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NCT03061201 Clinical Trial

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

Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT03061201
Other study ID # SB-525-1603
Secondary ID C3731001
Status Active, not recruiting
Phase Phase 2
First received
Last updated
Start date June 21, 2017
Est. completion date June 25, 2024

Study information
Verified date June 2024
Source Pfizer
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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)

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.

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 11
Est. completion date June 25, 2024
Est. primary completion date June 25, 2024
Accepts healthy volunteers No
Gender Male
Age group 18 Years and older
Eligibility 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

Study Design

Related Conditions & MeSH terms

Intervention

Biological:
• SB-525 (PF-07055480)
Single dose of investigational product SB-525 (PF-07055480)

Locations
Country Name City State
United States Emory University School of Medicine Atlanta Georgia
United States City of Hope Medical Center Duarte California
United States Arkansas Children's Hospital Little Rock Arkansas
United States University Of Miami Hospital and Clinics/Sylvester Comprehensive Cancer Center Miami Florida
United States Vanderbilt Hemostasis-Thrombosis Clinic Nashville Tennessee
United States Hemophilia Center of Western PA Pittsburgh Pennsylvania
United States UPMC Montefiore Clinical and Translational Research Center Pittsburgh Pennsylvania
United States UPMC, Investigational Drug Service Pittsburgh Pennsylvania
United States Midtown Ambulatory Care Center Sacramento California
United States UC Davis Ambulatory Care Clinic Sacramento California
United States UC Davis Comprehensive Cancer Center Sacramento California
United States UC Davis CTSC Clinical Research Center Sacramento California
United States UC Davis Hemophilia Treatment Center Sacramento California
United States UC Davis Investigational Drug Services Pharmacy Sacramento California
United States UC Davis Medical Center Sacramento California
United States University of California, San Francisco - Investigational Drug Service (IDS) Pharmacy San Francisco California
United States University of California, San Francisco - Outpatient Hematology Clinic San Francisco California
United States University of California, San Francisco -Moffitt Hospital San Francisco California
United States Washington Institute for Coagulation Seattle Washington
United States USF Health Morsani Center For Advanced Healthcare Tampa Florida

Sponsors (1)
Lead Sponsor Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary Incidence of adverse events and serious adverse events Up to 5 years after SB-525 (PF-07055480) infusion
Primary Changes in circulating FVIII activity Up to 5 years after SB-525 (PF-07055480) infusion
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