Clinical Trials on Congenital Afibrinogenemia in France

Total 1108 results

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
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
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
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
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

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
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

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

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
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

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
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
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
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
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
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
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
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
Sanifit Therapeutics S. A.
Clinipace Worldwide

Completed

Effect of SNF472 on Progression of Cardiovascular Calcification in End-Stage-Renal-Disease (ESRD) Patients on Hemodialysis (HD)

Cardiovascular Diseases | Cardiovascular Abnormalities | ESRD | Coronary Artery Calcification | Endstage Renal Disease | Calcifications, Vascular

Spain, United States, United Kingdom
Reata, a wholly owned subsidiary of Biogen

Terminated

A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON (FALCON)

Autosomal Dominant Polycystic Kidney | ADPKD

United States, Spain, United Kingdom, Australia, France, Japan, Germany, Belgium, Czechia, Italy
Novartis Pharmaceuticals

Recruiting

CINC424A2X01B Rollover Protocol

Acute Myeloid Leukemia | Primary Myelofibrosis | Polycythemia Vera | Thalassemia | Graft Versus Host Disease

Korea, Republic of, Italy, Belgium, Japan, Germany, Australia, Bulgaria, Chile, China, Denmark, Greece, Hungary, Israel, Lebanon, Poland, Portugal, Russian Federation, Slovakia, South Africa, Spain, Sweden, Thailand, India, Mexico, Turkey, Fran...
National Cancer Institute (NCI)

Suspended

A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)

Down Syndrome | Recurrent B Acute Lymphoblastic Leukemia

United States, Canada, Puerto Rico, Australia
Otsuka Pharmaceutical Development & Commercialization...
Otsuka Pharmaceutical Co., Ltd.

Completed

Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease (OVERTURE)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

United States, Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, France, Germany, Italy, Japan, Netherlands, Norway, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
Otsuka Pharmaceutical Development & Commercialization...

Completed

Efficacy and Safety of Tolvaptan in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease

Chronic Kidney Disease | Autosomal Dominant Polycystic Kidney Disease

Australia, Belgium, Italy, Spain, Sweden, United States, Canada, Netherlands, Czechia, Poland, Germany, Russian Federation, Hungary, Israel, Puerto Rico, Argentina, Denmark, South Africa, United Kingdom, Norway, France, Romania
National Cancer Institute (NCI)
Childrens Oncology Group (COG); LLS PedAL LLC

Recruiting

Highest Dose of Uproleselan in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Mixed Phenotype Acute Leukemia Relapsed or Refractory and That Expresses E-selectin Ligand on the Cell Membrane

Down Syndrome | Recurrent Acute Myeloid Leukemia | Recurrent Myelodysplastic Syndrome | Refractory Acute Myeloid Leukemia | Refractory Myelodysplastic Syndrome | Recurrent Mixed Phenotype Acute Leukemia | Refractory Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic...

United States
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Chemotherapy in Treating Children With Down Syndrome and Myeloproliferative Disorder, Acute Myelogenous Leukemia, or Myelodysplastic Syndrome

Myelodysplastic Syndromes | Leukemia | Myelodysplastic/Myeloproliferative Neoplasms

United States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland
Merck Sharp & Dohme LLC

Recruiting

Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)

Von Hippel-Lindau Disease | Pancreatic Neuroendocrine Tumor | Pheochromocytoma/Paraganglioma | Advanced Gastrointestinal Stromal Tumor | HIF-2α Mutated Cancers

Canada, United States, Denmark, France, Germany, Hungary, Italy, Russian Federation, Spain, Sweden, Turkey, Israel, United Kingdom, Netherlands, Australia, China, Singapore, Japan
National Cancer Institute (NCI)

Recruiting

A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia

Down Syndrome | B Acute Lymphoblastic Leukemia | B Lymphoblastic Lymphoma

United States, Canada, Puerto Rico, Australia, New Zealand
Children's Oncology Group
National Cancer Institute (NCI)

Active, not recruiting

Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome

Acute Myeloid Leukemia | Down Syndrome | Myelodysplastic Syndrome | Myeloproliferative Neoplasm | Myeloid Leukemia Associated With Down Syndrome

United States, Canada, Saudi Arabia, Puerto Rico, Australia, New Zealand

Clinical Trials on Congenital Afibrinogenemia in France

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