Clinical Trials on Infant, Extremely Low Birth Weight in France

Total 738 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
Vertex Pharmaceuticals Incorporated
Cystic Fibrosis Foundation

Terminated

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation (DISCOVER)

Cystic Fibrosis

United States
CF Therapeutics Development Network Coordinating...
Cystic Fibrosis Foundation

Completed

The EPIC Observational Study (EPIC OBS)

Cystic Fibrosis

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
Tenaya Therapeutics
Medical University of South Carolina; Johns Hopkins University; University of... and other collaborators

Recruiting

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) (RIDGE)

Arrhythmogenic Right Ventricular Cardiomyopathy

United States, Italy, United Kingdom, France, Sweden, Germany
Alexion Pharmaceuticals

Completed

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Wolman Disease | Lysosomal Acid Lipase Deficiency

United Kingdom, France, United States, Egypt, Ireland
Alexion Pharmaceuticals

Completed

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency (ARISE)

Lysosomal Acid Lipase Deficiency

France, Poland, United Kingdom, Spain, Mexico, Turkey, Japan, Australia, Russian Federation, United States, Germany, Italy, Czechia, Argentina, Croatia
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
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
University of Pennsylvania
National Heart, Lung, and Blood Institute (NHLBI); University Medical Center... and other collaborators

Enrolling by invitation

PROSpect: Prone and Oscillation Pediatric Clinical Trial

Acute Respiratory Distress Syndrome in Children

United States, Netherlands, Israel, Thailand, New Zealand, United Kingdom, Australia, Brazil, Canada, China, India, Italy, Malaysia, United Arab Emirates, Spain
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
Seattle Children's Hospital
National Heart, Lung, and Blood Institute (NHLBI); Cystic Fibrosis Foundation; CF Therapeutics Development Network Coordinating Center

Completed

Testing the Effect of Adding Chronic Oral Azithromycin to Inhaled Tobramycin in People With Cystic Fibrosis (CF) (TEACH)

Cystic Fibrosis

United States
Vertex Pharmaceuticals Incorporated

Completed

Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

Cystic Fibrosis

United States, France, United Kingdom, Germany, Belgium, Denmark, Canada, Australia, Sweden
Horizon Pharma USA, Inc.
Forest Laboratories

Completed

MP-376 (Aeroquin™, Levofloxacin for Inhalation) in Patients With Cystic Fibrosis

Cystic Fibrosis

United States, Australia, Israel, New Zealand, Canada
PTC Therapeutics
Cystic Fibrosis Foundation

Completed

Study of Ataluren (PTC124™) in Cystic Fibrosis

Cystic Fibrosis

United States, France, Belgium, United Kingdom, Spain, Canada, Germany, Israel, Italy, Netherlands, Sweden
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
French National Agency for Research on AIDS and...
Celgene Corporation

Terminated

Lenalidomide in Kaposi Disease Associated With HIV Infection (LENAKAP)

HIV Infection Associated Kaposi Disease

France
Vertex Pharmaceuticals Incorporated

Completed

Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation

Cystic Fibrosis

United States, Canada, Germany, United Kingdom
Boehringer Ingelheim

Recruiting

A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome

Netherton Syndrome

Belgium, United Kingdom, Australia, Germany, China, Malaysia, United States, Japan, France, Israel, Bulgaria, Italy, Finland, Switzerland, Netherlands, Portugal
Celtaxsys, Inc.

Completed

EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients

Cystic Fibrosis

United States, United Kingdom, Canada, Belgium, France, Germany, Italy
Gilead Sciences
Covance; ClinPhone, Inc.; Chiltern International Inc.

Completed

Aztreonam for Inhalation Solution vs Tobramycin Inhalation Solution in Patients With Cystic Fibrosis & Pseudomonas Aeruginosa

Cystic Fibrosis

Belgium, France, Germany, Italy, Netherlands, Spain, United States, Ireland, United Kingdom, Portugal, Austria, Denmark, Switzerland
Hospital de Clinicas de Porto Alegre

Completed

Nutritional Intervention and Glycemic Improvement in Patients With Pre-diabetic Cystic Fibrosis.

Cystic Fibrosis | Cystic Fibrosis-related Diabetes

Brazil
Vertex Pharmaceuticals Incorporated

Completed

A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Cystic Fibrosis

United States, Spain, Ireland, Belgium, Netherlands, United Kingdom, Australia, France, Canada, Denmark, Germany, Italy, Israel
University of Florida
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Characteristics of Prader-Willi Syndrome and Early-onset Morbid Obesity

Obesity | Prader-Willi Syndrome

United States
Vertex Pharmaceuticals Incorporated

Completed

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

Cystic Fibrosis

United States, United Kingdom, Belgium, Netherlands, France, Denmark, Israel, New Zealand, Australia, Ireland, Sweden, Canada, Germany, Poland, Switzerland, Italy, Austria, Hungary, Greece, Norway
Vertex Pharmaceuticals Incorporated

Completed

Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Cystic Fibrosis

United States, Spain, Ireland, Belgium, Netherlands, United Kingdom, Australia, France, Denmark, Germany, Italy, Canada
Vertex Pharmaceuticals Incorporated

Completed

A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Cystic Fibrosis

United States, Belgium, Netherlands, United Kingdom, Canada, Germany, Australia, France, Italy, Czechia, Austria, Greece, Sweden
Vertex Pharmaceuticals Incorporated

Completed

A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation

Cystic Fibrosis

United States, Australia, Canada, France, United Kingdom, Germany, Spain, Switzerland, Belgium, Israel, Netherlands, Denmark, Italy, Austria, Ireland, Sweden
Alexion Pharmaceuticals

Terminated

Efficacy and Safety Study of IV Ravulizumab in Patients With COVID-19 Severe Pneumonia

Pneumonia, Viral | Acute Respiratory Distress Syndrome | Acute Lung Injury | COVID-19 Severe Pneumonia

United States, Spain, United Kingdom, France, Japan
Boehringer Ingelheim

Completed

Tiotropium Bromide in Cystic Fibrosis

Cystic Fibrosis

United States, Australia, Austria, Belgium, Canada, Czech Republic, France, Germany, Hungary, Ireland, Israel, Italy, Poland, Portugal, Russian Federation, Slovakia, South Africa, Spain, Switzerland, United Kingdom
Anthera Pharmaceuticals

Completed

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

Cystic Fibrosis | Exocrine Pancreatic Insufficiency

United States
Vertex Pharmaceuticals Incorporated

Completed

A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation

Cystic Fibrosis

United States, Australia, Canada, Germany, Switzerland, Israel, Netherlands, Belgium, Italy, United Kingdom, France
Vertex Pharmaceuticals Incorporated

Completed

A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Cystic Fibrosis

United States, Belgium, Netherlands, United Kingdom, Canada, Australia, France, Germany, Italy, Czechia, Austria, Sweden, Greece
Corbus Pharmaceuticals Inc.
Cystic Fibrosis Foundation

Completed

Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

Cystic Fibrosis

United States, United Kingdom, Sweden, Canada, France, Austria, Germany, Netherlands, Italy, Belgium, Portugal, Bulgaria, Czechia, Greece, Hungary, Poland, Romania, Russian Federation, Serbia, Slovakia, Spain
Gilead Sciences

Completed

Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa (AIR-CF2)

Cystic Fibrosis

United States
Gilead Sciences

Completed

Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis (CF) Patients With Pseudomonas Aeruginosa (PA) (AIR-CF3)

Cystic Fibrosis

United States, Canada, Australia, New Zealand
Anthera Pharmaceuticals

Completed

Open-Label Phase III Long-Term Safety Trial of Liprotamase (DIGEST)

Cystic Fibrosis | Exocrine Pancreatic Insufficiency

United States
Mukoviszidose Institut gGmbH
Assistance Publique - Hôpitaux de Paris; Novo Nordisk A/S; Mucoviscidose-ABCF2

Completed

Early Diagnosis of Diabetes Mellitus in Patients With Cystic Fibrosis

Diabetes Mellitus | Cystic Fibrosis

Germany, Austria, France, Italy
Anthera Pharmaceuticals

Unknown

RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

Cystic Fibrosis | Exocrine Pancreatic Insufficiency

United States, Hungary, Israel, Lithuania, Poland, Spain, United Kingdom
Humanigen, Inc.

Completed

Study to Evaluate the Effect of KB001-A on Time-to-Need for Antibiotic Treatment (KB001-A)

Cystic Fibrosis

United States, Australia, Israel, New Zealand
Gilead Sciences

Completed

International Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa (AIR-CF1)

Cystic Fibrosis

United States, Canada, Australia, New Zealand
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
Nicole Hamblett
University of Washington; Dartmouth-Hitchcock Medical Center; Cystic Fibrosis...

Completed

Impact of Discontinuing Dornase Alfa in People With CF on Highly Effective CFTR Modulator Therapy-A SIMPLIFY Sub-Study (SIMPLIFY-DN)

Cystic Fibrosis

United States
Nicole Hamblett
University of Washington; Dartmouth-Hitchcock Medical Center; Cystic Fibrosis...

Completed

Impact of Discontinuing Hypertonic Saline in People With CF on Highly Effective CFTR Modulators- A SIMPLIFY Sub-Study (SIMPLIFY-HS)

Cystic Fibrosis

United States
Nicole Hamblett
University of Washington; Dartmouth-Hitchcock Medical Center; Cystic Fibrosis...

Completed

Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy (SIMPLIFY)

Cystic Fibrosis

United States
Horizon Pharma USA, Inc.

Completed

Safety, Tolerability and Efficacy of MP-376 Given for 28 Days to Cystic Fibrosis (CF) Patients

Cystic Fibrosis (CF)

Germany, United States, Netherlands
Vertex Pharmaceuticals Incorporated

Active, not recruiting

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

Cystic Fibrosis

United States, Spain, United Kingdom, Belgium, Netherlands, Denmark, Israel, New Zealand, France, Australia, Ireland, Germany, Sweden, Canada, Czechia, Switzerland, Portugal, Italy, Austria, Hungary, Norway, Poland, Greece
Rhythm Pharmaceuticals, Inc.

Completed

Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity

Obesity | Genetic Obesity | Obesity Due to Melanocortin 4 Receptor Deficiency

United States, United Kingdom, Germany, Netherlands, France, Canada, Greece, Israel, Spain
Proteostasis Therapeutics, Inc.

Completed

Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis

Healthy Volunteer - Complete | Cystic Fibrosis - Complete

United States, Belgium, United Kingdom, France, Germany, Canada, Denmark, New Zealand, Australia
Apellis Pharmaceuticals, Inc.

Completed

A Study of APL-9 in Adults With Mild to Moderate ARDS Due to COVID-19

Severe Acute Respiratory Syndrome | Coronavirus Infection | Covid-19 | COVID | Acute Respiratory Distress Syndrome | Coronavirus | Ards | Severe Acute Respiratory Syndrome Coronavirus 2 | Sars-CoV2

United States, Brazil

Clinical Trials on Infant, Extremely Low Birth Weight in France

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