Search clinical trials for: Hemophilia B, gene therapy

Total 293602 results

BioMarin Pharmaceutical

Enrolling by invitation

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) (GENEr8-LTE)

Hemophilia A

United Kingdom, United States
BioMarin Pharmaceutical

Active, not recruiting

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 (GENEr8-AAV5+)

Hemophilia A | Gene Therapy | Blood Disorder | Clotting Disorders

Taiwan, Korea, Republic of, United Kingdom, South Africa
Pfizer

Active, not recruiting

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

Hemophilia B

United States, Canada, Australia, Turkey
BioMarin Pharmaceutical

Active, not recruiting

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (GENEr8-3)

Hemophilia A

United States, Australia, Taiwan, Brazil
Yuzuncu Yıl University

Completed

Physiotherapy and Reaction Time in Hemophilia

Hemophilia Arthropathy

Turkey
Cairo University

Completed

Loaded Gait Training on Gross Motor Function in Cerebral Palsy

Cerebral Palsy

Egypt
Baxalta now part of Shire
Takeda Development Center Americas, Inc.

Completed

A Study of ADVATE in People With Hemophilia A in India

Hemophilia A

India
Pfizer

Active, not recruiting

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults (AFFINE)

Hemophilia A

Spain, Taiwan, Australia, United States, Japan, France, Turkey, Germany, Korea, Republic of, United Kingdom, Saudi Arabia, Canada, Greece, Sweden, Brazil, Italy
Spark Therapeutics

Completed

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Hematologic Diseases | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Gene Therapy | Blood Coagulation Disorder | Gene Transfer | Adeno-Associated Virus (AAV) | Factor VIII (FVIII) | Factor VIII (FVIII)... and other conditions

United States
Baxalta now part of Shire

Active, not recruiting

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

Hemophilia B

United States
City of Hope Medical Center
National Cancer Institute (NCI)

Active, not recruiting

Curcumin in Reducing Joint Pain in Breast Cancer Survivors With Aromatase Inhibitor-Induced Joint Disease

Breast Cancer | Joint Pain

United States
Eli Lilly and Company
Sigilon Therapeutics, Inc.

Terminated

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

Hemophilia A

United Kingdom, United States
Spark Therapeutics

Completed

A Gene Transfer Study for Hemophilia A

Hemophilia A

United States, Australia, Canada, Israel, Thailand
BioMarin Pharmaceutical

Active, not recruiting

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (BMN 270-301)

Hemophilia A

United States, Taiwan, United Kingdom, Australia, Israel, Belgium, Brazil, France, Germany, Italy, Korea, Republic of, South Africa, Spain
BioMarin Pharmaceutical

Active, not recruiting

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Severe Haemophilia A

United Kingdom
Celgene

Completed

A Study to Determine Dose, Safety, and Efficacy of Durvalumab as Monotherapy and in Combination Therapy in Subjects With Lymphoma or Chronic Lymphocytic Leukemia (FUSION NHL 001)

Lymphoma | Leukemia, Lymphocytic, Chronic, B-Cell

United States, United Kingdom, Germany, France, Italy, Netherlands, Japan
Galectin Therapeutics Inc.

Active, not recruiting

Study Evaluating the Efficacy and Safety of Belapectin for the Prevention of Esophageal Varices in NASH Cirrhosis (NAVIGATE)

Cirrhosis | NASH - Nonalcoholic Steatohepatitis | Prevention of Esophageal Varices

United States, France, Spain, Australia, Canada, Israel, Puerto Rico, Belgium, Argentina, Chile, Germany, Korea, Republic of, Mexico, Poland, United Kingdom
BioMarin Pharmaceutical

Completed

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg (GENEr8-2)

Hemophilia A

United States
Daniel Benjamin
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (PTN_POPS)

Heart Failure | Epilepsy | Pain | Pneumonia | Sepsis | Inflammation | Hypertension | Chronic Kidney Diseases | Hemophilia | Schizophrenia | Central Nervous System Infections | Nosocomial Pneumonia | Insomnia | Anxiety | Bipolar Disorder | Neutropenia | Staphylococcal Infections | Bradycardia | Seizures | Urinary Tract Infections | Pulmonary... and other conditions

United States, Canada, United Kingdom, Israel, Singapore
Shanghai Belief-Delivery BioMed Co., Ltd

Active, not recruiting

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

Hemophilia B

China
Freeline Therapeutics

Terminated

A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy

Hemophilia B

United Kingdom
Universita degli Studi della Basilicata
San Carlo Public Hospital, Potenza, Italy

Completed

Medical Cannabis for Treating Pain Related to Fibromyalgia (Bedrocan®-LD)

Fibromyalgia

Italy
Spark Therapeutics

Completed

Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

Hematologic Diseases | Hemophilia A | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Blood Coagulation Disorder | Factor VIII Deficiency

United States, Australia, Thailand, Canada
Rigel Pharmaceuticals

Completed

A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA

Warm Antibody Autoimmune Hemolytic Anemia

United States, Spain, Canada, Australia, Norway, France, Belarus, Germany, Netherlands, Belgium, United Kingdom, Austria, Czechia, Georgia, Italy, Russian Federation, Serbia, Ukraine, Romania, Denmark, Hungary, Bulgaria
University of Seville

Terminated

Therapeutic Exercise in Patients With Hemophilia (Hemofisio)

Hemophilia

Spain
Intercept Pharmaceuticals

Terminated

Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis (COBALT)

Liver Cirrhosis, Biliary

United States, Spain, Korea, Republic of, Australia, Finland, Israel, Lithuania, United Kingdom, Belgium, Netherlands, Estonia, Brazil, Italy, Serbia, Sweden, Argentina, Austria, Canada, Chile, Denmark, France, Germany, Hong Kong, Hun... and more
Fondazione Policlinico Universitario Agostino Gemelli...

Completed

Physical Activity and FVIII Elimination: Relevance to Personalized Therapy in Severe Hemophilia A

Hemophilia A

Italy
Haemnet
UniQure Biopharma B.V.

Completed

An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families (Exigency)

Hemophilia

United Kingdom
Cumhuriyet University

Completed

The Effect of Art Therapy on Pain, Emesis, Anxiety and Quality of Life, Breast Cancer

Breast Cancer

Turkey
University College, London

Terminated

A Factor IX Gene Therapy Study (FIX-GT) (FIX-GT)

Hemophilia B

Ireland, United Kingdom, Italy, United States
Novartis Pharmaceuticals

Completed

Risk Perception in Multiple Sclerosis

Multiple Sclerosis

United States
Freeline Therapeutics

Completed

AAV Gene Therapy Screening/Observational Protocol (ECLIPSE) (ECLIPSE)

Hemophilia B | Blood Coagulation Disorders, Inherited | Blood Coagulation Disorder | Haematologic Disease

United States, United Kingdom, Australia, Netherlands, Austria, France, Ireland, Italy, Turkey, Canada, Germany, South Africa
Institute of Hematology & Blood Diseases Hospital
East China University of Science and Technology

Active, not recruiting

Gene Therapy for Chinese Hemophilia B

Hemophilia B

China
CSL Behring

Completed

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

Hemophilia B

Germany, Denmark, Netherlands
Prof. Dr. Dr. Thomas Hilberg
Pfizer

Completed

iT-based Sports Therapy Application in Haemophilia (iT-BaSTAH)

Sports Physical Therapy | Haemophilia

Germany
Johns Hopkins All Children's Hospital
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Evaluation of the Duration of Therapy for Thrombosis in Children (Kids-DOTT)

Venous Thrombosis

United States, Australia, Canada, Austria, Israel, Netherlands
Actelion

Completed

Clinical Study to Assess the Long-term Safety and Tolerability of ACT 064992 in Patients With Symptomatic Pulmonary Arterial Hypertension (SERAPHIN OL)

Pulmonary Arterial Hypertension

United States, United Kingdom, Taiwan, Finland, Canada, Belarus, Belgium, Argentina, Australia, China, Colombia, Croatia, Germany, Hong Kong, India, Israel, Italy, Malaysia, Netherlands, Poland, Russian Federation, South Africa, Tha... and more
Ultragenyx Pharmaceutical Inc

Completed

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Hemophilia B

United States, United Kingdom
GlaxoSmithKline

Completed

Anemia Studies in Chronic Kidney Disease: Erythropoiesis Via a Novel Prolyl Hydroxylase Inhibitor Daprodustat-Dialysis (ASCEND-D)

Anaemia | Aspergillosis, Allergic Bronchopulmonary

Australia, Korea, Republic of, Spain, United States, Taiwan, Bulgaria, Mexico, Ukraine, Argentina, Belgium, South Africa, Singapore, Austria, Canada, Netherlands, Russian Federation, Sweden, Turkey, United Kingdom, Germany, Romania and more
Grifols Therapeutics LLC
Grifols Biologicals, LLC

Terminated

Alphanate in Immune Tolerance Induction Therapy

Hemophilia A, Congenital

United States, Spain, Italy, India, Canada, Russian Federation
National Institute of Allergy and Infectious Diseases...

Completed

A Study of Several Anti-HIV Drug Combinations in HIV-Infected Patients Who Have Used Indinavir

HIV Infections

United States
National Institute of Allergy and Infectious Diseases...
Bristol-Myers Squibb; Glaxo Wellcome

Completed

Comparison of Three Anti-HIV Drug Combinations in HIV-Infected Patients With No Symptoms of the Disease

HIV Infections

United States, Puerto Rico
National Institute of Allergy and Infectious Diseases...

Completed

Virologic Responses To New Nucleoside Regimens After Prolonged ZDV or ddI Monotherapy

HIV Infections

United States, Puerto Rico, Tanzania
National Institute of Allergy and Infectious Diseases...

Completed

A Study of Disease Progression and Anti-HIV Treatments

HIV Infections

United States, Tanzania, Puerto Rico
National Institute of Allergy and Infectious Diseases...
Bristol-Myers Squibb

Completed

The Effectiveness of Three Drug Combinations in HIV-Infected Patients Who Have Taken Zidovudine for More Than 12 Weeks

HIV Infections

United States, Puerto Rico
National Institute of Allergy and Infectious Diseases...

Completed

A Study of Indinavir Sulfate Plus Zidovudine (AZT) Plus Lamivudine in HIV-Infected Patients Who Have Taken AZT for Six or More Months

HIV Infections

United States, Puerto Rico
National Institute of Allergy and Infectious Diseases...
Bristol-Myers Squibb; Glaxo Wellcome

Completed

A Randomized, Double-Blind, Four-Arm Study Comparing Combination Nucleoside, Alternating Nucleoside, and Triple-Drug Therapy for the Treatment of Advanced HIV Disease (CD4 <= 50/mm3)

HIV Infections

United States, Puerto Rico
National Institute of Allergy and Infectious Diseases...
Hoffmann-La Roche; Glaxo Wellcome

Completed

A Randomized, Double Blind, Comparative Study of Dideoxycytidine (ddC) Alone or ddC/AZT Combination Versus Zidovudine (ZDV) Alone in Patients With HIV Infection Who Have Received Prior ZDV Therapy

HIV Infections

United States
National Institute of Allergy and Infectious Diseases...
Bristol-Myers Squibb; Glaxo Wellcome

Completed

A Randomized, Double-Blind Phase II/III Trial of Monotherapy vs. Combination Therapy With Nucleoside Analogs in HIV-Infected Persons With CD4 Cells of 200-500/mm3

HIV Infections

United States, Puerto Rico, Tanzania
National Institute of Allergy and Infectious Diseases...
Bristol-Myers Squibb; Glaxo Wellcome

Completed

A Randomized Comparative Trial of Zidovudine (AZT) Versus 2',3'-Dideoxyinosine (ddI) Versus AZT Plus ddI in Symptomatic HIV-Infected Children

HIV Infections

United States, Puerto Rico

Search clinical trials for: Hemophilia B, gene therapy

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