal bone formation, often associated with painful, recurrent episodes of soft tissue swelling (flare-ups). Lesions begin in early childhood and lead to progressive ankyloses of major joints with resultant loss of movement.

In this study, the ability of different palovarotene dosing regimens to prevent the formation of new HO will be evaluated in adult and pediatric participants with FOP.

design_allocation: Non-Randomized

ReferenceCitation: Shimono K, Tung WE, Macolino C, Chi AH, Didizian JH, Mundy C, Chandraratna RA, Mishina Y, Enomoto-Iwamoto M, Pacifici M, Iwamoto M. Potent inhibition of heterotopic ossification by nuclear retinoic acid receptor-γ agonists. Nat Med. 2011 Apr;17(4):454-60. doi: 10.1038/nm.2334. Epub 2011 Apr 3. Erratum in: Nat Med. 2012 Oct;18(10):1592.

ipd_sharing: No

lead_sponsor: Clementia Pharmaceuticals Inc.

location_country:
United Kingdom
Australia
Argentina
United States
France

location_facility: University of Pennsylvania, Center for FOP & Related Bone Disorders

location_facility: The Royal National Orthopaedic Hospital, Brockley Hill

location_facility: Queensland University of Technology (QUT) Institute of Health and Biomedical Innovation (IHBI)

location_facility: Hôpital Necker-Enfants Malades, Department of Genetics

location_facility: University of California San Francisco, Division of Endocrinology and Metabolism

location_facility: Hospital Italiano de Buenos Aires, Department of Pediatrics


type: Other Identifier

title: BETAEVAL_Global- The New BETACONNECT® Auto-injector: Adherence and EVALuation of MS Patients Treated With Betaferon®

summary:
Considering the significance of an early and consequent Multiple Sclerosis (MS) treatment as well as the challenge to achieve high adherence to treatment, evaluating the benefits of any new measure to improve adherence is important. The data storage capabilities of the BETACONNECT device, including the automated recording of injections, will facilitate the collection of reliable data on patient's injection behavior and adherence, which should be unaffected by recall bias or reporting bias.

To better understand the utilities of the new BETACONNECT device and characterize its contribution to adherence, we plan to prospectively follow-up MS patients using this device for 24 weeks. The study will take place in a real-life setting in Neurology centers across Europe.

lead_sponsor: Bayer

location_country:
Croatia
Bosnia and Herzegovina
Spain
Greece
Austria
Italy
Switzerland
Hungary
France
Belgium
Czechia

intervention_type: Device


title: Multi-center Phase 2 Study to Assess the Safety, Tolerability and Early Signs of Efficacy of Tid Orally Administered BAY63-2521 in Adult Delta F508 Homozygous Cystic Fibrosis Patients

summary: Assessment of the safety, tolerability and early signs of efficacy of three times a day orally administered BAY63-2521 in adult delta F508 homozygous Cystic Fibrosis patients not on treatment with Orkambi

ReferenceCitation: Derichs N, Taylor-Cousar JL, Davies JC, Fajac I, Tullis E, Nazareth D, Downey DG, Rosenbluth D, Malfroot A, Saunders C, Jensen R, Solomon GM, Vermeulen F, Kaiser A, Willmann S, Saleh S, Droebner K, Sandner P, Bear CE, Hoffmann A, Ratjen F, Rowe SM; Rio-CF Study Group. Riociguat for the treatment of Phe508del homozygous adults with cystic fibrosis. J Cyst Fibros. 2021 Nov;20(6):1018-1025. doi: 10.1016/j.jcf.2021.07.015. Epub 2021 Aug 19.

location_country:
United Kingdom
Netherlands
Canada
United States
France
Belgium
Germany


title: A Multicenter Randomized Trial, Comparing a 25G EUS Fine Needle Aspiration (FNA) Device With a 20G EUS ProCore Fine Needle Biopsy (FNB) Device

acronym: ASPRO

summary: The aim of this study is to compare the diagnostic accuracy of two EUS-guided tissue acquisition devices; the 25G Echotip Ultra Fine Needle Aspiration (FNA) device and the 20G Echotip ProCore Fine Needle Biopsy (FNB) device.

primary_purpose: Diagnostic

ReferenceCitation: van Riet PA, Giorgio Arcidiacono P, Petrone M, Quoc Nguyen N, Kitano M, Chang K, Larghi A, Iglesias-Garcia J, Giovannini M, van der Merwe S, Santo E, Baldaque-Silva F, Bucobo JC, Bruno MJ, Aslanian HR, Cahen DL, Farrell J. Combined versus single use 20 G fine-needle biopsy and 25 G fine-needle aspiration for endoscopic ultrasound-guided tissue sampling of solid gastrointestinal lesions. Endoscopy. 2020 Jan;52(1):37-44. doi: 10.1055/a-0966-8755. Epub 2019 Jul 22.

ReferenceCitation: van Riet PA, Larghi A, Attili F, Rindi G, Nguyen NQ, Ruszkiewicz A, Kitano M, Chikugo T, Aslanian H, Farrell J, Robert M, Adeniran A, Van Der Merwe S, Roskams T, Chang K, Lin F, Lee JG, Arcidiacono PG, Petrone M, Doglioni C, Iglesias-Garcia J, Abdulkader I, Giovannini M, Bories E, Poizat F, Santo E, Scapa E, Marmor S, Bucobo JC, Buscaglia JM, Heimann A, Wu M, Baldaque-Silva F, Moro CF, Erler NS, Biermann K, Poley JW, Cahen DL, Bruno MJ. A multicenter randomized trial comparing a 25-gauge EUS fine-needle aspiration device with a 20-gauge EUS fine-needle biopsy device. Gastrointest Endosc. 2019 Feb;89(2):329-339. doi: 10.1016/j.gie.2018.10.026. Epub 2018 Oct 24.

lead_sponsor: Foundation for Liver Research

location_country:
Spain
Italy
Australia
Sweden
United States
France
Israel
Belgium
Japan
Netherlands

location_facility: University Hospital of Santiago de Compostella


title: Patient-Specific Simulation Used for Case Rehearsal Prior to EVAR Procedure - Evaluation of Clinical Performance With and Without Prior Rehearsal Using the Procedure Rehearsal Studio. Multi-center Randomized Study

acronym: PRS

summary: The purpose of this study is to evaluate the procedural and clinical benefits of patient specific pre-procedure rehearsal for operators with various experience levels as a tool for optimizing Endovascular Aneurysm Repair (EVAR) procedures.

status: Unknown status

lead_sponsor: Simbionix US Corporation

location_country:
United States
Italy
France

location_facility: University of South Florida, Tampa General Hospital


title: An Open-label, Randomised, Multicentre, Single-dose, Parallel Group Trial to Evaluate Pharmacokinetics and Pharmacodynamics of Empagliflozin in Children and Adolescents From 10 to Less Than 18 Years of Age With Type 2 Diabetes Mellitus

lead_sponsor: Boehringer Ingelheim

ReferenceCitation: Bjornstad P, Laffel L, Tamborlane WV, Simons G, Hantel S, von Eynatten M, George J, Marquard J, Cherney DZI. Acute Effect of Empagliflozin on Fractional Excretion of Sodium and eGFR in Youth With Type 2 Diabetes. Diabetes Care. 2018 Aug;41(8):e129-e130. doi: 10.2337/dc18-0394. Epub 2018 Jun 25.

ReferenceCitation: Laffel LMB, Tamborlane WV, Yver A, Simons G, Wu J, Nock V, Hobson D, Hughan KS, Kaspers S, Marquard J. Pharmacokinetic and pharmacodynamic profile of the sodium-glucose co-transporter-2 inhibitor empagliflozin in young people with Type 2 diabetes: a randomized trial. Diabet Med. 2018 Aug;35(8):1096-1104. doi: 10.1111/dme.13629. Epub 2018 May 6.

location_country:
United States
South Africa
Israel
France
Mexico

location_facility: 1245.87.01013 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.97203 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.33001 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.01002 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.01004 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.01001 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.52001 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.27002 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.01012 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.27003 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.97202 Boehringer Ingelheim Investigational Site

summary: The aim of the study is to generate pharmacokinetic and pharmacodynamic data to identify the safe-effective dose of empagliflozin in children and adolescents aged 10 to less than 18 years with type 2 diabetes mellitus.


title: Long Term Experience With Abatacept in Routine Clinical Practice

acronym: ACTION

summary: The purpose of this study is to estimate the retention rate of Abatacept over 24 months in routine clinical practice in every participating country depending on the treatment line.

ReferenceCitation: Alten R, Mariette X, Lorenz HM, Nüßlein H, Galeazzi M, Navarro F, Chartier M, Heitzmann J, Poncet C, Rauch C, Le Bars M. Predictors of abatacept retention over 2 years in patients with rheumatoid arthritis: results from the real-world ACTION study. Clin Rheumatol. 2019 May;38(5):1413-1424. doi: 10.1007/s10067-019-04449-w. Epub 2019 Feb 21. Erratum in: Clin Rheumatol. 2019 Apr 11;:.

ReferenceCitation: Alten R, Mariette X, Lorenz HM, Galeazzi M, Cantagrel A, Nüßlein HG, Chartier M, Elbez Y, Rauch C, Le Bars M. Real-world predictors of 12-month intravenous abatacept retention in patients with rheumatoid arthritis in the ACTION observational study. RMD Open. 2017 Dec 29;3(2):e000538. doi: 10.1136/rmdopen-2017-000538. eCollection 2017.

ReferenceCitation: Alten R, Burkhardt H, Feist E, Krüger K, Rech J, Rubbert-Roth A, Voll RE, Elbez Y, Rauch C. Abatacept used in combination with non-methotrexate disease-modifying antirheumatic drugs: a descriptive analysis of data from interventional trials and the real-world setting. Arthritis Res Ther. 2018 Jan 2;20(1):1. doi: 10.1186/s13075-017-1488-5.

ReferenceCitation: Alten R, Nüßlein HG, Mariette X, Galeazzi M, Lorenz HM, Cantagrel A, Chartier M, Poncet C, Rauch C, Le Bars M. Baseline autoantibodies preferentially impact abatacept efficacy in patients with rheumatoid arthritis who are biologic naïve: 6-month results from a real-world, international, prospective study. RMD Open. 2017 Feb 13;3(1):e000345. doi: 10.1136/rmdopen-2016-000345. eCollection 2017.

lead_sponsor: Bristol-Myers Squibb

location_country:
Denmark
Spain
Greece
Austria
Ireland
Italy
Canada
Czech Republic
Netherlands
Switzerland
France
Belgium
Germany


title: A Phase 3b, Prospective, Randomized, Open-label, Blind Evaluator (PROBE) Study Evaluating the Efficacy and Safety of (LMW) Heparin/Edoxaban Versus Dalteparin in Venous Thromboembolism Associated With Cancer

summary: The primary objective is to demonstrate the non-inferiority of edoxaban (preceded by a short course of LMWH) compared with dalteparin for the prevention of the combined outcome of recurrent venous thromboembolism (VTE) or major bleeding in subjects with VTE associated with cancer during a 12-month study period. If non-inferiority is established, LMWH/edoxaban will be compared with dalteparin for superiority.

ReferenceCitation: Raskob GE, van Es N, Verhamme P, Carrier M, Di Nisio M, Garcia D, Grosso MA, Kakkar AK, Kovacs MJ, Mercuri MF, Meyer G, Segers A, Shi M, Wang TF, Yeo E, Zhang G, Zwicker JI, Weitz JI, Büller HR; Hokusai VTE Cancer Investigators. Edoxaban for the Treatment of Cancer-Associated Venous Thromboembolism. N Engl J Med. 2018 Feb 15;378(7):615-624. doi: 10.1056/NEJMoa1711948. Epub 2017 Dec 12.

ReferenceCitation: van Es N, Di Nisio M, Bleker SM, Segers A, Mercuri MF, Schwocho L, Kakkar A, Weitz JI, Beyer-Westendorf J, Boda Z, Carrier M, Chlumsky J, Décousus H, Garcia D, Gibbs H, Kamphuisen PW, Monreal M, Ockelford P, Pabinger I, Verhamme P, Grosso MA, Büller HR, Raskob GE. Edoxaban for treatment of venous thromboembolism in patients with cancer. Rationale and design of the Hokusai VTE-cancer study. Thromb Haemost. 2015 Nov 25;114(6):1268-76. doi: 10.1160/TH15-06-0452. Epub 2015 Aug 13.

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:
Italy
Hungary
United States
France
Belgium
Netherlands


primary_purpose: Other

title: A Multicenter Study for the Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody

lead_sponsor: Swedish Orphan Biovitrum

publications_result: Jordan MB, Locatelli F, Allen C, Cesaro S, Rizzari C, Rao A, Degar B, Garrington T, Sevilla J, Putti MC, Fagioli F, Ahlmann M, Dapena Diaz JL, Henry M, Grom A, De Benedetti F, de Min C. Post-Transplant Outcomes of Children with Primary Hemophagocytic Lymphohistiocytosis Treated with Emapalumab.Transplant Cell Ther. 2021; 27(Supplement 3): S118.

ReferenceCitation: Laveille C, Jacqmin P, de Graaf K, de Min C. Population Pharmacokinetic Analysis of Emapalumab, a Fully Human, Anti-Interferon Gamma Monoclonal Antibody, in Children with Primary Hemophagocytic Lymphohistiocytosis. Blood 2020; 136 (Supplement 1): 20

ReferenceCitation: Locatelli F, Jordan MB, Allen C, Cesaro S, Rizzari C, Rao A, Degar B, Garrington TP, Sevilla J, Putti MC, Fagioli F, Ahlmann M, Dapena Diaz JL, Henry M, De Benedetti F, Grom A, Lapeyre G, Jacqmin P, Ballabio M, de Min C. Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis. N Engl J Med. 2020 May 7;382(19):1811-1822. doi: 10.1056/NEJMoa1911326.

location_country:
United Kingdom
Italy
Spain
United States
France

location_facility: Fondazione MBBM c/o Ospedale San Gerardo Clinica Pediatrica

location_facility: Hospital Universitario Vall d'Hebron Servei de Hematologia i Oncologia

location_facility: Cincinnati Children's Hospital - Division of Immunobiology

location_facility: Sant Joan de Déu Hospital - Pediatric Rheumatology Department

location_facility: University of North Carolina at Chapel Hill

location_facility: Ospedale Pediatrico Bambino Gesu - UO Reumatologia

location_facility: Hospital Universitario Niño Jesús Servicio de Hemato-Oncología Pediátrica

location_facility: UCL Institute of Child Health Great Ormond Street Hospital

location_facility: Spectrum Health Helen Devos Children's Hospital

location_facility: Great Ormond Street Hospital - Department of Haematology

location_facility: Ospedale della Donna e del Bambino - U.O.C. Oncoematologia Pediatrica

summary: International, multicenter, long-term, follow-up study that will enrol HLH participants who have received emapalumab in previous clinical trials, in the context of the clinical development program for emapalumab or under compassionate use (CU).


acronym: INDIGO

title: Open Label Study of the Efficacy and Long Term Safety of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis

lead_sponsor: Mirum Pharmaceuticals, Inc.

ReferenceCitation: Loomes KM, Squires RH, Kelly D, Rajwal S, Soufi N, Lachaux A, Jankowska I, Mack C, Setchell KDR, Karthikeyan P, Kennedy C, Dorenbaum A, Desai NK, Garner W, Jaecklin T, Vig P, Miethke A, Thompson RJ. Maralixibat for the treatment of PFIC: Long-term, IBAT inhibition in an open-label, Phase 2 study. Hepatol Commun. 2022 Sep;6(9):2379-2390. doi: 10.1002/hep4.1980. Epub 2022 May 4.

location_country:
Poland
United States
United Kingdom
France

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: The Children's Memorial Health Institute

summary: This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus associated with PFIC.


acronym: AIM-IT

intervention_type: Biological

title: A Controlled, Randomized, Two-arm, Open-label, Assessor-blinded, Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

ReferenceCitation: Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D; HGT-HIT-094 Study Group. Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study. Mol Genet Metab. 2022 Sep-Oct;137(1-2):127-139. doi: 10.1016/j.ymgme.2022.07.017. Epub 2022 Aug 2.

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

location_facility: Children's Hospital and Research Center at Oakland

location_facility: Hospital Infantil Universitario Niño Jesus

location_facility: Women's and Children's Hospital, 72 King William Road

summary: Study HGT-HIT-094 is a multicenter study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter syndrome and cognitive impairment who have previously received and tolerated a minimum of 4 months of therapy with Elaprase.


title: A Prospective, Multicenter, Single-Arm Trial With the Lutonix Drug Coated Balloon for Treatment of Long Lesions in Femoropopliteal Arteries

summary: To demonstrate efficacy and safety of the Lutonix® Drug Coated Balloon for treatment of long TASC II Class C and D lesions (≥ 14 cm) lesions in the SFA

lead_sponsor: C. R. Bard

location_country: Austria Switzerland France Belgium Germany

location_facility: Krankenhaus Barmherzige Brüder Regensburg

location_facility: Luzerner Kantonsspital, Division of Angiology


title: The Robotic Vascular and Endovascular Registry (ROVER)

acronym: ROVER

summary:
The purpose of the registry is to gather both retrospective and prospective case data on the use of the commercially available Magellan Robotic System and Magellan Robotic Catheters in accordance with the approved intended use. For prospective cases, follow-up patient data will be collected at 14 days (± 5 days) post procedure to assess treatment success, primary patency of intended targeted vessel region, and adverse events.

The data will be analyzed for medical education, societal presentation, and/or publication by the investigators.

Over the next 2+ years, physicians who meet the selection criteria will be invited to participate in the registry.

lead_sponsor: Hansen Medical

location_country:
Germany
United States
France

location_facility:
Keck Medical Center of USC
Miami Valley Hospital
Good Samaritan Hospital
The Methodist Hospital
Miami Cardiac & Vascular Institute
Herz- und Gefäßzentrum Bad Bevensen
University Hospital of Strasbourg


type: Registry Identifier

type: U.S. NIH Grant/Contract

title: A Randomized Two-Arm Phase II Study of Trametinib Alone and in Combination With GSK2141795 in Patients With Advanced Uveal Melanoma

summary: This randomized phase II trial studies how well trametinib with or without Akt inhibitor GSK2141795 (GSK2141795) works in treating patients with uveal melanoma that has spread to other parts of the body (metastatic). Trametinib and GSK2141795 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether trametinib is more effective with or without GSK2141795 in treating patients with metastatic uveal melanoma.

lead_sponsor: National Cancer Institute (NCI)

location_country:
United States
United Kingdom
France

location_facility: Columbia University/Herbert Irving Cancer Center

location_facility: Vanderbilt University/Ingram Cancer Center

location_facility: Emory University Hospital/Winship Cancer Institute


title: An Open-Label, Single Arm, Phase 1 Study of the Pharmacokinetics and Safety of Carfilzomib in Subjects With Advanced Malignancies and Varying Degrees of Hepatic Impairment

summary: The purpose of this study is to compare the safety and efficacy of carfilzomib, including measuring the amount of the study drug in the blood at certain times following dosing. This study is being done in people with varying degrees of liver function to see if they respond differently to the study drug.

ReferenceCitation: Brown J, Plummer R, Bauer TM, Anthony S, Sarantopoulos J, De Vos F, White M, Schupp M, Ou Y, Vaishampayan U. Pharmacokinetics of carfilzomib in patients with advanced malignancies and varying degrees of hepatic impairment: an open-label, single-arm, phase 1 study. Exp Hematol Oncol. 2017 Oct 3;6:27. doi: 10.1186/s40164-017-0086-1. eCollection 2017.

location_country:
Netherlands
United States
United Kingdom
France

location_facility: Sir Bobby Robson Cancer Trials Research Centre

location_facility: Northern Ireland Cancer Trials Centre - Queen's University Belfast TriaIs Centre


intervention_type: Genetic

title: A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

lead_sponsor: bluebird bio

ReferenceCitation: Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4.

ipd_sharing_description: Bluebird bio is committed to transparency and appropriately de-identified patient-level datasets and supporting documents may be shared following attainment of applicable marketing approvals associated with this study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. Note that given the rarity of the disease and the identifiable nature of the participants in this study, it is anticipated that the sharing of data from this study may be limited. For enquiries, please contact us at datasharing@bluebirdbio.com.

location_country:
United Kingdom
Australia
Argentina
United States
France
Germany

location_facility: Great Ormond Street Hospital for Children NHS Foundation Trust

location_facility: Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center

location_facility: Boston Children's Hospital/Massachusetts General Hospital

summary: This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells were collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells were transplanted back into the participant following myeloablative conditioning. Participants in this study will be continuously followed in study LTF-304.


title: Practice of Ventilation in Critically Ill Patients Without Acute Respiratory Distress Syndrome - An International Observational Study

acronym: PRoVENT

summary:
The purpose of this international, multicenter study is to determine ventilation practice and outcomes of intubated and ventilated intensive care unit (ICU) patients. Ventilation characteristics and outcomes will be compared between patients without the acute respiratory distress syndrome (ARDS), patients at risk for ARDS, and patients with mild, moderate or severe ARDS.

Participating centres worldwide will include adult patients undergoing mechanical ventilation in the ICU during a 7-day period. Patients data will be collected during the first 7 days in the ICU, or until ICU discharge. Follow up is until day 90. Primary endpoint is the tidal volume size used during mechanical ventilation. Secondary endpoints are development of ARDS in patients without ARDS at the onset of mechanical ventilation, worsening of ARDS in patients with ARDS at the onset of mechanical ventilation, pulmonary infection, other pulmonary complications, need for tracheostomy, extra-pulmonary complications, duration of ventilation, length of ICU and hospital stay, and ICU, hospital and 90-day mortality.

ReferenceCitation: Hemmes SN, Serpa Neto A, Schultz MJ. Intraoperative ventilatory strategies to prevent postoperative pulmonary complications: a meta-analysis. Curr Opin Anaesthesiol. 2013 Apr;26(2):126-33. doi: 10.1097/ACO.0b013e32835e1242.

ReferenceCitation: Serpa Neto A, Cardoso SO, Manetta JA, Pereira VG, Espósito DC, Pasqualucci Mde O, Damasceno MC, Schultz MJ. Association between use of lung-protective ventilation with lower tidal volumes and clinical outcomes among patients without acute respiratory distress syndrome: a meta-analysis. JAMA. 2012 Oct 24;308(16):1651-9. doi: 10.1001/jama.2012.13730.

ReferenceCitation: Determann RM, Royakkers A, Wolthuis EK, Vlaar AP, Choi G, Paulus F, Hofstra JJ, de Graaff MJ, Korevaar JC, Schultz MJ. Ventilation with lower tidal volumes as compared with conventional tidal volumes for patients without acute lung injury: a preventive randomized controlled trial. Crit Care. 2010;14(1):R1. doi: 10.1186/cc8230. Epub 2010 Jan 7.

ReferenceCitation: Severgnini P, Selmo G, Lanza C, Chiesa A, Frigerio A, Bacuzzi A, Dionigi G, Novario R, Gregoretti C, de Abreu MG, Schultz MJ, Jaber S, Futier E, Chiaranda M, Pelosi P. Protective mechanical ventilation during general anesthesia for open abdominal surgery improves postoperative pulmonary function. Anesthesiology. 2013 Jun;118(6):1307-21. doi: 10.1097/ALN.0b013e31829102de.

ReferenceCitation: Serpa Neto A, Barbas CSV, Artigas A, et al. Rationale and Study Design of Provent-An International Multicenter Observational Study on Practice of Ventilation in Critically Ill Patients without ARDS. J Clin Trials 2013;3;[in press]

ReferenceCitation: Simonis FD, Barbas CSV, Artigas-Raventós A, Canet J, Determann RM, Anstey J, Hedenstierna G, Hemmes SNT, Hermans G, Hiesmayr M, Hollmann MW, Jaber S, Martin-Loeches I, Mills GH, Pearse RM, Putensen C, Schmid W, Severgnini P, Smith R, Treschan TA, Tschernko EM, Vidal Melo MF, Wrigge H, de Abreu MG, Pelosi P, Schultz MJ, Neto AS; PRoVENT investigators; PROVE Network investigators. Potentially modifiable respiratory variables contributing to outcome in ICU patients without ARDS: a secondary analysis of PRoVENT. Ann Intensive Care. 2018 Mar 21;8(1):39. doi: 10.1186/s13613-018-0385-7.

ReferenceCitation: Neto AS, Barbas CSV, Simonis FD, Artigas-Raventós A, Canet J, Determann RM, Anstey J, Hedenstierna G, Hemmes SNT, Hermans G, Hiesmayr M, Hollmann MW, Jaber S, Martin-Loeches I, Mills GH, Pearse RM, Putensen C, Schmid W, Severgnini P, Smith R, Treschan TA, Tschernko EM, Melo MFV, Wrigge H, de Abreu MG, Pelosi P, Schultz MJ; PRoVENT; PROVE Network investigators. Epidemiological characteristics, practice of ventilation, and clinical outcome in patients at risk of acute respiratory distress syndrome in intensive care units from 16 countries (PRoVENT): an international, multicentre, prospective study. Lancet Respir Med. 2016 Nov;4(11):882-893. doi: 10.1016/S2213-2600(16)30305-8. Epub 2016 Oct 4.

lead_sponsor: Prof. Dr. Marcus J. Schultz

location_country: Brazil Spain Italy Austria Netherlands France Chile Belgium Germany

location_facility: Hospital Universitari Germans Trias I Pujol

location_facility: Hospital Clínico de la Pontificia Universidad Católica de Chile


title: A Randomised, Double-blind, Placebo-controlled, Phase I/II Trial of RNActive®-Derived Cancer Vaccine (CV9104) in Asymptomatic or Minimally Symptomatic Patients With Metastatic Castrate-refractory Prostate Cancer

summary: The purpose of this study is to determine whether the new RNActive®-derived prostate cancer vaccine CV9104 prolongs survival in patients with asymptomatic or minimally symptomatic metastatic prostate cancer that is castrate resistant.

lead_sponsor: CureVac

location_country:
Poland
Spain
United Kingdom
Czech Republic
Switzerland
Sweden
France
Germany

location_facility: CHUV Centre Pluridisciplinaire d'Oncologie

location_facility: Complejo Hospitalario Universitario Santiago Departamento de Oncología

location_facility: Kantonsspital Graubünden Department Innere Medizin Hämatologie und Onkologie

location_facility: Urologische Klinik und Poliklinik der Technischen Universität München Klinikum Rechts der Isar

location_facility: Universitätsklinikum Münster Klinik und Poliklinik für Urologie

location_facility: Universitätsspital Basel Medizinische Onkologie

location_facility: Kantonsspital St. Gallen Department Innere Medizin Hämatologie Medizinische Onkologie

location_facility: Skånes Universitetssjukhus Malmö Urologmottagningen

location_facility: Szpital Sw. Elżbiety - Mokotowskie Centrum Medyczne

location_facility: NZOZ Magodent, Centrum Medyczne Ostrobramska, Oncologii Klinicznej i Chemíoterapii

location_facility: Multiscan, a.s, Oddělení klinické a radiační onkologie

location_facility: Universitätsklinikum Aachen Klinik für Urologie

location_facility: Hospital Clínico Virgen de la Victoria Campus Universitario de Teatinos s/n

location_facility: Profesorskie Centrum Medyczne OPTIMUM Wrocław

location_facility: Ortenau Klinikum Urologie und Kinderurologie

location_facility: Nottingham City Hospital Department of Oncology

location_facility: Urologische Klinik Dr. Castingius München

location_facility: Nationales Zentrum für Tumorerkrankungen Medizinische Onkologie

location_facility: Hospital de Madrid Norte Sanchinarro Centro Integral Oncológico Clara Campal (CIOCC)

location_facility: Krajská zdravotní, a.s. - Nemocnice Chomutov, o.z.Onkologické oddělení

location_facility: Thomayerova nemocnice, Urologické oddělení

location_facility: Vivantes Klinikum Am Urban Klinik für Urologie

location_facility: Szpital Uniwersytecki, Katedra i Klinika Urologii i Onkologii Urologicznej

location_facility: Universitätsklinikum Dresden Klinik und Poliklinik für Urologie

location_facility: Chirurgische Universitätsklinik Freiburg Klinik für Urologie

location_facility: Centralny Szpital Kliniczny MSWiA, Klinika Onkologii I Hematologii

location_facility: Clínica Universitaria de Navarra Departamento de Oncología

location_facility: Karolinska Universitetssjukhuset Solna Urologiska kliniken

location_facility: Fakultní nemocnice Olomouc, Urologická klinika

location_facility: Instituto Valenciano de Oncología Unidad de Investigación Clínica

location_facility: Krajská zdravotní, a.s. - Masarykova nemocnice Ústí nad Labem

location_facility: Instytut M. Curie-Skłodowskiej Centrum Onkologii


title: Clinical Study of the aura6000™ Targeted Hypoglossal Implantable Neurostimulation (THN) Sleep Therapy System

summary: The objective of the study is to confirm the safety and efficacy in patients utilizing the aura6000 System for the treatment of Obstructive Sleep Apnea (OSA).

ReferenceCitation: Zaidi FN, Meadows P, Jacobowitz O, Davidson TM. Tongue anatomy and physiology, the scientific basis for a novel targeted neurostimulation system designed for the treatment of obstructive sleep apnea. Neuromodulation. 2013 Jul-Aug;16(4):376-86; discussion 386. doi: 10.1111/j.1525-1403.2012.00514.x. Epub 2012 Aug 31. Review.

ReferenceCitation: Mwenge GB, Rombaux P, Dury M, Lengelé B, Rodenstein D. Targeted hypoglossal neurostimulation for obstructive sleep apnoea: a 1-year pilot study. Eur Respir J. 2013 Feb;41(2):360-7. doi: 10.1183/09031936.00042412. Epub 2012 May 17.

lead_sponsor: ImThera Medical, Inc.

location_country:
Italy
United States
France
Belgium
Germany

location_facility:
Advanced Sleep Research
G.B. Morgagni- L. Pierantoni Hospital
Sharp Rees Stealy Medical Center
Porter Adventist Hospital
St. Joseph-Stift
Clinique Univ. Saint-Luc
Pitié-Salpêtrière Hospital
Orange Regional Medical Center
Chicago ENT


title: Randomized Multi-Center Study Comparing No Drainage to Preoperative Biliary Drainage Using Metal Stents in Patients With Resectable Pancreatic or Periampullary Cancer

summary: The purpose of this study is to demonstrate that preoperative biliary drainage using self-expanding metal stents (SEMS) does not negatively impact overall surgical outcomes in patients undergoing pancreaticoduodenectomy for treatment of pancreatic or periampullary cancer.

lead_sponsor: Boston Scientific Corporation

location_country:
Hong Kong
Italy
Australia
United States
France
China
India
Belgium
Japan

location_facility: Prince of Wales Hospital, the Chinese University of Hong Kong

location_facility: Fondazione Policlinico Universitario Agostino Gemelli

location_facility: Xijing Hospital of Digestive Diseases Fourth Military Medical University


title: A Phase 2, Multicenter, Open-Label, Randomized Study to Investigate the Safety and Efficacy of GS-7977 and Ribavirin Administered for 48 Weeks in Patients Infected With Chronic HCV With Cirrhosis and Portal Hypertension With or Without Liver Decompensation

summary: This study will evaluate the antiviral efficacy of combination therapy with sofosbuvir (SOF) plus ribavirin (RBV) for 48 weeks in adults with compensated and decompensated chronic hepatitis C virus (HCV) infection. Approximately 50 adults will be randomized (1:1) to receive study drug for 48 weeks or take part in an untreated observational arm for the first 24 weeks followed by study drug for another 48 weeks.

lead_sponsor: Gilead Sciences

location_country:
Spain
New Zealand
Australia
United States
France


title: Noninvasive and Continuous Hemoglobin Monitoring for Surgical Blood Management

acronym: NACHO

summary:
This is a multi-center cluster-randomized trial with the following Specific Aims:

To evaluate if continuous noninvasive hemoglobin monitoring will reduce the RBC transfusions in patients undergoing surgeries associated with a significant risk of bleeding.
To evaluate if patients monitored with continuous noninvasive hemoglobin experience less frequent complications and shorter hospital stay compared with patients who are not being monitored with continuous noninvasive hemoglobin.

Accordingly, the study hypotheses are defined as follows:

The primary null hypothesis is that continuous noninvasive hemoglobin monitoring will not reduce the RBC transfusions in patients undergoing surgeries associated with a significant risk of bleeding.
The secondary hypothesis is that in patients monitored with continuous noninvasive hemoglobin, there will be earlier warning of critical drops in hemoglobin, and thus, there will be less frequent complications compared with patients who are not being monitored with continuous noninvasive hemoglobin.

lead_sponsor: Society for the Advancement of Blood Management, Inc

location_country:
Japan
United States
Italy
France

location_facility: The Ohio State University Wexner Medical Center

location_facility: Sapporo Medical University School of Medicine


title: A Multicenter, Open Label, Phase 1B Study of Escalating Doses of RO5045337 Administered Orally, With Cytarabine Administered A) Subcutaneously, or B) Intravenously, in Patients With Acute Myelogenous Leukemia (AML)

summary: This multi-center, open-label, Phase 1b study will evaluate the safety, pharmacokinetics and efficacy of RO5045337 in combination with cytarabine in patients with acute myelogenous leukemia. In Arm A, cohorts of previously untreated patients deemed unsuitable for standard induction therapy will receive escalating oral doses of RO5045377 and cytarabine 20 mg/m2 subcutaneously daily for Days 1 to 10 of each 28-day cycle. In Arm B, cohorts of patients who have relapsed or are refractory after at least one cytarabine/anthracycline containing regimen will receive escalating oral doses of RO5045377 on Days 1 to 5 and cytarabine 1 gm/m2 intravenously on Days 1 to 6 of each 28-day cycle. Patients will receive up to 4 cycles of therapy, patients in Arm A who achieve hematologic response may continue additional cycles until disease progression.

lead_sponsor: Hoffmann-La Roche

location_country:
United Kingdom
Italy
Canada
United States
France


title: Xarelto® for Long-term and Initial Anticoagulation in Venous Thromboembolism (VTE)

acronym: XALIA

summary:
Following the findings of the clinical trials in drug development, this global non-interventional cohort field study will investigate rivaroxaban under clinical practice conditions in comparison with current standard of care for patients with acute deep vein thrombosis (DVT).

The main goal is to analyze long-term safety in the use of rivaroxaban in the treatment of acute DVT in routine clinical practice.

ReferenceCitation: Ageno W, Mantovani LG, Haas S, Kreutz R, Monje D, Schneider J, Bugge JP, Gebel M, Turpie AGG. Patient Management Strategies and Long-Term Outcomes in Isolated Distal Deep-Vein Thrombosis versus Proximal Deep-Vein Thrombosis: Findings from XALIA. TH Open. 2019 Mar 26;3(1):e85-e93. doi: 10.1055/s-0039-1683968. eCollection 2019 Jan.

ReferenceCitation: Ageno W, Mantovani LG, Haas S, Kreutz R, Monje D, Schneider J, van Eickels M, Gebel M, Zell E, Turpie AG. Safety and effectiveness of oral rivaroxaban versus standard anticoagulation for the treatment of symptomatic deep-vein thrombosis (XALIA): an international, prospective, non-interventional study. Lancet Haematol. 2016 Jan;3(1):e12-21. doi: 10.1016/S2352-3026(15)00257-4. Epub 2015 Dec 8.

lead_sponsor: Bayer

location_country:
Spain
Italy
United Kingdom
Ukraine
Belgium
Portugal
Slovenia
Austria
Israel
Norway
Sweden
Canada
Switzerland
Hungary
Germany
Moldova, Republic of
Denmark
Greece
Czech Republic
France
Netherlands


title: Xarelto® on Prevention of Stroke and Non-central Nervous System Systemic Embolism in Patients With Non-valvular Atrial Fibrillation

acronym: XANTUS

summary: This is an international observational study in patients with non-valvular atrial fibrillation who are prescribed rivaroxaban under routine treatment conditions to prevent stroke or non-central nervous system systemic embolism.

ReferenceCitation: Kirchhof P, Haas S, Amarenco P, Hess S, Lambelet M, van Eickels M, Turpie AGG, Camm AJ; XANTUS Investigators*. Impact of Modifiable Bleeding Risk Factors on Major Bleeding in Patients With Atrial Fibrillation Anticoagulated With Rivaroxaban. J Am Heart Assoc. 2020 Mar 3;9(5):e009530. doi: 10.1161/JAHA.118.009530. Epub 2020 Feb 21.

ReferenceCitation: Amarenco P, Haas S, Hess S, Kirchhof P, Lambelet M, Bach M, Turpie AGG, Camm AJ. Outcomes associated with non-recommended dosing of rivaroxaban: results from the XANTUS study. Eur Heart J Cardiovasc Pharmacother. 2019 Apr 1;5(2):70-79. doi: 10.1093/ehjcvp/pvy041.

ReferenceCitation: Kirchhof P, Radaideh G, Kim YH, Lanas F, Haas S, Amarenco P, Turpie AGG, Bach M, Lambelet M, Hess S, Camm AJ; Global XANTUS program Investigators. Global Prospective Safety Analysis of Rivaroxaban. J Am Coll Cardiol. 2018 Jul 10;72(2):141-153. doi: 10.1016/j.jacc.2018.04.058.

ReferenceCitation: Camm AJ, Turpie AGG, Hess S, Amarenco P, Lambelet M, Haas S, van Eickels M, Kirchhof P; XANTUS Investigators. Outcomes after catheter ablation and cardioversion in patients with non-valvular atrial fibrillation: results from the prospective, observational XANTUS study. Europace. 2018 Jun 1;20(6):e87-e95. doi: 10.1093/europace/eux127.

ReferenceCitation: Camm AJ, Amarenco P, Haas S, Hess S, Kirchhof P, Kuhls S, van Eickels M, Turpie AG; XANTUS Investigators. XANTUS: a real-world, prospective, observational study of patients treated with rivaroxaban for stroke prevention in atrial fibrillation. Eur Heart J. 2016 Apr 7;37(14):1145-53. doi: 10.1093/eurheartj/ehv466. Epub 2015 Sep 1.

ReferenceCitation: Camm AJ, Amarenco P, Haas S, Hess S, Kirchhof P, van Eickels M, Turpie AG. XANTUS: rationale and design of a noninterventional study of rivaroxaban for the prevention of stroke in patients with atrial fibrillation. Vasc Health Risk Manag. 2014 Jul 17;10:425-34. doi: 10.2147/VHRM.S63298. eCollection 2014.

location_country:
United Kingdom
Ukraine
Belgium
Czechia
Poland
Portugal
Slovenia
Austria
Russian Federation
Israel
Norway
Sweden
Slovakia
Canada
Hungary
Germany
Moldova, Republic of
Denmark
Ireland
France
Netherlands


title: A Phase Ib/II Open-label, Multi-center Study of the Combination of MEK162 Plus AMG 479 (Ganitumab) in Adult Patients With Selected Advanced Solid Tumors

lead_sponsor: Pfizer

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

location_country:
Spain
Italy
United Kingdom
Australia
Canada
United States
France
Belgium

location_facility: University of Utah / Huntsman Cancer Institute Huntsman

location_facility: Massachusetts General Hospital Mass General 2

summary:
This is a multi-center, open-label, phase Ib/II study. First, the aim of the phase Ib part is to estimate the MTD(s) and/or to identify the recommended phase II dose(s) (RP2D) for the combination of MEK162 and AMG 479 (ganitumab), followed by the phase II part to assess the clinical efficacy and to further assess the safety of the combination in selected patient populations. The dose escalation part of the study will be guided by a Bayesian Logistic Regression Model (BLRM). At least 18 patients are expected to be enrolled in the dose escalation part.

Following MTD/ RP2D declaration, patients will be enrolled in three phase II arms to assess efficacy of the combination as well as to better understand the safety, tolerability, PK, antibody concentrations and PD of the combination at MTD/RP2D. Phase II arm 1 will consist of approximately 25 patients with KRAS-mutant colorectal adenocarcinoma. Phase II arm 2 will consist of approximately 20 patients with metastatic pancreatic adenocarcinoma. Phase II arm 3 will consist of approximately 28 patients with mutant BRAFV600 melanoma.

Patients will be treated until progression of disease, unacceptable toxicity develops, or withdrawal of informed consent, whichever occurs first. All patients will be followed up - at minimum patients must complete the safety follow-up assessments 30 days after the last dose of the study treatment.


title: An Exploratory Non-interventional Study of Ex-vivo Spiking Followed by Thromboelastography as a Method for Predicting the Efficacy of Recombinant Activated Human FVII (rFVIIa, NovoSeven®) in Adult Congenital Haemophilia A or B Patients With Inhibitors

summary: This trial is conducted in Europe and the United States of America (USA). The aim of this trial is to evaluate the basal and spiked TEG® (Thromboelastography) or ROTEM® (Thromboelastometry) profiles of frequently bleeding haemophilia subjects with inhibitors in a non-bleeding state.

lead_sponsor: Novo Nordisk A/S

location_country:
Denmark
United States
United Kingdom
France


title: Diabetes Attitudes Wishes and Needs (DAWN) 2 Study: A Multinational, Multi-stakeholder Survey Study of Psychosocial Issues in Diabetes and Patient-centred Diabetes Care

summary:
The study is conducted in Africa, Asia, Europe, Japan, North America and South America.

The multinational surveys will explore the experiences and unmet needs of people with diabetes, as well as those of family members of people with diabetes, and of healthcare professionals treating people with diabetes.

ReferenceCitation: Peyrot M, Burns KK, Davies M, Forbes A, Hermanns N, Holt R, Kalra S, Nicolucci A, Pouwer F, Wens J, Willaing I, Skovlund SE. Diabetes Attitudes Wishes and Needs 2 (DAWN2): a multinational, multi-stakeholder study of psychosocial issues in diabetes and person-centred diabetes care. Diabetes Res Clin Pract. 2013 Feb;99(2):174-84. doi: 10.1016/j.diabres.2012.11.016. Epub 2012 Dec 27.

ReferenceCitation: Nicolucci A, Kovacs Burns K, Holt RI, Comaschi M, Hermanns N, Ishii H, Kokoszka A, Pouwer F, Skovlund SE, Stuckey H, Tarkun I, Vallis M, Wens J, Peyrot M; DAWN2 Study Group. Diabetes Attitudes, Wishes and Needs second study (DAWN2™): cross-national benchmarking of diabetes-related psychosocial outcomes for people with diabetes. Diabet Med. 2013 Jul;30(7):767-77. doi: 10.1111/dme.12245. Erratum in: Diabet Med. 2013 Oct;30(10):1266.

ReferenceCitation: Kovacs Burns K, Nicolucci A, Holt RI, Willaing I, Hermanns N, Kalra S, Wens J, Pouwer F, Skovlund SE, Peyrot M; DAWN2 Study Group. Diabetes Attitudes, Wishes and Needs second study (DAWN2™): cross-national benchmarking indicators for family members living with people with diabetes. Diabet Med. 2013 Jul;30(7):778-88. doi: 10.1111/dme.12239.

ReferenceCitation: Holt RI, Nicolucci A, Kovacs Burns K, Escalante M, Forbes A, Hermanns N, Kalra S, Massi-Benedetti M, Mayorov A, Menéndez-Torre E, Munro N, Skovlund SE, Tarkun I, Wens J, Peyrot M; DAWN2 Study Group. Diabetes Attitudes, Wishes and Needs second study (DAWN2™): cross-national comparisons on barriers and resources for optimal care--healthcare professional perspective. Diabet Med. 2013 Jul;30(7):789-98. doi: 10.1111/dme.12242.

ReferenceCitation: Funnell M. Beyond the data: moving towards a new DAWN in diabetes. Diabet Med. 2013 Jul;30(7):765-6. doi: 10.1111/dme.12244.

ReferenceCitation: Peyrot M, Egede LE, Campos C, Cannon AJ, Funnell MM, Hsu WC, Ruggiero L, Siminerio LM, Stuckey HL. Ethnic differences in psychological outcomes among people with diabetes: USA results from the second Diabetes Attitudes, Wishes, and Needs (DAWN2) study. Curr Med Res Opin. 2014 Nov;30(11):2241-54. doi: 10.1185/03007995.2014.947023. Epub 2014 Aug 18.

ReferenceCitation: Peyrot M, Egede LE, Funnell MM, Hsu WC, Ruggiero L, Siminerio LM, Stuckey HL. US ethnic group differences in self-management in the 2nd diabetes attitudes, wishes and needs (DAWN2) study. J Diabetes Complications. 2018 Jun;32(6):586-592. doi: 10.1016/j.jdiacomp.2018.03.002. Epub 2018 Mar 8.

ReferenceCitation: Nefs G, Pouwer F. The role of hypoglycemia in the burden of living with diabetes among adults with diabetes and family members: results from the DAWN2 study in The Netherlands. BMC Public Health. 2018 Jan 18;18(1):156. doi: 10.1186/s12889-018-5064-y.

ReferenceCitation: Barnard KD, Holt RI, Dyson PA, Cummings MH, Kanumilli N, O'Neill S, Hall G. Could the Discrepancy in Perceived Emotional Care Received and Provided Be a Barrier to Active Diabetes Self-management? Insights From the Second Diabetes Attitudes, Wishes and Needs (DAWN2) Study. Diabetes Care. 2016 Feb;39(2):e20-1. doi: 10.2337/dc15-0674. Epub 2015 Dec 30.

ReferenceCitation: Peyrot M, Egede LE, Funnell MM, Hsu WC, Ruggiero L, Siminerio LM, Stuckey HL. Ethnic differences in family member diabetes involvement and psychological outcomes: results from the second Diabetes Attitudes, Wishes and Needs (DAWN2) study in the USA. Curr Med Res Opin. 2015;31(7):1297-307. doi: 10.1185/03007995.2015.1043251. Epub 2015 May 20.

location_country:
Denmark
Poland
Spain
Italy
Russian Federation
Netherlands
United Kingdom
Canada
Algeria
United States
France
China
India
Mexico
Japan
Germany
Turkey


title: Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry

summary:
This observational registry is conducted in Europe, Asia, Africa and the United States of America (USA).

The purpose of the registry is to evaluate the efficacy and safety of activated recombinant human factor VII (rFVIIa) during bleeding episodes and for the prevention of bleeding during invasive procedures/surgery in patients with Glanzmann's thrombasthenia (GT) with past or present refractoriness to platelet transfusions. Attention will be directed towards complications related to thrombo-embolic events and concomitant medications especially antifibrinolytics.

ReferenceCitation: Zotz RB, Poon MC, Di Minno G, D'Oiron R; Glanzmann Thrombasthenia Registry Investigators . The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes. TH Open. 2019 Sep 12;3(3):e286-e294. doi: 10.1055/s-0039-1696657. eCollection 2019 Jul.

ReferenceCitation: Di Minno G, Zotz RB, d'Oiron R, Bindslev N, Di Minno MN, Poon MC; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes of non-surgical bleeding episodes in patients with Glanzmann thrombasthenia. Haematologica. 2015 Aug;100(8):1031-7. doi: 10.3324/haematol.2014.121475. Epub 2015 May 22.

ReferenceCitation: Poon MC, d'Oiron R, Zotz RB, Bindslev N, Di Minno MN, Di Minno G; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment and outcomes in surgical intervention. Haematologica. 2015 Aug;100(8):1038-44. doi: 10.3324/haematol.2014.121384. Epub 2015 May 22.

location_country:
Spain
Italy
Austria
Netherlands
United Kingdom
Switzerland
Sweden
Hungary
Algeria
United States
France
Belgium
Germany
Bulgaria
Pakistan


title: A Clinical Evaluation of the Abbott Vascular ESPRIT BVS (Bioresorbable Vascular Scaffold) System for the Treatment of Subjects With Symptomatic Claudication From Occlusive Vascular Disease of the Superficial Femoral (SFA) or Common or External Iliac Arteries.

summary: The purpose of the ESPRIT I Clinical Investigation is to evaluate the safety and performance of the ESPRIT BVS in subjects with symptomatic claudication from occlusive vascular disease of the superficial femoral (SFA) or common or external iliac arteries.

ReferenceCitation: Lammer J, Bosiers M, Deloose K, Schmidt A, Zeller T, Wolf F, Lansink W, Sauguet A, Vermassen F, Lauwers G, Scheinert D, Popma JJ, McGreevy R, Rapoza R, Schwartz LB, Jaff MR. Bioresorbable Everolimus-Eluting Vascular Scaffold for Patients With Peripheral Artery Disease (ESPRIT I): 2-Year Clinical and Imaging Results. JACC Cardiovasc Interv. 2016 Jun 13;9(11):1178-87. doi: 10.1016/j.jcin.2016.02.051.

lead_sponsor: Abbott Medical Devices

location_country: Austria Germany France Belgium

location_facility:
Abbott Vascular International BVBA
Sint-Blasius Hospital
Oost Limburg Ziekenhuis
Medical University of Vienna
Herzzentrum Bad Krozingen
Clinique Pasteur
Park-Krankenhaus Leipzig
Gent University Hospital


title: Allogeneic Stem Cell Transplantation in Children and Adolescents With Acute Lymphoblastic Leukaemia

acronym: ALL-SCT-BFMi

summary:
With this protocol the ALL-SCT BFM international study group wants

to evaluate whether hematopoietic stem cell transplantation (HSCT) from matched family or unrelated donors (MD) is equivalent to the HSCT from matched sibling donors (MSD).
to evaluate the efficacy of hematopoietic stem cell transplantation (HSCT)from mismatched family or unrelated donors (MMD) as compared to HSCT from matched sibling donors or matched donors.
to determine whether therapy has been carried out according to the main HSCT protocol recommendations. The standardisation of the treatment options during HSCT from different donor types aims at the achievement of an optimal comparison of survival after HSCT with survival after chemotherapy only.
to prospectively evaluate and compare the incidence of acute and chronic Graft-versus-Host-Disease (GvHD) after HSCT from matched sibling donor (MSD), from matched donor (MD) and from mismatched donor (MMD).

ReferenceCitation: Peters C, Schrauder A, Schrappe M, von Stackelberg A, Stary J, Yaniv I, Gadner H, Klingebiel T; BFM Study Group, the IBFM-Study Group and the Paediatric Disease Working Party of the EBMT. Allogeneic haematopoietic stem cell transplantation in children with acute lymphoblastic leukaemia: the BFM/IBFM/EBMT concepts. Bone Marrow Transplant. 2005 Mar;35 Suppl 1:S9-11.

ReferenceCitation: Pulsipher MA, Peters C, Pui CH. High-risk pediatric acute lymphoblastic leukemia: to transplant or not to transplant? Biol Blood Marrow Transplant. 2011 Jan;17(1 Suppl):S137-48. doi: 10.1016/j.bbmt.2010.10.005. Review.

ReferenceCitation: Peters C, Schrappe M, von Stackelberg A, Schrauder A, Bader P, Ebell W, Lang P, Sykora KW, Schrum J, Kremens B, Ehlert K, Albert MH, Meisel R, Matthes-Martin S, Gungor T, Holter W, Strahm B, Gruhn B, Schulz A, Woessmann W, Poetschger U, Zimmermann M, Klingebiel T. Stem-cell transplantation in children with acute lymphoblastic leukemia: A prospective international multicenter trial comparing sibling donors with matched unrelated donors-The ALL-SCT-BFM-2003 trial. J Clin Oncol. 2015 Apr 10;33(11):1265-74. doi: 10.1200/JCO.2014.58.9747. Epub 2015 Mar 9.

ReferenceCitation: Tasian SK, Peters C. Targeted therapy or transplantation for paediatric ABL-class Ph-like acute lymphocytic leukaemia? Lancet Haematol. 2020 Dec;7(12):e858-e859. doi: 10.1016/S2352-3026(20)30369-0.

ReferenceCitation: Balduzzi A, Dalle JH, Wachowiak J, Yaniv I, Yesilipek A, Sedlacek P, Bierings M, Ifversen M, Sufliarska S, Kalwak K, Lankester A, Toporski J, Di Maio L, Glogova E, Poetschger U, Peters C. Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study. Biol Blood Marrow Transplant. 2019 Nov;25(11):2197-2210. doi: 10.1016/j.bbmt.2019.07.011. Epub 2019 Jul 15.

lead_sponsor: St. Anna Kinderkrebsforschung

location_country:
Denmark
Poland
Italy
Austria
Czech Republic
Sweden
France
Israel
Turkey
Netherlands
Slovakia

location_facility: Department of Transplantation, University Children's Hospital

location_facility: Pediatric Immuno-Hematology Unit Robert Debré Hospital

location_facility: Department of Pediatric Hematology-Oncology and Pediatric Stem Cell Transplantation, Akdeniz University School of Medicine

location_facility: Universitätsklinik für Kinder- und Jugendheilkunde, Klin. Abt. f. Hämato-Onkologie

location_facility: Department of Paediatric Haematology and Oncology HSCT-Unit

location_facility: Clinica Pediatrica dell'Universita di Milano Bicocca, Hospitale San Gerardo

location_facility: Department of Pediatrics, Gülhane Military Medical Academy

location_facility: Department of Pediatric Hematology, Oncology and BMT, Istanbul School of Medicine

location_facility: Department of Paediatric Haematology and Oncology, Wilhelmina Children's Hospital

location_facility: University Hospital, Collegium Medicum UMK, Pediatric Hematology and Oncology

location_facility: Dept. of Paediatrics - BMT Unit, School of Medicine, University of Ankara

location_facility: Department of Pediatric Oncology, Lund University Hospital

location_facility: Wroclaw Medical University, Dept. of Children Hematology and Oncology

location_facility: Department of Pediadric Oncology, Hematology and Transplantology, University of Medical Sciences

location_facility: Radboud University - Nijmegen Medical Centre

location_facility: Children's University Hospital - Hematology - Oncology

location_facility: Schneider Children's Medical Center of Israel

location_facility: Department of Pediatric Bone Marrow Transplantation Unit, University Childrens´ Hospital

intervention_type: Radiation


title: A Post Marketing, Multi-Center, Trial to Measure Human Anti-Human Antibodies (HAHA) to Soliris (Eculizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria

summary: How does long term treatment with Soliris affect HAHA in PNH patients?

lead_sponsor: Alexion Pharmaceuticals

location_country:
United Kingdom
Ireland
Italy
Australia
Netherlands
Switzerland
United States
France
Belgium
Germany

location_facility: Azienda Ospediliera Universitatia Careggi

location_facility: Institut fur Klinische Transfusionmedizin und Immungenetick


title: C1 Inhibitor Treatment Registry to Assess the Safety and Immunological Profile of Ruconest in the Treatment of HAE Attacks

summary: This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.

lead_sponsor: Pharming Technologies B.V.

location_country:
Croatia
Poland
Slovenia
Italy
Sweden
Hungary
North Macedonia
France
Norway
Germany
Bulgaria
Slovakia
Czechia

location_facility: Semmelweis University, 3rd Department of Internal Medicine


title: An Observational Registry of Abatacept in Patients With Juvenile Idiopathic Arthritis

summary: The purpose of this study is to examine the long-term safety of Abatacept for the treatment of juvenile idiopathic arthritis (JIA) with particular in interest in the occurrence of serious infections, autoimmune disorders, and malignancies.

lead_sponsor: Bristol-Myers Squibb

location_country:
Spain
Italy
United Kingdom
United States
Peru
Romania
South Africa
Portugal
Austria
Russian Federation
Puerto Rico
Israel
Mexico
Slovakia
Brazil
Canada
Hungary
Germany
Denmark
Greece
Latvia
France
Netherlands

location_facility: Centro de Reumatologia Pediatrico de Puerto Rico


title: A Prospective, Randomized, Multicenter Study to Demonstrate the Superiority of the Barricaid® to Discectomy for Primary Lumbar Disc Herniation: Extended Follow-Up of the Barricaid® Annular Closure Device (ACD) Randomized Control Trial (RCT) Postmarket Cohort for Lumbar Disc Herniation and Interaction With Other Risk Factors

summary: A randomized study to demonstrate the superiority of the Barricaid® when used in conjunction with limited discectomy, compared to limited discectomy alone, with regard to preventing reherniation and the recurrence of pain or dysfunction.

ReferenceCitation: Thomé C, Klassen PD, Bouma GJ, Kuršumović A, Fandino J, Barth M, Arts M, van den Brink W, Bostelmann R, Hegewald A, Heidecke V, Vajkoczy P, Fröhlich S, Wolfs J, Assaker R, Van de Kelft E, Köhler HP, Jadik S, Eustacchio S, Hes R, Martens F; Annular Closure RCT Study Group. Annular closure in lumbar microdiscectomy for prevention of reherniation: a randomized clinical trial. Spine J. 2018 Dec;18(12):2278-2287. doi: 10.1016/j.spinee.2018.05.003. Epub 2018 May 3.

ReferenceCitation: Martens F, Vajkoczy P, Jadik S, Hegewald A, Stieber J, Hes R. Patients at the Highest Risk for Reherniation Following Lumbar Discectomy in a Multicenter Randomized Controlled Trial. JB JS Open Access. 2018 Apr 16;3(2):e0037. doi: 10.2106/JBJS.OA.17.00037. eCollection 2018 Jun 28.

ReferenceCitation: Klassen PD, Hsu WK, Martens F, Inzana JA, van den Brink WA, Groff MW, Thomé C. Post-lumbar discectomy reoperations that are associated with poor clinical and socioeconomic outcomes can be reduced through use of a novel annular closure device: results from a 2-year randomized controlled trial. Clinicoecon Outcomes Res. 2018 Jun 26;10:349-357. doi: 10.2147/CEOR.S164129. eCollection 2018.

ReferenceCitation: Ament J, Thaci B, Yang Z, Kulubya E, Hsu W, Bouma G, Kim KD. Cost-effectiveness of a Bone-anchored Annular Closure Device Versus Conventional Lumbar Discectomy in Treating Lumbar Disc Herniations. Spine (Phila Pa 1976). 2019 Jan 1;44(1):5-16. doi: 10.1097/BRS.0000000000002746.

ReferenceCitation: Kuršumović A, Kienzler JC, Bouma GJ, Bostelmann R, Heggeness M, Thomé C, Miller LE, Barth M; Annular Closure RCT study group. Morphology and Clinical Relevance of Vertebral Endplate Changes Following Limited Lumbar Discectomy With or Without Bone-anchored Annular Closure. Spine (Phila Pa 1976). 2018 Oct 15;43(20):1386-1394. doi: 10.1097/BRS.0000000000002632.

ReferenceCitation: Klassen PD, Bernstein DT, Köhler HP, Arts MP, Weiner B, Miller LE, Thomé C. Bone-anchored annular closure following lumbar discectomy reduces risk of complications and reoperations within 90 days of discharge. J Pain Res. 2017 Aug 26;10:2047-2055. doi: 10.2147/JPR.S144500. eCollection 2017. Erratum in: J Pain Res. 2017 Dec 06;10 :2739.

ReferenceCitation: Thomé C, Kuršumovic A, Klassen PD, Bouma GJ, Bostelmann R, Martens F, Barth M, Arts M, Miller LE, Vajkoczy P, Hes R, Eustacchio S, Nanda D, Köhler HP, Brenke C, Flüh C, Van de Kelft E, Assaker R, Kienzler JC, Fandino J; Annular Closure RCT Study Group. Effectiveness of an Annular Closure Device to Prevent Recurrent Lumbar Disc Herniation: A Secondary Analysis With 5 Years of Follow-up. JAMA Netw Open. 2021 Dec 1;4(12):e2136809. doi: 10.1001/jamanetworkopen.2021.36809.

ReferenceCitation: Kienzler JC, Fandino J, Van de Kelft E, Eustacchio S, Bouma GJ; Barricaid® Annular Closure RCT Study Group. Risk factors for early reherniation after lumbar discectomy with or without annular closure: results of a multicenter randomized controlled study. Acta Neurochir (Wien). 2021 Jan;163(1):259-268. doi: 10.1007/s00701-020-04505-4. Epub 2020 Oct 21.

ReferenceCitation: van den Brink W, Flüh C, Miller LE, Klassen PD, Bostelmann R. Lumbar disc reherniation prevention with a bone-anchored annular closure device: 1-year results of a randomized trial. Medicine (Baltimore). 2019 Nov;98(44):e17760. doi: 10.1097/MD.0000000000017760.

ReferenceCitation: Kienzler JC, Klassen PD, Miller LE, Assaker R, Heidecke V, Fröhlich S, Thomé C; Annular Closure RCT Study Group. Three-year results from a randomized trial of lumbar discectomy with annulus fibrosus occlusion in patients at high risk for reherniation. Acta Neurochir (Wien). 2019 Jul;161(7):1389-1396. doi: 10.1007/s00701-019-03948-8. Epub 2019 May 15.

lead_sponsor: Intrinsic Therapeutics

location_country: Austria Netherlands Switzerland France Belgium Germany

location_facility: Neurochirurgische Universitätsklinik Knappschafts-Krankenhaus Bochum-Langendreer

location_facility: Orthopädische Klinik und Poliklinik - Universitätsmedizin Rostock


title: AZA PH GL 2003 CL 001 - Extension A Multicenter, Randomized, Open-label, Parallel-group, Phase 3 Trial of Subcutaneous Azacitidine Plus Best Supportive Care Versus Conventional Care Regimens Plus Best Supportive Care for the Treatment of Myelodysplastic Syndromes (MDS)

summary: At the conclusion of study AZA PH GL 2003 CL 001 (NCT00071799), eligible participants could be enrolled in an optional extension phase in order to continue treatment with azacitidine until it became commercially available; the continued treatment was for ethical and safety reasons only and not to provide additional efficacy data.

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

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

ReferenceCitation: Gore SD, Fenaux P, Santini V, Bennett JM, Silverman LR, Seymour JF, Hellström-Lindberg E, Swern AS, Beach CL, List AF. A multivariate analysis of the relationship between response and survival among patients with higher-risk myelodysplastic syndromes treated within azacitidine or conventional care regimens in the randomized AZA-001 trial. Haematologica. 2013 Jul;98(7):1067-72. doi: 10.3324/haematol.2012.074831. Epub 2013 Apr 12.

ReferenceCitation: Savona MR, Kolibaba K, Conkling P, Kingsley EC, Becerra C, Morris JC, Rifkin RM, Laille E, Kellerman A, Ukrainskyj SM, Dong Q, Skikne BS. Extended dosing with CC-486 (oral azacitidine) in patients with myeloid malignancies. Am J Hematol. 2018 Oct;93(10):1199-1206. doi: 10.1002/ajh.25216. Epub 2018 Sep 3.

ReferenceCitation: Seymour JF, Fenaux P, Silverman LR, Mufti GJ, Hellström-Lindberg E, Santini V, List AF, Gore SD, Backstrom J, McKenzie D, Beach CL. Effects of azacitidine compared with conventional care regimens in elderly (≥ 75 years) patients with higher-risk myelodysplastic syndromes. Crit Rev Oncol Hematol. 2010 Dec;76(3):218-27. doi: 10.1016/j.critrevonc.2010.04.005. Epub 2010 May 6.

ReferenceCitation: Garcia-Manero G, Scott BL, Cogle CR, Boyd TE, Kambhampati S, Hetzer J, Dong Q, Kumar K, Ukrainskyj SM, Beach CL, Skikne BS. CC-486 (oral azacitidine) in patients with myelodysplastic syndromes with pretreatment thrombocytopenia. Leuk Res. 2018 Sep;72:79-85. doi: 10.1016/j.leukres.2018.08.001. Epub 2018 Aug 3.

ReferenceCitation: Ma X, Steensma DP, Scott BL, Kiselev P, Sugrue MM, Swern AS. Selection of patients with myelodysplastic syndromes from a large electronic medical records database and a study of the use of disease-modifying therapy in the United States. BMJ Open. 2018 Jul 23;8(7):e019955. doi: 10.1136/bmjopen-2017-019955.

ReferenceCitation: de Lima M, Oran B, Champlin RE, Papadopoulos EB, Giralt SA, Scott BL, William BM, Hetzer J, Laille E, Hubbell B, Skikne BS, Craddock C. CC-486 Maintenance after Stem Cell Transplantation in Patients with Acute Myeloid Leukemia or Myelodysplastic Syndromes. Biol Blood Marrow Transplant. 2018 Oct;24(10):2017-2024. doi: 10.1016/j.bbmt.2018.06.016. Epub 2018 Jun 20.

ReferenceCitation: Wehmeyer J, Zaiss M, Losem C, Schmitz S, Niemeier B, Harde J, Hannig CV, Harich HD, Müller J, Klausmann M, Tessen HW, Potthoff K. Impact of performance status and transfusion dependency on outcome of patients with myelodysplastic syndrome, acute myeloid leukemia and chronic myelomonocytic leukemia treated with azacitidine (PIAZA study). Eur J Haematol. 2018 Dec;101(6):766-773. doi: 10.1111/ejh.13160. Epub 2018 Oct 25.

ReferenceCitation: Platzbecker U, Middeke JM, Sockel K, Herbst R, Wolf D, Baldus CD, Oelschlägel U, Mütherig A, Fransecky L, Noppeney R, Bug G, Götze KS, Krämer A, Bochtler T, Stelljes M, Groth C, Schubert A, Mende M, Stölzel F, Borkmann C, Kubasch AS, von Bonin M, Serve H, Hänel M, Dührsen U, Schetelig J, Röllig C, Kramer M, Ehninger G, Bornhäuser M, Thiede C. Measurable residual disease-guided treatment with azacitidine to prevent haematological relapse in patients with myelodysplastic syndrome and acute myeloid leukaemia (RELAZA2): an open-label, multicentre, phase 2 trial. Lancet Oncol. 2018 Dec;19(12):1668-1679. doi: 10.1016/S1470-2045(18)30580-1. Epub 2018 Nov 12.

ReferenceCitation: Boutault R, Peterlin P, Boubaya M, Sockel K, Chevallier P, Garnier A, Guillaume T, Le Bourgeois A, Debord C, Godon C, Le Bris Y, Theisen O, Kroschinsky F, Moreau P, Béné MC, Platzbecker U, Eveillard M. A novel complete blood count-based score to screen for myelodysplastic syndrome in cytopenic patients. Br J Haematol. 2018 Dec;183(5):736-746. doi: 10.1111/bjh.15626. Epub 2018 Nov 8.

ReferenceCitation: Wenk C, Garz AK, Grath S, Huberle C, Witham D, Weickert M, Malinverni R, Niggemeyer J, Kyncl M, Hecker J, Pagel C, Mulholland CB, Müller-Thomas C, Leonhardt H, Bassermann F, Oostendorp RAJ, Metzeler KH, Buschbeck M, Götze KS. Direct modulation of the bone marrow mesenchymal stromal cell compartment by azacitidine enhances healthy hematopoiesis. Blood Adv. 2018 Dec 11;2(23):3447-3461. doi: 10.1182/bloodadvances.2018022053.

ReferenceCitation: Zeidan AM, Klink AJ, McGuire M, Feinberg B. Treatment sequence of lenalidomide and hypomethylating agents and the impact on clinical outcomes for patients with myelodysplastic syndromes. Leuk Lymphoma. 2019 Aug;60(8):2050-2055. doi: 10.1080/10428194.2018.1551538. Epub 2019 Jan 14.

ReferenceCitation: Leung KK, Nguyen A, Shi T, Tang L, Ni X, Escoubet L, MacBeth KJ, DiMartino J, Wells JA. Multiomics of azacitidine-treated AML cells reveals variable and convergent targets that remodel the cell-surface proteome. Proc Natl Acad Sci U S A. 2019 Jan 8;116(2):695-700. doi: 10.1073/pnas.1813666116. Epub 2018 Dec 24.

ReferenceCitation: Craddock C, Slade D, De Santo C, Wheat R, Ferguson P, Hodgkinson A, Brock K, Cavenagh J, Ingram W, Dennis M, Malladi R, Siddique S, Mussai F, Yap C. Combination Lenalidomide and Azacitidine: A Novel Salvage Therapy in Patients Who Relapse After Allogeneic Stem-Cell Transplantation for Acute Myeloid Leukemia. J Clin Oncol. 2019 Mar 1;37(7):580-588. doi: 10.1200/JCO.18.00889. Epub 2019 Jan 17.

ReferenceCitation: Silverman LR, Fenaux P, Mufti GJ, Santini V, Hellström-Lindberg E, Gattermann N, Sanz G, List AF, Gore SD, Seymour JF. Continued azacitidine therapy beyond time of first response improves quality of response in patients with higher-risk myelodysplastic syndromes. Cancer. 2011 Jun 15;117(12):2697-702. doi: 10.1002/cncr.25774. Epub 2011 Jan 10.

lead_sponsor: Celgene

location_country:
Poland
Spain
Greece
Italy
Australia
Netherlands
United Kingdom
Hungary
France
Germany
Bulgaria


title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study of the Safety, Tolerability and Efficacy of E2007 in Parkinson's Disease Patients With "Wearing Off" Motor Fluctuations and "On" Period Dyskinesias

summary: The purpose of this study is to investigate the safety, tolerability and efficacy of E2007 in Parkinson's Disease patients who have "wearing off" motor fluctuations and "on" period dyskenisias.

lead_sponsor: Eisai Limited

location_country:
Serbia
Spain
Italy
Czech Republic
France
Germany

location_facility: Klinikum der Heinrich-Heine- Universit?t

location_facility: Reparto di Neurologia - Ospedale Misericordia

location_facility: Universit?tsklinikum Rostock Klinik f?r Neurologie

location_facility: Klinikum der Friedrich-Wilhelms- Univerit?t Bonn

location_facility: First Faculty of Medicine Charles University

location_facility: Humboldt Universit?t Charite Neurologische Klinik

location_facility: Universit?tskrankenh aus Hamburg Eppendorf

location_facility: Hospital Clinic I Provincial de Barcelona

location_facility: Unit? Operativa Parkinson e Disordini del Movimento

location_facility: Zentralkrankenhaus Reinkenheide Neurologische Klinik

location_facility: Centre D'Investigation Clinique Pavillon Riser - Hopital Purpan

location_facility: Dept. of Neurology - Second Faculty of Medicine Charles University

location_facility: Department of Neurology, Regional Hospital Pardubice

location_facility: Parkinson's Competence Network Germany Dept. of Neurology - Philipps-University Marburg

location_facility: Clinic of Neurology, Faculty Hospital Olomouc


title: Continued Access to DRV/Rtv in HIV-1 Infected Children and Adolescents (Rollover Patients From C212, C228, C230)

lead_sponsor: Janssen Sciences Ireland UC

ReferenceCitation: Violari A, Masenya M, Blanche S, Vanveggel S, Hufkens V, Chetty P, Opsomer M. The DIANA Study: Continued Access to Darunavir/Ritonavir (DRV/r) and Long-Term Safety Follow-Up in HIV-1-Infected Pediatric Patients Aged 3 to < 18 Years. Drug Saf. 2021 Apr;44(4):439-446. doi: 10.1007/s40264-020-01032-0. Epub 2020 Dec 24.

location_country:
Brazil
Spain
United Kingdom
Argentina
Ukraine
India
France
South Africa

summary: The primary objective of this study is to continue to provide Darunavir (DRV) to pediatric patients who previously received DRV in any of three pediatric clinical studies sponsored by Tibotec Pharmaceuticals and continue to benefit from using it, in countries where DRV is not yet commercially available for the pediatric patient, is not reimbursed or cannot be accessed through another source (like access program or government program).


title: A Phase I/II Trial of Temsirolimus Plus Neratinib for Patients With Metastatic HER2-Amplified or Triple Negative Breast Cancer

summary: This is an open-label, single arm, multi-center, multi-national, adaptive design, dose-escalation Phase 1/2 study to determine the maximum tolerated dose (MTD) of temsirolimus with daily neratinib, and to determine the safety and efficacy of this combination when given to patients with advanced breast carcinoma, specifically trastuzumab-refractory HER2-amplified disease or triple-negative disease.

lead_sponsor: Puma Biotechnology, Inc.

location_country:
Denmark
Hong Kong
Spain
United Kingdom
United States
France

location_facility: Memorial Sloan Kettering Cancer Center (MSKCC)

location_facility: Edinburgh Cancer Center, Western General Hospital

location_facility: Weill Cornell Medical College - New York - Presbyterian Hospital

location_facility: Hospital Universitario Sant Joan de Reus

location_facility: Hospital Universitari Arnau de Vilanova de Lleida


title: NordiNet® International Outcome Study-Observational Prospective Study on Patients Treated With Norditropin®

summary: This observational study is conducted globally. The aim of the study is to investigate the effectiveness and safety of real-life treatment with Norditropin®. The study population will consist of children and adults who are on treatment with Norditropin® in accordance with normal clinical practice.

ReferenceCitation: Weber MM, Biller BM, Pedersen BT, Pournara E, Christiansen JS, Höybye C. The effect of growth hormone (GH) replacement on blood glucose homeostasis in adult nondiabetic patients with GH deficiency: real-life data from the NordiNet(®) International Outcome Study. Clin Endocrinol (Oxf). 2017 Feb;86(2):192-198. doi: 10.1111/cen.13256. Epub 2016 Nov 21.

ReferenceCitation: Sävendahl L, Pournara E, Pedersen BT, Blankenstein O. Is safety of childhood growth hormone therapy related to dose? Data from a large observational study. Eur J Endocrinol. 2016 May;174(5):681-91. doi: 10.1530/EJE-15-1017. Epub 2016 Feb 22.

ReferenceCitation: Lee PA, Sävendahl L, Oliver I, Tauber M, Blankenstein O, Ross J, Snajderova M, Rakov V, Pedersen BT, Christesen HT. Comparison of response to 2-years' growth hormone treatment in children with isolated growth hormone deficiency, born small for gestational age, idiopathic short stature, or multiple pituitary hormone deficiency: combined results from two large observational studies. Int J Pediatr Endocrinol. 2012 Jul 12;2012(1):22. doi: 10.1186/1687-9856-2012-22.

ReferenceCitation: Höybye C, Sävendahl L, Christesen HT, Lee P, Pedersen BT, Schlumpf M, Germak J, Ross J. The NordiNet® International Outcome Study and NovoNet® ANSWER Program®: rationale, design, and methodology of two international pharmacoepidemiological registry-based studies monitoring long-term clinical and safety outcomes of growth hormone therapy (Norditropin®). Clin Epidemiol. 2013 Apr 26;5:119-27. doi: 10.2147/CLEP.S42602. Print 2013.

ReferenceCitation: Sävendahl L, Blankenstein O, Oliver I, Christesen HT, Lee P, Pedersen BT, Rakov V, Ross J. Gender influences short-term growth hormone treatment response in children. Horm Res Paediatr. 2012;77(3):188-94. doi: 10.1159/000337570. Epub 2012 Apr 12.

ReferenceCitation: Blankenstein O, Snajderova M, Blair J, Pournara E, Pedersen BT, Petit IO. Real-life GH dosing patterns in children with GHD, TS or born SGA: a report from the NordiNet® International Outcome Study. Eur J Endocrinol. 2017 Aug;177(2):145-155. doi: 10.1530/EJE-16-1055. Epub 2017 May 18.

ReferenceCitation: Christesen HT, Pedersen BT, Pournara E, Petit IO, Júlíusson PB. Short Stature: Comparison of WHO and National Growth Standards/References for Height. PLoS One. 2016 Jun 9;11(6):e0157277. doi: 10.1371/journal.pone.0157277. eCollection 2016.

ReferenceCitation: Biller BMK, Höybye C, Carroll P, Gordon MB, Birkegård AC, Kelepouris N, Nedjatian N, Weber MM. Pregnancy outcomes in women receiving growth hormone replacement therapy enrolled in the NordiNet® International Outcome Study (IOS) and the American Norditropin® Studies: Web-Enabled Research (ANSWER) Program. Pituitary. 2021 Aug;24(4):611-621. doi: 10.1007/s11102-021-01138-3. Epub 2021 Mar 12.

ReferenceCitation: Savendahl L, Polak M, Backeljauw P, Blair JC, Miller BS, Rohrer TR, Hokken-Koelega A, Pietropoli A, Kelepouris N, Ross J. Long-Term Safety of Growth Hormone Treatment in Childhood: Two Large Observational Studies: NordiNet IOS and ANSWER. J Clin Endocrinol Metab. 2021 May 13;106(6):1728-1741. doi: 10.1210/clinem/dgab080.

ReferenceCitation: Weber MM, Gordon MB, Höybye C, Jørgensen JOL, Puras G, Popovic-Brkic V, Molitch ME, Ostrow V, Holot N, Pietropoli A, Biller BMK. Growth hormone replacement in adults: Real-world data from two large studies in US and Europe. Growth Horm IGF Res. 2020 Feb;50:71-82. doi: 10.1016/j.ghir.2019.09.002. Epub 2019 Oct 26.

ReferenceCitation: Sävendahl L, Polak M, Backeljauw P, Blair J, Miller BS, Rohrer TR, Pietropoli A, Ostrow V, Ross J. Treatment of Children With GH in the United States and Europe: Long-Term Follow-Up From NordiNet® IOS and ANSWER Program. J Clin Endocrinol Metab. 2019 Oct 1;104(10):4730-4742. doi: 10.1210/jc.2019-00775. Erratum in: J Clin Endocrinol Metab. 2020 Jun 1;105(6):.

location_country:
Luxembourg
Saudi Arabia
Italy
Serbia
Spain
United Kingdom
Lithuania
Belgium
Czechia
Slovenia
Russian Federation
Argentina
Israel
Norway
Sweden
Switzerland
Hungary
Germany
Denmark
Ireland
Finland
France
Netherlands


title: Patient Characteristics in Advanced Renal Cell Carcinoma and Daily Practice Treatment With Nexavar

summary: Renal cell carcinoma accounts for roughly 3 % of all cancer. It is a rather aggressive cancer type, which means that patients who present with an advanced disease have a rather poor prognosis. When this study has been started the standard therapy for patients has been cytokines, which might be accompanied by significant toxicities or might fail the therapeutic goal. In these cases sorafenib can be a feasible therapeutic option. This non-interventional study has been created and is being conducted to collect clinical data on the patients' therapy with sorafenib in an everyday treatment schedule. The main goal of this study focuses on patient characteristics and tumor status in RCC treated with sorafenib as well as the treatment duration and safety of sorafenib under everyday treatment conditions.

location_country:
Poland
Slovenia
Greece
Austria
Mexico
Netherlands
Argentina
Czech Republic
Indonesia
Russian Federation
Sweden
Korea, Republic of
France
China
Colombia
Philippines
Germany
Slovakia


title: Prophylactic Treatment With Recombinant Factor VIIa (rFVIIa, NovoSeven®) in Haemophilia Patients With Inhibitors

acronym: PROPACT

summary: This study is conducted in Europe and North and South America. The primary aim of this observational study is to evaluate the frequency and pattern of bleeding episodes in haemophilia patients receiving preventative treatment with activated recombinant human factor VII. The secondary aim is to evaluate which patients are selected for this treatment, the dose and dose intervals used, and the safety of activated recombinant human factor VII when used as prevention. The study also aims to increase understanding of the unmet medical need and clinical relevance of preventative treatment in haemophilia patients.

time_perspective: Retrospective

location_country:
Croatia
Spain
Ireland
Italy
United Kingdom
Argentina
Czech Republic
Canada
Switzerland
Sweden
United States
France
Germany
Slovakia


title: A Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Subcutaneous Methylnaltrexone for the Treatment of Opioid-Induced Constipation in Subjects With Cancer-Related Pain

summary: This purpose of this study is to evaluate the safety and effectiveness of a subcutaneous (injection beneath the skin) form of methylnaltrexone in subjects who have cancer-related pain and constipation from taking opioids. The length of participation will be up to 7 weeks.

status: Withdrawn

lead_sponsor: Bausch Health Americas, Inc.

location_country:
United States
Spain
Canada
France


title: A Multicentre, Randomised, Double-Blind, Placebo Controlled, Phase II Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable Implants of Afamelanotide (CUV1647) for the Prophylactic Treatment of Pre-Cancerous Skin Lesions of the Head, Forearms and Hands in Immune Compromised, Organ Transplant Patients.

summary:
The purpose of this study is to determine whether afamelanotide (CUV1647) is effective in reducing the number of actinic keratoses and squamous cell carcinomas developing in immune compromised organ transplant recipients, who are at particularly high risk, over a 24 month test period.

The number of lesions formed on the head, hands and forarms will be monitored over this 24 month test period.

primary_purpose: Prevention

lead_sponsor: Clinuvel Pharmaceuticals Limited

location_country: Italy Australia Switzerland Sweden France Belgium Germany

location_facility:
Karolinska University Hospital
University Hospital
Charité Univeritätsklinikum
Ospedali Riuniti di Bergamo
The Queen Elizabeth Hospital
Kiel University Hospital
University of Padua
Universitätsspital Zürich
Hospital Erasme
The Princess Alexandra Hospital
The Royal Melbourne Hospital
Gent University Hospital


title: A Phase III Study With Freeze Dried HL10, Three Dosages of 200 mg/kg Ideal Body Weight Versus Standard Therapy in ALI/ARDS Patients

summary: The purpose of the study is to evaluate the efficacy and safety of three doses of HL 10 given at fixed time intervals compared to standard therapy

ReferenceCitation: Lu Q, Zhang M, Girardi C, Bouhemad B, Kesecioglu J, Rouby JJ. Computed tomography assessment of exogenous surfactant-induced lung reaeration in patients with acute lung injury. Crit Care. 2010;14(4):R135. doi: 10.1186/cc9186. Epub 2010 Jul 15.

ReferenceCitation: Kesecioglu J, Beale R, Stewart TE, Findlay GP, Rouby JJ, Holzapfel L, Bruins P, Steenken EJ, Jeppesen OK, Lachmann B. Exogenous natural surfactant for treatment of acute lung injury and the acute respiratory distress syndrome. Am J Respir Crit Care Med. 2009 Nov 15;180(10):989-94. doi: 10.1164/rccm.200812-1955OC. Epub 2009 Aug 27.

lead_sponsor: LEO Pharma

location_country:
Denmark
Spain
United Kingdom
Austria
Netherlands
Finland
Canada
Sweden
France
Belgium
Norway
Germany

location_facility: Allgemeines Krankenhaus, Klinik für Anästhesie un Allgemeine Intensivmedizin

location_facility: Hôpital Pitié Salpétrière, Département d'Anesthésie Réanimation

location_facility: University Hospital of Lund, Department of Intensive Care

location_facility: Ullevål University Hospital, INtensive Care Department

location_facility: Klinik für Anästhesiologie der RWTH Aachen

location_facility: Mount Sinai Hospital, Critical Care Unit

location_facility: St Thomas Hospital, Adult Intensive Care

location_facility: Kuopio University Hospital, Intensive Care Unit