Gene therapy for patients with Hemophilia A
Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
This study is testing a new gene therapy for adult men with severe hemophilia A to see if it can help increase their factor VIII levels and reduce bleeding episodes, making treatment easier than current options.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 18 Years and up |
| Sex | Male |
| Sponsor | ASC Therapeutics Industry-sponsored |
| Locations | 1 site (Little Rock, Arkansas) |
| Trial ID | NCT04676048 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and preliminary efficacy of ASC618, a gene therapy using an AAV vector that encodes a modified version of human factor VIII, aimed at treating hemophilia A. The therapy is designed to address the limitations of current treatment methods, which require frequent intravenous injections due to the short half-life of existing therapies. Participants will be male adults with severe or moderately severe hemophilia A who have previously received FVIII replacement therapy. The study will assess how well this gene therapy can improve factor VIII levels and reduce bleeding episodes.
Who should consider this trial
Good fit: Ideal candidates for this study are adult males aged 18 and older with severe or moderately severe hemophilia A who have a history of receiving FVIII therapy.
Not a fit: Patients with pre-existing immunity to the AAV8 vector or those with certain medical conditions, such as high titer FVIII inhibitors or significant liver fibrosis, may not benefit from this study.
Why it matters
Potential benefit: If successful, this gene therapy could significantly reduce the need for frequent injections and improve the quality of life for hemophilia A patients.
How similar studies have performed: Other studies exploring gene therapy for hemophilia have shown promising results, indicating that this approach may be viable and beneficial.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male ≥18 years of age * Severe or moderately severe hemophilia A (FVIII activity ≤ 2 IU/dL) as evidenced by * medical history * Received FVIII prophylactic or on-demand replacement therapy for ≥ 150 accumulated * days (exposure days) * ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months * BMI ≤ 30 * Agree to use double-barrier contraceptive until at least 3 consecutive semen samples are negative after ASC-618 infusion Exclusion Criteria: * Pre-existing immunity to AAV8 vector as defined by AAV8 total antibodies and neutralizing antibodies qualified tests. * Current inhibitors, or history of high titer FVIII inhibitors * Presence of \> Grade 2 liver fibrosis on elastography/Fibroscan or comparable imaging methodology * History of chronic renal disease * Active infection or any immunosuppressive disorder * History of cardiac surgery and need anticoagulant therapy * Any cardiovascular / genetic risk factors for thromboembolic disorders * Evidence of active Hepatitis B, Hepatitis C, Human Immunodeficiency Virus (HIV)-1/2 or syphilis infection. * Receipt of any vector or gene transfer agent * Current antiviral therapy for hepatitis B or C
Where this trial is running
Little Rock, Arkansas
- Arkansas Children's Hospital — Little Rock, Arkansas, United States (Recruiting)
Study contacts
- Study coordinator: Clinical Trial Manager, PhD
- Email: gil.gonen@asctherapeutics.com
- Phone: (408) 495-3891
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.