Active, currently enrolling
A Phase 3, single-arm, open-label, multicenter study of the safety and efficacy of dirloctocogene samoparvovec (SPK-8011, adeno-associated viral vector with B-domain deleted human factor VIII gene) in adults with severe or moderately severe hemophilia A (KEYSTONE 1)
NCT No.: NCT06297486
Study Type: Clinical Trial
Phase:
Phase III
Region: California - Northern
Acronym:
Official Title
A Phase 3, single-arm, open-label, multicenter study of the safety and efficacy of dirloctocogene samoparvovec (SPK-8011, adeno-associated viral vector with B-domain deleted human factor VIII gene) in adults with severe or moderately severe hemophilia A (KEYSTONE 1)
Purpose
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.
Detailed Description
Eligibility Criteria
Inclusion Criteria
- Have a negative anti-AAV-Spark200 neutralizing antibody (NAb) test result.
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Are adult males with severe or moderately severe hemophilia A, defined as endogenous FVIII activity =3%, as documented by a certified laboratory (historically or during the Screening Period) and where the FVIII:C level is measured more than 96 hours after the prior dose of an extended-half-life FVIII
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Have =150 documented exposure days to an FVIII protein product such as recombinant, plasma-derived, or extended half-life FVIII product
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Have no prior history of hypersensitivity or anaphylaxis associated with the administration of any FVIII product.
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Have screening hepatic ultrasound without evidence of cirrhosis and no laboratory or clinical evidence per the Investigator's judgment of advanced liver disease or cirrhosis.
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Exclusion Criteria
- Have an inherited or acquired bleeding disorder other than hemophilia A
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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.
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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.
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Keywords and/or Specific Medical Conditions
Sponsors
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