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RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

ASC618 Gene Therapy in Hemophilia A Patients

Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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

Who May Be Eligible (Plain English)

Who May Qualify: - 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 Who Should NOT Join This Trial: - 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 weakened immune system Virus (HIV)-1/2 or syphilis infection. - Receipt of any vector or gene transfer agent - Current antiviral therapy for hepatitis B or C Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

BIOLOGICAL

ASC618

ASC618 will be given as a single IV infusion

Locations (1)

Arkansas Children's Hospital
Little Rock, Arkansas, United States