Clinical trial NCT03876301: Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
Official title: A Multi-Center, Observational Study in Males With Hemophilia A
Study type: OBSERVATIONAL
Overall status: COMPLETED
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
Lead sponsor: Spark Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2019-03-15
Enrollment: 25
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of bleeding events, annualized
timeFrame: 12 months
Secondary outcomes:
measure: Dose and total FVIIII consumption
timeFrame: 12 months
measure: Annualized number of infusions (AIR)
timeFrame: 12 months
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Madison
country: United States
facility: Mississippi Center for Advanced Medicine
state: Mississippi
city: Seattle
country: United States
facility: Bloodworks Northwest
state: Washington
city: Melbourne
country: Australia
facility: The Alfred Hospital
state: Victoria
city: Murdoch
country: Australia
facility: Fiona Stanley Hospital
state: Western Austrailia
city: Vancouver
country: Canada
facility: Providence Hematology/St. Paul's Hosptial
state: British Columbia
city: Hamilton
country: Canada
facility: McMaster University / Royal Prince Alfred Hospital
state: Ontario
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital, Mahidol University
Study officials:
References: