Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Factor VIII Deficiency, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Hematologic Diseases, Hemophilia A, Hemorrhagic Disorders
Conditions
Keywords
Factor VIII, Factor VIII Protein, Recombinant
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.
Interventions
There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.
Sponsors
Spark Therapeutics, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of bleeding events, annualized | 12 months | Annualized bleeding rate (ABR) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Dose and total FVIIII consumption | 12 months | Total FVIII replacement therapy consumption and the corresponding dose |
| Annualized number of infusions (AIR) | 12 months | Number of reported infusions over the study period |
Countries
Australia, Canada, Thailand, United States
Outcome results
None listed