Gene Therapy for Hemophilia A

(KEYSTONE 1 Trial)

Recruiting at26 trial locations

Age: 18+

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Spark Therapeutics, Inc.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis.

Research Team

Eligibility Criteria

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.

Inclusion Criteria

Have a negative test for inhibitor against FVIII (ie, <0.6 Bethesda units [BU]) during screening

Have a negative anti-AAV-Spark200 neutralizing antibody (NAb) test result

Have no prior history of hypersensitivity or anaphylaxis associated with the administration of any FVIII product

Exclusion Criteria

Have inherited or acquired thrombophilia, have signs of thromboembolic disease in the Investigator's judgment, or are on current treatment for thromboembolic disease. A history of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing is not considered an exclusion criterion

Have concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study or that would, in the opinion of the Investigator or Sponsor, preclude the candidate's safe participation in and completion of the study, or the interpretation of the study results

I have a bleeding disorder that is not hemophilia A.

Treatment Details

Interventions

• SPK-8011 (Gene Therapy)

Trial Overview 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.

Participant Groups

Group I: Cohort C Experimental Treatment 1 Intervention

Participants with severe or moderately severe hemophilia A without FVIII inhibitors using emicizumab prophylaxis

Group II: Cohort B Experimental Treatment 1 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 Treatment 1 Intervention

Participants with severe or moderately severe hemophilia A without FVIII inhibitors using routine FVIII prophylaxis

Who Is Running the Clinical Trial?

Spark Therapeutics, Inc.

Lead Sponsor

Trials

16

Recruited

410+