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

Study Results

Results pending

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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Recruitment Status

COMPLETED

Total Enrollment

25 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-01-21

Study Completion Date

2023-05-02

Brief Summary

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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.

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Detailed Description

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Conditions

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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

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Observational Cohort

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

Standard of Care FVIII Replacement therapy

Intervention Type DRUG

There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.

Interventions

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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.

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No