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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03432520




Registration number
NCT03432520
Ethics application status
Date submitted
24/01/2018
Date registered
14/02/2018
Date last updated
29/07/2024

Titles & IDs
Public title
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
Scientific title
A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A
Secondary ID [1] 0 0
SPK-8011/8016-LTFU
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Treatment: Other - SPK-8011

Treatment: Other: SPK-8011
Observational long-term safety follow-up study of subjects previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of FVIII Inhibitors
Timepoint [1] 0 0
4 years
Primary outcome [2] 0 0
Incidence of all adverse events
Timepoint [2] 0 0
4 years
Primary outcome [3] 0 0
Incidence(s) of new or exacerbation of adverse events of interest
Timepoint [3] 0 0
4 years
Primary outcome [4] 0 0
Annual bleeding rate
Timepoint [4] 0 0
4 years
Primary outcome [5] 0 0
FVIII activity levels
Timepoint [5] 0 0
4 years
Primary outcome [6] 0 0
Total FVIII consumption
Timepoint [6] 0 0
4 years
Primary outcome [7] 0 0
Number of FVIII infusions
Timepoint [7] 0 0
4 years
Secondary outcome [1] 0 0
Joint Health Assessment
Timepoint [1] 0 0
4 years
Secondary outcome [2] 0 0
Target Joint Assessment
Timepoint [2] 0 0
4 years
Secondary outcome [3] 0 0
Haem-A-QoL
Timepoint [3] 0 0
4 years
Secondary outcome [4] 0 0
EQ-5D-5L
Timepoint [4] 0 0
4 years
Secondary outcome [5] 0 0
Activities Assessments
Timepoint [5] 0 0
4 years
Secondary outcome [6] 0 0
Health Economics Assessment
Timepoint [6] 0 0
4 years

Eligibility
Key inclusion criteria
Inclusion Criteria

Participants are eligible to be included in the study only if all of the following criteria apply:

1. Have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored gene therapy study; and
2. Understand the purpose and risks of the study and provide signed and dated informed consent before undergoing any study-specific procedures.
Minimum age
18 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply:

1.Unable or unwilling to comply with the schedule of visits and study assessments as described in this study 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
14/08/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2032
Actual
Sample size
Target
Accrual to date
Final
29
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital Department of Cell & Molecular Therapies - Sydney
Recruitment hospital [2] 0 0
The Alfred Hospital & Monash Medical Centre - Melbourne
Recruitment postcode(s) [1] 0 0
2050 - Sydney
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Massachusetts
Country [2] 0 0
United States of America
State/province [2] 0 0
Mississippi
Country [3] 0 0
United States of America
State/province [3] 0 0
Missouri
Country [4] 0 0
United States of America
State/province [4] 0 0
Oregon
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
United States of America
State/province [6] 0 0
Virginia
Country [7] 0 0
Canada
State/province [7] 0 0
Ontario

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Spark Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.
Trial website
https://clinicaltrials.gov/study/NCT03432520
Trial related presentations / publications
George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205.
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trial Director
Address 0 0
Spark Therapeutics, Inc.
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

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03432520