Brief Title
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
Official Title
A Multi-Center, Observational Study in Males With Hemophilia A
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 with clinically severe hemophilia A receiving prophylactic FVIII replacement
therapy as standard of care. The data collected from this study may assist in providing
baseline information for comparison to the Spark's investigational hemophilia A gene therapy
in future Phase 3 studies.
Study Type
Observational
Primary Outcome
Number of bleeding events, annualized
Secondary Outcome
Dose and total FVIIII consumption
Condition
Blood Coagulation Disorder
Intervention
Standard of Care FVIII Replacement therapy
Study Arms / Comparison Groups
Observational Cohort
Description: Adult males with clinically severe hemophilia A, who are negative for neutralizing antibody (NAb) to AAV-Spark200
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
55
Start Date
January 21, 2019
Completion Date
June 2022
Primary Completion Date
December 2021
Eligibility Criteria
Inclusion Criteria:
1. Able and willing to provide written informed consent.
2. Males ≥18 years of age.
3. Clinically severe hemophilia A
4. Previous exposure to FVIII therapy
5. No prior history of hypersensitivity or anaphylaxis associated with an FVIII or
intravenous immunoglobulin administration.
6. No measurable inhibitor against FVIII
7. Willing to participate and receive treatment in a future Spark hemophilia A gene
therapy study.
Exclusion Criteria:
1. Documented active hepatitis B or C within the past 12 months of Screening
2. Currently on antiviral therapy to treat hepatitis B or C;
3. Documented significant liver disease within the past 6 months of Screening
4. Have serological evidence of HIV-1 or HIV-2
5. Anti-AAV-Spark 200 neutralizing titers ≥1:1
6. Previously received SPK-8011;
7. Previously dosed with any investigational or approved gene therapy product at any time
or treated with an investigational drug within the last 12 weeks;
8. Planned surgical procedure in the next 12 months requiring FVIII prophylactic
treatment.
9. 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.
10. Unable or unwilling to comply with the schedule of visits and/or study assessments
described in the protocol.
Gender
Male
Ages
18 Years - N/A
Contacts
Spencer K Sullivan, MD, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT03876301
Organization ID
SPK-8011-301
Responsible Party
Sponsor
Study Sponsor
Spark Therapeutics
Study Sponsor
Spencer K Sullivan, MD, Principal Investigator, Mississippi Center for Advanced Medicine
Verification Date
October 2020