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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03876301
Other study ID # SPK-8011-301
Secondary ID
Status Completed
Phase
First received
Last updated
Start date January 21, 2019
Est. completion date May 2, 2023

Study information

Verified date July 2023
Source Spark Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 25
Est. completion date May 2, 2023
Est. primary completion date May 2, 2023
Accepts healthy volunteers
Gender Male
Age group 18 Years and older
Eligibility 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.

Study Design


Intervention

Drug:
Standard of Care FVIII Replacement therapy
There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.

Locations

Country Name City State
Australia The Alfred Hospital Melbourne Victoria
Australia Fiona Stanley Hospital Murdoch Western Austrailia
Canada McMaster University / Royal Prince Alfred Hospital Hamilton Ontario
Canada Providence Hematology/St. Paul's Hosptial Vancouver British Columbia
Thailand Ramathibodi Hospital, Mahidol University Bangkok
United States Emory University Atlanta Georgia
United States Children's Hospital of Michigan Detroit Michigan
United States University of Florida Gainesville Florida
United States Mississippi Center for Advanced Medicine Madison Mississippi
United States University of California San Francisco San Francisco California
United States Bloodworks Northwest Seattle Washington

Sponsors (1)

Lead Sponsor Collaborator
Spark Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Thailand, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of bleeding events, annualized Annualized bleeding rate (ABR) 12 months
Secondary Dose and total FVIIII consumption Total FVIII replacement therapy consumption and the corresponding dose 12 months
Secondary Annualized number of infusions (AIR) Number of reported infusions over the study period 12 months
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