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Hemophilia B Gene Therapy With AAV8 Vector

NCT01620801 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2019-03-12

No results posted yet for this study

Summary

Hemophilia B is a bleeding disease in males due to very low levels of coagulation factor IX (FIX) in the blood. The current treatment is intravenous injection of FIX clotting factor concentrates, in response to bleeding. This study will focus on the severe, most common type of hemophilia B. This study plans to use a virus called adeno-associated virus (AAV), which in nature causes no disease, and can be engineered to deliver the human FIX gene (AAV8-hFIX19 vector) to liver cells, where FIX is normally made. This study will use the AAV8-hFIX19 vector.

Conditions

  • Hemophilia B

Interventions

BIOLOGICAL

AAV8-hFIX19

One-time IV vector administration.

Sponsors & Collaborators

  • Children's Hospital of Philadelphia

    collaborator OTHER

  • University of Pittsburgh

    collaborator OTHER

  • Royal Prince Alfred Hospital, Sydney, Australia

    collaborator OTHER

  • St. James's Hospital, Ireland

    collaborator OTHER

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Clinical Director · Spark Therapeutics, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-10-31
Primary Completion
2016-03-31
Completion
2016-03-31

Countries

  • United States
  • Australia

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 NCT01620801 on ClinicalTrials.gov