title: Multi-center, Randomized, Parallel, Adaptive, Controlled Trial in Adult and Pediatric Patients With Type 1 Diabetes Using Hybrid Closed Loop System and Control (CSII, MDI and SAP) at Home

summary: The purpose of this study is to evaluate the safety and effectiveness of the Hybrid Closed Loop system (HCL) in adult and pediatric patients with type 1 diabetes in the home setting. A diverse population of patients with type 1 diabetes will be studied. The study population will have a large range for duration of diabetes and glycemic control, as measured by glycosylated hemoglobin (A1C). They will be enrolled in the study regardless of their prior diabetes regimen, including using Multiple Daily Injections (MDI), Continuous Subcutaneous Insulin Infusion (CSII) or Sensor-Augmented Pump therapy (SAP)

lead_sponsor: Medtronic Diabetes

location_country:
Spain
Italy
United Kingdom
New Zealand
Canada
Sweden
United States
France
Germany

location_facility: Diabeteszentrum für Kinder und Jugendliche, Kinder- und Jugendkrankenhaus Auf der Bult

location_facility: Diabetes and Glandular Disease Clinic, P.A.

location_facility: Cambridge University Hospitals NHS Foundation Trust - Addenbrooke's Hospital

location_facility: Indiana University Health Riley Hospital for Children

location_facility: Dept Internal Medicine, Örebro University Hospital

location_facility: Center of Excellence in Diabetes & Endocrinology

location_facility: University of South Florida Diabetes Center

location_facility: Texas Children's Hospital / Baylor University

location_facility: Westminster Endocrine & Diabetes Research Society


ipd_sharing: Yes

status: Active, not recruiting

primary_purpose: Treatment

acronym: Next MONARCH 1

intervention_type: Drug

title: A Randomized, Open-Label, Phase 2 Study of Abemaciclib Plus Tamoxifen or Abemaciclib Alone, in Women With Previously Treated Hormone Receptor-Positive, HER2-Negative, Metastatic Breast Cancer

study_completion_date_type: Anticipated

lead_sponsor: Eli Lilly and Company

type: Other Identifier

type: EudraCT Number

source: euctr

design_allocation: Randomized

study_start_date_type: Actual

study_type: Interventional

ReferenceType: derived

ReferenceCitation: Hamilton E, Cortes J, Ozyilkan O, Chen SC, Petrakova K, Manikhas A, Jerusalem G, Hegg R, Huober J, Zhang W, Chen Y, Martin M. nextMONARCH Phase 2 randomized clinical trial: overall survival analysis of abemaciclib monotherapy or in combination with tamoxifen in patients with endocrine-refractory HR + , HER2- metastatic breast cancer. Breast Cancer Res Treat. 2022 Aug;195(1):55-64. doi: 10.1007/s10549-022-06662-9. Epub 2022 Jul 12.

ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

enrollment_type: Actual

location_country:
Taiwan
Brazil
Spain
Italy
Austria
Mexico
Russian Federation
Argentina
United States
France
Turkey
Belgium
Germany
Czechia

location_facility: The Center for Cancer and Blood Disorders

location_facility: Clinica de Pesquisas e Centro de Estudos em Oncologia Ginecológica e Mamária LTDA

location_facility: University of Wisconsin-Madison Hospital and Health Clinic

location_facility: For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

location_facility: St-Petersburg scientifical practical center of specialized kinds of medical care (oncological)

location_facility: Republic Oncology Dispensary of MoH of Republic Tatarstan

location_facility: Saint-Petersburg city clinical oncology dispensary

summary: The main purpose of this study is to evaluate the safety and efficacy of abemaciclib plus tamoxifen or abemaciclib alone in women with previously treated hormone receptor-positive (HR+), human epidermal growth factor receptor 2 negative (HER2-), metastatic breast cancer.

lead_sponsor_type: industriel

status_simplified: Ongoing


title: Phase 3B Open-Label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

summary: The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.

status: Terminated

study_completion_date_type: Actual

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
United Kingdom
Italy
Canada
United States
Israel
France
Bulgaria

location_facility: Institut de Myologie GH Pitié-Salpêtrière

location_facility: Washington University School of Medicine, St. Louis

location_facility: Hadassah-Hebrew University Medical Center

status_simplified: Unknown


status: Completed

acronym: EDITION JUNIOR

title: 6-Month, Multicenter, Randomized, Open-label, 2-Arm, Parallel-group Study Comparing the Efficacy and Safety of a New Formulation of Insulin Glargine and Lantus® Injected Once Daily in Children and Adolescents Age 6 - 17 Years With Type 1 Diabetes Mellitus With a 6-month Safety Extension Period

lead_sponsor: Sanofi

ReferenceCitation: Danne T, Tamborlane WV, Malievsky OA, Franco DR, Kawamura T, Demissie M, Niemoeller E, Goyeau H, Wardecki M, Battelino T. Efficacy and Safety of Insulin Glargine 300 Units/mL (Gla-300) Versus Insulin Glargine 100 Units/mL (Gla-100) in Children and Adolescents (6-17 years) With Type 1 Diabetes: Results of the EDITION JUNIOR Randomized Controlled Trial. Diabetes Care. 2020 Jul;43(7):1512-1519. doi: 10.2337/dc19-1926. Epub 2020 May 19.

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

location_country:
Serbia
Spain
Italy
United Kingdom
United States
Romania
Japan
Czechia
Poland
Russian Federation
Argentina
Israel
Mexico
Sweden
Brazil
Canada
Hungary
North Macedonia
Germany
Bulgaria
Denmark
Latvia
France
Chile

location_facility:
Investigational Site Number 8400035
Investigational Site Number 4840002
Investigational Site Number 3480004
Investigational Site Number 6420005
Investigational Site Number 2760003
Investigational Site Number 2030005
Investigational Site Number 0760003
Investigational Site Number 1240005
Investigational Site Number 8260004
Investigational Site Number 3920005
Investigational Site Number 7240001
Investigational Site Number 6880002
Investigational Site Number 6420004
Investigational Site Number 3480006
Investigational Site Number 1000005
Investigational Site Number 3800005
Investigational Site Number 3480002
Investigational Site Number 0760001
Investigational Site Number 8400015
Investigational Site Number 8400032
Investigational Site Number 3480003
Investigational Site Number 3800001
Investigational Site Number 8260001
Investigational Site Number 0320007
Investigational Site Number 1520007
Investigational Site Number 6160005
Investigational Site Number 0760005
Investigational Site Number 1000004
Investigational Site Number 1520004
Investigational Site Number 2030001
Investigational Site Number 2500003
Investigational Site Number 6160003
Investigational Site Number 6160007
Investigational Site Number 8400030
Investigational Site Number 6430004
Investigational Site Number 3920003
Investigational Site Number 1240003
Investigational Site Number 8400029
Investigational Site Number 8400005
Investigational Site Number 6430003
Investigational Site Number 3920004
Investigational Site Number 0760002
Investigational Site Number 0320002
Investigational Site Number 3800003
Investigational Site Number 4280002
Investigational Site Number 2080001
Investigational Site Number 3800004
Investigational Site Number 3920006
Investigational Site Number 4840005
Investigational Site Number 2760001
Investigational Site Number 6420003
Investigational Site Number 1520006
Investigational Site Number 6160006
Investigational Site Number 7240004
Investigational Site Number 3480007
Investigational Site Number 0320004
Investigational Site Number 6430002
Investigational Site Number 4840004
Investigational Site Number 6420007
Investigational Site Number 3920002
Investigational Site Number 3760002
Investigational Site Number 6420006
Investigational Site Number 8400021
Investigational Site Number 7520002
Investigational Site Number 8400010
Investigational Site Number 8400016
Investigational Site Number 1000001
Investigational Site Number 4840003
Investigational Site Number 0760004
Investigational Site Number 6430001
Investigational Site Number 0760006
Investigational Site Number 0320001
Investigational Site Number 2030003
Investigational Site Number 0320005
Investigational Site Number 1240002
Investigational Site Number 0320006
Investigational Site Number 2760004
Investigational Site Number 8070001
Investigational Site Number 3480005
Investigational Site Number 2500002
Investigational Site Number 3760003
Investigational Site Number 8400008
Investigational Site Number 0320003
Investigational Site Number 8400037
Investigational Site Number 1520002
Investigational Site Number 3760001
Investigational Site Number 3760006
Investigational Site Number 2760002
Investigational Site Number 3800006
Investigational Site Number 1520003
Investigational Site Number 8260002
Investigational Site Number 3920007
Investigational Site Number 4280001
Investigational Site Number 4840001
Investigational Site Number 7240003
Investigational Site Number 6880004
Investigational Site Number 1240006
Investigational Site Number 3480001
Investigational Site Number 8400038
Investigational Site Number 6880003
Investigational Site Number 7240002
Investigational Site Number 7240006
Investigational Site Number 8260005
Investigational Site Number 6160001
Investigational Site Number 6160004
Investigational Site Number 8400034
Investigational Site Number 7240005

summary:
Primary Objective:

To compare the efficacy of a new formulation of insulin glargine (HOE901-U300) to Lantus in terms of change of HbA1c from baseline to endpoint (month 6) in children and adolescents with type 1 diabetes mellitus.

.

Secondary Objectives:

To compare HOE901-U300 and Lantus in terms of:

Percentage of participants reaching target HbA1c and fasting plasma glucose (FPG).
To assess the safety of HOE901-U300 including analysis of events of hypoglycemia, events of hyperglycemia with ketosis, and development of anti-insulin-antibodies.

status_simplified: Completed


title: A Phase I/II, Multi-center, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis

summary: This trial will consist of three arms: Part A, Part B, and Part C. Part A has two groups. The first group will enroll adult subjects with cystic fibrosis (CF) into a single ascending dose (SAD) treatment group. The second group will enroll adult subjects with CF, including those on background treatment with ORKAMBI® and those not on a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, into a multiple ascending dose (MAD) treatment group. Part B will enroll adult subjects with CF currently on stable ORKAMBI® background therapy for a minimum of 3 months into a Phase II treatment group consisting of two cohorts. Part C will enroll adult subjects with CF, including those on background treatment with KALYDECO® and those not on a CFTR modulator, into a Phase II treatment group consisting of three cohorts. Approximately 136 subjects will be enrolled.

lead_sponsor: Proteostasis Therapeutics, Inc.

location_country:
Denmark
Canada
United States
France
Germany

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Quebec

location_facility: Groupe Hospitalier Pellegrin - Hôpital des Enfants

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: University of Florida College of Medicine

location_facility: Northwestern University Memorial Hospital

location_facility: Universitaetsklinikum Frankfurt-Zentrum der Inneren Medizin

location_facility: St. Luke's Cystic Fibrosis Center of Idaho

location_facility: University of Kansas Medical Center Research Institute, Inc.

location_facility: University of Oklahoma Health Sciences Center

location_facility: Institut de Recherches Cliniques de Montreal

location_facility: St. Paul's Hospital Pacific Lung Research Center


title: A Randomized, Open-Label Phase II/III Study With Dendritic Cells Loaded With Allogeneic Tumour Cell Lysate (PheraLys) in Subjects With Mesothelioma as Maintenance Treatment (MesoPher) After Chemotherapy

acronym: DENIM

summary: This study is to evaluate the overall survival (OS) rate (determined from the time of randomization in the study) of subjects who receive dendritic cell immunotherapy with MesoPher plus best supportive care (BSC) compared to BSC alone.

status: Recruiting

enrollment_type: Anticipated

ipd_sharing: Undecided

lead_sponsor: Amphera BV

location_country:
United Kingdom
Italy
France
Belgium
Netherlands

location_facility: Universitá Politecnica delle Marche - Ospedali Riuniti di Ancona

location_facility: Centre Hospitalier Régional Universitaire de Lille


title: Characterization of Acute and Long Term Response to Left Ventricle Only Pacing Combined With MultiPoint Pacing and SyncAV

summary:
The objective of this clinical investigation is to evaluate the clinical benefits of left ventricle (LV) only pacing combined with automatic adjustment of AV timing (SyncAV) in patients receiving cardiac resynchronization therapy (CRT) after 6 months of therapy.

This clinical investigation is a prospective, two-arm, randomized 1:1, multicenter feasibility study designed to evaluate the effectiveness of LV only with multipoint pacing (MPP) and SyncAV compared to bi-ventricular pacing with MPP and SyncAV. The clinical investigation will be conducted at approximately 7 centers in Europe and Canada. Approximately 120 subjects will be enrolled in the study. No site may enroll more than 33% of the total subjects.

Data will be collected at enrollment, CRT implant procedure, hospital pre-discharge, one and 6 months post implant. Enrollment data collection will include demographics, cardiovascular history, medication, echocardiography measurements and quality of life questionnaire. CRT implant procedure data collection will include implanted system information and lead location. The electrical conduction recording procedure will include surface ECG and device IEGM recordings during various pacing configurations at implant or up to 45 days post implant. In patients who consent to invasive measurements (expected target of at least 80 patients), a hemodynamic recording procedure will include invasive hemodynamic measurements during various pacing configurations which may take place during device implant or up to 45 days post implant. Hospital pre-discharge data collection will take place within 3 days after the CRT implant, electrical conduction recordings visit or hemodynamic recordings visit and will include system information, surface ECG, and device IEGMs.

In a subset of patients from selected centers that have access to this technology (expected 20 patients), non-invasive electrical activation data will be collected with body surface mapping within 45 days of the implant procedure.

Patients will be randomized 1:1 to receive either biventricular pacing with multipoint pacing (MPP) or LV-only pacing with MPP at the one-month (± 15 days) visit. The 6-month (± 15 days) post randomization follow up visit will include surface ECG, IEGMs, echocardiographic parameters and quality of life questionnaire.

Subjects participating in this clinical investigation will follow the hospital center standard of care from implant to 6 month follow up. The expected duration of enrollment is 1.5 year. The total duration of the clinical investigation is expected to be 2 years.

status: Enrolling by invitation

ipd_sharing: No

ipd_sharing_description: No participant data will be shared to other researchers

lead_sponsor: Abbott Medical Devices

location_country:
Canada
Italy
United Kingdom
France

location_facility:
CHU Rennes
John Radcliffe Hospital
Montreal Heart Institute
Queen Elizabeth Hospital
St. Bartholomew's Hospital
Policlinico Casilino

intervention_type: Device


acronym: PETITE

title: A Multicenter, Open-Label, Single-Arm Study to Evaluate the Pharmacokinetics, Efficacy, and Safety of Brivaracetam in Neonates With Repeated Electroencephalographic Seizures

lead_sponsor: UCB Biopharma SRL

ipd_sharing_description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

location_country:
United Kingdom
Ireland
France
Belgium
Germany

summary: The purpose of the study is to evaluate the pharmacokinetics (PK) of brivaracetam (BRV) in neonates who have seizures that are not adequately controlled with previous antiepileptic drug (AED) treatment, and to identify the optimal BRV dose (Exploratory Cohort) for the treatment of subjects enrolled into the Confirmatory Cohorts of this study.


title: Performance of a New REsting Pressure Index During Invasive Angiography Compared To Adenosine Hyperemic FFR

acronym: PREDICT

summary: To test the feasibility and diagnostic accuracy of a new automated pressure derived resting index (Pd/Pamin), using FFR as gold standard, in de novo coronary lesions in which invasive physiological evaluation is warranted.

status: Unknown status

study_type: Observational

time_perspective: Prospective

ReferenceType: background

ReferenceCitation: Levine GN, Bates ER, Blankenship JC, Bailey SR, Bittl JA, Cercek B, Chambers CE, Ellis SG, Guyton RA, Hollenberg SM, Khot UN, Lange RA, Mauri L, Mehran R, Moussa ID, Mukherjee D, Nallamothu BK, Ting HH; American College of Cardiology Foundation; American Heart Association Task Force on Practice Guidelines; Society for Cardiovascular Angiography and Interventions. 2011 ACCF/AHA/SCAI Guideline for Percutaneous Coronary Intervention. A report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines and the Society for Cardiovascular Angiography and Interventions. J Am Coll Cardiol. 2011 Dec 6;58(24):e44-122. doi: 10.1016/j.jacc.2011.08.007. Epub 2011 Nov 7.

ReferenceCitation: Task Force on Myocardial Revascularization of the European Society of Cardiology (ESC) and the European Association for Cardio-Thoracic Surgery (EACTS); European Association for Percutaneous Cardiovascular Interventions (EAPCI), Wijns W, Kolh P, Danchin N, Di Mario C, Falk V, Folliguet T, Garg S, Huber K, James S, Knuuti J, Lopez-Sendon J, Marco J, Menicanti L, Ostojic M, Piepoli MF, Pirlet C, Pomar JL, Reifart N, Ribichini FL, Schalij MJ, Sergeant P, Serruys PW, Silber S, Sousa Uva M, Taggart D. Guidelines on myocardial revascularization. Eur Heart J. 2010 Oct;31(20):2501-55. doi: 10.1093/eurheartj/ehq277. Epub 2010 Aug 29.

ReferenceCitation: Mamas MA, Horner S, Welch E, Ashworth A, Millington S, Fraser D, Fath-Ordoubadi F, Neyses L, El-Omar M. Resting Pd/Pa measured with intracoronary pressure wire strongly predicts fractional flow reserve. J Invasive Cardiol. 2010 Jun;22(6):260-5.

ReferenceCitation: Sen S, Escaned J, Malik IS, Mikhail GW, Foale RA, Mila R, Tarkin J, Petraco R, Broyd C, Jabbour R, Sethi A, Baker CS, Bellamy M, Al-Bustami M, Hackett D, Khan M, Lefroy D, Parker KH, Hughes AD, Francis DP, Di Mario C, Mayet J, Davies JE. Development and validation of a new adenosine-independent index of stenosis severity from coronary wave-intensity analysis: results of the ADVISE (ADenosine Vasodilator Independent Stenosis Evaluation) study. J Am Coll Cardiol. 2012 Apr 10;59(15):1392-402. doi: 10.1016/j.jacc.2011.11.003. Epub 2011 Dec 7.

ReferenceCitation: Ganz P, Abben R, Friedman PL, Garnic JD, Barry WH, Levin DC. Usefulness of transstenotic coronary pressure gradient measurements during diagnostic catheterization. Am J Cardiol. 1985 Apr 1;55(8):910-4.

ReferenceCitation: Grüntzig AR, Senning A, Siegenthaler WE. Nonoperative dilatation of coronary-artery stenosis: percutaneous transluminal coronary angioplasty. N Engl J Med. 1979 Jul 12;301(2):61-8.

ReferenceCitation: Berry C, van 't Veer M, Witt N, Kala P, Bocek O, Pyxaras SA, McClure JD, Fearon WF, Barbato E, Tonino PA, De Bruyne B, Pijls NH, Oldroyd KG. VERIFY (VERification of Instantaneous Wave-Free Ratio and Fractional Flow Reserve for the Assessment of Coronary Artery Stenosis Severity in EverydaY Practice): a multicenter study in consecutive patients. J Am Coll Cardiol. 2013 Apr 2;61(13):1421-7. doi: 10.1016/j.jacc.2012.09.065. Epub 2013 Feb 6.

ReferenceCitation: Ganz P, Harrington DP, Gaspar J, Barry WH. Phasic pressure gradients across coronary and renal artery stenoses in humans. Am Heart J. 1983 Dec;106(6):1399-406.

lead_sponsor: Centro Hospitalar Lisboa Ocidental

location_country:
United States
Italy
Portugal
France

location_facility: Centro Hospitalar de Lisboa Ocidental - Hospital de Santa Cruz

location_facility: Centro Hospitalar de Lisboa Central (CHLC) - Hospital de Santa Marta

location_facility: Department of Cardiology, University Hospital

location_facility: Institute of Cardiology, Catholic University of the Sacred Heart

location_facility: Hospital Prof. Doutor Fernando da Fonseca

lead_sponsor_type: academique


title: Assessment of the Efficacy and Safety of a New Medical Device in the Local Treatment of Diabetic Foot Ulcers: a Prospective, Randomized, Controlled, Double-blind, European Multicentre Clinical Trial

acronym: DISCOVERY

summary: Evaluation of the efficacy and safety of a device with LU3103209 compound versus the same device, free of LU3103209 compound, in the local management of DFUs.

lead_sponsor: Laboratoires URGO

location_country:
Poland
Spain
Italy
United Kingdom
France
Czechia

location_facility:
Pr JIRKOVSKA
Dr KORZON
Pr LAZARO
Pr PIAGGESI
Pr PETIT
Pr EDMONDS


title: Study of Early Endocrine Profile in Infants With Prader-Willi Syndrome (PWS) in Order to Unravel the Switch From Early Feeding Difficulties to Obesity and Hyperphagia.

summary:
The present project aims to determine the underlying mechanisms for the switch from failure to thrive to excessive weight gain and hyperphagia with impaired satiety in PWS. The primary objective is to describe the evolution of circulating hormones involved in feeding and appetite regulation during the 4 first years of life. The secondary objective is to make this blood bank available for other research projects and particularly the investigation of hormones involved in hypogonadism.

Over the last ten years, the age at diagnosis in PWS has fallen significantly and the majority of cases is now diagnosed during the 1st trimester of life giving the possibility to collect precise clinical data and serum samples at early stages. The investigators of the project are involved in the care of patients with PWS and have a devoted clinic and an organized network in their country through clinical networks or patient associations.

design_allocation: Non-Randomized

primary_purpose: Basic Science

ReferenceCitation: Cadoudal T, Buléon M, Sengenès C, Diene G, Desneulin F, Molinas C, Eddiry S, Conte-Auriol F, Daviaud D, Martin PG, Bouloumié A, Salles JP, Tauber M, Valet P. Impairment of adipose tissue in Prader-Willi syndrome rescued by growth hormone treatment. Int J Obes (Lond). 2014 Sep;38(9):1234-40. doi: 10.1038/ijo.2014.3. Epub 2014 Jan 10.

ReferenceCitation: Beauloye V, Diene G, Kuppens R, Zech F, Winandy C, Molinas C, Faye S, Kieffer I, Beckers D, Nergårdh R, Hauffa B, Derycke C, Delhanty P, Hokken-Koelega A, Tauber M. High unacylated ghrelin levels support the concept of anorexia in infants with prader-willi syndrome. Orphanet J Rare Dis. 2016 May 4;11(1):56. doi: 10.1186/s13023-016-0440-0.

lead_sponsor: University Hospital, Toulouse

location_country:
United Kingdom
Netherlands
Sweden
France
Belgium
Germany

location_facility: Unité d'Endocrinologie Pédiatrique / Université Catholique de Louvain

location_facility: Metabolic & Molecular Imaging Group / MRC Clinical Sciences Centre / Imperial College London / Hammersmith Hospital

location_facility: Department of Pediatrics / Division of Endocrinology / Erasmus University Medical Center / Sophia Children's Hospital Rotterdam

location_facility: Department of Endocrinology / University Children's Hospital

intervention_type: Other


title: Phase 2/3 Study of Masitinib in Combination With Gemcitabine Versus Gemcitabine Alone in Advanced/Metastatic Epithelial Ovarian Cancer Patients in Second Line Being Refractory to First Line Platinum Treatment or in Third Line

summary: The objective is to evaluate the efficacy and safety of masitinib in combination with gemcitabine in refractory ovarian cancer patients.

lead_sponsor: AB Science

location_country:
Spain
United Kingdom
Russian Federation
Ukraine
Algeria
France

location_facility: Complejo Hospitalario Universitario de Santiago

location_facility: Centre hospitalo-universitaire de Beni Messous

location_facility: Lviv State Oncological Regional Treatment and Diagnostic Center


intervention_type: Biological

title: Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients

lead_sponsor: Promethera Therapeutics

location_country: Poland France Spain Belgium

location_facility: Instytut - Pomnik Centrum Zdrowia Dziecka

location_facility: Hospital Universitari Vall d'Hebron de Barcelona

summary: The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.


title: A Comparison of Glycosade® and Uncooked Cornstarch (UCCS) for the Dietary Management of Hepatic Glycogen Storage Diseases (GSD)

acronym: Glyde

summary: To compare efficacy of Glycosade® with uncooked corn starch (UCCS for the dietary management of hepatic glycogen storage diseases (GSD).

lead_sponsor: Vitaflo International, Ltd

location_country:
Netherlands
United States
United Kingdom
France

location_facility: National Hospital for Neurology and Neurosurgery

location_facility: Great Ormond Street Hospital for Children

intervention_type: Dietary Supplement


intervention_type: Combination Product

title: A Phase 1/2 Study of Durvalumab and Monalizumab in Adult Subjects With Select Advanced Solid Tumors

lead_sponsor: MedImmune LLC

type: Other Grant/Funding Number

ipd_sharing_description: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure

location_country:
Spain
Italy
United Kingdom
Australia
New Zealand
Canada
Hungary
Korea, Republic of
United States
France
Belgium

summary: This is a multicenter, open-label, dose-escalation, dose-exploration and dose-expansion study to evaluate the safety, tolerability, antitumor activity, PK, pharmacodynamics, and immunogenicity of Durvalumab (MEDI4736) in combination with monalizumab (IPH2201) in Adult Subjects with selected advanced solid tumors and the combination of durvalumab and monalizumab (IPH2201) standard of care systemic therapy with or without biological agent and monalizumab (IPH2201) with biological agent administered to subjects with recurrent or metastatic colorectal cancer (CRC).


title: A Multicentre Observational Study to Evaluate Clinical Outcomes of the G7 Acetabular System

summary: The primary purpose of this study is to evaluate the clinical and radiographic performance of the G7 Acetabular Cup System.

time_perspective: Retrospective

lead_sponsor: Zimmer Biomet

location_country:
Spain
Ireland
Netherlands
United States
France
Germany

location_facility: Midwestern Regional Orthopaedic Hospital


title: An Exploratory Phase 2 a Study to Assess Safety, Tolerability, Pharmacodynamic and Pharmacokinetics of VAS203 in Patients With Moderate and Severe Traumatic Brain Injury

acronym: NOSTRA

summary:
In the exploratory multi-center Phase 2 a study safety, tolerability, pharmacodynamics and pharmacokinetics of the Nitric Oxide Synthase inhibitor VAS203 is assessed in patients with moderate and severe traumatic brain injury. Traumatic brain injury patients (32 males) receive 15, 20 and 30 mg/kg VAS203, respectively, by continuous infusion in three cohorts (Cohort 1 open; Cohorts 2 and 3 double blind, randomised placebo-controlled). End of Study for all patients will be Day 14; adverse events and concomitant medications will be documented throughout the study.

Objectives are to assess safety and tolerability of VAS203, to evaluate concentrations of metabolites of VAS203 in plasma and microdialysate and to assess pharmacodynamic effects of VAS203 on surrogate parameters. Safety parameter will include vital signs (blood pressure heart rate, respiration rate, oxygen saturation and blood gases), fluid balance, ECG, laboratory examinations (clinical chemistry, liver function, haematology/coagulation, urinalysis, renal parameters) and adverse events. Concentration of VAS203 will be determined in plasma and microdialysate. Pharmacodynamic parameters will include intracranial pressure (ICP), biochemical parameters in microdialysate (nitrite/nitrate, arginine, citrulline, pyruvate, lactate, glucose), Partial Oxygen Pressure in brain parenchyma and Therapy Intensity Level (TIL).

ReferenceType: result

ReferenceCitation: Stover JF, Belli A, Boret H, Bulters D, Sahuquillo J, Schmutzhard E, Zavala E, Ungerstedt U, Schinzel R, Tegtmeier F; NOSTRA Investigators. Nitric oxide synthase inhibition with the antipterin VAS203 improves outcome in moderate and severe traumatic brain injury: a placebo-controlled randomized Phase IIa trial (NOSTRA). J Neurotrauma. 2014 Oct 1;31(19):1599-606. doi: 10.1089/neu.2014.3344. Epub 2014 Jul 28.

lead_sponsor: Vasopharm GmbH

location_country:
Spain
United Kingdom
Austria
Switzerland
France

location_facility: Hospital Clinic University of Barcelona Surgical Intensive Care Unit

location_facility: Vall d'Hebron University Hospital Department of Neurosurgery

location_facility: Southampton University Hospital Division of Clinical Neurosciences

location_facility: University Hospital Zuerich Surgical Intensive Care

location_facility: Medical University Innsbruck Department of Neurology


title: A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.

lead_sponsor: BioMarin Pharmaceutical

location_facility: Institute of Genetic Medicine International Centre for Life

location_facility: Policlinico Universitario Agostino Gemelli

summary: The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.


title: A Phase IIb, Open-label Study to Assess the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of Multiple Subcutaneous Doses of PRO045 in Subjects With Duchenne Muscular Dystrophy

summary: The purpose of the study is to see whether PRO045 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 45 in the DNA for the dystrophin protein.


title: Clinical Evaluation of Therapy™ Cool Flex Ablation Catheter for the Treatment of Paroxysmal Atrial Fibrillation

acronym: FACT-AF

summary:
The purpose of this study is to observe the performance of Therapy™ Cool Flex™ catheter by documenting the procedural parameters, major complications and recurrence of AF after ablation using Cool Flex ablation catheter for the treatment of paroxysmal atrial fibrillation.

This study is an observational clinical study which is not based on any specific endpoints.

primary_purpose: Other

location_country:
Portugal
United Kingdom
Italy
Australia
France
Germany

location_facility:
Hospital Santa Cruz
Ospendale dell'Angelo
Hopital du Haut Leveque
Heart Hospital
Universitat Leipzig Herzzentrum
Royal Adelaide Hospital


title: A Multi-Center, Single Arm, Prospective Study of the WallFlex™ Biliary Partially-covered Stent for the Palliative Treatment of Malignant Bile Duct Obstruction

summary: This is a a prospective study of the WallFlex™ Biliary Partially-covered Stent designed to collect data to support regulatory clearance by the FDA in the United States and to determine the functionality of the WallFlex™ Biliary Partially Covered Stent as a Palliative treatment for malignant bile duct obstruction.

lead_sponsor: Boston Scientific Corporation

location_country: Italy Netherlands India France Belgium Germany

location_facility: EVK Krankenhaus der Universitat Dusseldorf

location_facility: Academisch Medisch Centrum Universiteit van Amsterdam


summary: Study compares the efficacy and safety of FLUTIFORM® with Seretide® in the treatment of mild to moderate persistent asthma in pediatric subjects.

lead_sponsor: Mundipharma Research Limited

location_country: Poland Hungary France Romania Germany Czechia


title: A Phase II, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Efficacy and Safety Study of NV1FGF in Patients With Severe Peripheral Artery Occlusive Disease.

acronym: TALISMAN 201

summary: This study is a double-blind study to evaluate the efficacy and safety of NV1FGF, a pCOR plasmid constructed by inserting the gene coding for the FGF compared to placebo in patients with severe Peripheral Arterial Occlusive Disease, Fontaine's stage IV. The efficacy was assessed by the complete healing of at least one ulcer in the treated limb, 25 week post-baseline and secondary by the rate of amputation and death.

ReferenceCitation: Nikol S, Baumgartner I, Van Belle E, Diehm C, Visoná A, Capogrossi MC, Ferreira-Maldent N, Gallino A, Wyatt MG, Wijesinghe LD, Fusari M, Stephan D, Emmerich J, Pompilio G, Vermassen F, Pham E, Grek V, Coleman M, Meyer F; TALISMAN 201 investigators. Therapeutic angiogenesis with intramuscular NV1FGF improves amputation-free survival in patients with critical limb ischemia. Mol Ther. 2008 May;16(5):972-8. doi: 10.1038/mt.2008.33. Epub 2008 Apr 1.

ReferenceCitation: Niebuhr A, Henry T, Goldman J, Baumgartner I, van Belle E, Gerss J, Hirsch AT, Nikol S. Long-term safety of intramuscular gene transfer of non-viral FGF1 for peripheral artery disease. Gene Ther. 2012 Mar;19(3):264-70. doi: 10.1038/gt.2011.85. Epub 2011 Jun 30.

lead_sponsor: Sanofi

location_country:
United Kingdom
Italy
Switzerland
France
Belgium
Germany

location_facility:
Istituto Dermopatico dell'Immacolata
St George's Hospital
Hôpital Européen Georges Pompidou
Klinikum Karlsbad-Langensteinbach
Universitätsklinik
Ghent University Hospital


acronym: ACTIVATE-Kids

title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

lead_sponsor: Agios Pharmaceuticals, Inc.

location_country:
Spain
Canada
United States
Israel
France

location_facility: Hospital Universitario Infantil Nino Jesus

location_facility: Centre hospitalier Universitaire de Sainte-Justine

summary: Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving blood transfusions. Participants will be randomized 2:1 to receive either mitapivat or matching placebo. Randomization will be stratified by age (1 to < 6 years, 6 to < 12 years, 12 to < 18 years). Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 12-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat for up to 5 years in the open-label extension (OLE) period.


title: User Evaluation New Ambient Experience Content

summary:
The study aims to investigate the effect of Philips Ambient Experience with specially designed paediatric content on the experience of pediatric patients undergoing an MRI examination, their caregiver, and clinical staff, on workflow and on clinical outcome. The designed paediatric content exists of personal selection of a character, video projection on the wall and/or a screen visible when in the bore, audio, and room ambience lighting.

The study will be conducted in collaboration with six partner hospitals within the EU. At each hospital, 50 pediatric patients between 6 and 12 years old will be included in the study.

study_start_date_type: Anticipated

status: Not yet recruiting

primary_purpose: Supportive Care

lead_sponsor: Philips Electronics Nederland B.V. acting through Philips CTO organization

location_country: Denmark Poland Spain France Belgium Germany

location_facility: Polish Mother's Hospital / Institute of Polish Mother's Health Center

intervention_type: Behavioral


title: A Prospective, 5-Year Global Study on MAST™ Minimally Invasive Fusion Procedures for the Treatment of the Degenerative Lumbar Spine

summary:
The purpose of this study is

to evaluate the effectiveness of MAST techniques for anterior/lateral and posterior approaches in Degenerative Disc Disease (DDD) patients with spondylolisthesis (≥ grade I).
To assess how single or double level MAST(Minimal Access Spinal Technologies) fusion procedures PLIF (Posterior Lumbar Interbody Fusion), TLIF (Transforaminal Lumbar Interbody Fusion), DLIF (Direct Lateral Interbody Fusion), OLIF (Oblique Lumbar Interbody Fusion), ALIF (Anterior Lumbar Interbody Fusion), or MIDLF (Midline Lumbar Interbody Fusion) are used in surgical practice and to describe long-term safety and effectiveness in a broad patient population of patients with degenerative lumbar disc disease .

lead_sponsor: Medtronic Spinal and Biologics

location_country:
Denmark
Brazil
Portugal
Spain
Italy
Austria
Mexico
Russian Federation
Argentina
United Kingdom
Korea, Republic of
France
China
Belgium
Germany
Slovakia
Czechia

location_facility: Scientific Research Institute of Traumatology and Orthopedics

location_facility: East Suffolk and North Essex NHS Foundation Trust (was formerly Ipswich Hospital NHS)

location_facility: National Health Insurance Corporation Ilsan Hospital

location_facility: Hospital Naval General de Alta Especialidad

location_facility: Department of Orthopedics, Korea University Ansan Hospital

location_facility: The First Affiliated Hospital of Dalian Medical University

location_facility: Department of Neurological Surgery, Chung-Ang University Hospital

location_facility: Nizhny Novgorod Research Institute of Traumatology and Orthopedics of Public Health Ministry

location_facility: Hospital Universitario Marques de Valdecilla


title: Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis

summary: The primary objectives of the study are to estimate and rank-order the longitudinal standardized mean changes over 6 months and over 12 months, for a set of outcome measures administered to participants with amyotrophic lateral sclerosis (ALS), in order to identify measures that are more sensitive to disease progression than Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). The secondary objectives of this study are: To evaluate the test-retest reproducibility of each outcome measure; To determine correlations between 6 and 12-month changes in all exploratory measures with 18 and 24-month changes in ALSFRS-R and survival; To assess correlations between/among the various measures; To obtain biological samples in order to identify molecular correlates to the clinical measures and to further characterize previously identified and novel molecular biomarkers of disease progression for incorporation into future clinical studies.

lead_sponsor: Biogen

location_country:
United Kingdom
Ireland
Netherlands
Canada
Switzerland
United States
France
Belgium
Germany

location_facility: Montreal Neurological Institute Clinical Research Unit

location_facility: Washington University School of Medicine

location_facility: Hopital Gui de Chauliac, Service de Neurologie

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: University of California San Diego Medical Center


title: A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma

summary:
This is a Phase I, open-label, dose escalation and dose expansion study with BID (suspension) and TID (tablet) oral dose of tazemetostat. Subjects will be screened for eligibility within 14 days of the planned first dose of tazemetostat. A treatment cycle will be 28 days. Response assessment will be evaluated after 8 weeks of treatment and subsequently every 8 weeks while on study.

The study has two parts: Dose Escalation and Dose Expansion.

Dose escalation for subjects with the following relapsed/refractory malignancies:

Rhabdoid tumors:
Atypical teratoid rhabdoid tumor (ATRT)
Malignant rhabdoid tumor (MRT)
Rhabdoid tumor of kidney (RTK)
Selected tumors with rhabdoid features
INI1-negative tumors:
Epithelioid sarcoma
Epithelioid malignant peripheral nerve sheath tumor
Extraskeletal myxoid chondrosarcoma
Myoepithelial carcinoma
Renal medullary carcinoma
Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) (with Sponsor approval)
Synovial Sarcoma with a SS18-SSX rearrangement Dose Escalation cohorts are closed to enrollment.

Dose Expansion at the MTD or the RP2D

Cohort 1 - ATRT (closed to enrollment)
Cohort 2 - MRT/RTK/selected tumors with rhabdoid features (closed to enrollment)

Cohort 3 - INI-negative tumors:

Epithelioid sarcoma
Epithelioid malignant peripheral nerve sheath tumor
Extraskeletal myxoid chondrosarcoma
Myoepithelial carcinoma
Renal medullary carcinoma
Chordoma (poorly differentiated or de-differentiated)
Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) with Sponsor approval
Cohort 4 - Tumor types eligible for Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement (closed to enrollment)

lead_sponsor: Epizyme, Inc.

location_country:
Denmark
United Kingdom
Italy
Australia
Netherlands
Canada
United States
France
Germany

location_facility: Great Ormond Street Hospital for Children NHS Foundation Trust

location_facility: St. Jude Children's Research Hospital, Inc.

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

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Texas Children's Cancer and Hematology Center

location_facility: University of California San Francisco - Benioff Children's Hospital

location_facility: Istituto Giannina Gaslini- UOSD Centro di Neuro-Oncologia

location_facility: Lady Cilento/Queensland Children's Hospital

location_facility: Prinses Maxima Centrum voor Kinderoncologie

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: The Childrens Hospital at Westmead Oncology Unit

location_facility: Massachusetts General Hospital - Cancer Center

location_facility: Rigshospitalet Department of Oncology Blegdamsvej

location_facility: Oregon Health & Science University (OHSU)

location_facility: Westfalische Wilhelms - Universitat Munster Padiatrische

location_facility: Central Manchester University Hospital - Royal Manchester Children's Hospital

location_facility: Erasmus University Medical Center - Sophia Children's Hospital


title: Effect of Intense Sport Practice in Athletes With Non-ischemic Scar

acronym: EISCAR

summary:
The prognostic relevance of isolated non-ischemic LGE (i.e. with no underlying "labelled" cardiomyopathy) is unclear, and current guidelines to not state on the clearance of athlete with this type of findings as regards to competitive or intense sport practice.

The principal objective of the study is to evaluate during a five-years follow up, the clinical outcome of athletes with this kind of findings. The secondary objective is the determination of prognostic factors. The management and follow-up of the athletes will be let at the appraisal of each center.

lead_sponsor: Rennes University Hospital

location_country:
Luxembourg
United Kingdom
Australia
France
Belgium
Germany

location_facility:
St. George's University of London
Hospital Center of Luxembourg
Saarland University
Baker IDI Heart and Diabetes Institute
University Hospital Gasthuisberg
CHU de Rennes


acronym: AQUA

title: Open-label Phase-4 Study to Examine the Change of Vision-related Quality of Life in Subjects With Diabetic Macular Edema (DME) During Treatment With Intravitreal Injections of 2 mg Aflibercept According to EU Label for the First Year of Treatment

lead_sponsor: Bayer

location_country:
Poland
Portugal
Spain
Italy
Austria
United Kingdom
Canada
Lithuania
Switzerland
Hungary
France
Germany
Slovakia
Czechia

summary: To evaluate the change in quality of life (NEI VFQ 25) in subjects with DME during the first year of treatment with aflibercept according to the EU Label.


title: Comprehensive Nano Post Market Data Collection

summary: This data collection project is intended to provide clinical outcomes data to engineering, marketing as well as fulfill the post market surveillance requirements of various regulatory authorities. The study will be a prospective and one-arm.

location_country:
United Kingdom
Austria
Canada
Korea, Republic of
France

location_facility:
Kingston General Hospital
Privatklinik Leech
Chonnam National University Hospital
University of Liverpool
Hospial Lapeyronie
Samsung Medical Centre


acronym: CheckMate 436

title: A Phase I/ II Study to Evaluate the Safety and Preliminary Efficacy of Nivolumab in Combination With Brentuximab Vedotin in Subjects With Relapsed Refractory Non Hodgkin Lymphomas With CD30 Expression (CheckMate 436: CHECKpoint Pathway and Nivolumab Clinical Trial Evaluation 436)

lead_sponsor: Bristol-Myers Squibb

ReferenceCitation: Zinzani PL, Santoro A, Gritti G, Brice P, Barr PM, Kuruvilla J, Cunningham D, Kline J, Johnson NA, Mehta-Shah N, Manley T, Francis S, Sharma M, Moskowitz AJ. Nivolumab Combined With Brentuximab Vedotin for Relapsed/Refractory Primary Mediastinal Large B-Cell Lymphoma: Efficacy and Safety From the Phase II CheckMate 436 Study. J Clin Oncol. 2019 Nov 20;37(33):3081-3089. doi: 10.1200/JCO.19.01492. Epub 2019 Aug 9.

location_country:
Spain
Italy
United Kingdom
Canada
United States
France

location_facility: Washington University School Of Medicine

location_facility: The Ohio State University Comprehensive Cancer Center

location_facility: Alma Mater Studiorum - Universita' Di Bologna

location_facility: U of Washington / Seattle Cancer Care Alliance

location_facility: University Of Miami Sylvester Comprehensive Cancer Center

summary: The purpose of this study is to determine whether Nivolumab, in combination with brentuximab vedotin, is safe and effective in patients with certain subtypes of non-Hodgkin's lymphomas with CD30 expression that have not responded to treatment or have come back. The subtypes we are studying are Diffuse Large B-Cell Lymphoma (DLBCL), Peripheral T-Cell Lymphoma (PTCL), Cutaneous T-Cell Lymphoma (CTCL), Primary Mediastinal Large B-Cell Lymphoma (PMBL) and Mediastinal Gray Zone Lymphoma (MGZL).


title: A Phase 3 Open-Label Extension Study to Evaluate the Long-Term Safety of Omiganan Topical Gel in Subjects With Rosacea

summary: This study evaluates the long-term safety of once-daily application of Omiganan topical gel in subjects with severe papulopustular rosacea.

lead_sponsor: Maruho Co., Ltd.

location_country:
United Kingdom
Australia
New Zealand
Canada
Sweden
United States
France
Netherlands


title: A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated Velmanase Alfa (Recombinant Human Alpha-mannosidase) Treatment in Pediatric Patients Below 6 Years of Age With Alpha-Mannosidosis

lead_sponsor: Chiesi Farmaceutici S.p.A.

location_country: Denmark Italy Austria France Germany

summary: The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years


acronym: JAVELIN Medley

title: A PHASE 1B/2 OPEN-LABEL STUDY TO EVALUATE SAFETY, CLINICALACTIVITY, PHARMACOKINETICS AND PHARMACODYNAMICS OFAVELUMAB (MSB0010718C) IN COMBINATION WITH OTHER CANCERIMMUNOTHERAPIES IN PATIENTS WITH ADVANCED MALIGNANCIES

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:
Poland
Taiwan
United Kingdom
Australia
Canada
United States
France
Japan

location_facility: Investigational Pharmacy, Karmanos Cancer Center

location_facility: NYU Laura and Isaac Perlmutter Cancer Center

location_facility: University of Iowa Hospitals and Clinics

location_facility: UT Southwestern Simmons Comprehensive Cancer Center

location_facility: Weill Cornell Medical College/New York Presbyterian Hospital

location_facility: The Sarah Cannon Research Institute / Tennessee Oncology, PLLC

location_facility: British Columbia Cancer Agency - Vancouver Centre

location_facility: H Lee Moffitt Cancer Center and Research Institute

location_facility: Karmanos Cancer Institute Weisberg Cancer Treatment Center

location_facility: Centre Hospitalier de l'Université de Montréal (CHUM)

location_facility: UC San Diego Medical Center - La Jolla (Jacobs Medical Center / Thornton Pavilion)

location_facility: UCLA Hematology-Oncology Infusion Center

location_facility: Mount Sinai Comprehensive Cancer Center - Aventura

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: PH 145294, Centre Hospitalier de l'Universite de Montreal (CHUM), Oncology Research Pharmacy

location_facility: University of Texas Southwestern Medical Center

location_facility: Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - Panstwowy Instytut Badawczy

location_facility: UCLA Clinical Research Unit (Adminstration Office)

location_facility: Sanford Cancer Center Oncology Clinic & Pharmacy

location_facility: University of Pittsburgh Cancer Institute

location_facility: Investigational Drug Services, National Taiwan University Hospital

summary: This is a Phase 1b/2 dose-optimization study to evaluate safety, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of avelumab (MSB0010718C) in combination with other cancer immunotherapies in patients with locally advanced or metastatic solid tumors. The primary purpose is to assess the safety and early signs of efficacy of various avelumab combinations with other cancer immunotherapies, optimizing dosing regimens as appropriate, in a limited series of indications.


title: A Phase 1b Study to Evaluate the Safety and Efficacy of MEDI4736 as Monotherapy and in Combination With Tremelimumab or AZD9150 in Subjects With Relapsed or Refractory Diffuse Large B-cell Lymphoma.

summary: The purpose of this study is to evaluate the safety, tolerability, and efficacy of MEDI4736 (durvalumab) alone and in combination with either tremelimumab or AZD9150 in adult subjects with relapsed or refractory dIffuse large B-cell lymphoma.

lead_sponsor: MedImmune LLC

location_country:
Ireland
United States
United Kingdom
France


title: Open-label, Multicentre, Multiple-dose Trial to Evaluate Pharmacokinetics, Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Orfadin in Patients Diagnosed With Hereditary Tyrosinemia Type 1

summary: The purpose of this study is to look at the steady-state serum concentrations of nitisinone when switching from twice daily and once daily dosing.

lead_sponsor: Swedish Orphan Biovitrum

location_country: Denmark France Belgium Sweden Germany

location_facility: Swedish Orphan Biovitrum Investigational Site

location_facility: Swedish Orphan Biovitrum Investigational site


acronym: ASCEND-Peds

title: A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase Deficiency

lead_sponsor: Genzyme, a Sanofi Company

ReferenceCitation: Diaz GA, Jones SA, Scarpa M, Mengel KE, Giugliani R, Guffon N, Batsu I, Fraser PA, Li J, Zhang Q, Ortemann-Renon C. One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency. Genet Med. 2021 Aug;23(8):1543-1550. doi: 10.1038/s41436-021-01156-3. Epub 2021 Apr 19. Erratum in: Genet Med. 2022 Oct;24(10):2209.

location_country:
Brazil
United Kingdom
Italy
United States
France
Germany

location_facility:
Investigational Site Number 250002
Investigational Site Number 840001
Investigational Site Number 826001
Investigational Site Number 276001
Investigational Site Number 076001
Investigational Site Number 380001

summary:
Primary Objective:

To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.

Secondary Objective:

To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.


title: A Multicenter, Randomized, Double-blind, Placebo-controlled, 4-arm, 26 Week Parallel-Group Study to Evaluate Efficacy, Safety and Tolerability of 2 Oral Doses and 2 Regimes of Tideglusib vs Placebo in Mild-to-Moderate AD Patients

acronym: ARGO

summary:
The main purpose of this study is to evaluate the cognitive changes after administration of tideglusib versus placebo at two oral doses and two treatment regimes for 26 weeks in patients with mild to moderate Alzheimer's disease.

After the 26 week core treatment period, the patients may continue in the study under blinded conditions for an optional extension period up to a maximum of 39 additional weeks (total study duration up to 65 weeks), until the last patient in the study has completed the 26 week of treatment.

lead_sponsor: Noscira SA

location_country:
Spain
United Kingdom
Finland
France
Belgium
Germany


title: Safety and Efficacy Study Comparing a New hMG Formulation (hMG-IBSA) to a Reference Product (Menopur®) in Patients Undergoing Ovarian Stimulation for in Vitro Fertilisation (IVF)

summary: The purpose of the non-inferiority study is to evaluate the clinical efficacy and the safety of two different subcutaneous hMG preparations when administered to patients undergoing controlled ovarian stimulation for IVF.

lead_sponsor: IBSA Institut Biochimique SA

location_country:
Denmark
United Kingdom
Switzerland
Hungary
France

location_facility: First Dept. Obstetric and Gynaecology, Semmelweiss University

location_facility: Groupe Hospitalier Cochin - Saint Vincent de Paul


title: Single-Arm Study to Assess the Efficacy of UVADEX® (Methoxsalen) Sterile Solution in Conjunction With the THERAKOS® CELLEX® Photopheresis System in Pediatric Patients With Steroid-Refractory Acute Graft-vs-Host Disease (aGvHD)

summary: This is a single-arm, open-label, multicenter study of the efficacy of UVADEX® (methoxsalen) Sterile Solution in conjunction with THERAKOS® CELLEX® Photopheresis Systems (ECP) in pediatric participants with steroid-refractory aGvHD. The study is composed of Screening, Treatment, and Follow-up Periods.

ipd_sharing_description:
Because we work in the rare disease space, to eliminate risk of patient identification we do not share individual patient data.

We post summary aggregate results for applicable clinical trials in the registry, and statistical endpoints and discussion in publications; with each referencing the other.

lead_sponsor: Therakos, Inc., a Mallinckrodt Company

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

location_facility: Universitaetsklinikum Ulm, Kinder- und Jugendmedizin

location_facility: Hospital Infantil Universitario "Nino Jesus"

location_facility: Children's Healthcare of Atlanta, Emory - Children's Center

location_facility: Universitätsklinikum Leipzig, Klinik und Poliklinik für Kinder- und Jugendmedizin, Abteilung für Hämatologie und Internistische Onkologie

location_facility: Klinikum rechts der Isar, TU München, Klinik- und Poliklinik für Kinder- und Jugendmedizin, Kinderklinik München Schwabing

location_facility: University Hospitals Rainbow Babies & Children's

location_facility: Vanderbilt University Medical Center - Ingram Cancer Institute

location_facility: U.O.C. Clinica di Oncoematologia Pediatrica Azienda Ospedaliera di Padova

intervention_type: Procedure


title: An International 7-Week, Double-Blind, Placebo-Controlled, 2-Parallel Group Study to Assess the Efficacy of Dianicline 40 mg BID as an Aid to Smoking Cessation in Cigarette Smokers

acronym: EURODIAN

summary: The primary objective is to demonstrate the efficacy of dianicline as an aid to smoking cessation in cigarette smokers. The main secondary objectives are: to assess the craving for cigarettes, nicotine withdrawal symptoms and the safety of dianicline.

lead_sponsor: Sanofi

location_country: Denmark Spain France Belgium Norway Sweden


title: A Phase 1 Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors

summary: This is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antineoplastic activity of avapritinib (formerly BLU-285), administered orally (PO), in adult patients with unresectable GIST or other relapsed or refractory solid tumors. The study consists of 2 parts, a dose-escalation part (Part 1) and an expansion part (Part 2).

ReferenceCitation: Kang YK, George S, Jones RL, Rutkowski P, Shen L, Mir O, Patel S, Zhou Y, von Mehren M, Hohenberger P, Villalobos V, Brahmi M, Tap WD, Trent J, Pantaleo MA, Schöffski P, He K, Hew P, Newberry K, Roche M, Heinrich MC, Bauer S. Avapritinib Versus Regorafenib in Locally Advanced Unresectable or Metastatic GI Stromal Tumor: A Randomized, Open-Label Phase III Study. J Clin Oncol. 2021 Oct 1;39(28):3128-3139. doi: 10.1200/JCO.21.00217. Epub 2021 Aug 3.

ReferenceCitation: von Mehren M, Heinrich MC, Shi H, Iannazzo S, Mankoski R, Dimitrijević S, Hoehn G, Chiroli S, George S. Clinical efficacy comparison of avapritinib with other tyrosine kinase inhibitors in gastrointestinal stromal tumors with PDGFRA D842V mutation: a retrospective analysis of clinical trial and real-world data. BMC Cancer. 2021 Mar 19;21(1):291. doi: 10.1186/s12885-021-08013-1.

ReferenceCitation: Jones RL, Serrano C, von Mehren M, George S, Heinrich MC, Kang YK, Schöffski P, Cassier PA, Mir O, Chawla SP, Eskens FALM, Rutkowski P, Tap WD, Zhou T, Roche M, Bauer S. Avapritinib in unresectable or metastatic PDGFRA D842V-mutant gastrointestinal stromal tumours: Long-term efficacy and safety data from the NAVIGATOR phase I trial. Eur J Cancer. 2021 Mar;145:132-142. doi: 10.1016/j.ejca.2020.12.008. Epub 2021 Jan 16.

ReferenceCitation: Joseph CP, Abaricia SN, Angelis MA, Polson K, Jones RL, Kang YK, Riedel RF, Schoffski P, Serrano C, Trent J, Tetzlaff ED, Si TD, Zhou T, Doyle A, Bauer S, Roche M, Havnaer T. Optimal Avapritinib Treatment Strategies for Patients with Metastatic or Unresectable Gastrointestinal Stromal Tumors. Oncologist. 2021 Apr;26(4):e622-e631. doi: 10.1002/onco.13632. Epub 2021 Jan 5.

ReferenceCitation: Heinrich MC, Jones RL, von Mehren M, Schöffski P, Serrano C, Kang YK, Cassier PA, Mir O, Eskens F, Tap WD, Rutkowski P, Chawla SP, Trent J, Tugnait M, Evans EK, Lauz T, Zhou T, Roche M, Wolf BB, Bauer S, George S. Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial. Lancet Oncol. 2020 Jul;21(7):935-946. doi: 10.1016/S1470-2045(20)30269-2. Erratum in: Lancet Oncol. 2020 Sep;21(9):e418.

ReferenceCitation: Gebreyohannes YK, Wozniak A, Zhai ME, Wellens J, Cornillie J, Vanleeuw U, Evans E, Gardino AK, Lengauer C, Debiec-Rychter M, Sciot R, Schöffski P. Robust Activity of Avapritinib, Potent and Highly Selective Inhibitor of Mutated KIT, in Patient-derived Xenograft Models of Gastrointestinal Stromal Tumors. Clin Cancer Res. 2019 Jan 15;25(2):609-618. doi: 10.1158/1078-0432.CCR-18-1858. Epub 2018 Oct 1.

lead_sponsor: Blueprint Medicines Corporation

location_country:
Poland
Spain
Italy
United Kingdom
Netherlands
Korea, Republic of
United States
France
Belgium
Germany

location_facility: Fondazione IRCCS - Istituto Nazinale dei Tumori

location_facility: Leuven Cancer Institute University Hospitals Leuven

location_facility: Vall d' Hebron Institute of Oncology (VHIO)

location_facility: Centrum Onkologii-Instytut im. Marii Skłodowskiej-Curie w Warszawie

location_facility: Scottsdale Healthcare Hospitals DBA HonorHealth


title: Randomized, Multicenter, Double-blind, Placebo-controlled, Two-arm Parallel Group Trial of Rimonabant 20-mg od, for Inhibition of Atherosclerosis Progression Assessed by Carotid Artery Intima-media Thickness (CIMT), in Overweight Patients With Additional Risk Factors

acronym: AUDITOR

summary:
Objectives:

Primary: To evaluate the effect of rimonabant 20-mg once daily in comparison with placebo, on the quantitative progression of atherosclerosis as assessed by carotid artery intima-media thickness (CIMT)
Secondary: To evaluate the safety and tolerability of the above rimonabant regimen in the study population of atherosclerosis patients.

ReferenceCitation: O'Leary DH, Reuwer AQ, Nissen SE, Després JP, Deanfield JE, Brown MW, Zhou R, Zabbatino SM, Job B, Kastelein JJ, Visseren FL; AUDITOR investigators. Effect of rimonabant on carotid intima-media thickness (CIMT) progression in patients with abdominal obesity and metabolic syndrome: the AUDITOR Trial. Heart. 2011 Jul;97(14):1143-50. doi: 10.1136/hrt.2011.223446. Epub 2011 May 24.

location_country:
Spain
United Kingdom
Canada
United States
France
Netherlands


title: Testing the Usefulness of Lantus When Initiated Prematurely In Patients With Type 2 Diabetes

summary:
Primary objective:

The primary objective of this trial is:

To evaluate the efficacy of initiating Lantus in combination with oral antidiabetics drugs compared to oral antidiabetic treatment optimised by enhancing hygienic and dietary measures in type 2 diabetics whose blood glucose control is acceptable but not optimal on maximum oral treatment, based on the number of patients achieving a HbA1c value < 7% at the end of treatment.

Secondary objectives:

The secondary objectives of this trial are to compare between the two treatment groups:

The variation in HbA1c between baseline and end of trial.
The frequency of episodes of symptomatic hypoglycaemia (diurnal and nocturnal), severe hypoglycaemia (diurnal and nocturnal) and asymptomatic hypoglycaemia.
Mean blood glucose levels at different times of the day.
The variation in weight and lipid in each group between baseline and end of trial.
The incidence of adverse events.

location_country:
Croatia
Slovenia
Russian Federation
Czech Republic
France
Romania


title: A Multi-centre Study to Define Novel Individualised Dosing Regimens to Maximise Antibiotic Effectiveness for Treatment of Pneumonia in Intensive Care Units

acronym: PNEUDOS

summary:
Pneumonia is the most common infection in intensive care unit (ICU) patients and occurs in 10% of all ICU admissions. Unfortunately, ICU patient outcomes remain poor with a high mortality rate associated with pneumonia despite recent therapeutic advances.

Previous studies of antibiotics used in ICU patients, which includes ceftriaxone, meropenem and piperacillin/tazobactam, have quantified major differences in pharmacokinetics (PK) between ICU and non-ICU patients, with ICU patients displaying a unique spectrum of plasma concentration-time profiles. These PK differences can lead to suboptimal antibiotic concentrations in blood, which have been associated with a reduced likelihood of clinical cure for pneumonia. Furthermore, highlighting the importance of optimised dosing for pneumonia is that multi-drug resistant (MDR) pathogens emerge during antibiotic therapy in approximately half of the ICU patients, frequently emerging from the lung.

Previous work has highlighted how infection site concentrations determine patient outcome. For pneumonia, the infection site is best described as the epithelial lining fluid (ELF) in the lung.

Although optimal antibiotic therapy should be considered a priority for ICU patients with pneumonia to improve the persisting poor outcomes, the dosing regimens that can achieve therapeutic concentrations at the infection site (i.e., ELF) in ICU patients with pneumonia remain unknown.

The PNEUDOS study aims to address this significant knowledge gap by defining novel individualised dosing regimens that can maximise antibiotic efficacy by achieving therapeutic concentrations in the blood and ELF of ICU patients with pneumonia. These dosing regimens can then be validated in future clinical trials.

ReferenceCitation: Abdul-Aziz MH, Alffenaar JC, Bassetti M, Bracht H, Dimopoulos G, Marriott D, Neely MN, Paiva JA, Pea F, Sjovall F, Timsit JF, Udy AA, Wicha SG, Zeitlinger M, De Waele JJ, Roberts JA; Infection Section of European Society of Intensive Care Medicine (ESICM); Pharmacokinetic/pharmacodynamic and Critically Ill Patient Study Groups of European Society of Clinical Microbiology and Infectious Diseases (ESCMID); Infectious Diseases Group of International Association of Therapeutic Drug Monitoring and Clinical Toxicology (IATDMCT); Infections in the ICU and Sepsis Working Group of International Society of Antimicrobial Chemotherapy (ISAC). Antimicrobial therapeutic drug monitoring in critically ill adult patients: a Position Paper(.). Intensive Care Med. 2020 Jun;46(6):1127-1153. doi: 10.1007/s00134-020-06050-1. Epub 2020 May 7. Review.

lead_sponsor: The University of Queensland

location_country:
Hong Kong
Australia
France
Belgium
Malaysia

location_facility: Centre Hospitalier Universitaire de Nimes


title: Phase 1/2a Open Label, Multicenter Clinical Trial of a Novel Small Molecule EBNA1 Inhibitor, VK 2019, in Patients With Epstein Barr Virus Positive Nasopharyngeal Cancer, With Pharmacokinetic and Pharmacodynamic Correlative Studies

summary: VK-2019-001 is a 1/2a trial of the oral EBNA-1 targeting agent VK-2019 in patients with EBV-positive recurrent or metastatic NPC to determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D), as well as to evaluate the PK profile of VK-2019.

ReferenceCitation: Young LS. Epstein-Barr virus at 50-future perspectives. Chin J Cancer. 2014 Nov;33(11):527-8. doi: 10.5732/cjc.014.10208.

ReferenceCitation: Young LS, Dawson CW. Epstein-Barr virus and nasopharyngeal carcinoma. Chin J Cancer. 2014 Dec;33(12):581-90. doi: 10.5732/cjc.014.10197. Epub 2014 Nov 21.

ReferenceCitation: Tsao SW, Tsang CM, Lo KW. Epstein-Barr virus infection and nasopharyngeal carcinoma. Philos Trans R Soc Lond B Biol Sci. 2017 Oct 19;372(1732). pii: 20160270. doi: 10.1098/rstb.2016.0270. Review.

lead_sponsor: Cullinan Oncology, LLC

location_country:
Hong Kong
United States
France
China
Singapore

location_facility: Stanford University, School of Medicine, Stanford Cancer Institute

location_facility: The University of Texas - MD Anderson Cancer Center

location_facility: Hong Kong University - Queen Mary Hospital


title: A Prospective Observational, Long Term Follow up Study of Patients Treated With Imlifidase Prior to Kidney Transplantation

summary:
The rationale for the current protocol is to collect data from extended follow up in subjects that have received a kidney transplant following imlifidase dosing to provide a better understanding regarding the long-term outcome for these subjects. Data of parameters such as patient and graft survival, comorbidity, treatment of graft rejection episodes and quality of life as well as anti-drug antibody levels will be collected.

This prospective, observational follow up study of subjects who have received imlifidase prior to kidney transplantation will provide important data to future prescribers and patients of the potential long-term benefits of imlifidase mediated transplantation.

lead_sponsor: Hansa Biopharma AB

location_country:
Sweden
United States
France

location_facility:
Cedars-Sinai Medical Center
Necker Hospital
Karolinska University Hospital
The Johns Hopkins Hospital
Uppsala University Hospital
New York University School of Medicine


title: A Multicentre Open-label Phase IIa Study With Escalating Dose of MS1819- SD, to Investigate the Efficacy and Safety of a Yarrowia Lipolytica Lipase Preparation for the Compensation of Exocrine Pancreatic Insufficiency Caused by Chronic Pancreatitis and/or Distal Pancreatectomy

summary:
This is a Phase IIa study sponsored by AzurRx SAS and Syneos Health is a local representative sponsor and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by chronic pacreatitis (CP) and/or distal pancreatectomy. The new medication is called MS1819 Spray Dried (MS1819-SD) which is a lipase produced by the LIP2 gene of Yarrowia lipolytica using recombinant DNA technology.

The primary purpose of this study is to investigate the safety of escalating doses of study drug MS1819-SD in people with chronic pancreatitis. This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common with CP patients. The deficiency in this enzyme can be responsible of greasy diarrhea, fecal urge and weight loss.

The design of the study is open-label, meaning that all eligible participants will receive the study drug MS1819-SD. The MS1819-SD dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of four treatment periods.

The total duration of the MS1819-SD treatment phase is of 48-60 days, The total duration of patient participation in the study is of 74-93 days.

Approximately twelve patients will be enrolled in this study.

lead_sponsor: AzurRx SAS

location_country:
New Zealand
Australia
France

location_facility: Mackay Institute of Research and Innovation


title: A Phase Ib/II Open Label Study to Assess the Safety and Pharmacokinetics of NUC-3373, a Nucleotide Analogue, Given in Combination With Standard Agents Used in Colorectal Cancer Treatment

summary: This is a three-part study of NUC-3373 administered by intravenous (IV) infusion across two administration schedules, either as monotherapy or as part of various combinations with agents commonly used to treat CRC (leucovorin, oxaliplatin, irinotecan, bevacizumab, cetuximab and panitumumab). The primary objective is to identify a recommended dose and schedule for NUC-3373 when combined with these agents.

lead_sponsor: NuCana plc

location_country:
United States
United Kingdom
France

location_facility: The Beatson West of Scotland Cancer Centre


title: Long Term Follow-up of Integra® Cadence™ Total Ankle System in Primary Ankle Joint Replacement

summary: This study will evaluate the long term performance and safety data for the Cadence™ Total Ankle System (CTAS) when used for primary arthroplasty in patients with primary arthritis (e.g. degenerative disease), secondary arthritis (e.g. post-traumatic, avascular necrosis, if minimally 2/3 of the talus is preserved), and systemic arthritis of the ankle (e.g. rheumatoid arthritis, hemochromatosis)

lead_sponsor: Smith & Nephew, Inc.

location_country:
United Kingdom
Spain
Canada
France
Belgium

location_facility:
Universitair Ziekenhuis Gent
CHRU Tours
North Cumbria University Hospitals
Foot and Ankle Institute
Clinica Nostra Senhora del Remei
St. Michael's Hospital


acronym: PALL

title: A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B-cell Acute Lymphoblastic Leukaemia

lead_sponsor: Institut de Recherches Internationales Servier

ReferenceCitation: Benjamin R, Graham C, Yallop D, Jozwik A, Mirci-Danicar OC, Lucchini G, Pinner D, Jain N, Kantarjian H, Boissel N, Maus MV, Frigault MJ, Baruchel A, Mohty M, Gianella-Borradori A, Binlich F, Balandraud S, Vitry F, Thomas E, Philippe A, Fouliard S, Dupouy S, Marchiq I, Almena-Carrasco M, Ferry N, Arnould S, Konto C, Veys P, Qasim W; UCART19 Group. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020 Dec 12;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5.

ipd_sharing_description:
Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

sponsored by Servier
with a first patient enrolled as of 1 January 2004 onwards
for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

location_country:
United Kingdom
United States
Spain
France

location_facility: University of Texas Southwestern Medical Center

summary: This study aims to evaluate the safety and feasibility of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).


acronym: Phase 1 dry powder

title: Single-dose Study Testing Rivaroxaban Granules for Oral Suspension Formulation in Children From 2 Months to 12 Years With Previous Thrombosis

lead_sponsor: Bayer

location_country:
Spain
Italy
Finland
Canada
Hungary
United States
France
Belgium

location_facility: Hopital Necker les enfants malades - Paris

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: Hôpital Arnaud de Villeneuve - Montpellier

location_facility: A.O.U. Città della Salute e della Scienza di Torino

location_facility: Ciutat Sanitària i Universitaria de la Vall d'Hebron

location_facility: University of Semmelweis/ Semmelweis Egyetem

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: Turun yliopistollinen keskussairaala, kantasairaala

summary: To characterize the pharmacokinetic profile of rivaroxaban administered as granules for suspension formulation and to document safety and tolerability


title: A Phase 2a, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of AMG 714 in Adult Patients With Type II Refractory Celiac Disease, an In Situ Small Bowel T Cell Lymphoma

summary: Protocol CELIM-RCD-002 is designed to evaluate the efficacy and safety of AMG 714 for the treatment of adult patients with type II refractory celiac disease (RCD-II), an in-situ small bowel T-cell lymphoma.

ReferenceCitation: Cellier C, Bouma G, van Gils T, Khater S, Malamut G, Crespo L, Collin P, Green PHR, Crowe SE, Tsuji W, Butz E, Cerf-Bensussan N, Macintyre E, Parnes JR, Leon F, Hermine O, Mulder CJ; RCD-II Study Group Investigators. Safety and efficacy of AMG 714 in patients with type 2 refractory coeliac disease: a phase 2a, randomised, double-blind, placebo-controlled, parallel-group study. Lancet Gastroenterol Hepatol. 2019 Dec;4(12):960-970. doi: 10.1016/S2468-1253(19)30265-1. Epub 2019 Sep 4.

ipd_sharing_description: Publication in peer-reviewed journals

lead_sponsor: Amgen

location_country:
Spain
Finland
United States
France
Netherlands


title: A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of lGIV-C as a Corticosteroid Sparing Agent in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis

summary: This is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) as a corticosteroid (CS)-sparing agent in subjects with CS-dependent Myasthenia Gravis (MG).

ReferenceCitation: Bril V, Szczudlik A, Vaitkus A, Rozsa C, Kostera-Pruszczyk A, Hon P, Bednarik J, Tyblova M, Köhler W, Toomsoo T, Nowak RJ, Mozaffar T, Freimer ML, Nicolle MW, Magnus T, Pulley MT, Rivner M, Dimachkie MM, Distad BJ, Pascuzzi RM, Babiar D, Lin J, Coll MQ, Griffin R, Mondou E. A Randomized, Double-Blind, Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis. Neurology. 2022 Oct 21. pii: 10.1212/WNL.0000000000201501. doi: 10.1212/WNL.0000000000201501. [Epub ahead of print]

ReferenceCitation: Dalakas MC, Meisel A. Immunomodulatory effects and clinical benefits of intravenous immunoglobulin in myasthenia gravis. Expert Rev Neurother. 2022 Apr;22(4):313-318. doi: 10.1080/14737175.2022.2057223. Epub 2022 Apr 5. Review.

lead_sponsor: Grifols Therapeutics LLC

location_country:
Poland
Estonia
Canada
Lithuania
Hungary
United States
France
Belgium
Germany
Czechia

location_facility: Universitaetsklinikum Giessen und Marburg GmbH Standort Marburg

location_facility: Ohio State University Wexner Medical Center, Neurology Department

location_facility: University of Florida at Shands Jacksonville

location_facility: Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii Klinicznej

location_facility: CHU Strasbourg - Nouvel Hôpital Civil, Clinique Neurologique

location_facility: III Szpital Miejski w Lodzi im. Dr K. Jonschera

location_facility: Samodzielny Publiczny Centralny Szpital Kliniczny

location_facility: Jahn Ferenc Del-pesti Korhaz es Rendelointezet, Neurologiai Osztaly

location_facility: University Health Network (UHN) - Toronto General Hospital

location_facility: London Health Sciences Centre- University Hospital

location_facility: CHU de Toulouse - Hôpital Purpan, Service de Neurologie Générale

location_facility: Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont

location_facility: Uniwersyteckie Centrum Kliniczne, Dept of Neurology

location_facility: Charité Universitaetsmedizin Berlin, Klinik für Neurologie

location_facility: Pest Megyei Flor Ferenc Korhaz, Neurologia es Stroke Osztaly

location_facility: Krankenhaus Martha-Maria Halle-Doelau, Klinik fuer Neurologie

location_facility: Fakultni nemocnice Brno, Dept of Neurologicka klinika

location_facility: Fachkrankenhaus Hubertusburg gGmbH, Klinik f. Neurologie

location_facility: Universitaetsklinikum Jena, Klinik fuer Neurologie

location_facility: Yale University School of Medicine, Department of Neurology

location_facility: Houston Methodist Neurological Institute

location_facility: Vseobecna fakultni nemocnice v Praze, Dept of Neurologicka klinika

location_facility: Universitaetsklinikum Hamburg-Eppendorf, Klinik und Poliklinik

location_facility: University of Kansas Medical Center Research Institute, Inc.

location_facility: Hospital of Lithuanian University of Health Sciences Kaunas Clinics


title: Pilot Study 3 of Outpatient Control-to-Range: Safety and Efficacy With Day-and-Night In-Home Use

summary:
The purpose of this study is to see if an automated insulin management system ("study system") can safely be used at home to manage blood sugar. The study system includes (1) a CGM that measures glucose levels, (2) a computer program on a smartphone that determines how much insulin is needed and allows the study participant user to control the whole system, and (3) an insulin pump that delivers the insulin. The CGM will be from Dexcom. The pump will be from Roche. The CGM and pump are similar to the devices that are currently available for people to purchase and use. However, the smartphone device, the CGM sensor type used with it, and the overall study system can only be used for research at this time.

The study will be completed by about 24 individuals at 6 centers in the United States and Europe. This study has several phases and will take about 11-14 weeks to complete depending on whether the study participant is a CGM user or not.

At selected sites (based on subject eligibility and availability), approximately 10-20 subjects who exhibit safe and competent use of the system at home will be given the option to continue home use of the system in Day-and-Night Closed-Loop mode for up to 5 months.

ReferenceCitation: Anderson SM, Raghinaru D, Pinsker JE, Boscari F, Renard E, Buckingham BA, Nimri R, Doyle FJ 3rd, Brown SA, Keith-Hynes P, Breton MD, Chernavvsky D, Bevier WC, Bradley PK, Bruttomesso D, Del Favero S, Calore R, Cobelli C, Avogaro A, Farret A, Place J, Ly TT, Shanmugham S, Phillip M, Dassau E, Dasanayake IS, Kollman C, Lum JW, Beck RW, Kovatchev B; Control to Range Study Group. Multinational Home Use of Closed-Loop Control Is Safe and Effective. Diabetes Care. 2016 Jul;39(7):1143-50. doi: 10.2337/dc15-2468. Epub 2016 Apr 13.

lead_sponsor: Jaeb Center for Health Research

location_country:
United States
Italy
Israel
France

location_facility: National Center for Childhood Diabetes- Schneider Children's Medical Center


title: A Prospective, Randomized, Intra-patient, Comparative, Open, Multi-centre Study to Evaluate the Efficacy of a Single-Use NPWT System on the Prevention of Incision Healing Complications in Patients Undergoing Reduction Mammoplasty

summary: The purpose of this study is to see if the use of a single-use negative pressure wound therapy system (NPWT) will have an effect on the reduction of post-surgical incision healing complications following breast reduction surgery and to assess the medium-term aesthetic appearance and quality of the resultant scar, compared with standard of care dressings.

primary_purpose: Prevention

location_country:
Netherlands
United States
South Africa
France

location_facility: Northwestern University Feinberg School of Medicine


title: A 24 Week Off Drug Extension, Parallel Group, Study Assessing Durability of Effect on Skeletal Muscle Strength and Function Following a 6-month Double-blind, Placebo Controlled Study Evaluating Bimagrumab in Older Adults With Sarcopenia (InvestiGAIT Extension)

summary: This extension study was a 24-week off-drug follow-up of the core CBYM338E2202 (NCT ) study and the main objective was to determine the long-term durability of bimagrumab (BYM338) effect after a 6-month treatment period.

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

lead_sponsor: Novartis Pharmaceuticals

location_country:
Denmark
Taiwan
Spain
Russian Federation
Australia
Switzerland
Korea, Republic of
United States
France
Belgium
Japan
Czechia


title: A Multicenter Open-Label Single-Arm Phase II Study Evaluating the Safety and Efficacy of Bevacizumab in Combination With Carboplatin and Paclitaxel in Patients With Metastatic, Recurrent or Persistent Cervical Cancer

lead_sponsor: Hoffmann-La Roche

ReferenceCitation: Redondo A, Colombo N, McCormack M, Dreosti L, Nogueira-Rodrigues A, Scambia G, Lorusso D, Joly F, Schenker M, Ruff P, Estevez-Diz M, Irahara N, Donica M, Gonzalez-Martín A. Primary results from CECILIA, a global single-arm phase II study evaluating bevacizumab, carboplatin and paclitaxel for advanced cervical cancer. Gynecol Oncol. 2020 Oct;159(1):142-149. doi: 10.1016/j.ygyno.2020.07.026. Epub 2020 Aug 4.

location_country:
Poland
Brazil
Portugal
Romania
Greece
Italy
Mexico
Russian Federation
Argentina
Serbia
Spain
Costa Rica
France
Panama
South Africa
Colombia
Turkey
Bulgaria

location_facility: Russian Oncology Research Center n.a. N.N. Blokhin Dpt of Clinical Pharmacology and Chemotherapy

location_facility: Istanbul Uni of Medicine Faculty; Oncology Dept

location_facility: IPO do Porto; Servico de Oncologia Medica

location_facility: Wielkopolskie Centrum Onkologii im. M. Sklodowskiej-Curie

location_facility: Hospital Universitario La Paz; Servicio de Oncologia

location_facility: Instituto Nacional de Cancer - INCa; Pesquisa Clinica

location_facility: Institut Gustave Roussy; Oncologie Medicale

location_facility: Irccs Istituto Europeo Di Oncologia (IEO); Oncologia Medica

location_facility: St. Petersburg Oncology & Gynecology; City Clinical Oncology Dispensary

location_facility: Universita' Cattolica Del Sacro Cuore; Reparto Ginecologia Oncologica

location_facility: Instituto Nacional de Cancerologia; Oncology

location_facility: Centrum Onkologii Instytut im. M.Sklodowskiej-Curie; Klinika Ginekologii Onkologicznej

location_facility: Istituto Tumori Napoli;Unità Operativa Oncologia Medica Uro-Ginecologica

location_facility: Centro Oncologico MD Anderson International Espana

location_facility: Centrum Onkologii - Instytut M.Sklodowskiej-Curie; Klinika Ginekologii Onkologicznej

location_facility: Centro Oncologico Riojano Integral (CORI)

location_facility: Istituto Nazionale dei Tumori; Divisione Oncologia Chirurgica e Ginecologica

location_facility: Ankara Baskent University Medicine Faculty; Gynaecology

location_facility: Hospital Universitario la Fe; Servicio de Oncologia

location_facility: Alexandras General Hospital of Athens; Oncology Department

location_facility: Complejo Hospitalario Universitario de Santiago (CHUS) ; Servicio de Oncologia

location_facility: Complex Oncological Center - Plovdiv, EOOD

location_facility: University of Pretoria; Department of Medical Oncology

location_facility: Instituto do Cancer do Estado de Sao Paulo - ICESP

location_facility: Instituto Valenciano Oncologia; Oncologia Medica

location_facility: Centre Francois Baclesse; Urologie Gynecologie

summary: This study is to assess safety as defined by the frequency and severity of gastrointestinal (GI) perforation/fistula, GI-vaginal fistula and genitourinary (GU) fistula in participants treated with bevacizumab 15 milligrams per kilogram (mg/kg) in combination with paclitaxel and carboplatin, all repeated every 3 weeks, for recurrent, persistent or metastatic cervical cancer. In addition, this study will include evaluation of the overall safety profile of bevacizumab in combination with paclitaxel and carboplatin in this setting, assessment of GI perforation/fistula, GI-vaginal fistula and GU fistula events over time, and evaluation of efficacy.


title: A Randomized Phase III Multicenter Trial of Neoadjuvant Docetaxel (Taxotere®) Plus Cisplatin and 5-Fluorouracil (TPF) Versus Neoadjuvant Cisplatin Plus 5-Fluorouracil Followed by Concomitant Chemoradiotherapy to Improve the Overall Survival and Progression Free Survival in Patients With Locally Advanced Squamous Cell Carcinoma of the Head and Neck

summary:
1.To compare overall survival after treatment with the test tri-therapy (TPF: docetaxel plus cisplatin and 5FU) or the control treatment (PF: cisplatin plus 5-FU) followed by chemoradiotherapy in patients with locally advanced SCCHN.
2.The main secondary endpoint is progression free survival (PFS). The other secondary endpoints are to evaluate and compare improvement of local symptoms; time-to-treatment failure; quality of life; clinical complete response rate (CR and CR/PR); toxicity and to evaluate the relationship of tumor markers and response to therapy.

ReferenceCitation: Posner MR, Hershock DM, Blajman CR, Mickiewicz E, Winquist E, Gorbounova V, Tjulandin S, Shin DM, Cullen K, Ervin TJ, Murphy BA, Raez LE, Cohen RB, Spaulding M, Tishler RB, Roth B, Viroglio Rdel C, Venkatesan V, Romanov I, Agarwala S, Harter KW, Dugan M, Cmelak A, Markoe AM, Read PW, Steinbrenner L, Colevas AD, Norris CM Jr, Haddad RI; TAX 324 Study Group. Cisplatin and fluorouracil alone or with docetaxel in head and neck cancer. N Engl J Med. 2007 Oct 25;357(17):1705-15.

ReferenceCitation: Lorch JH, Goloubeva O, Haddad RI, Cullen K, Sarlis N, Tishler R, Tan M, Fasciano J, Sammartino DE, Posner MR; TAX 324 Study Group. Induction chemotherapy with cisplatin and fluorouracil alone or in combination with docetaxel in locally advanced squamous-cell cancer of the head and neck: long-term results of the TAX 324 randomised phase 3 trial. Lancet Oncol. 2011 Feb;12(2):153-9. doi: 10.1016/S1470-2045(10)70279-5. Epub 2011 Jan 11.

location_country:
Portugal
Russian Federation
Argentina
Canada
United States
France


title: Dose-Ranging Pharmacodynamic Assessment of Platelet Aggregation Inhibition With Clopidogrel in Children of Blalock-Taussig Shunt Age Categories (Neonates and Infants/Toddlers)

summary: PICOLO is a double blind placebo controlled phase II dose ranging, dose escalating study in patients of Blalock-Taussig age categories (neonates and infants/toddlers), to determine the dose providing inhibition of platelet aggregation similar to adults.

ReferenceCitation: Li JS, Yow E, Berezny KY, Bokesch PM, Takahashi M, Graham TP Jr, Sanders SP, Sidi D, Bonnet D, Ewert P, Jennings LK, Michelson AD; PICOLO Investigators. Dosing of clopidogrel for platelet inhibition in infants and young children: primary results of the Platelet Inhibition in Children On cLOpidogrel (PICOLO) trial. Circulation. 2008 Jan 29;117(4):553-9. doi: 10.1161/CIRCULATIONAHA.107.715821. Epub 2008 Jan 14.

ReferenceCitation: Pasquali SK, Yow E, Jennings LK, Li JS. Platelet activity associated with concomitant use of clopidogrel and proton pump inhibitors in children with cardiovascular disease. Congenit Heart Dis. 2010 Nov-Dec;5(6):552-5. doi: 10.1111/j.1747-0803.2010.00461.x.

location_country:
Italy
Canada
United States
France
Belgium
Germany


title: An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II

summary: Glycogen Storage Disease Type II ("GSD-II"; also known as Pompe disease) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with GSD-II, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.

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

lead_sponsor: Genzyme, a Sanofi Company

location_country:
United States
Israel
United Kingdom
France

location_facility: University of Florida College of Medicine


title: A PHASE I TRIAL OF BUTYRATE AND GANCICLOVIR IN EBV-ASSOCIATED MALIGNANCIES

summary:
RATIONALE: The Epstein Barr virus can cause cancer and lymphoproliferative disorders. Ganciclovir is an antiviral drug that acts against the Epstein Barr virus. Arginine butyrate may make virus cells more sensitive to ganciclovir. Combining ganciclovir and arginine butyrate may kill more Epstein Barr virus cells and tumor cells.

PURPOSE: Phase I trial to study the effectiveness of arginine butyrate plus ganciclovir in treating patients who have cancer or lymphoproliferative disorders that are associated with the Epstein Barr virus.

ReferenceCitation: Faller DV, Mentzer SJ, Perrine SP. Induction of the Epstein-Barr virus thymidine kinase gene with concomitant nucleoside antivirals as a therapeutic strategy for Epstein-Barr virus-associated malignancies. Curr Opin Oncol. 2001 Sep;13(5):360-7. Review.

ReferenceCitation: Mentzer SJ, Perrine SP, Faller DV. Epstein--Barr virus post-transplant lymphoproliferative disease and virus-specific therapy: pharmacological re-activation of viral target genes with arginine butyrate. Transpl Infect Dis. 2001 Sep;3(3):177-85. Review.

ReferenceCitation: Perrine SP, Hermine O, Small T, Suarez F, O'Reilly R, Boulad F, Fingeroth J, Askin M, Levy A, Mentzer SJ, Di Nicola M, Gianni AM, Klein C, Horwitz S, Faller DV. A phase 1/2 trial of arginine butyrate and ganciclovir in patients with Epstein-Barr virus-associated lymphoid malignancies. Blood. 2007 Mar 15;109(6):2571-8. Epub 2006 Nov 21.

ReferenceCitation: Mentzer SJ, Fingeroth J, Reilly JJ, Perrine SP, Faller DV. Arginine butyrate-induced susceptibility to ganciclovir in an Epstein-Barr-virus-associated lymphoma. Blood Cells Mol Dis. 1998 Jun;24(2):114-23.

lead_sponsor: Boston Medical Center

location_country:
Germany
United States
Italy
France

location_facility: Cancer Research Center at Boston Medical Center

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

location_facility: Methodist Cancer Center at Methodist Hospital


title: A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

summary: A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the MPS II.

lead_sponsor: JCR Pharmaceuticals Co., Ltd.

location_country:
Germany
United States
Brazil
France

location_facility: UCSF Benioff Children's Hospital Oakland

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


title: Repetitive Transcranial Magnetic Stimulation of the Precentral-gyrus in the Relief of Fibromyalgia Pain: an International Multicenter Controlled Adaptative Trial

acronym: TMSFFI

summary: There are currently several points concerning rTMS for pain relief that have a huge impact in the practical application of rTMS in clinical practice, but which have not been approached by previous studies. Also, an even more important issue is the frequency of the sessions in each treatment protocol. The idea of performing stimulation in chronic pain patients on a consecutive daily basis for long-term treatments seem unrealistic and potentially costly. However, it has been reported in animals, healthy subjects and FM patients that the analgesic effects of rTMS can outlast the stimulation session for several minutes and even days. This has led to the proposal that pain treatment with rTMS could be performed in consecutive daily induction sessions, followed by spaced (weekly, fortnightly and even monthly) maintenance sessions. This approach is similar to what is performed routinely in patients with major depression treated by rTMS to the dorsolateral prefrontal cortex, and has successfully been tested in FM patients in a single- center study. Should this approach prove effective in larger prospective multicentric studies, rTMS could enter the armamentarium of non-invasive, non- pharmacological, low-adverse event therapeutic options to relieve fibromyalgia- related pain and associate symptoms. Hence, our aim is to conduct a multicentric international clinical trial on the influence of rTMS in the control of pain in a consecutive adaptive trial design where conventional rTMS (10Hz) and patterned rTMS (theta-burst) will be studied. Stimulations will be performed in a clinical practice-friendly approach, where daily maintenance sessions will be followed by weekly, and fortnightly stimulation sessions. There have been very scarce adaptive trials in the pain and non-invasive stimulation fields. This methodological approach allows for the use of lower number of patients in consecutive trials and, among other qualities, it allows for the imputation of data from the first trial into the second one, thus decreasing the duration of the studies and the number of participants.

lead_sponsor: University of Sao Paulo

location_country: Japan France Brazil

location_facility: Ambulatório de Fisioterapia do Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

location_facility: Centre d'Evaluation et de Traitement de la Douleur Hôpital Ambroise Paré

location_facility: Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (HC/FMUSP)

location_facility: Department of Neuromodulation Osaka University