Clinical Trials on Genetic Diseases, X-Linked in France

Total 2319 results

Vertex Pharmaceuticals Incorporated

Completed

A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)

Cystic Fibrosis

United States, France, Germany
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)

Hypochondroplasia

United States, Norway, Australia, Singapore, United Kingdom, Canada, France, Germany, Portugal, Spain, Sweden
National Cancer Institute (NCI)
Multicenter Hemophilia Cohort Study

Completed

The Second Multicenter Hemophilia Cohort Study

Hepatocellular Carcinoma | Non-Hodgkin Lymphoma | Liver Decompensation

United States, Germany, Italy, Brazil, Canada, France, Greece, Sweden
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
Genzyme, a Sanofi Company

Completed

A Multi-centre, Open Label, Single-arm Study Intended to Further Investigate the Safety and Efficacy of Plerixafor as a Front-line Mobilisation Agent in Combination With G-CSF in Patients With Lymphoma or MM (Multiple Myeloma). (PREDICT)

Transplantation | Lymphoma (Non-Hodgkin's Lymphoma) | Hodgkin's Disease or Multiple Myeloma | Front Line Mobilization

Netherlands, France, Germany, Sweden, Spain, Italy, United Kingdom
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Recruiting

Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRD (PHYOX7)

End Stage Renal Disease | Primary Hyperoxaluria Type 2 | Primary Hyperoxaluria Type 1

Germany, Spain, United States, United Kingdom, Italy, United Arab Emirates, Lebanon, Morocco, France, Romania
Genzyme, a Sanofi Company

Completed

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) (ENCORE)

Gaucher Disease, Type 1

United States, France, Germany, Italy, Brazil, Australia, Argentina, Canada, Egypt, Russian Federation, Spain, United Kingdom
Bioverativ Therapeutics Inc.

Completed

Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A (ASPIRE)

Hemophilia A

United States, Canada, Italy, Australia, Austria, Belgium, Brazil, France, Germany, Hong Kong, India, Ireland, Israel, Japan, Netherlands, New Zealand, Poland, South Africa, Spain, Sweden, Switzerland, United Kingdom
Horizon Pharma USA, Inc.
Forest Laboratories

Completed

Trial of Aeroquin Versus Tobramycin Inhalation Solution (TIS) in Cystic Fibrosis (CF) Patients (TIS)

Cystic Fibrosis

United States, Ireland, United Kingdom, France, Germany, Israel
Alnylam Pharmaceuticals

Recruiting

BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1) (BONAPH1DE)

Primary Hyperoxaluria Type 1

France, Spain, United States, Israel, Belgium, Switzerland, Germany, United Kingdom, Italy, Canada
Pharmaxis
ethica Clinical Research Inc.; Europe: KasaConsult bvba, Hoegaarden, Belgium; Argentina: Resolution Latin America; Buenos Aires, Argentina

Completed

Long Term Administration of Inhaled Mannitol in Cystic Fibrosis

Cystic Fibrosis

United States, France, Belgium, Canada, Argentina, Germany, Netherlands
Vertex Pharmaceuticals Incorporated

Completed

A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation

Cystic Fibrosis

United States, France, United Kingdom, Germany, Belgium, Australia, Denmark, Canada, Sweden
Vertex Pharmaceuticals Incorporated

Completed

Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation

Cystic Fibrosis

Spain, Belgium, Netherlands, France, Canada, Germany, Sweden, Italy, Czechia, Switzerland, Portugal, Austria, Hungary, Norway, Poland
PTC Therapeutics

Terminated

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

Cystic Fibrosis

United States, Spain, France, Belgium, United Kingdom, Australia, Brazil, Bulgaria, Canada, Germany, Israel, Italy, Netherlands, Poland, Argentina, Greece
Merck Sharp & Dohme LLC

Completed

Study of Denufosol Tetrasodium Inhalation Solution in Patients With Cystic Fibrosis (CF) Lung Disease

Cystic Fibrosis

United States, Australia, Canada, New Zealand
Johnson & Johnson Pharmaceutical Research & Development...

Completed

A Study of the Efficacy and Tolerability of Pancrelipase Microtablet (MT) Capsules for the Treatment of Cystic Fibrosis-dependent Exocrine Pancreatic Insufficiency

Malabsorption Syndromes | Cystic Fibrosis | Steatorrhea | Exocrine Pancreatic Insufficiency

United States, Canada
Novo Nordisk A/S

Completed

Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome

Obesity | Metabolism and Nutrition Disorder

France, United States, Turkey, Italy, Australia, New Zealand, Canada, Netherlands
University of Southern California
National Institute on Aging (NIA); Alzheimer's Therapeutic Research Institute; Alzheimer's Clinical Trials Consortium

Recruiting

Trial-Ready Cohort-Down Syndrome (TRC-DS) (TRC-DS)

Dementia | Alzheimer Disease | Down Syndrome

United States, Spain, United Kingdom
University College, London
CHDI Foundation, Inc.

Recruiting

HDClarity: a Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease (HDClarity)

Huntington's Disease

United States, United Kingdom, France, Canada, Germany, Italy, Spain, New Zealand, Poland
Pfizer

Completed

Transthyretin Amyloidosis Outcome Survey (THAOS) (THAOS)

Transthyretin Amyloidosis | Transthyretin Gene Mutations

United States, France, Spain, Taiwan, Israel, Korea, Republic of, Denmark, Canada, Netherlands, Japan, Sweden, Brazil, Mexico, Argentina, Portugal, Belgium, Bulgaria, Cyprus, Germany, Italy, Malaysia, Romania, Saudi Arabia, Turkey, United...
Hospices Civils de Lyon

Not yet recruiting

Pregnancy in Women With Rare Multisystemic Vascular Diseases: COGRare5 Study (COGRare5)

Arteriovenous Malformations | Marfan Syndrome | Osler Rendu Disease | Vascular Anomaly | Lymphedema Primary | Cerebrospinal; Disorder

France
Agios Pharmaceuticals, Inc.

Recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period (ACTIVATE-Kids)

Pediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic Anemia

United States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
Janssen Research & Development, LLC

Completed

A Study of JNJ-64251330 in Participants With Familial Adenomatous Polyposis

Adenomatous Polyposis Coli

United States, Germany, Korea, Republic of, France, Netherlands, Spain, Puerto Rico
Ardea Biosciences, Inc.

Completed

Lesinurad Monotherapy in Gout Subjects Intolerant to Xanthine Oxidase Inhibitors (LIGHT)

Gout

United States, Canada, South Africa, New Zealand, Belgium, Australia, Germany
Bayer

Completed

A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A (PROTECT-VIII)

Hemophilia A

Korea, Republic of, Taiwan, Turkey, Singapore, United States, Netherlands, Austria, Denmark, United Kingdom, Belgium, Italy, France, Japan, Germany, Israel, Poland, Canada, Norway, Romania, Colombia
Sanofi
Regeneron Pharmaceuticals

Completed

Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy (ODYSSEY FH I)

Hypercholesterolemia

United States, Austria, Canada, Czech Republic, Denmark, France, Israel, Netherlands, Norway, Russian Federation, South Africa, Spain, Sweden, United Kingdom
Pfizer

Terminated

Study Evaluating Safety Of Patients Switching To ReFacto AF In Usual Care Settings

Hemophilia A

Netherlands, Spain, Greece, Italy, France, Belgium, Germany, Austria, Sweden, Hungary, Finland, United Kingdom, Denmark, Romania
ApoPharma

Terminated

Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias (FIRST)

Iron Overload | Sickle Cell Disease | Other Anemias

United States, Turkey, United Kingdom, Egypt, Saudi Arabia, Brazil, Canada, Tunisia
Pfizer

Completed

Prevalence and Characteristics of Transthyretin Amyloidosis in Patients With Left Ventricular Hypertrophy of Unknown Etiology (TTRACK)

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Italy, Romania, France, Australia, Austria, Portugal, Slovakia, Slovenia, Spain, United Kingdom
BioCryst Pharmaceuticals

Completed

Study to Evaluate the Urate-Lowering Activity and Safety of Oral BCX4208 Administered in Subjects With Gout

Gout

United States
Pfizer

Terminated

Carotid B-mode Ultrasound Study to Compare Anti-Atherosclerotic Effect of Torcetrapib/Atorvastatin to Atorvastatin. (RADIANCE 2)

Mixed Hyperlipidemia

United States, Canada, Czech Republic, Finland, France, Netherlands
Federation Francophone de Cancerologie Digestive

Recruiting

Prospective National Cohort Evaluating Predictive Biomarkers of Resistance to Immunotherapy in Patients With MSI/dMMR Metastatic Colorectal Cancer (CORESIM) (CORESIM)

Colorectal Cancer Metastatic | Microsatellite Instability-High Colorectal Cancer

France
Anthera Pharmaceuticals

Completed

SIMPLICITY: Studying Impacts on Malabsorption With Liprotamase in Cystic Fibrosis (SIMPLICITY)

Cystic Fibrosis | Exocrine Pancreatic Insufficiency

United States
Bayer
Novartis

Completed

Study to Evaluate the Safety and Efficacy of Ciprofloxacin (Inhaled) in Patients With Cystic Fibrosis

Cystic Fibrosis

United States, Israel, Germany, Canada, Australia, Sweden, Denmark, Norway, United Kingdom
Takeda

Completed

MPC-004 for the Treatment of an Acute Gout Flare (AGREE)

Gout

United States
KemPharm Denmark A/S

Completed

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

Niemann-Pick Disease, Type C

Spain, France, United Kingdom, Switzerland, Denmark, Poland, Italy, Germany
Novartis Pharmaceuticals

Recruiting

Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia

Myeloid Leukemia, Philadelphia Positive

Korea, Republic of, Germany, France, Greece, Hungary, Italy, Japan, Russian Federation, Thailand, Turkey, China, United States, Netherlands, Poland
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A

Severe Hemophilia A

United States, United Kingdom, Spain, Hong Kong, Israel, New Zealand, Sweden, Australia, Brazil, Japan, Austria, Belgium, Italy, India, Switzerland, Canada, France, Germany, South Africa
Mandos LLC

Completed

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

Niemann-Pick Disease, Type C

United States, Australia, United Kingdom, Germany, France, New Zealand, Singapore, Spain, Turkey
Novo Nordisk A/S

Completed

A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A (pathfinder™5)

Congenital Bleeding Disorder | Haemophilia A

France, United States, Switzerland, Malaysia, Brazil, Canada, Japan, United Kingdom, Israel, Portugal, Ukraine, Greece, Lithuania, Puerto Rico, Turkey, Italy
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
Vertex Pharmaceuticals Incorporated

Completed

A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor

Cystic Fibrosis

United States, Australia, Canada, United Kingdom, Germany, Belgium, Austria, Ireland, Italy, France
Vertex Pharmaceuticals Incorporated

Completed

Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation

Cystic Fibrosis

United States, France, United Kingdom, Germany, Australia, New Zealand, Belgium
Vertex Pharmaceuticals Incorporated

Completed

A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation

Cystic Fibrosis

United States, Germany, United Kingdom, Canada, Australia, Switzerland, France, Ireland, Belgium, Denmark, Poland
AbbVie

Completed

A Phase 2 Study of ABBV-3067 Alone and in Combination With ABBV-2222

Cystic Fibrosis

United States, Belgium, Canada, Czechia, France, Hungary, Netherlands, New Zealand, Poland, Slovakia, United Kingdom, Serbia
Merck KGaA, Darmstadt, Germany

Completed

Predictive Markers in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) Children Treated With SAIZEN®

Growth Hormone Deficiency | Turner Syndrome

Argentina, Australia, Austria, Canada, France, Germany, Italy, Norway, Russian Federation, Singapore, Spain, Sweden, United Kingdom
Amicus Therapeutics

Active, not recruiting

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Pompe Disease

United States, Netherlands, United Kingdom, Australia, Germany, New Zealand
Novartis Pharmaceuticals

Recruiting

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies (V-PEDS-OLE)

Heterozygous or Homozygous Familial Hypercholesterolemia

Netherlands, Israel, Hungary, Italy, Germany, Turkey, Spain, United States, France, Norway, Russian Federation, South Africa, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, Poland, Taiwan, Malaysia
Cellaion SA

Terminated

HepaStem Long-Term Safety Registry (PROLONGSTEM)

Urea Cycle Disorder | Crigler-Najjar Syndrome | Acute on Chronic Liver Failure

Belgium, Spain, France, Bulgaria, Poland
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

Clinical Trials on Genetic Diseases, X-Linked in France

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