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RECRUITING
NCT04676048
PHASE1/PHASE2

ASC618 Gene Therapy in Hemophilia A Patients

Sponsor: ASC Therapeutics

View on ClinicalTrials.gov

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

BIOLOGICAL

ASC618

ASC618 will be given as a single IV infusion

Locations (1)

Arkansas Children's Hospital

Little Rock, Arkansas, United States