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

Hemophilia A Clinical Trial

Official title:

A Multi-Center, Observational Study in Males With Hemophilia A

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03876301
• Other study ID #: SPK-8011-301
• Status: Completed
• Start date: January 21, 2019
• Est. completion date: May 2, 2023

Study information

• Verified date: July 2023
• Source: Spark Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: May 2, 2023
• Est. primary completion date: May 2, 2023
• Gender: Male
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

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

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
Australia	The Alfred Hospital	Melbourne	Victoria
Australia	Fiona Stanley Hospital	Murdoch	Western Austrailia
Canada	McMaster University / Royal Prince Alfred Hospital	Hamilton	Ontario
Canada	Providence Hematology/St. Paul's Hosptial	Vancouver	British Columbia
Thailand	Ramathibodi Hospital, Mahidol University	Bangkok
United States	Emory University	Atlanta	Georgia
United States	Children's Hospital of Michigan	Detroit	Michigan
United States	University of Florida	Gainesville	Florida
United States	Mississippi Center for Advanced Medicine	Madison	Mississippi
United States	University of California San Francisco	San Francisco	California
United States	Bloodworks Northwest	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Spark Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Thailand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of bleeding events, annualized	Annualized bleeding rate (ABR)	12 months
Secondary	Dose and total FVIIII consumption	Total FVIII replacement therapy consumption and the corresponding dose	12 months
Secondary	Annualized number of infusions (AIR)	Number of reported infusions over the study period	12 months