What is ASC618 studying for Hemophilia A?

ASC618 is being studied as a gene therapy for Hemophilia A to evaluate its safety and early effectiveness. This trial aims to see if a single infusion can increase factor VIII activity and reduce bleeding episodes, improving management of this bleeding disorder. The study is sponsored by ASC Therapeutics.

Who may be eligible for this Hemophilia A clinical trial?

Adult males aged 18 or older with severe or moderately severe Hemophilia A and low factor VIII levels may be eligible. Participants need to have received factor VIII treatment for at least 150 days and have a body mass index of 30 or less. Please review eligibility criteria for more details.

Is there any cost to participate in this Hemophilia A clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal expenses such as travel to the study site may not be covered and can vary. Participants should consider their own travel arrangements.

Actively Recruiting

Phase 1

Phase 2

Age: 18Years +

MALE

ID04676048

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

Led by ASC Therapeutics · Updated on 2023-02-01

Participants Needed

Research Sites

156 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating a gene therapy called ASC618 for adult men with severe or moderately severe Hemophilia A, a condition where the blood does not clot properly due to low levels of factor VIII. The study aims to assess the safety and early effectiveness of this therapy, which uses a liver-targeted viral vector to deliver a modified human factor VIII gene. This approach addresses challenges with current treatments that require frequent intravenous injections due to their short half-life. Participants will receive a single intravenous infusion of ASC618. This gene therapy uses an AAV vector to deliver a codon-optimized human factor VIII gene designed to increase factor VIII activity in the liver. The study is conducted as a phase 12 trial, focusing on the treatments safety and preliminary effects. During the 12 months following infusion, participants will be monitored for adverse events and serious adverse events. Researchers will measure changes in factor VIII activity levels, annual factor VIII consumption, and annual bleeding rates. The study involves regular evaluations to track these outcomes and ensure participant safety throughout the follow-up period.

CONDITIONS

Brief Title

ASC618 Gene Therapy in Hemophilia A Patients

Who Can Participate

Age: 18Years +

MALE

Eligibility Criteria

You may qualify if you...

Male 18 years of age or older
Diagnosed with severe or moderately severe Hemophilia A with factor VIII activity of 2 IU/dL or less
Received factor VIII prophylactic or on-demand replacement therapy for at least 150 accumulated exposure days
Experienced 12 or more bleeding episodes in the past 12 months if receiving on-demand therapy
Body mass index (BMI) of 30 or less
Agree to use double-barrier contraception until at least 3 consecutive semen samples test negative after ASC618 infusion

You will not qualify if you...

Pre-existing immunity to AAV8 vector as shown by antibody tests
Current inhibitors or history of high titer factor VIII inhibitors
More than Grade 2 liver fibrosis on imaging tests
History of chronic kidney disease
Active infection or immunosuppressive disorder
History of cardiac surgery requiring anticoagulant therapy
Any cardiovascular or genetic risk factors for thromboembolic disorders
Active infection with Hepatitis B, Hepatitis C, HIV-1/2, or syphilis
Previous receipt of any vector or gene transfer agent
Currently on antiviral therapy for hepatitis B or C

Research Team

Clinical Trial Manager, PhD

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