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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03003533




Registration number
NCT03003533
Ethics application status
Date submitted
16/12/2016
Date registered
28/12/2016
Date last updated
15/05/2020

Titles & IDs
Public title
A Gene Transfer Study for Hemophilia A
Scientific title
Gene Transfer, Dose-Finding Safety, Tolerability, and Efficacy Study of SPK-8011 [a Recombinant Adeno-Associated Viral Vector With Human Factor VIII Gene] in Individuals With Hemophilia A
Secondary ID [1] 0 0
SPK-8011-101
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
Interventional
Description of intervention(s) / exposure
Other interventions - SPK-8011

Experimental: SPK-8011 - All participants who meet the eligibility criteria will receive an outpatient single intravenous (i.v.) administration of SPK-8011.


Other interventions: SPK-8011
A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of study-related adverse events, including clinically significant abnormal laboratory values - adverse events
Timepoint [1] 0 0
52 weeks
Primary outcome [2] 0 0
Changes from baseline in FVIII activity levels after a single outpatient administration of SPK-8011 - changes in FVIII activity levels
Timepoint [2] 0 0
52 weeks
Secondary outcome [1] 0 0
Kinetic assessment of SPK-8011 including shedding of vector DNA in bodily fluids - vector shedding
Timepoint [1] 0 0
52 weeks
Secondary outcome [2] 0 0
Number of participants requiring a course of steroid therapy for the elevations in liver enzymes - number of participants requiring steroids
Timepoint [2] 0 0
52 weeks

Eligibility
Key inclusion criteria
- Males age18 years or older

- Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma
FVIII activity levels = 2% of normal

- Have received >150 exposure days (EDs) to FVIII concentrates or cryoprecipitate

- Have experienced >10 bleeding events over the previous 12 months only if receiving
on-demand therapy and having FVIII baseline level 1-2% of normal

- Have no prior history of allergic reaction to any FVIII product

- Have no measurable inhibitor against Factor VIII as assessed by the central laboratory
and have no prior history of inhibitors to FVIII protein

- Agree to use reliable barrier contraception
Minimum age
18 Years
Maximum age
No limit
Gender
Males
Can healthy volunteers participate?
No
Key exclusion criteria
- Evidence of active hepatitis B or C

- Currently on antiviral therapy for hepatitis B or C

- Have significant underlying liver disease

- Have serological evidence* of HIV-1 or HIV-2 with CD4 counts =200/mm3 (* participants
who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are
eligible to enroll)

- Have detectable antibodies reactive with AAV-Spark200 capsid

- Participated in a gene transfer trial within the last 52 weeks or in a clinical trial
with an investigational product within the last 12 weeks

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1/Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
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)
NSW
Recruitment hospital [1] 0 0
Royal Prince Alfred Hosptial - Camperdown
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
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
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
Mississippi
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
North Carolina
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Virginia
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario

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

Ethics approval
Ethics application status

Summary
Brief summary
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the
safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals
with hemophilia A.
Trial website
https://clinicaltrials.gov/show/NCT03003533
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Lindsey George, MD
Address 0 0
Children's Hospital of Philadelphia
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Director
Address 0 0
Country 0 0
Phone 0 0
1-855-SPARKTX
Fax 0 0
Email 0 0
clinicaltrials@sparktx.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03003533