LTFU for Gene Transfer Subjects With Hemophilia B

Plain-language summary

This protocol provides long-term follow-up for people who received the AAV2-hFIX16 vector into the liver for severe hemophilia B between 2001 and 2009. Following US FDA guidelines for long-term follow-up (LTFU) of investigational gene therapy products, it records clinical outcomes and any adverse events. Participants undergo annual history/physical examination, blood tests and periodic liver ultrasound, with data collected for up to 15 years after gene transfer.

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

Completed

Sponsor

Spark Therapeutics, Inc.

Start date

8/1/2007

Estimated completion

12/1/2017

Countries

United States

Related genes

Locations

• Philadelphia, United States
• Pittsburgh, United States

Eligibility criteria

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

Inclusion Criteria

-Adult subjects who participated in prior intrahepatic AAV2-hFIX16 gene transfer studies

Exclusion Criteria

* Subjects who will not consent for study
* Subjects who the investigators believe are not capable of performing endpoints of the study