NCT04676048 ASC618 Gene Therapy in Hemophilia A Patients
| NCT ID | NCT04676048 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | ASC Therapeutics |
| Condition | Hemophilia A |
| Study Type | INTERVENTIONAL |
| Enrollment | 12 participants |
| Start Date | 2022-08-03 |
| Primary Completion | 2023-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 12 participants in total. It began in 2022-08-03 with a primary completion date of 2023-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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
Eligibility 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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT04676048 clinical trial?
This trial is open to male participants only, aged 18 Years or older, studying Hemophilia A. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04676048 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT04676048 currently recruiting?
Yes, NCT04676048 is actively recruiting participants. Contact the research team at gil.gonen@asctherapeutics.com for enrollment information.
Where is the NCT04676048 trial being conducted?
This trial is being conducted at Little Rock, United States.
Who is sponsoring the NCT04676048 clinical trial?
NCT04676048 is sponsored by ASC Therapeutics. The trial plans to enroll 12 participants.