Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults (AFFINE)

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ClinicalTrials.gov Identifier: NCT04370054

Recruitment Status : Active, not recruiting

First Posted : April 30, 2020

Last Update Posted : September 28, 2023

View this study on the modernized ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Pfizer

Tracking Information

First Submitted Date ^ICMJE

April 21, 2020

First Posted Date ^ICMJE

April 30, 2020

Last Update Posted Date

September 28, 2023

Actual Study Start Date ^ICMJE

August 18, 2020

Estimated Primary Completion Date

July 1, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Total annualized bleeding rate (ABR) [ Time Frame: 15 months ]

Original Primary Outcome Measures ^ICMJE

Annualized bleeding rate (ABR) [ Time Frame: 12 months ]

Change History

Current Secondary Outcome Measures ^ICMJE

FVIII activity levels [ Time Frame: 15 months ]
Annualized bleeding rate (ABR) [ Time Frame: 15 months ]
Annualized infusion rate (AIR) of exogenous Factor VIII Activity [ Time Frame: 15 months ]
Annualized FVIII consumption [ Time Frame: 15 months ]
Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location [ Time Frame: 15 months ]
Change in joint health using HJHS (Hemophilia Joint Health Score) [ Time Frame: 12 months ]
HJHS is a validated outcome tool developed for the assessment of joint health in people with hemophilia. The ordinal joint score assesses 9 items in 6 index joints.
Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL) [ Time Frame: 12 months ]
HAL is a disease specific measure of the impact of hemophilia on functional abilities in adults. The instrument consists of 42 items across 7 domains, utilizing a past month recall period. Each item is rated on a scale of 0-6 with higher recoded scores indicating more functional limitations.
Patient Reported Outcome (PRO) instrument - Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) [ Time Frame: 12 months ]
Haem-A-QoL is a disease specific measure of health-related quality of life in patients with hemophilia. Intended for adults, the instrument uses a 4-week recall period to assess health across 10 domains consisting of 46 items. Each item is rated on a scale of 0-6 with lower scores indicating better health-related quality of life.
Incidence and severity of AEs [ Time Frame: 5-year study period ]

Original Secondary Outcome Measures ^ICMJE

Steady state FVIII activity levels [ Time Frame: 12 months ]
Annualized infusion rate (AIR) of exogenous Factor VIII Activity [ Time Frame: 12 months ]
Annualized FVIII consumption [ Time Frame: 12 months ]
Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location [ Time Frame: 12 months ]
Change in joint health using HJHS (Hemophilia Joint Health Score) [ Time Frame: 12 months ]
HJHS is a validated outcome tool developed for the assessment of joint health in people with hemophilia. The ordinal joint score assesses 9 items in 6 index joints.
Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL) [ Time Frame: 12 months ]
HAL is a disease specific measure of the impact of hemophilia on functional abilities in adults. The instrument consists of 42 items across 7 domains, utilizing a past month recall period. Each item is rated on a scale of 0-6 with higher recoded scores indicating more functional limitations.
Patient Reported Outcome (PRO) instrument - Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) [ Time Frame: 12 months ]
Haem-A-QoL is a disease specific measure of health-related quality of life in patients with hemophilia. Intended for adults, the instrument uses a 4-week recall period to assess health across 10 domains consisting of 46 items. Each item is rated on a scale of 0-6 with lower scores indicating better health-related quality of life.
Incidence and severity of AEs [ Time Frame: 5-year study period ]

Current Other Pre-specified Outcome Measures

Not Provided

Original Other Pre-specified Outcome Measures

Not Provided

Descriptive Information

Brief Title ^ICMJE

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

Official Title ^ICMJE

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%)

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.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase ^ICMJE

Phase 3

Study Design ^ICMJE

Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment

Condition ^ICMJE

Hemophilia A

Intervention ^ICMJE

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

Single IV infusion

Other Name: Gene Therapy

Study Arms ^ICMJE

Experimental: PF-07055480 (giroctocogene fitelparvovec)

Single administration of PF-07055480

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

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Active, not recruiting

Actual Enrollment ^ICMJE

Original Estimated Enrollment ^ICMJE

Estimated Study Completion Date ^ICMJE

September 28, 2028

Estimated Primary Completion Date

July 1, 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Sex/Gender ^ICMJE

Sexes Eligible for Study:	Male
Gender Based Eligibility:	Yes

Ages ^ICMJE

18 Years to 64 Years (Adult)

Accepts Healthy Volunteers ^ICMJE

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Listed Location Countries ^ICMJE

Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Korea, Republic of, Saudi Arabia, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT04370054

Other Study ID Numbers ^ICMJE

C3731003
2019-004451-37 ( EudraCT Number )

Has Data Monitoring Committee

Yes

U.S. FDA-regulated Product

Studies a U.S. FDA-regulated Drug Product:	Yes
Studies a U.S. FDA-regulated Device Product:	No

IPD Sharing Statement ^ICMJE

Plan to Share IPD:	Yes
Plan Description:	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.
URL:	https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

Current Responsible Party

Pfizer

Original Responsible Party

Same as current

Current Study Sponsor ^ICMJE

Pfizer

Original Study Sponsor ^ICMJE

Same as current

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Study Director:

Pfizer CT.gov Call Center

Pfizer

PRS Account

Pfizer

Verification Date

September 2023

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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