Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
- Conditions
- Hemophilia A
- Registration Number
- NCT03432520
- Lead Sponsor
- Spark Therapeutics, Inc.
- Brief Summary
This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.
- Detailed Description
This study will follow patients with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any prior Spark-sponsored SPK-8011 or SPK-8016 study. Participants will be followed for up to a total of 10 years post infusion (including the time on the dosing study).
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Male
- Target Recruitment
- 29
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) Up to 9 years Number of Participants with Adverse Events of Special Interest (AESIs) Up to 9 years Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development Up to 9 years Number of Participants with Clinical Laboratory Abnormalities Up to 9 years Number of Participants with Vital Sign Abnormalities Up to 9 years Number of Participants with Physical Exam Abnormalities Up to 9 years
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (12)
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Pennsylvania State University Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Mississippi Center for Advanced Medicine
🇺🇸Madison, Mississippi, United States
Hemophilia Center of Western Pennsylvania
🇺🇸Pittsburgh, Pennsylvania, United States
Royal Prince Alfred Hospital Department of Cell & Molecular Therapies
🇦🇺Sydney, New South Wales, Australia
The Alfred Hospital & Monash Medical Centre
🇦🇺Melbourne, Victoria, Australia
St. Michael's Hospital
🇨🇦Toronto, Ontario, Canada
Truman Medical Centers
🇺🇸Kansas City, Missouri, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
Thomas Jefferson University
🇺🇸Philadelphia, Pennsylvania, United States