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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04370054




Registration number
NCT04370054
Ethics application status
Date submitted
21/04/2020
Date registered
30/04/2020
Date last updated
4/03/2024

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
Other interventions - PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy

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


Other interventions: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Single IV infusion
Intervention code [1] 0 0
Other interventions
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
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 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
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single
IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor
VIII Gene Therapy) in adult male participants with moderately severe or severe hemophilia A
(FVIII:C=1%) for the study duration of 5 years. The study will enroll eligible participants
who have been followed on routine prophylaxis with FVIII products in the Lead-In study
C0371004.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04370054
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04370054