Study of a Gene Therapy Treatment for Hemophilia A (NCT06297486) | Clinical Trial Compass
WithdrawnPhase 3
Study of a Gene Therapy Treatment for Hemophilia A
Stopped: Strategic Decision
United States0Started 2024-03-13
Plain-language summary
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.
Who can participate
Age range18 Years
SexMALE
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Have a negative anti-AAV-Spark200 neutralizing antibody (NAb) test result.
* 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
* Have ≥150 documented exposure days to an FVIII protein product such as recombinant, plasma-derived, or extended half-life FVIII product
* Have no prior history of hypersensitivity or anaphylaxis associated with the administration of any FVIII product.
* 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.
* Have a negative test for inhibitor against FVIII (ie, \<0.6 Bethesda units \[BU\]) during screening.
* Have no documented FVIII inhibitor (ie, \<0.6 BU), FVIII half-life \<6 hours, or FVIII recovery \<66% in the 5 years prior to screening.
* Candidates who completed successful immune tolerance induction (ITI) at least 5 years before screening are eligible, provided they have had no evidence of inhibitor recurrence (permanent or temporary) within 5 years prior to screening as may be indicated by detection of an inhibitor, FVIII half-life \<6 hours, or FVIII recovery \<66% since completing ITI.
* If human immunodeficiency virus (HIV)-positive at screening, ha…
What they're measuring
1
Annualized Bleed Rate (ABR) for All Bleeds [Cohort A]