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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02484092




Registration number
NCT02484092
Ethics application status
Date submitted
18/06/2015
Date registered
29/06/2015

Titles & IDs
Public title
A Gene Therapy Study for Hemophilia B
Scientific title
Gene Therapy, Open-label, Dose-escalation Study of PF-06838435 (SPK-9001) [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B
Secondary ID [1] 0 0
SPK-9001-101
Secondary ID [2] 0 0
C0371005
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia B 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-9001

Experimental: SPK-9001 - Single intravenous (i.v.) infusion of SPK-9001 \[an adeno-associated viral (AAV) vector with human factor IX gene\] Intervention: Gene Therapy / Gene Transfer


Treatment: Other: SPK-9001
A novel, bioengineered adeno-associated viral vector carrying human factor IX variant
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings
Timepoint [1] 0 0
Baseline up to Week 52
Primary outcome [2] 0 0
Number of Participants With Clinically Significant Change From Baseline in Vital Signs
Timepoint [2] 0 0
Baseline up to Week 52
Primary outcome [3] 0 0
Number of Participants With Clinical Laboratory Abnormalities Reported as TEAE
Timepoint [3] 0 0
Baseline up to Week 52
Primary outcome [4] 0 0
Number of Participants With Drug -Related TEAEs and Serious Adverse Events (SAEs)
Timepoint [4] 0 0
Baseline up to Week 52
Primary outcome [5] 0 0
Number of Participants With Positive Immune Reponses Against Adeno-associated Virus Vector (AAV) Capsid
Timepoint [5] 0 0
Baseline up to Week 52
Primary outcome [6] 0 0
Number of Participants Who Reached > 150% Vector-derived FIX:C Activity Level After SPK-9001 Infusion
Timepoint [6] 0 0
Baseline up to Week 52
Primary outcome [7] 0 0
Number of Participants With FIX Inhibitor
Timepoint [7] 0 0
Baseline up to Week 52
Primary outcome [8] 0 0
Incremental Recovery of FIX Product
Timepoint [8] 0 0
Day 0 and Week 52
Secondary outcome [1] 0 0
FIX:C Activity
Timepoint [1] 0 0
Baseline up to Week 52
Secondary outcome [2] 0 0
Change From Baseline in FIX:C Antigen Level at Steady State
Timepoint [2] 0 0
Week 12 up to Week 52

Eligibility
Key inclusion criteria
* Able to provide informed consent and comply with requirements of the study
* Males =18 y.o. with confirmed diagnosis of hemophilia B (=2 IU/dL or =2% endogenous factor IX)
* Received =50 exposure days to factor IX products
* A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions
* No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein
* Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences
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 (* subjects who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible to enroll)
* Neutralizing antibodies reactive with AAV-Spark100 above and/or below a defined titre
* Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational drug within the last 12 weeks
* Unable or unwilling to comply with study assessments

Study design
Purpose of the study
Treatment
Allocation to intervention
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 2
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
18/11/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
8/04/2019
Sample size
Target
Accrual to date
Final
15
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown/Sydney
Recruitment postcode(s) [1] 0 0
2050 - Camperdown/Sydney
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
Mississippi
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
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
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests


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/NCT02484092