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

Sponsor

Spark Therapeutics (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT03876301

Collaborator

(none)

Enrollment

Locations

58.3

Anticipated Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
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	Drug: Standard of Care FVIII Replacement therapy

Study Design

Study Type:

Observational

Anticipated Enrollment :

55 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Actual Study Start Date :

Jan 21, 2019

Anticipated Primary Completion Date :

Jun 1, 2023

Anticipated Study Completion Date :

Dec 1, 2023

Arms and Interventions

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

Arm

Intervention/Treatment

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

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

Outcome Measures

Primary Outcome Measures

Number of bleeding events, annualized [12 months]
Annualized bleeding rate (ABR)

Secondary Outcome Measures

Dose and total FVIIII consumption [12 months]
Total FVIII replacement therapy consumption and the corresponding dose
Annualized number of infusions (AIR) [12 months]
Number of reported infusions over the study period

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

Male

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California San Francisco	San Francisco	California	United States	94117
2	University of Florida	Gainesville	Florida	United States	32610
3	Emory University	Atlanta	Georgia	United States	30322
4	Children's Hospital of Michigan	Detroit	Michigan	United States	48201
5	Mississippi Center for Advanced Medicine	Madison	Mississippi	United States	39110
6	Bloodworks Northwest	Seattle	Washington	United States	98104
7	The Alfred Hospital	Melbourne	Victoria	Australia	3004
8	Fiona Stanley Hospital	Murdoch	Western Austrailia	Australia	6150
9	Providence Hematology/St. Paul's Hosptial	Vancouver	British Columbia	Canada	V621Y6
10	McMaster University / Royal Prince Alfred Hospital	Hamilton	Ontario	Canada	L8N3Z5
11	Ramathibodi Hospital, Mahidol University	Bangkok		Thailand	10400