NCT04676048

ASC618 Gene Therapy in Hemophilia A Patients

Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: ASC Therapeutics · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Conditions

• Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	ASC618	ASC618 will be given as a single IV infusion

Timeline

• Start date: 2022-08-03
• Primary completion: 2023-12-01
• Completion: 2026-12-01
• First posted: 2020-12-19
• Last updated: 2023-02-01

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study