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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04370054




Registration number
NCT04370054
Ethics application status
Date submitted
21/04/2020
Date registered
30/04/2020

Titles & IDs
Public title
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
Scientific title
Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants With Moderately Severe to Severe Hemophilia A(FVIII:C=1%)
Secondary ID [1] 0 0
2019-004451-37
Secondary ID [2] 0 0
C3731003
Universal Trial Number (UTN)
Trial acronym
AFFINE
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 - PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy

Experimental: PF-07055480 (giroctocogene fitelparvovec) - Single administration of PF-07055480


Treatment: Other: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Single IV infusion
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Total annualized bleeding rate (ABR)
Timepoint [1] 0 0
15 months
Secondary outcome [1] 0 0
FVIII activity levels
Timepoint [1] 0 0
15 months
Secondary outcome [2] 0 0
Annualized bleeding rate (ABR)
Timepoint [2] 0 0
15 months
Secondary outcome [3] 0 0
Annualized infusion rate (AIR) of exogenous Factor VIII Activity
Timepoint [3] 0 0
15 months
Secondary outcome [4] 0 0
Annualized FVIII consumption
Timepoint [4] 0 0
15 months
Secondary outcome [5] 0 0
Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location
Timepoint [5] 0 0
15 months
Secondary outcome [6] 0 0
Change in joint health using HJHS (Hemophilia Joint Health Score)
Timepoint [6] 0 0
Yearly up to 5 years
Secondary outcome [7] 0 0
Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL)
Timepoint [7] 0 0
Yearly up to 5 years
Secondary outcome [8] 0 0
Patient Reported Outcome (PRO) instrument - Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)
Timepoint [8] 0 0
Yearly up to 5 years
Secondary outcome [9] 0 0
Incidence and severity of AEs
Timepoint [9] 0 0
5-year study period

Eligibility
Key inclusion criteria
Main inclusion Criteria

* Males who have been followed on routine Factor VIII prophylaxis therapy during the lead-in study (C0371004) and have > = 150 documented exposure days to a Factor VIII protein product
* Moderately severe to severe hemophilia A (Factor VIII activity < =1%)
* Suspension of FVIII prophylaxis therapy post study drug infusion

Main exclusion Criteria

* Anti-AAV6 neutralizing antibodies
* History of inhibitor to Factor VIII
* Laboratory values at screening visit that are abnormal or outside acceptable study limits
* Significant and/or unstable liver disease, biliary disease, significant liver fibrosis
* Conditions associated with increased thromboembolic risk such as inherited or acquired thrombophilia, or a history of thrombotic events
* Planned surgical procedure requiring Factor VIII surgical prophylactic factor treatment 12 months from screening visit
* Active hepatitis B or C
* Serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count =200 mm3 and/or viral load >20 copies/mL
Minimum age
18 Years
Maximum age
64 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria

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
Single group
Other design features
Phase
Phase 3
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
18/08/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
25/10/2028
Actual
Sample size
Target
Accrual to date
Final
76
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 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
Washington
Country [3] 0 0
Brazil
State/province [3] 0 0
SAO Paulo
Country [4] 0 0
Canada
State/province [4] 0 0
Ontario
Country [5] 0 0
France
State/province [5] 0 0
Paris
Country [6] 0 0
Germany
State/province [6] 0 0
Berlin
Country [7] 0 0
Germany
State/province [7] 0 0
Frankfurt am Main
Country [8] 0 0
Greece
State/province [8] 0 0
Athens
Country [9] 0 0
Italy
State/province [9] 0 0
RM
Country [10] 0 0
Italy
State/province [10] 0 0
Firenze
Country [11] 0 0
Italy
State/province [11] 0 0
Napoli
Country [12] 0 0
Japan
State/province [12] 0 0
Aichi
Country [13] 0 0
Japan
State/province [13] 0 0
Saitama
Country [14] 0 0
Korea, Republic of
State/province [14] 0 0
Seoul
Country [15] 0 0
Saudi Arabia
State/province [15] 0 0
Riyadh
Country [16] 0 0
Spain
State/province [16] 0 0
Barcelona
Country [17] 0 0
Spain
State/province [17] 0 0
Valladolid
Country [18] 0 0
Sweden
State/province [18] 0 0
Skane
Country [19] 0 0
Taiwan
State/province [19] 0 0
Taipei
Country [20] 0 0
Turkey
State/province [20] 0 0
Adana
Country [21] 0 0
Turkey
State/province [21] 0 0
Gaziantep
Country [22] 0 0
Turkey
State/province [22] 0 0
Izmir
Country [23] 0 0
United Kingdom
State/province [23] 0 0
London

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/NCT04370054