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NCT00515710 · ClinicalTrials.gov registry record
LTFU for Gene Transfer Subjects With Hemophilia B
A clinical trial, sponsored by Spark Therapeutics.
- Completed
- Registry status
- 4
- Enrollment target
NCT00515710: Completed study, sponsored by Spark Therapeutics.
NCT00515710 is a clinical trial that has completed, run by Spark Therapeutics. The registered enrollment target is 4 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00515710 has completed, sponsored by Spark Therapeutics.
- COMPLETED
- Registry status
- 4 participants
- Enrollment target
Study Summary
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.
Primary Outcome
Annual study visits will be comprised of history, physical examination, blood tests and urinalysis and may include hepatic ultrasound tests; additional medical information may be needed to determine the relationship of adverse events to the investigational gene therapy vector AAV2-hFIX16.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2007-08 |
| Est. Completion | 2017-12 |
What the finished NCT00515710 record still lists
NCT00515710 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 4 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT00515710 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00515710 about?
NCT00515710 is a clinical study titled "LTFU for Gene Transfer Subjects With Hemophilia B". 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 ...
What is the current status of trial NCT00515710?
This trial is currently completed. The enrollment target is 4 participants. The study started on 2007-08. Estimated completion is 2017-12.
Who is sponsoring clinical trial NCT00515710?
This trial is sponsored by Spark Therapeutics, which has 13 total clinical trials registered on ClinicalTrials.gov.
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