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

Primary Purpose

Hemophilia A

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring PF-07055480, SB-525, Hemophilia, Factor VIII, FVIII, Gene Therapy, AAV, AAV6, Annualized bleeding rate, ABR, giroctocogene fitelparvovec, AFFINE

Eligibility Criteria

18 Years - 64 Years (Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • NOW Physical Therapy
  • Clinical and Translational Research Unit (CTRU)
  • Lucile Packard Childrens Hospital
  • Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City
  • UCSF IDS Pharmacy
  • University of California, San Francisco - Clinical Research Center
  • University of California, San Francisco - Moffitt/Long Inpatient Hematology
  • University of California, San Francisco - Outpatient Hematology Clinic
  • Stanford Health Care
  • Washington Institute for Coagulation
  • University of Washington Medical Center - Translational Research Unit (TRU)
  • The Alfred Hospital
  • Centro de Hematologia e Hemoterapia de Campinas - Hemocentro UNICAMP
  • McMaster University Medical Centre - Hamilton Health Sciences
  • Juravinski Hospital - Hamilton Health Sciences
  • Hopital Necker
  • Vivantes Klinikum im Friedrichshain
  • Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik II
  • General Hospital of Athens "Hippokration"
  • General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center
  • Università degli studi di Roma "La Sapienza"- Policlinico Umberto I
  • Azienda Ospedaliero Universitaria Careggi SODc Malattie Emorragiche e della Coagulazione
  • Dip. di Medicina Clinica e Chirurgia, Università degli Studi di Napoli Federico II - UOC di Medicin
  • Nagoya University Hospital - Transfusion Medicine
  • Saitama Medical University Hospital
  • Kyung Hee University Hospital at Gangdong
  • King Faisal Specialist Hospital & Research Center
  • Hospital Universitari Vall d'Hebron
  • H.U. Rio Hortega
  • Skåne University Hospital
  • National Taiwan University Hospital
  • Adana Acibadem Hospital
  • Gaziantep University Sahinbey Research and Training Hospital
  • Ege University Medical Faculty, Pediatric Hematology
  • Ege University Medical Faculty
  • Guy's and St. Thomas' NHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PF-07055480 (giroctocogene fitelparvovec)

Arm Description

Single administration of PF-07055480

Outcomes

Primary Outcome Measures

Total annualized bleeding rate (ABR)

Secondary Outcome Measures

FVIII activity levels
Annualized bleeding rate (ABR)
Annualized infusion rate (AIR) of exogenous Factor VIII Activity
Annualized FVIII consumption
Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location
Change in joint health using HJHS (Hemophilia Joint Health Score)
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)
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)
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

Full Information

First Posted
April 21, 2020
Last Updated
September 26, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT04370054
Brief Title
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
Acronym
AFFINE
Official 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%)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 18, 2020 (Actual)
Primary Completion Date
July 1, 2024 (Anticipated)
Study Completion Date
September 28, 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
Keywords
PF-07055480, SB-525, Hemophilia, Factor VIII, FVIII, Gene Therapy, AAV, AAV6, Annualized bleeding rate, ABR, giroctocogene fitelparvovec, AFFINE

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
68 (Actual)

8. Arms, Groups, and Interventions

Arm Title
PF-07055480 (giroctocogene fitelparvovec)
Arm Type
Experimental
Arm Description
Single administration of PF-07055480
Intervention Type
Biological
Intervention Name(s)
PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Other Intervention Name(s)
Gene Therapy
Intervention Description
Single IV infusion
Primary Outcome Measure Information:
Title
Total annualized bleeding rate (ABR)
Time Frame
15 months
Secondary Outcome Measure Information:
Title
FVIII activity levels
Time Frame
15 months
Title
Annualized bleeding rate (ABR)
Time Frame
15 months
Title
Annualized infusion rate (AIR) of exogenous Factor VIII Activity
Time Frame
15 months
Title
Annualized FVIII consumption
Time Frame
15 months
Title
Annualized bleeding rate (ABR) and total ABR of specific type by cause and by location
Time Frame
15 months
Title
Change in joint health using HJHS (Hemophilia Joint Health Score)
Description
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.
Time Frame
12 months
Title
Patient Reported Outcome (PRO) instrument - Hemophilia Activities List (HAL)
Description
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.
Time Frame
12 months
Title
Patient Reported Outcome (PRO) instrument - Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)
Description
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.
Time Frame
12 months
Title
Incidence and severity of AEs
Time Frame
5-year study period

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
NOW Physical Therapy
City
Mountain View
State/Province
California
ZIP/Postal Code
94040
Country
United States
Facility Name
Clinical and Translational Research Unit (CTRU)
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Lucile Packard Childrens Hospital
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City
City
Redwood City
State/Province
California
ZIP/Postal Code
94063
Country
United States
Facility Name
UCSF IDS Pharmacy
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
University of California, San Francisco - Clinical Research Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
University of California, San Francisco - Moffitt/Long Inpatient Hematology
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
University of California, San Francisco - Outpatient Hematology Clinic
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Stanford Health Care
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Washington Institute for Coagulation
City
Seattle
State/Province
Washington
ZIP/Postal Code
98101
Country
United States
Facility Name
University of Washington Medical Center - Translational Research Unit (TRU)
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
The Alfred Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3004
Country
Australia
Facility Name
Centro de Hematologia e Hemoterapia de Campinas - Hemocentro UNICAMP
City
Campinas
State/Province
SAO Paulo
ZIP/Postal Code
13083-878
Country
Brazil
Facility Name
McMaster University Medical Centre - Hamilton Health Sciences
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Juravinski Hospital - Hamilton Health Sciences
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8V 1C3
Country
Canada
Facility Name
Hopital Necker
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Vivantes Klinikum im Friedrichshain
City
Berlin
ZIP/Postal Code
10249
Country
Germany
Facility Name
Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik II
City
Frankfurt am Main
ZIP/Postal Code
60590
Country
Germany
Facility Name
General Hospital of Athens "Hippokration"
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
Università degli studi di Roma "La Sapienza"- Policlinico Umberto I
City
Roma
State/Province
RM
ZIP/Postal Code
00161
Country
Italy
Facility Name
Azienda Ospedaliero Universitaria Careggi SODc Malattie Emorragiche e della Coagulazione
City
Firenze
ZIP/Postal Code
50134
Country
Italy
Facility Name
Dip. di Medicina Clinica e Chirurgia, Università degli Studi di Napoli Federico II - UOC di Medicin
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Nagoya University Hospital - Transfusion Medicine
City
Nagoya
State/Province
Aichi
ZIP/Postal Code
466-8560
Country
Japan
Facility Name
Saitama Medical University Hospital
City
Iruma-gun
State/Province
Saitama
ZIP/Postal Code
350-0495
Country
Japan
Facility Name
Kyung Hee University Hospital at Gangdong
City
Seoul
ZIP/Postal Code
05278
Country
Korea, Republic of
Facility Name
King Faisal Specialist Hospital & Research Center
City
Riyadh
Country
Saudi Arabia
Facility Name
Hospital Universitari Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
H.U. Rio Hortega
City
Valladolid
ZIP/Postal Code
47012
Country
Spain
Facility Name
Skåne University Hospital
City
Malmö
State/Province
Skane
ZIP/Postal Code
205 02
Country
Sweden
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
10002
Country
Taiwan
Facility Name
Adana Acibadem Hospital
City
Adana
ZIP/Postal Code
01130
Country
Turkey
Facility Name
Gaziantep University Sahinbey Research and Training Hospital
City
Gaziantep
ZIP/Postal Code
27310
Country
Turkey
Facility Name
Ege University Medical Faculty, Pediatric Hematology
City
Izmir
ZIP/Postal Code
35100
Country
Turkey
Facility Name
Ege University Medical Faculty
City
Izmir
ZIP/Postal Code
35100
Country
Turkey
Facility Name
Guy's and St. Thomas' NHS Foundation Trust
City
London
ZIP/Postal Code
SE1 7EH
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=C3731003
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

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

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