Trials / Completed
CompletedNCT03734588
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 4 (actual)
- Sponsor
- Spark Therapeutics, Inc. · Industry
- Sex
- Male
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII.
Conditions
- Adeno-Associated Virus (AAV)
- Blood Coagulation Disorder
- Blood Coagulation Disorders, Inherited
- Coagulation Protein Disorders
- Factor VIII (FVIII)
- Factor VIII (FVIII) Deficiency
- Factor VIII (FVIII) Gene
- Factor VIII (FVIII) Protein
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Gene Therapy
- Gene Transfer
- Hematologic Diseases
- Hemorrhagic Disorders
- Recombinant
- Vector
- Inhibitors
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | SPK-8016 | adeno-associated viral vector |
Timeline
- Start date
- 2019-01-30
- Primary completion
- 2020-10-14
- Completion
- 2023-01-19
- First posted
- 2018-11-08
- Last updated
- 2024-02-23
- Results posted
- 2024-02-23
Locations
11 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03734588. Inclusion in this directory is not an endorsement.