Gene therapy for patients with Hemophilia A

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• SR604: a new treatment tested in adults with Hemophilia A/B or congenital Factor VII deficiency

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• Bone health in severe hemophilia A: emicizumab vs efanesoctocog alfa

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• NXT007 versus emicizumab to prevent bleeds in people with Hemophilia A

  This will test whether NXT007 prevents bleeding better than emicizumab in people aged 12 and older with Hemophilia A, including those with or without factor VIII inhibitors.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.