SPK-8011

GENETIC3 trials

Sponsors

Spark Therapeutics, Inc.

Conditions

Hemophilia A

Phase 1

A Gene Transfer Study for Hemophilia A

CompletedNCT03003533

Spark Therapeutics, Inc.Hemophilia A

Start: 2017-01-26End: 2023-12-05Updated: 2024-12-30

Phase 3

Study of a Gene Therapy Treatment for Hemophilia A

WithdrawnNCT06297486

Spark Therapeutics, Inc.Hemophilia A

Start: 2024-03-13End: 2035-09-04Updated: 2024-12-13

Unknown Phase

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

Active, not recruitingNCT03432520

Spark Therapeutics, Inc.Hemophilia A

Start: 2018-08-14End: 2032-12-31Updated: 2024-11-29

Related Papers

Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A

Blood Advances2024-07-183 citations

Long-Term FVIII Expression with Reduced Bleeding Following Gene Transfer for Hemophilia A: Follow-up on the Dirloctocogene Samoparvovec Phase I/II Trial

Hämostaseologie2024-02-011 citations

Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011

Blood2022-11-156 citations

Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A

Blood2022-11-151 citations

The Effects of Immunomodulation with Corticosteroids to Manage an AAV Capsid Immune response in the Phase I/II Study of SPK-8011

Blood2022-11-15

Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A.

2021-11-2412 citations

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

New England Journal of Medicine2021-11-17232 citations

Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors

Molecular Therapy — Methods & Clinical Development2020-03-1327 citations