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

Completed

• Conditions: Blood Coagulation Disorders, Inherited; Hemorrhagic Disorders; Blood Coagulation Disorder; Coagulation Protein Disorders; Hemophilia A; Genetic Diseases, Inborn; Factor VIII Deficiency; Hematologic Diseases; Genetic Diseases, X-Linked
• Interventions: Drug: Standard of Care FVIII Replacement therapy

• Registration Number: NCT03876301
• Lead Sponsor: Spark Therapeutics, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-01-21
• Study First Submitted: 2019-03-11
• Study First Submitted QC: 2019-03-13
• Study First Posted: 2019-03-15
• Primary Completion: 2023-05-02
• Study Completion: 2023-05-02
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-06
• Last Update Posted: 2023-07-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 25

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Observational Cohort	Standard of Care FVIII Replacement therapy	Adult males with clinically severe hemophilia A, who are negative for neutralizing antibody (NAb) to AAV-Spark200

Primary Outcome Measures

Name	Time	Method
Number of bleeding events, annualized	12 months	Annualized bleeding rate (ABR)

Secondary Outcome Measures

Name	Time	Method
Dose and total FVIIII consumption	12 months	Total FVIII replacement therapy consumption and the corresponding dose
Annualized number of infusions (AIR)	12 months	Number of reported infusions over the study period

Trial Locations

Locations (11)

University of Florida; 🇺🇸 Gainesville, Florida, United States

The Alfred Hospital; 🇦🇺 Melbourne, Victoria, Australia

Providence Hematology/St. Paul's Hosptial; 🇨🇦 Vancouver, British Columbia, Canada

Mississippi Center for Advanced Medicine; 🇺🇸 Madison, Mississippi, United States

University of California San Francisco; 🇺🇸 San Francisco, California, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States

Children's Hospital of Michigan; 🇺🇸 Detroit, Michigan, United States

Bloodworks Northwest; 🇺🇸 Seattle, Washington, United States

Fiona Stanley Hospital; 🇦🇺 Murdoch, Western Austrailia, Australia

Ramathibodi Hospital, Mahidol University; 🇹🇭 Bangkok, Thailand

McMaster University / Royal Prince Alfred Hospital; 🇨🇦 Hamilton, Ontario, Canada