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

Completed

Conditions studied: 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

In brief

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.

Key facts

Study ID

NCT03876301

Run by

Spark Therapeutics, Inc.

People needed

25

Starts

2019-01-21

Expected to finish

2023-05-02

Last updated by the study team

2023-07-10

Who can join

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

You may qualify if…

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

You may not qualify if…

• Documented active hepatitis B or C within the past 12 months of Screening
• Currently on antiviral therapy to treat hepatitis B or C;
• Documented significant liver disease within the past 6 months of Screening
• Have serological evidence of HIV-1 or HIV-2
• Anti-AAV-Spark 200 neutralizing titers ≥1:1
• Previously received SPK-8011;
• Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks;
• Planned surgical procedure in the next 12 months requiring FVIII prophylactic treatment.
• 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.
• Unable or unwilling to comply with the schedule of visits and/or study assessments described in the protocol.

Where it is running

• University of California San Francisco — San Francisco, California, United States
• University of Florida — Gainesville, Florida, United States
• Emory University — Atlanta, Georgia, United States
• Children's Hospital of Michigan — Detroit, Michigan, United States
• Mississippi Center for Advanced Medicine — Madison, Mississippi, United States
• Bloodworks Northwest — Seattle, Washington, United States
• The Alfred Hospital — Melbourne, Victoria, Australia
• Fiona Stanley Hospital — Murdoch, Western Austrailia, Australia
• Providence Hematology/St. Paul's Hosptial — Vancouver, British Columbia, Canada
• McMaster University / Royal Prince Alfred Hospital — Hamilton, Ontario, Canada
• Ramathibodi Hospital, Mahidol University — Bangkok, Thailand

Full record on ClinicalTrials.gov

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