LTFU for Gene Transfer Subjects With Hemophilia B

Completed

Conditions studied: Hemophilia B

In brief

Several subjects enrolled in a multi-site, gene transfer clinical study to evaluate the intrahepatic administration of AAV2-hFIX16 vector for the treatment of severe hemophilia B between 2001 and 2009. As the US FDA has established guidelines for the long-term follow-up (LTFU) of subjects receiving investigational gene therapy products, this protocol seeks to characterize the clinical outcome and the type and seriousness of adverse events following the AAV gene transfer. The primary study tools will consist of annual history/physical examination and blood tests, as well as periodic liver ultrasound, to characterize clinical outcomes. Where possible, data will be obtained for up to 15 years following hepatic AAV2-hFIX16 gene transfer.

Key facts

Study ID

NCT00515710

Run by

Spark Therapeutics, Inc.

People needed

4

Starts

2007-08-01

Expected to finish

2017-12-01

Last updated by the study team

2020-05-15

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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

Where it is running

• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Hemophilia Center of Western Pennsylvania — Pittsburgh, Pennsylvania, United States

Full record on ClinicalTrials.gov

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