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

Plain-language summary

This is a long-term follow-up study evaluating the ongoing safety and efficacy of the gene therapies SPK-8011 and SPK-8016 in males with hemophilia A. Only people who already received a single intravenous administration of SPK-8011 or SPK-8016 in the Spark-sponsored studies SPK-8011-101 or SPK-8016-101 are eligible. No new treatment is given; participants are monitored over time for the effects of the therapy they already received.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Active, not recruiting

Sponsor

Spark Therapeutics, Inc.

Intervention

SPK-8011, SPK-8016

Start date

8/14/2018

Estimated completion

12/1/2032

Countries

United States, Australia, Canada

Related genes

Locations

• Boston, United States
• Madison, United States
• Kansas City, United States
• Portland, United States
• Hershey, United States
• Philadelphia, United States
• Philadelphia, United States
• Pittsburgh, United States
• Richmond, United States
• Sydney, Australia
• Melbourne, Australia
• Toronto, Canada

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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 either Study SPK-8011-101 or SPK-8016-101, respectively.
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.