Trials / Completed
CompletedNCT03876301
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
A Multi-Center, Observational Study in Males With Hemophilia A
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 25 (actual)
- Sponsor
- Spark Therapeutics, Inc. · Industry
- Sex
- Male
- Age
- 18 Years
- Healthy volunteers
- —
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.
Conditions
- Blood Coagulation Disorder
- Blood Coagulation Disorders, Inherited
- Coagulation Protein Disorders
- Hemophilia A
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Hematologic Diseases
- Hemorrhagic Disorders
- Factor VIII Deficiency
Interventions
| Type | Name | Description |
|---|---|---|
| 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. |
Timeline
- Start date
- 2019-01-21
- Primary completion
- 2023-05-02
- Completion
- 2023-05-02
- First posted
- 2019-03-15
- Last updated
- 2023-07-10
Locations
11 sites across 4 countries: United States, Australia, Canada, Thailand
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03876301. Inclusion in this directory is not an endorsement.