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A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

Primary Purpose

Hemophilia A

Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
SB-525 (PF-07055480)
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Male ≥18 years of age
  • Severe hemophilia A (past evidence of circulating FVIII activity of < 1% normal)
  • Treated or exposed to FVIII concentrates or cryoprecipitate for at least 150 exposure days
  • ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months
  • Agree to use double barrier contraceptive until at least 3 consecutive semen samples are negative for AAV 2/6 after SB-525 infusion

Exclusion Criteria:

  • Presence of neutralizing antibodies
  • Current inhibitor, or history of FVIII inhibitor (except for transient low titer inhibitor detected in childhood)
  • History of hypersensitivity response to FVIII
  • History of Hepatitis B or HIV-1/2 infection
  • History of Hepatitis C, unless viral assays in two samples, collected at least 6 months apart, are negative
  • Evidence of any bleeding disorder in addition to hemophilia A
  • Markers of hepatic inflammation or overt or occult cirrhosis
  • History of chronic renal disease or creatinine ≥ 1.5 mg/dL
  • Presence of liver mass on magnetic resonance imaging (MRI), or, positive alpha fetoprotein
  • Presence of > grade 2 liver fibrosis on elastography for subjects with history of treated Hepatitis C or suspicion of chronic liver disease

Sites / Locations

  • Arkansas Children's Hospital
  • City of Hope Medical Center
  • Midtown Ambulatory Care Center
  • UC Davis Ambulatory Care Clinic
  • UC Davis Comprehensive Cancer Center
  • UC Davis CTSC Clinical Research Center
  • UC Davis Hemophilia Treatment Center
  • UC Davis Investigational Drug Services Pharmacy
  • UC Davis Medical Center
  • University of California, San Francisco - Investigational Drug Service (IDS) Pharmacy
  • University of California, San Francisco - Outpatient Hematology Clinic
  • University of California, San Francisco -Moffitt Hospital
  • University Of Miami Hospital and Clinics/Sylvester Comprehensive Cancer Center
  • USF Health Morsani Center For Advanced Healthcare
  • Emory University School of Medicine
  • Hemophilia Center of Western PA
  • UPMC Montefiore Clinical and Translational Research Center
  • UPMC, Investigational Drug Service
  • Vanderbilt Hemostasis-Thrombosis Clinic
  • Washington Institute for Coagulation

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Sequential dose escalation

Arm Description

SB-525 (PF-07055480) is administered as a single infusion

Outcomes

Primary Outcome Measures

Incidence of adverse events and serious adverse events
Changes in circulating FVIII activity

Secondary Outcome Measures

Full Information

First Posted
February 15, 2017
Last Updated
September 19, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT03061201
Brief Title
A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
Official Title
A PHASE 1/2, OPEN-LABEL, ADAPTIVE, DOSE-RANGING STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF SB-525 (PF-07055480) (RECOMBINANT AAV2/6 HUMAN FACTOR 8 GENE THERAPY) IN ADULT SUBJECTS WITH SEVERE HEMOPHILIA A
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 21, 2017 (Actual)
Primary Completion Date
June 18, 2024 (Anticipated)
Study Completion Date
June 18, 2024 (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
The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity after dosing with SB-525 (PF-07055480)
Detailed Description
The proposed clinical study uses a recombinant adeno-associated virus 2/6 (AAV2/6) vector encoding the cDNA for the B-domain deleted human F8 (hF8). The secreted FVIII has the same amino acid sequence as approved recombinant anti hemophilic factors (Refacto® and Xyntha®). The SB-525 (PF-07055480) vector encodes a liver-specific promotor module and AAV2/6 exhibits liver tropism, thus providing the potential for long-term hepatic production of FVIII in hemophilia A subjects. The constant production of FVIII after a single SB-525 (PF-07055480) administration may provide potential benefit in durable protection against bleeding and the complications thereof without lifelong repetitive IV factor replacement administration.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Dose selection based on safety and kinetics of circulating FVIII levels observed in previously dosed participants.
Masking
None (Open Label)
Masking Description
Open Label
Allocation
N/A
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Sequential dose escalation
Arm Type
Experimental
Arm Description
SB-525 (PF-07055480) is administered as a single infusion
Intervention Type
Biological
Intervention Name(s)
SB-525 (PF-07055480)
Intervention Description
Single dose of investigational product SB-525 (PF-07055480)
Primary Outcome Measure Information:
Title
Incidence of adverse events and serious adverse events
Time Frame
Up to 5 years after SB-525 (PF-07055480) infusion
Title
Changes in circulating FVIII activity
Time Frame
Up to 5 years after SB-525 (PF-07055480) infusion

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male ≥18 years of age Severe hemophilia A (past evidence of circulating FVIII activity of < 1% normal) Treated or exposed to FVIII concentrates or cryoprecipitate for at least 150 exposure days ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months Agree to use double barrier contraceptive until at least 3 consecutive semen samples are negative for AAV 2/6 after SB-525 infusion Exclusion Criteria: Presence of neutralizing antibodies Current inhibitor, or history of FVIII inhibitor (except for transient low titer inhibitor detected in childhood) History of hypersensitivity response to FVIII History of Hepatitis B or HIV-1/2 infection History of Hepatitis C, unless viral assays in two samples, collected at least 6 months apart, are negative Evidence of any bleeding disorder in addition to hemophilia A Markers of hepatic inflammation or overt or occult cirrhosis History of chronic renal disease or creatinine ≥ 1.5 mg/dL Presence of liver mass on magnetic resonance imaging (MRI), or, positive alpha fetoprotein Presence of > grade 2 liver fibrosis on elastography for subjects with history of treated Hepatitis C or suspicion of chronic liver disease
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
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
City of Hope Medical Center
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Facility Name
Midtown Ambulatory Care Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
UC Davis Ambulatory Care Clinic
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
UC Davis Comprehensive Cancer Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
UC Davis CTSC Clinical Research Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
UC Davis Hemophilia Treatment Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
UC Davis Investigational Drug Services Pharmacy
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
UC Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of California, San Francisco - Investigational Drug Service (IDS) Pharmacy
City
San Francisco
State/Province
California
ZIP/Postal Code
94143-0622
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
University of California, San Francisco -Moffitt Hospital
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
University Of Miami Hospital and Clinics/Sylvester Comprehensive Cancer Center
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
USF Health Morsani Center For Advanced Healthcare
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Emory University School of Medicine
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Hemophilia Center of Western PA
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
UPMC Montefiore Clinical and Translational Research Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
UPMC, Investigational Drug Service
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Vanderbilt Hemostasis-Thrombosis Clinic
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Washington Institute for Coagulation
City
Seattle
State/Province
Washington
ZIP/Postal Code
98101
Country
United States

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=SB-525-1603
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

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