Phase 2
N=25
A Gene Transfer Study for Hemophilia A
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT03003533 ↗Enrolled (actual)
25
Serious AEs
20.0%
Results posted
Dec 2024
Primary outcome: Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 2; 3; 9; 11 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- SPK-8011 (Genetic)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Spark Therapeutics, Inc.
- Primary completion
- Dec 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
2; 3; 9; 11 | — |
| PRIMARY Number of Participants Who Received Corticosteroids for Presumed Immune Response |
0; 2; 7; 10 | — |
| PRIMARY Peak Factor VIII (FVIII) Activity Levels Assessed by One-Stage Coagulation Assay (OSA) |
11.5; 20.0; 38.7; 55.1 | — |
| PRIMARY Nominal FVIII Level by OSA at Week 52/EOS |
5.4; 7.8; 8.4; 4.1 | — |
| PRIMARY Spontaneous Bleeds Annualized Bleeding Rate |
NA; 0.3; 0.0; 0.0 | — |
| PRIMARY Total Annualized FVIII Infusion Rate |
0.3; 3.6; 7.4; 1.6 | — |
| SECONDARY Time to Achieve Peak FVIII Activity Level |
279.5; 141.0; 56.4; 54.6 | — |
| SECONDARY Number of Participants With Vector-shedding Confirmed Below Quantifiable Limits (BQL) of SPK-8011-101 in Bodily Fluids |
2; 3; 9; 11; 2; 3 | — |
| SECONDARY Incidence of Immune Response to the BDD-hFVIII Transgene |
0; 0; 0; 0 | — |
Summary
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
Eligibility Criteria
Inclusion Criteria
- Males age 18 years or older
- Confirmed diagnosis of hemophilia A as evidenced by their medical history with baseline FVIII activity levels 150 exposure days (EDs) to FVIII concentrates or cryoprecipitate
- Have no prior history of allergic reaction to any FVIII product
- Have no measurable inhibitor against FVIII as assessed by the central laboratory and have no prior history of inhibitors to FVIII protein and no clinical signs or symptoms of decreased response to FVIII administration
- Agree to use reliable barrier contraception
Exclusion Criteria
- Evidence of active hepatitis B or C
- Currently on antiviral therapy for hepatitis B or C
- Have significant underlying liver disease
- Have serological evidence* of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 and who are on an antiretroviral drug regimen (* participants who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible to enroll)
- Have detectable antibodies reactive with AAV-Spark200 capsid
- Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks
Data sourced from ClinicalTrials.gov (NCT03003533). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.