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

NCT03876301 | Completed FDA Drug

• 1 other identifier: SPK-8011-301
• Study Type: observational
• Target: 25
• Locations: 4 countries
• Sites: 11

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.

Conditions

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

Keywords

Factor VIII; Factor VIII Protein; Recombinant

Outcome Measures

Primary Outcomes (1)

• Number of bleeding events, annualized

  Annualized bleeding rate (ABR)

  12 months

Secondary Outcomes (2)

• Dose and total FVIIII consumption

  12 months

• Annualized number of infusions (AIR)

  12 months

Study Arms (1)

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

Interventions

Standard of Care FVIII Replacement therapy DRUG

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

Observational Cohort

Eligibility Criteria

• Age: 18 Years+
• Gender Eligibility Details: Genetically male
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

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.

Sponsors & Collaborators

• Spark Therapeutics, Inc.: lead

Study Sites (11)

University of California San Francisco

San Francisco, California, 94117, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Mississippi Center for Advanced Medicine

Madison, Mississippi, 39110, United States

Location

Bloodworks Northwest

Seattle, Washington, 98104, United States

Location

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

Fiona Stanley Hospital

Murdoch, Western Austrailia, 6150, Australia

Location

Providence Hematology/St. Paul's Hosptial

Vancouver, British Columbia, V621Y6, Canada

Location

McMaster University / Royal Prince Alfred Hospital

Hamilton, Ontario, L8N3Z5, Canada

Location

Ramathibodi Hospital, Mahidol University

Bangkok, 10400, Thailand

Location

MeSH Terms

Conditions

Blood Coagulation Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Hematologic Diseases; Hemorrhagic Disorders

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 11, 2019
• First Posted: March 15, 2019
• Study Start: January 21, 2019
• Primary Completion: May 2, 2023
• Study Completion: May 2, 2023
• Last Updated: July 10, 2023

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

TH (1); AU (2); CA (2); US (6)