Status:
COMPLETED
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
Lead Sponsor:
Spark Therapeutics, Inc.
Conditions:
Blood Coagulation Disorder
Blood Coagulation Disorders, Inherited
Eligibility:
MALE
18+ years
Brief Summary
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients wit...
Eligibility Criteria
Inclusion
- Able and willing to provide written informed consent.
- Males ≥18 years of age.
- Clinically severe hemophilia A
- Previous exposure to FVIII therapy
- No prior history of hypersensitivity or anaphylaxis associated with an FVIII or intravenous immunoglobulin administration.
- No measurable inhibitor against FVIII
- Willing to participate and receive treatment in a future Spark hemophilia A gene therapy study.
Exclusion
- Documented active hepatitis B or C within the past 12 months of Screening
- Currently on antiviral therapy to treat hepatitis B or C;
- Documented significant liver disease within the past 6 months of Screening
- Have serological evidence of HIV-1 or HIV-2
- Anti-AAV-Spark 200 neutralizing titers ≥1:1
- Previously received SPK-8011;
- Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks;
- Planned surgical procedure in the next 12 months requiring FVIII prophylactic treatment.
- Any history of chronic infection or other chronic disease, concurrent clinically significant major disease (such as liver abnormalities or type I diabetes) including active malignancy, except for non-melanoma skin cancer, any other condition or any other unspecified reasons that, in opinion of the Investigator or Sponsor, makes the participant unsuitable for participation and dosing in a future clinical study for Spark's hemophilia A gene therapy.
- Unable or unwilling to comply with the schedule of visits and/or study assessments described in the protocol.
Key Trial Info
Start Date :
January 21 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 2 2023
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT03876301
Start Date
January 21 2019
End Date
May 2 2023
Last Update
July 10 2023
Active Locations (11)
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1
University of California San Francisco
San Francisco, California, United States, 94117
2
University of Florida
Gainesville, Florida, United States, 32610
3
Emory University
Atlanta, Georgia, United States, 30322
4
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201