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LTFU for Gene Transfer Subjects With Hemophilia B

NCT00515710 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 4

Last updated 2020-05-15

No results posted yet for this 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.

Conditions

  • Hemophilia B

Sponsors & Collaborators

  • Children's Hospital of Philadelphia

    collaborator OTHER

  • The Hemophilia Center of Western Pennsylvania

    collaborator OTHER

  • Royal Prince Alfred Hospital, Sydney, Australia

    collaborator OTHER

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Clinical Director · Spark Therapeutics, Inc.

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-08-31
Primary Completion
2017-12-31
Completion
2017-12-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00515710 on ClinicalTrials.gov