NCT03003533

A Gene Transfer Study for Hemophilia A

Gene-transfer, Open-label, Dose-escalation Study of SPK-8011 [Adeno-associated Viral Vector With B-domain Deleted Human Factor VIII Gene] in Individuals With Hemophilia A

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Spark Therapeutics, Inc. · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.

Detailed description

Hemophilia A is a condition in which blood is unable to clot effectively. It is caused by a mutation or deletion in the gene that is responsible for producing blood-clotting factor VIII protein. Individuals with hemophilia A suffer from repeated bleeding episodes, often into the joints, which can cause chronic joint disease and sometime results in death due to the inability of the blood to clot efficiently. This chronic joint disease can have significant physical, psychosocial, and quality-of-life effects, including financial burden. The current standard of care includes the use of factor-based therapies which are given either as prophylaxis or to treat bleeding, as well as new non-factor prophylaxis therapies.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
GENETIC	SPK-8011	A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene

Timeline

• Start date: 2017-01-26
• Primary completion: 2023-12-05
• Completion: 2023-12-05
• First posted: 2016-12-28
• Last updated: 2024-12-30
• Results posted: 2024-12-30

Locations

16 sites across 5 countries: United States, Australia, Canada, Israel, Thailand

Regulatory

• FDA-regulated drug study