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Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03876301
Recruitment Status : Active, not recruiting
First Posted : March 15, 2019
Last Update Posted : February 22, 2023
Information provided by (Responsible Party):
Spark Therapeutics

Brief Summary:
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.

Condition or disease Intervention/treatment
Blood Coagulation Disorder Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Hemophilia A Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemorrhagic Disorders Factor VIII Deficiency Drug: Standard of Care FVIII Replacement therapy

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Study Type : Observational
Estimated Enrollment : 55 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Multi-Center, Observational Study in Males With Hemophilia A
Actual Study Start Date : January 21, 2019
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : December 2023

Resource links provided by the National Library of Medicine

Group/Cohort Intervention/treatment
Observational Cohort
Adult males with clinically severe hemophilia A, who are negative for neutralizing antibody (NAb) to AAV-Spark200
Drug: Standard of Care FVIII Replacement therapy
There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.

Primary Outcome Measures :
  1. Number of bleeding events, annualized [ Time Frame: 12 months ]
    Annualized bleeding rate (ABR)

Secondary Outcome Measures :
  1. Dose and total FVIIII consumption [ Time Frame: 12 months ]
    Total FVIII replacement therapy consumption and the corresponding dose

  2. Annualized number of infusions (AIR) [ Time Frame: 12 months ]
    Number of reported infusions over the study period

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Genetically male
Sampling Method:   Non-Probability Sample
Study Population
Adult males with clinically severe hemophilia A (i.e., ≤2% IU/dL FVIII activity level), who meet eligibility criteria for neutralizing antibody (NAb) to AAV-Spark200.

Inclusion Criteria:

  1. Able and willing to provide written informed consent.
  2. Males ≥18 years of age.
  3. Clinically severe hemophilia A
  4. Previous exposure to FVIII therapy
  5. No prior history of hypersensitivity or anaphylaxis associated with an FVIII or intravenous immunoglobulin administration.
  6. No measurable inhibitor against FVIII
  7. Willing to participate and receive treatment in a future Spark hemophilia A gene therapy study.

Exclusion Criteria:

  1. Documented active hepatitis B or C within the past 12 months of Screening
  2. Currently on antiviral therapy to treat hepatitis B or C;
  3. Documented significant liver disease within the past 6 months of Screening
  4. Have serological evidence of HIV-1 or HIV-2
  5. Anti-AAV-Spark 200 neutralizing titers ≥1:1
  6. Previously received SPK-8011;
  7. Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks;
  8. Planned surgical procedure in the next 12 months requiring FVIII prophylactic treatment.
  9. Any history of chronic infection or other chronic disease, concurrent clinically significant major disease (such as liver abnormalities or type I diabetes) including active malignancy, except for non-melanoma skin cancer, any other condition or any other unspecified reasons that, in opinion of the Investigator or Sponsor, makes the participant unsuitable for participation and dosing in a future clinical study for Spark's hemophilia A gene therapy.
  10. Unable or unwilling to comply with the schedule of visits and/or study assessments described in the protocol.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03876301

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United States, California
University of California San Francisco
San Francisco, California, United States, 94117
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
United States, Michigan
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
United States, Mississippi
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States, 39110
United States, Washington
Bloodworks Northwest
Seattle, Washington, United States, 98104
Australia, Victoria
The Alfred Hospital
Melbourne, Victoria, Australia, 3004
Australia, Western Austrailia
Fiona Stanley Hospital
Murdoch, Western Austrailia, Australia, 6150
Canada, British Columbia
Providence Hematology/St. Paul's Hosptial
Vancouver, British Columbia, Canada, V621Y6
Canada, Ontario
McMaster University / Royal Prince Alfred Hospital
Hamilton, Ontario, Canada, L8N3Z5
Ramathibodi Hospital, Mahidol University
Bangkok, Thailand, 10400
Sponsors and Collaborators
Spark Therapeutics
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Principal Investigator: Spencer K Sullivan, MD Mississippi Center for Advanced Medicine
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Responsible Party: Spark Therapeutics
ClinicalTrials.gov Identifier: NCT03876301    
Other Study ID Numbers: SPK-8011-301
First Posted: March 15, 2019    Key Record Dates
Last Update Posted: February 22, 2023
Last Verified: February 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Spark Therapeutics:
Factor VIII
Factor VIII Protein
Additional relevant MeSH terms:
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Hemostatic Disorders
Hemophilia A
Hematologic Diseases
Blood Coagulation Disorders
Hemorrhagic Disorders
Coagulation Protein Disorders
Blood Coagulation Disorders, Inherited
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Pathologic Processes
Vascular Diseases
Cardiovascular Diseases