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ASC618 Gene Therapy in Hemophilia A Patients
Sponsor: ASC Therapeutics
Summary
Currently, hemophilia A patients are managed with prophylactic or on-demand replacement therapy with recombinant FVIII or alternative therapeutics. The major challenges of current treatment regimens, such as the short half-life of hemophilia therapeutics with the need for frequent IV injections, encourage the current efforts for gene transfer therapy. This study will evaluate the safety and preliminary efficacy of ASC618, an AAV vector encoding B-domain deleted codon-optimized human factor VIII under a synthetic liver-directed promoter
Official title: Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
Key Details
Gender
MALE
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
12
Start Date
2022-08-03
Completion Date
2026-12
Last Updated
2023-02-01
Healthy Volunteers
No
Conditions
Interventions
ASC618
ASC618 will be given as a single IV infusion
Locations (1)
Arkansas Children's Hospital
Little Rock, Arkansas, United States