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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03307980




Registration number
NCT03307980
Ethics application status
Date submitted
29/09/2017
Date registered
12/10/2017

Titles & IDs
Public title
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
Scientific title
A FACTOR IX (FIX) GENE TRANSFER, MULTI CENTER EVALUATION OF THE LONG TERM SAFETY AND EFFICACY STUDY OF PF 06838435 AND A DOSE ESCALATION SUBSTUDY IN INDIVIDUALS WITH HEMOPHILIA B
Secondary ID [1] 0 0
SPK-9001-LTFU-101
Secondary ID [2] 0 0
C0371003
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 - PF-06838435 (formerly SPK-9001)

Experimental: PF-06838435 Dose-Escalation - Single intravaneous infusion of PF-06838435. After 2 participants receive initial dose, data will be evaluated and a decision will be made to escalate or reduce the dose being evaluated, increase the number of participants receiving the dose, or stop dosing. Multiple iterations may be undertaken.


Treatment: Other: PF-06838435 (formerly SPK-9001)
Gene Therapy: 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
Incidence of PF-06838435 related adverse events
Timepoint [1] 0 0
Baseline up to Year 6
Secondary outcome [1] 0 0
Incidence of clinically significant changes from baseline
Timepoint [1] 0 0
Baseline up to 52 weeks
Secondary outcome [2] 0 0
Incidence of protocol-defined medically important events
Timepoint [2] 0 0
Baseline up to 52 weeks
Secondary outcome [3] 0 0
Immune response against AAV capsid protein and hFIX transgene
Timepoint [3] 0 0
Baseline up to 52 weeks
Secondary outcome [4] 0 0
Coagulation Clotting Assay for FIX activity levels
Timepoint [4] 0 0
Baseline up to Year 6
Secondary outcome [5] 0 0
Mean and standard deviation of vector-derived FIX Activity levels
Timepoint [5] 0 0
Baseline up to 52 weeks
Secondary outcome [6] 0 0
Mean and standard deviation of FIX Antigen levels
Timepoint [6] 0 0
Baseline up to 52 weeks
Secondary outcome [7] 0 0
Annualized bleeding rate (ABR)
Timepoint [7] 0 0
Baseline up to Year 6
Secondary outcome [8] 0 0
Annualized (factor FIX) infusion rate
Timepoint [8] 0 0
Baseline up to Year 6
Secondary outcome [9] 0 0
Total factor consumption (IU)
Timepoint [9] 0 0
Baseline up to Year 6
Secondary outcome [10] 0 0
Total number of bleeding events
Timepoint [10] 0 0
Baseline up to Year 6
Secondary outcome [11] 0 0
Haem-A-QoL
Timepoint [11] 0 0
Baseline up to Year 6
Secondary outcome [12] 0 0
EQ-5D-5L
Timepoint [12] 0 0
Baseline up to Year 6
Secondary outcome [13] 0 0
Brief Pain Inventory
Timepoint [13] 0 0
Year 2 up to Year 6
Secondary outcome [14] 0 0
McGill Pain Questionnaire
Timepoint [14] 0 0
Baseline up to 52 weeks

Eligibility
Key inclusion criteria
This study is currently only enrolling into the dose-escalation substudy with subsequent long-term follow-up. The Eligibility Criteria for entry into the dose-escalation substudy is presented below:



1. Able to provide informed consent and comply with requirements of the study
2. Males age 18 to 65 years with confirmed diagnosis of hemophilia B (=2 IU/dL or =2% endogenous factor IX)
3. Received =50 exposure days to factor IX products
4. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein
5. Agree to refrain from donating sperm and either abstain from intercourse or use reliable barrier contraception until 3 consecutive semen samples are negative for vector sequences
Minimum age
18 Years
Maximum age
65 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Evidence of active hepatitis B or C
2. Currently on antiviral therapy for hepatitis B or C
3. Have significant underlying liver disease
4. 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)
5. Neutralizing antibody titers to the capsid portion of PF-06838435 above the established threshold
6. Sensitivity to heparin or heparin induced thrombocytopenia; sensitivity to any of the study interventions, or components thereof, or drug or other allergy
7. Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within 3 months of screening visit
8. Any concurrent clinically significant major disease or condition
9. Unable or unwilling to comply with the study procedures

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/06/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
6/06/2029
Actual
Sample size
Target
Accrual to date
Final
21
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - 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
Louisiana
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
Pennsylvania
Country [6] 0 0
Canada
State/province [6] 0 0
Ontario
Country [7] 0 0
Canada
State/province [7] 0 0
Quebec
Country [8] 0 0
Canada
State/province [8] 0 0
Saskatchewan
Country [9] 0 0
Turkey
State/province [9] 0 0
Istanbul
Country [10] 0 0
Turkey
State/province [10] 0 0
Izmir

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?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03307980