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

Spark Therapeutics

Status: Completed

Conditions: Genetic Diseases, X-Linked; Factor VIII Deficiency; Coagulation Protein Disorders; Genetic Diseases, Inborn; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Hemophilia A; Hematologic Diseases; Blood Coagulation Disorder

Treatments: Drug: Standard of Care FVIII Replacement therapy

Study type: Observational

Funder types: Industry

Identifiers

NCT03876301

SPK-8011-301

Details and patient eligibility

About

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.

Enrollment: 25 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

25 participants in 1 patient group

Observational Cohort

Description:

Adult males with clinically severe hemophilia A, who are negative for neutralizing antibody (NAb) to AAV-Spark200

Treatment:

Drug: Standard of Care FVIII Replacement therapy