Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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WITHDRAWN
PHASE3
INTERVENTIONAL
2024-03-13
2035-09-04
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort A (Primary Cohort)
Participants with severe or moderately severe hemophilia A without FVIII inhibitors using routine FVIII prophylaxis
SPK-8011
Participants will receive a single dose of SPK-8011, administered by intravenous (IV) infusion, on Day 1.
Cohort B
Participants with severe or moderately severe hemophilia A without FVIII inhibitors using on-demand FVIII replacement therapy
SPK-8011
Participants will receive a single dose of SPK-8011, administered by intravenous (IV) infusion, on Day 1.
Cohort C
Participants with severe or moderately severe hemophilia A without FVIII inhibitors using emicizumab prophylaxis
SPK-8011
Participants will receive a single dose of SPK-8011, administered by intravenous (IV) infusion, on Day 1.
Interventions
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SPK-8011
Participants will receive a single dose of SPK-8011, administered by intravenous (IV) infusion, on Day 1.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
* 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.
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.
18 Years
MALE
No
Sponsors
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Spark Therapeutics, Inc.
INDUSTRY
Responsible Party
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Locations
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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
University of Utah
Salt Lake City, Utah, United States
Bloodworks NW
Seattle, Washington, United States
Countries
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Other Identifiers
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2023-504537-46
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
SPK-8011-302
Identifier Type: -
Identifier Source: org_study_id