Study of a Gene Therapy Treatment for Hemophilia A

Withdrawn before enrolling · Phase 3

Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID

NCT06297486

Run by

Spark Therapeutics, Inc.

People needed

0

Starts

2024-03-13

Expected to finish

2035-09-04

Last updated by the study team

2024-12-13

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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, 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.
• Meet the following inclusion criteria by cohort:
• Cohort A: have documented history of prior treatment with FVIII prophylaxis (defined as receiving a prescribed dose and frequency of FVIII infusions with the intent to treat continuously for 52 weeks per year) for a minimum of 6 months prior to screening; and are willing to continue their FVIII prophylaxis during the Lead-In Period of this study (minimum of 24 weeks).
• Cohort B: have documented history of prior treatment with FVIII on demand for a minimum of 6 months that shows ≥5 treated bleeds in the last 6 months prior to screening.
• Cohort C: have documented history of prior treatment with emicizumab prophylaxis for a minimum of 6 months prior to screening.

You may not qualify if…

• Have an inherited or acquired bleeding disorder other than hemophilia A
• 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.

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• Loma Linda University Health — Loma Linda, California, United States
• Orthopaedic Institute for Children/Orthopaedic Hemophilia Treatment Center — Los Angeles, California, United States
• Kaiser Permanente-Oakland Medical Center — Oakland, California, United States
• Kaiser Permanente-Roseville Medical Center — Roseville, California, United States
• Kaiser Permanente -Sacramento Medical Center — Sacramento, California, United States
• Kaiser Permanente -San Francisco Medical Center — San Francisco, California, United States
• University of California - San Francisco — San Francisco, California, United States
• Kaiser Permanente- Santa Clara Medical Center — Santa Clara, California, United States
• Kaiser Permanente-Vallejo Medical Center — Vallejo, California, United States
• Kaiser Permanente -Walnut Creek Medical Center — Walnut Creek, California, United States
• University of Florida — Gainesville, Florida, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Newark Beth Israel — Newark, New Jersey, United States
• Weill Cornell Medical Hospital — New York, New York, United States
• University of North Carolina - Chapel Hill — Chapel Hill, North Carolina, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• University Hospitals Cleveland — Cleveland, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Oregon Health & Sciences University — Portland, Oregon, United States
• Penn State Hershey Medical Center — Hershey, Pennsylvania, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• Cook Children's Hospital — Fort Worth, Texas, United States
• The University of Texas Health Science Center at Houston-Gulf States Hemophilia & Thrombosis Center — Houston, Texas, United States

Full record on ClinicalTrials.gov

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