Clinical Trials on Congenital Athymia in France

Total 1119 results

Soleno Therapeutics, Inc.

Completed

Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period

Prader-Willi Syndrome

United States, United Kingdom
Seaside Therapeutics, Inc.

Completed

Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome (Harbor-A)

Fragile X Syndrome

United States
Seaside Therapeutics, Inc.

Terminated

An Open Label Extension Study in Subjects With Fragile X Syndrome (209FX303)

Fragile X Syndrome

United States
Hoffmann-La Roche

Completed

A Study of RO4917523 in Patients With Fragile X Syndrome

Fragile X Syndrome

France, Spain, United States, Peru, Canada, United Kingdom, Chile, Argentina, Mexico, Sweden
Soleno Therapeutics, Inc.

Completed

A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome

United States, United Kingdom
Otsuka Pharmaceutical Development & Commercialization...

Recruiting

A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Autosomal Recessive Polycystic Kidney (ARPKD)

United States, United Kingdom, Belgium, Poland, France, Germany, Italy
National Cancer Institute (NCI)

Active, not recruiting

Testing Cabozantinib in Patients With Advanced Pancreatic Neuroendocrine and Carcinoid Tumors

Carcinoid Tumor | Functioning Pancreatic Neuroendocrine Tumor | Intermediate Grade Lung Neuroendocrine Neoplasm | Locally Advanced Pancreatic Neuroendocrine Tumor | Locally Advanced Unresectable Digestive System Neuroendocrine Neoplasm | Low Grade Lung Neuroendocrine Neoplasm | Metastatic Digestive... and other conditions

United States
Acceleron Pharma Inc. (a wholly owned subsidiary...

Terminated

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth Disease

United States
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) (ENERGIZE)

Non-Transfusion-dependent Alpha-Thalassemia | Non-Transfusion-dependent Beta-Thalassemia

Spain, Taiwan, Thailand, United Kingdom, Malaysia, United States, Netherlands, Bulgaria, Turkey, Italy, Canada, Brazil, France, United Arab Emirates, Denmark, Greece, Lebanon, Saudi Arabia
Pharnext S.C.A.
Premier Research Group plc; Eurofins Optimed; Synteract HCR (Syneos Health); Gr... and other collaborators

Active, not recruiting

Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A (PLEO-CMT-FU)

Charcot-Marie-Tooth Disease, Type IA

United States, Belgium, Canada, France, Netherlands, Spain, United Kingdom
Novo Nordisk A/S

Completed

Observational Prospective Study on Patients Treated With Norditropin®

Chronic Kidney Disease | Turner Syndrome | Noonan Syndrome | Chronic Renal Insufficiency | Growth Hormone Disorder | Growth Hormone Deficiency in Children | Adult Growth Hormone Deficiency | Genetic Disorder | Foetal Growth Problem | Small for Gestational Age

Israel, Saudi Arabia, Spain, Argentina, Russian Federation, Germany, Netherlands, Italy, Belgium, United Kingdom, Slovenia, Ireland, Sweden, Denmark, Hungary, Finland, Norway, France, Czechia, Lithuania, Luxembourg, Serbia, Switzerland
Zynerba Pharmaceuticals, Inc.

Recruiting

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome (RECONNECT)

Fragile X Syndrome

United States, Australia, United Kingdom, Ireland
Seaside Therapeutics, Inc.

Completed

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome (Harbor-C)

Fragile X Syndrome

United States
Novartis Pharmaceuticals

Completed

Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell Disease (STEADFAST)

Sickle Cell Disease (SCD)

United Kingdom, Spain, Greece, Brazil, Turkey, United States, France, Netherlands, Lebanon, Panama, Ireland
Peloton Therapeutics, Inc., a subsidiary of Merck...

Active, not recruiting

A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004)

VHL - Von Hippel-Lindau Syndrome | VHL Gene Mutation | VHL Syndrome | VHL Gene Inactivation | VHL-Associated Renal Cell Carcinoma | VHL-Associated Clear Cell Renal Cell Carcinoma

United States, Denmark, United Kingdom, France
Novo Nordisk A/S

Completed

Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B (paradigm™ 3)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Austria, Spain, Germany, Macedonia, The Former Yugoslav Republic of, Taiwan, United Kingdom, Italy, Malaysia, Thailand, France, Greece, Turkey, Japan, South Africa, Romania
Novo Nordisk A/S

Completed

Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773 (paradigm™ 4)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Austria, Spain, Germany, Macedonia, The Former Yugoslav Republic of, Taiwan, United Kingdom, Italy, Malaysia, Thailand, France, Greece, Russian Federation, Turkey, Japan, South Africa, Romania
Novo Nordisk A/S

Completed

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (paradigm™6)

Congenital Bleeding Disorder | Haemophilia B

Spain, United Kingdom, Australia, Taiwan, France, Malaysia, United States, Austria, Canada, Israel, Japan, Thailand, Algeria, Argentina
Novo Nordisk A/S

Completed

Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients (paradigm™ 2)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Germany, Macedonia, The Former Yugoslav Republic of, United Kingdom, Italy, Malaysia, Hungary, Thailand, France, Russian Federation, Turkey, Japan, South Africa, Canada
Marinus Pharmaceuticals

Active, not recruiting

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy (TrustTSC)

Tuberous Sclerosis Complex

United States, Spain, United Kingdom, Israel, Italy, Germany, France, Canada
SWOG Cancer Research Network
National Cancer Institute (NCI); Genentech, Inc.

Recruiting

Testing Drug Treatments After CAR T-cell Therapy in Patients With Relapsed/Refractory Diffuse Large B-cell Lymphoma

Diffuse Large B-Cell Lymphoma | Recurrent Diffuse Large B-Cell Lymphoma | Refractory Diffuse Large B-Cell Lymphoma | Primary Mediastinal (Thymic) Large B-Cell Lymphoma | Grade 3b Follicular Lymphoma | Transformed Follic Lymph to Diff Large B-Cell Lymphoma | Transformed Marg Zone Lymph to Diff...

United States
Pharnext SA

Completed

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT) (PLEO-CMT)

Charcot-Marie-Tooth Disease Type 1A

United States, United Kingdom, Belgium, Canada, France, Netherlands, Spain, Germany
Pharnext S.C.A.
Worldwide Clinical Trials

Active, not recruiting

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PREMIER)

Charcot-Marie-Tooth Disease

United States, Spain, Belgium, Italy, Israel, Canada, Denmark, France, Germany
Novo Nordisk A/S

Recruiting

A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (REAL 8)

SGA, Turner Syndrome, Noonan Syndrome, ISS

Korea, Republic of, United States, Ireland, United Kingdom, Finland, France, Netherlands, Japan, Thailand, Belgium, China, Israel, Brazil, India, Portugal, Austria, Bulgaria, Canada, Croatia, Germany, Greece, Malaysia, Poland, Saudi Arabia and more
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A (pathfinder™6)

Congenital Bleeding Disorder | Haemophilia A

United States, Spain, Australia, France, Thailand, Italy, Malaysia, Austria, Greece, Japan, Taiwan, Canada, Israel, Algeria, Argentina, Bulgaria, Germany, Romania
Children's Oncology Group
National Cancer Institute (NCI)

Recruiting

Study of Kidney Tumors in Younger Patients

Renal Cell Carcinoma | Wilms Tumor | Clear Cell Sarcoma of the Kidney | Papillary Renal Cell Carcinoma | Kidney Medullary Carcinoma | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Congenital Mesoblastic Nephroma | Renal Cell Carcinoma Associated With Xp11.2 Translocations... and other conditions

United States, Canada, Puerto Rico, Australia, New Zealand, Israel, Switzerland
Novo Nordisk A/S

Completed

Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United States, Spain, Taiwan, Turkey, Poland, Croatia, Italy, Malaysia, Brazil, United Kingdom, Hungary, Israel, Thailand, Japan, South Africa, Canada, France, Argentina
Novo Nordisk A/S

Completed

A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A (pathfinder8)

Congenital Bleeding Disorder | Haemophilia A

United States, Netherlands, Spain, Switzerland, Taiwan, Turkey, United Kingdom, Croatia, Germany, Brazil, France, Japan, Australia, Hungary, Israel, Korea, Republic of, Lithuania, Portugal, Ukraine, Greece, Italy, Denmark, Norway, Canada and more
Novo Nordisk A/S

Completed

Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A. (pathfinder™3)

Congenital Bleeding Disorder | Haemophilia A

France, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, United Kingdom, Croatia, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Italy, Japan, Norway, Bulgaria
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A (guardian™ 3)

Congenital Bleeding Disorder | Haemophilia A

United States, Macedonia, The Former Yugoslav Republic of, Serbia, Malaysia, Brazil, Turkey, Lithuania, Russian Federation, Italy, Japan, Poland, Puerto Rico, Taiwan
Novo Nordisk A/S

Completed

Efficacy of NovoSeven® in Bleeding Prophylaxis in Hemophilia

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

France, United States, Spain, Brazil, Philippines, South Africa, Poland, Turkey, Romania, Bulgaria, Russian Federation, Argentina
Palvella Therapeutics, Inc.

Recruiting

CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention Of Basal Cell Carcinomas (BCCs) In Patients With Gorlin Syndrome

BCCs in Gorlin Syndrome Patients

United States, United Kingdom
Sol-Gel Technologies, Ltd.

Terminated

Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)

Basal Cell Nevus Syndrome

United States, France, Belgium, Denmark, Germany, Italy, Netherlands, Spain, United Kingdom
Reata, a wholly owned subsidiary of Biogen

Completed

A Phase 2/3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients With Alport Syndrome - CARDINAL (CARDINAL)

Alport Syndrome

United States, United Kingdom, Japan, Australia, France, Germany, Puerto Rico, Spain
Novo Nordisk A/S

Completed

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A (pathfinder™2)

Congenital Bleeding Disorder | Haemophilia A

France, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, Japan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Puerto Rico, Norway, Korea, Republic of, Bulgaria, Russian...
Columbia University
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

A Randomized Trial of Unruptured Brain AVMs (ARUBA)

Arteriovenous Malformations, Cerebral

United States, Brazil, Canada, Australia, Austria, Finland, France, Germany, Italy, Korea, Republic of, Netherlands, Spain, Switzerland, United Kingdom
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Study of Blood Samples From Newborns With Down Syndrome

Myeloid Proliferations Associated With Down Syndrome

United States, Canada, Australia, Puerto Rico
Hoffmann-La Roche

Completed

A Study of Two Vismodegib Regimens in Participants With Multiple Basal Cell Carcinomas

Basal Cell Carcinoma

United States, Spain, France, Germany, Netherlands, Russian Federation, Italy, Mexico, Austria, Canada
Novartis Pharmaceuticals

Active, not recruiting

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

Tuberous Sclerosis Complex

United States, Spain, Belgium, Taiwan, Turkey, Australia, Korea, Republic of, Thailand, France, Hungary, United Kingdom, Italy, Japan, Canada, Russian Federation, Poland, Mexico, Colombia
SOTIO Biotech AG
SOTIO Biotech a.s.

Active, not recruiting

Study of SO-C101 and SO-C101 in Combination With Pembro in Adult Patients With Advanced/Metastatic Solid Tumors

Melanoma | Renal Cell Carcinoma | Cervical Cancer | Hepatocellular Carcinoma | Gastric Cancer | Ovarian Cancer | Non Small Cell Lung Cancer | Mesothelioma | Bladder Cancer | Triple Negative Breast Cancer | Small-cell Lung Cancer | Head and Neck Squamous Cell Carcinoma | Thyroid | Anal Cancer | Merkel Cell Carcinoma | Biliary... and other conditions

United States, France, Spain, Czechia
Novartis Pharmaceuticals

Completed

A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures (EXIST-3)

Tuberous Sclerosis Complex-associated Refractory Seizures

United States, Colombia, Belgium, France, Japan, Taiwan, Turkey, Spain, Thailand, Korea, Republic of, Australia, Hungary, United Kingdom, Canada, Norway, Germany, Italy, Russian Federation, Denmark, Argentina, Netherlands, Greece, P... and more
Acceleron Pharma Inc. (a wholly owned subsidiary...

Terminated

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

Charcot-Marie-Tooth Disease | Facioscapulohumeral Muscular Dystrophy

United States, Spain, Canada
PellePharm, Inc.

Completed

Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome)

Basal Cell Nevus Syndrome

United States, Spain, Germany, United Kingdom, Italy, France, Belgium, Canada, Denmark, Netherlands
Otsuka Pharmaceutical Development & Commercialization...

Completed

Tolvaptan Extension Study in Participants With ADPKD (TEMPO 4/4)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

United States, Australia, Belgium, Germany, Italy, Poland, Romania, United Kingdom, Argentina, Canada, France, Netherlands, Russian Federation
Novo Nordisk A/S

Completed

Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors (adept™2)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Taiwan, United Kingdom, Thailand, Serbia, Croatia, Italy, Poland, Romania, Hungary, Malaysia, United States, Austria, Brazil, Greece, Japan, Puerto Rico, Russian Federation, South Africa, Turkey
University of California, San Francisco
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

Epilepsy Phenome/Genome Project (EPGP)

Epilepsy | Infantile Spasms | Lennox-Gastaut Syndrome | Polymicrogyria | Localization-related Epilepsy | Periventricular Heterotopias

United States, Argentina, Australia
Celgene

Recruiting

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

United States, Japan, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Israel, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, Lebanon, China, Netherlands
Otsuka Pharmaceutical Development & Commercialization...
Otsuka Pharmaceutical Co., Ltd.

Completed

Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD) (TEMPO3:4)

Polycystic Kidney Disease, Autosomal Dominant

United States, Belgium, United Kingdom, Russian Federation, France, Australia, Netherlands, Italy, Japan, Denmark, Romania, Poland, Canada, Argentina, Germany
Atara Biotherapeutics

Recruiting

A Study to Evaluate Tabelecleucel in Participants With Epstein-barr Virus-associated Diseases

Lymphoproliferative Disorders | Leiomyosarcoma | Stem Cell Transplant Complications | Solid Organ Transplant Complications | Epstein-Barr Virus (EBV)-Associated Diseases | EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD) | Allogeneic Hematopoietic Cell Transplant | EBV+ Sarcomas | EBV+ Lymphoproliferative... and other conditions

United States, Austria, Belgium, France, Italy, Spain, United Kingdom
Novartis Pharmaceuticals

Completed

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) (EXIST-2)

Lymphangioleiomyomatosis (LAM) | Tuberous Sclerosis Complex (TSC)

United States, United Kingdom, Germany, Italy, Russian Federation, Netherlands, Japan, Canada, Poland, France, Spain

Clinical Trials on Congenital Athymia in France

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