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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03876301




Registration number
NCT03876301
Ethics application status
Date submitted
11/03/2019
Date registered
15/03/2019
Date last updated
15/05/2020

Titles & IDs
Public title
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
Scientific title
A Multi-Center, Observational Study in Males With Hemophilia A
Secondary ID [1] 0 0
SPK-8011-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Blood Coagulation Disorder 0 0
Blood Coagulation Disorders, Inherited 0 0
Coagulation Protein Disorders 0 0
Hemophilia A 0 0
Genetic Diseases, Inborn 0 0
Genetic Diseases, X-Linked 0 0
Hematologic Diseases 0 0
Hemorrhagic Disorders 0 0
Factor VIII Deficiency 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Clotting disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Treatment: Drugs - Standard of Care FVIII Replacement therapy

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


Treatment: Drugs: 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 code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of bleeding events, annualized - Annualized bleeding rate (ABR)
Timepoint [1] 0 0
12 months
Secondary outcome [1] 0 0
Dose and total FVIIII consumption - Total FVIII replacement therapy consumption and the corresponding dose
Timepoint [1] 0 0
12 months
Secondary outcome [2] 0 0
Annualized number of infusions (AIR) - Number of reported infusions over the study period
Timepoint [2] 0 0
12 months

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Gender
Males
Can healthy volunteers participate?
Key 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.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC,Western Austraili
Recruitment hospital [1] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [2] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
3004 - Melbourne
Recruitment postcode(s) [2] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Michigan
Country [5] 0 0
United States of America
State/province [5] 0 0
Mississippi
Country [6] 0 0
United States of America
State/province [6] 0 0
Washington
Country [7] 0 0
Canada
State/province [7] 0 0
British Columbia
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario
Country [9] 0 0
Thailand
State/province [9] 0 0
Bangkok

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Spark Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT03876301
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Spencer K Sullivan, MD
Address 0 0
Mississippi Center for Advanced Medicine
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications