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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 | Clinical Trials | Clareo Health

COMPLETEDPHASE2

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

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

NCT03061201•Pfizer

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - No limit years

Sex

MALE

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

Locations (22)

Arkansas Children's Hospital

Little Rock, Arkansas, United States

City of Hope Medical Center

Duarte, California, United States

Midtown Ambulatory Care Center