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Gene Therapy

Gene Therapy for Hemophilia A (KEYSTONE 1 Trial)

Phase 3
Recruiting
Research Sponsored by Spark Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Have ≥150 documented exposure days to an FVIII protein product such as recombinant, plasma-derived, or extended half-life FVIII product
If human immunodeficiency virus (HIV)-positive at screening, have an adequate cluster of differentiation 4 (CD4) count (>200/mm3) and undetectable viral load (<50 genome copies [gc]/mL), are on an antiretroviral drug regimen, and have completed at least 12 weeks of this treatment regimen prior to screening
Must not have
Have an inherited or acquired bleeding disorder other than hemophilia A
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial aims to test if SPK-8011 is better at preventing bleeding episodes in people with hemophilia A who do not have inhibitors compared to the standard treatment with FVIII prophylaxis

Who is the study for?
Adult males with severe or moderately severe Hemophilia A, having FVIII activity ≤3%. They must have used FVIII products for at least 150 days and not be allergic to them. No history of liver disease, cirrhosis, or inhibitors against FVIII in the past 5 years. HIV-positive participants need controlled virus levels and CD4 count >200/mm3.
What is being tested?
The trial is testing SPK-8011's ability to prevent bleeding episodes in comparison with routine prophylaxis using Factor VIII (FVIII) in men with Hemophilia A without inhibitors. It will assess if gene therapy can reduce the frequency of bleeds better than standard treatments.
What are the potential side effects?
Potential side effects are not specified here but may include reactions similar to other gene therapies such as immune responses, injection site reactions, headaches, muscle aches, fever or changes in liver function tests.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have used a Factor VIII product for at least 150 days.
Select...
I am HIV-positive with a CD4 count over 200, an undetectable viral load, and have been on HIV medication for at least 12 weeks.
Select...
I haven't had issues with FVIII inhibitors or low FVIII activity in the past 5 years.
Select...
I have been treated with FVIII for at least 6 months and had 5 or more bleeds treated in the last 6 months.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have a bleeding disorder that is not hemophilia A.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Group I: Cohort CExperimental Treatment1 Intervention
Participants with severe or moderately severe hemophilia A without FVIII inhibitors using emicizumab prophylaxis
Group II: Cohort BExperimental Treatment1 Intervention
Participants with severe or moderately severe hemophilia A without FVIII inhibitors using on-demand FVIII replacement therapy
Group III: Cohort A (Primary Cohort)Experimental Treatment1 Intervention
Participants with severe or moderately severe hemophilia A without FVIII inhibitors using routine FVIII prophylaxis
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
SPK-8011
2017
Completed Phase 2
~30

Find a Location

Who is running the clinical trial?

Spark Therapeutics, Inc.Lead Sponsor
14 Previous Clinical Trials
354 Total Patients Enrolled
6 Trials studying Hemophilia A
91 Patients Enrolled for Hemophilia A
~57 spots leftby May 2027
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