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ASC618 Gene Therapy in Hemophilia A Patients
Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
Who can join
Ages 18 and older · Men
Full 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
About the study
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
What is being tested
- ASC618 (biological)
Sponsor: ASC Therapeutics · Participants: 12 · Started: Aug 3, 2022
Contact the study team
- Clinical Trial Manager, PhD · Phone: (408) 495-3891
Official record on ClinicalTrials.gov — NCT04676048
Locations in the U.S.
| Arkansas | Arkansas Children's Hospital, Little Rock |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.