A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
Last updated on July 2021Recruitment
- Recruitment Status
- Active, not recruiting
- Estimated Enrollment
- 20
Summary
- Conditions
- Hemophilia A
- Type
- Interventional
- Phase
- Phase 2
- Design
- Allocation: N/AIntervention Model: Sequential AssignmentIntervention 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 LabelPrimary Purpose: Treatment
Participation Requirements
- Age
- Between 18 years and 125 years
- Gender
- Only males
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) ve...
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.
Tracking Information
- NCT #
- NCT03061201
- Collaborators
- Not Provided
- Investigators
- Study Director: Pfizer CT.gov Call Center Pfizer
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