Register a trial

The ANZCTR website is back online for trial registration and updates. We apologise for any inconvenience caused while the site was inactive.

Please note that the ANZCTR will be unattended from Friday 18th April until Tuesday 22nd April due to the Easter long weekend. Submissions and updates will not be processed during that time.



Reset your password and enable multi-factor authentication (MFA)


For ANZCTR account holders: to help ensure the cyber safety of your account, you’ll need to reset your password and set-up multi-factor authentication (MFA) as per the instructions below.


  1. Go to the Login page, click ‘reset password’ and follow the instructions.
  2. Check your email for the link to set a new password.
  3. Create a new password that meets requirements. New passwords must include at least one lowercase letter, one uppercase letter, one number and one special character (e.g. !#$%&@).
  4. Return to the Login page and enter your new password. A verification code will be sent to your email.
  5. Check your email for the code and enter it on the Login page. If the code is entered incorrectly, you can re-enter the correct one or request a new one.

Learn more about MFA and its importance on the Australian Signals Directorate website.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03003533
Ethics application status
Date submitted
16/12/2016
Date registered
28/12/2016

Titles & IDs
Public title
A Gene Transfer Study for Hemophilia A
Scientific title
Gene-transfer, Open-label, Dose-escalation Study of SPK-8011 [Adeno-associated Viral Vector With B-domain Deleted 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
Treatment: Other - SPK-8011

Experimental: SPK-8011 5x10^11 vg/kg - Participants received a single intravenous (IV) infusion of SPK-8011 5x10\^11 vector genomes per kilogram (vg/kg) body weight.

Experimental: SPK-8011 1x10^12 vg/kg - Participants received a single IV infusion of SPK-8011 1x10\^12 vg/kg.

Experimental: SPK-8011 2x10^12 vg/kg - Participants received a single IV infusion of SPK-8011 2x10\^12 vg/kg.

Experimental: SPK-8011 1.5x10^12 vg/kg - Participants received a single IV infusion of SPK-8011 1.5x10\^12 vg/kg.


Treatment: Other: SPK-8011
A novel, bio-engineered, recombinant adeno-associated viral vector carrying human factor VIII gene
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
From date of first dose to Week 52/End of Study (EOS) Visit
Primary outcome [2] 0 0
Number of Participants Who Received Corticosteroids for Presumed Immune Response
Timepoint [2] 0 0
Up to Week 52/EOS Visit
Primary outcome [3] 0 0
Peak Factor VIII (FVIII) Activity Levels Assessed by One-Stage Coagulation Assay (OSA)
Timepoint [3] 0 0
Up to Week 52/EOS visit
Primary outcome [4] 0 0
Nominal FVIII Level by OSA at Week 52/EOS
Timepoint [4] 0 0
Up to Week 52/EOS Visit
Primary outcome [5] 0 0
Spontaneous Bleeds Annualized Bleeding Rate
Timepoint [5] 0 0
Week 5 up to Week 52/EOS Visit
Primary outcome [6] 0 0
Total Annualized FVIII Infusion Rate
Timepoint [6] 0 0
Week 5 up to Week 52/EOS Visit
Secondary outcome [1] 0 0
Time to Achieve Peak FVIII Activity Level
Timepoint [1] 0 0
Up to Week 52/EOS Visit
Secondary outcome [2] 0 0
Number of Participants With Vector-shedding Confirmed Below Quantifiable Limits (BQL) of SPK-8011-101 in Bodily Fluids
Timepoint [2] 0 0
Up to Week 52/EOS Visit
Secondary outcome [3] 0 0
Incidence of Immune Response to the BDD-hFVIII Transgene
Timepoint [3] 0 0
Up to Week 52/EOS Visit

Eligibility
Key inclusion criteria
* Males age 18 years or older
* Confirmed diagnosis of hemophilia A as evidenced by their medical history with baseline FVIII activity levels <=2%
* Have received >150 exposure days (EDs) to FVIII concentrates or cryoprecipitate
* Have no prior history of allergic reaction to any FVIII product
* Have no measurable inhibitor against FVIII as assessed by the central laboratory and have no prior history of inhibitors to FVIII protein and no clinical signs or symptoms of decreased response to FVIII administration
* Agree to use reliable barrier contraception
Minimum age
18 Years
Maximum age
No limit
Sex
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 and who are on an antiretroviral drug regimen (* 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
Non-randomised trial
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
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/01/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/12/2023
Sample size
Target
Accrual to date
Final
25
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Royal Prince Alfred Hosptial - Camperdown
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
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
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Mississippi
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Oregon
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Virginia
Country [9] 0 0
Canada
State/province [9] 0 0
Ontario
Country [10] 0 0
Israel
State/province [10] 0 0
Tel Hashomer
Country [11] 0 0
Thailand
State/province [11] 0 0
Bangkok

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
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

Data sharing statement


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03003533