Search clinical trials for: Factor VIII gene therapy

Total 304588 results

Spark Therapeutics, Inc.

Recruiting

Study of a Gene Therapy Treatment for Hemophilia A (KEYSTONE 1)

Hemophilia A

United States
St. Jude Children's Research Hospital

Not yet recruiting

Liver Biopsy Following Gene Therapy For Hemophilia

Hemophilia A | Hemophilia B

United States
Pfizer

Recruiting

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Hemophilia A | Hemophilia B

Australia, United States, Turkey
Christian Medical College, Vellore, India
Dr. H. Trent Spencer, Professor, Emory University of Medicine, Atlanta Ga...

Recruiting

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

Hemophilia A

India
BioMarin Pharmaceutical

Recruiting

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors (GENEr8-INH)

Hemophilia A With Inhibitor | Hemophilia A With Anti Factor VIII

United States, United Kingdom, Taiwan, Israel, Korea, Republic of, South Africa, Brazil, Italy, Germany
Medical College of Wisconsin

Recruiting

Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A

Hemophilia A

United States
BioMarin Pharmaceutical

Active, not recruiting

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)

Hemophilia A

Japan
Bayer
Ultragenix pharmaceutical

Active, not recruiting

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle".

Hemophilia A

Netherlands, United States, Germany, United Kingdom, France, Bulgaria
Takeda

Completed

A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions

Hemophilia

France
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
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
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
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
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
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
AbbVie
Radiation Therapy Oncology Group

Completed

A Study of ABT-414 in Participants With Newly Diagnosed Glioblastoma (GBM) With Epidermal Growth Factor Receptor (EGFR) Amplification (Intellance1)

Glioblastoma | Gliosarcoma

United States, Argentina, Australia, Austria, Belgium, Brazil, Canada, China, Colombia, Czechia, France, Germany, Hong Kong, Ireland, Israel, Italy, Korea, Republic of, Mexico, Netherlands, New Zealand, Portugal, Russian Federation, Singapor... and more
Zhujiang Hospital
Guangdong Province, Department of Science and Technology

Withdrawn

The Relationship Between Clotting Factor VIII and Bleeding Adverse Reactions in Patients Under the Therapies of Thrombolysis, Anticoagulation and Anti-platelet

Ischemic Stroke | Bleeding

China
Grifols Therapeutics LLC
Grifols Biologicals, LLC

Terminated

Alphanate in Immune Tolerance Induction Therapy

Hemophilia A, Congenital

United States, Spain, Italy, India, Canada, Russian Federation
Bayer

Completed

Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A." (POTTER 10)

Hemophilia A

Italy
Hospices Civils de Lyon

Unknown

TGA (Thrombin Generation Assay) and Prophylaxis in Haemophilia (HOPE)

Haemophilia A

France
Shenzhen Geno-Immune Medical Institute

Unknown

Lentiviral FVIII Gene Therapy

Hemophilia A

China
Hampshire Hospitals NHS Foundation Trust
Uppsala University; University of Wales Cardiff United Kingdom (UK)

Completed

Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A

Severe Haemophilia A

United Kingdom
Bayer

Completed

A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A (Leopold II)

Hemophilia A

China, United States, Ukraine, Taiwan, Mexico, Thailand, Romania, Colombia, Turkey, Serbia, Russian Federation, South Africa, Japan, Indonesia, Argentina, Czech Republic, India, Slovakia
Nara Medical University
Shire

Unknown

Basic Study of Combination Therapy Based on APCC-induced FVllI Activation in Hemophilia A Patients With Inhibitors

Hemophilia A With Inhibitor

Japan
Bayer

Completed

Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment (SPINART)

Hemophilia A

United States, Bulgaria, Romania, Argentina
Oslo University Hospital

Completed

A Comparison Study of Bypassing Agent Therapy With and Without Tranexamic Acid in Haemophilia A Patients With Inhibitor (BPATXAS)

Hereditary Factor VIII Deficiency Disease With Inhibitor

Norway
Sheba Medical Center

Completed

Combination Therapy of Low Doses of rFVIIa and FEIBA for Severe Hemophilia A Patients With an Inhibitor to Factor VIII

Hemophilia A
Bayer

Completed

Prophylaxis Versus On-demand Therapy Through Economic Report (POTTER)

Hemophilia A

Italy
Merck Sharp & Dohme LLC

Completed

Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia

HIV Infections | Hemophilia A

Canada, United States
Ankara City Hospital Bilkent

Recruiting

Evaluation of Sarcopenia and Related Factors in Patients Diagnosed With Psoriatic Arthritis

Psoriatic Arthritis

Turkey
Zhongshan Hospital Xiamen University

Not yet recruiting

Efficacy of Self vs. Physician Application of Triamcinolone-Econazole in Otomycosis

Otomycosis

China
Peter MacCallum Cancer Centre, Australia
AbbVie

Not yet recruiting

MRD-Directed Consolidation With Epcor-only or Epcor-R2 Post Anti-CD19 CAR TCell Therapy for Large B-Cell Lymphoma (EpLCART)

Relapsed/Refractory Large B-cell Lymphoma

Australia
IRCCS Azienda Ospedaliero-Universitaria di Bologna

Recruiting

RISK FACTORS FOR NON-RESPONSE TO HORMONAL MEDICAL THERAPY IN PATIENTS WITH ENDOMETRIOSIS

Endometriosis

Italy
The First Affiliated Hospital of Soochow University

Not yet recruiting

Treatment of High-risk Newly Diagnosed Multiple Myeloma With Minimal Residual Disease Detection

Multiple Myeloma | Newly Diagnosed | MRD | High Risk
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

Not yet recruiting

FOLFOX Plus Lenvatinib Adjuvant Therapy for Hepatocellular Carcinoma Post-Liver Transplantation

Liver Cancer | Liver Transplant

China
Kyverna Therapeutics

Not yet recruiting

A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects With Systemic Sclerosis

Systemic Sclerosis | Systemic Sclerosis - Diffuse Cutaneous | Systemic Sclerosis - 2013 ACR/EULAR Classification Criteria
Children's Oncology Group

Not yet recruiting

A Study Using Risk Factors to Determine Treatment for Children With Favorable Histology Wilms Tumors (FHWT)

Stage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor
Celgene
Bristol-Myers Squibb

Recruiting

A Study to Compare the Efficacy and Safety of Idecabtagene Vicleucel With Lenalidomide Maintenance Therapy Versus Lenalidomide Maintenance Therapy Alone in Adult Participants With Newly Diagnosed Multiple Myeloma Who Have Suboptimal Response After Autologous Stem Cell Transplantation (KarMMa-9)

Multiple Myeloma

Austria, United States, Spain, Australia, Japan, France, Israel, Italy, Canada, Greece, Belgium, Norway, Romania, Czechia, Denmark, Germany, Korea, Republic of, United Kingdom, Poland
Essen Biotech

Not yet recruiting

Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease (BAH243)

Sickle Cell Disease | Sickle-Cell Disease With Crisis

China
CSL Behring

Not yet recruiting

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy

Hemophilia B
Brazilian Clinical Research Institute
Alliança Diagnostic

Not yet recruiting

Anti-inflammatory and Anti-thrombotic Therapy With colcHicine and Low Dose Rivaroxaban for Major Adverse Cardiovascular Events Reduction in Ischemic Stroke (ARCHIMEDES)

Ischemic Stroke
Bristol-Myers Squibb

Not yet recruiting

A Study to Assess the Effectiveness and Safety of Ozanimod in Chinese Adults With Relapsing Multiple Sclerosis

Relapsing Multiple Sclerosis

China
St. Jude Children's Research Hospital
AbbVie

Not yet recruiting

Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)

T-cell Lymphoma | T-cell Acute Lymphoblastic Leukemia | Mixed Phenotype Acute Leukemia

United States
Mayo Clinic

Not yet recruiting

Stereotactic Body Radiation Therapy Plus Androgen Receptor Pathway Inhibitor and Androgen Deprivation Therapy for Treatment of Metastatic, Recurrent Hormone-Sensitive Prostate Cancer, DIVINE Trial (DIVINE)

Recurrent Prostate Cancer | Castration-resistant Prostate Cancer | Stage IVB Prostate Cancer AJCC v8 | Recurrent Castration-Sensitive Prostate Carcinoma

United States
Lyvgen Biopharma Holdings Limited

Not yet recruiting

A Study of LVGN6051 Combination Therapy in Patient With Head and Neck Squamous Cell Carcinoma (HNSCC)

Head and Neck Squamous Cell Carcinoma

China
Second Affiliated Hospital, School of Medicine,...

Recruiting

A Prospective Study of Whole-Brain RT Combined With Thiotepa Sheath Injection Combined With Systemic Therapy for the Primary Disease in the Treatment of Meningeal Metastases in Solid Tumors

Solid Tumor Leptomeningeal Metastasis

China

Search clinical trials for: Factor VIII gene therapy

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