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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-06-21

Study Completion Date

2024-07-16

Brief Summary

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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)

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Detailed Description

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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.

Conditions

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Hemophilia A

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Dose selection based on safety and kinetics of circulating FVIII levels observed in previously dosed participants.

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Open Label

Study Groups

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Sequential dose escalation

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

Group Type EXPERIMENTAL

SB-525 (PF-07055480)

Intervention Type BIOLOGICAL

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

Interventions

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SB-525 (PF-07055480)

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

Intervention Type BIOLOGICAL

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status