NCT03432520

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

A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 29 (actual)
• Sponsor: Spark Therapeutics, Inc. · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Hemophilia A

Interventions

Type	Name	Description
GENETIC	SPK-8011	Observational long-term safety follow-up study of participants previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study
GENETIC	SPK-8016	Observational long-term safety follow-up study of participants previously treated with SPK-8016 in any Spark-sponsored SPK-8016 study

Timeline

• Start date: 2018-08-14
• Primary completion: 2032-12-01
• Completion: 2032-12-01
• First posted: 2018-02-14
• Last updated: 2024-11-29

Locations

12 sites across 3 countries: United States, Australia, Canada

Regulatory

• FDA-regulated drug study