LTFU for Gene Transfer Subjects With Hemophilia B

NCT00515710 | Completed DMC

• 1 other identifier: AAV2-hFIX16-LTFU-01
• Study Type: observational
• Target: 4
• Locations: 1 country
• Sites: 2

Brief 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

Keywords

Hemophilia B; Gene Transfer; Adeno-Associated Virus (AAV); Coagulation Factor IX; Long-term Follow-up; Delayed Adverse Events

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of intra-hepatic administration of AAV2-hFIX. Toxicity related to the administration of AAV2-hFIX will be evaluated locally and systemically.

  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.

  Long-term follow-up up to 15 years

Study Arms (1)

1

Prior gene therapy study subjects receiving AAV2-hFIX16.

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Prior gene therapy study subjects receiving AAV2-hFIX16.

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

Sponsors & Collaborators

• Spark Therapeutics, Inc.: lead
• Children's Hospital of Philadelphia: collaborator
• The Hemophilia Center of Western Pennsylvania: collaborator
• Royal Prince Alfred Hospital, Sydney, Australia: collaborator

Study Sites (2)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15213, United States

Location

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Clinical Director

  Spark Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 10, 2007
• First Posted: August 14, 2007
• Study Start: August 1, 2007
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2017
• Last Updated: May 15, 2020

Record last verified: 2020-05

Locations

US (2)