Actively Recruiting
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
12
Participants Needed
1
Research Sites
156 weeks
Total Duration
On this page
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 1/2 trial, focusing on the treatment's 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
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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey: What to Expect
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 12 months post-infusion
Participants receive a single intravenous infusion of ASC618 gene therapy.
Approximately 12 follow-up visits over 12 months
Trial Site Locations
Total: 1 location
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
Research Team
C
Clinical Trial Manager, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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