Brief Title
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
Official Title
A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A
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. Subjects will be followed for up to a total of 5 years post infusion (including the time on the dosing study).
Study Type
Observational
Primary Outcome
Incidence of FVIII Inhibitors
Secondary Outcome
Joint Health Assessment
Condition
Hemophilia A
Intervention
SPK-8011
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Genetic
Estimated Enrollment
40
Start Date
August 14, 2018
Completion Date
December 2032
Primary Completion Date
December 2032
Eligibility Criteria
Inclusion Criteria Participants are eligible to be included in the study only if all of the following criteria apply: 1. Have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored gene therapy study; and 2. Understand the purpose and risks of the study and provide signed and dated informed consent before undergoing any study-specific procedures. Exclusion Criteria Participants are excluded from the study if any of the following criteria apply: 1.Unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Clinical Trial Director, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT03432520
Organization ID
SPK-8011/8016-LTFU
Responsible Party
Sponsor
Study Sponsor
Spark Therapeutics
Study Sponsor
Clinical Trial Director, Study Director, Spark Therapeutics, Inc.
Verification Date
February 2023