ASC618 Gene Therapy in Hemophilia A Patients
Key facts
- Where
- Little Rock, United StatesSee the site
- Phase
- Phase 1/2
Where in United States
- Arkansas Children's HospitalLittle Rock, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- ASC Therapeutics
- Trial code
- NCT04676048
- Record updated
- 1 February 2023
- Original description
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.Read full original descriptionShow less
Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
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
Inclusion: 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: 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; 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; Current antiviral therapy for hepatitis B or C; Presence of > Grade 2 liver fibrosis on elastography/Fibroscan or comparable imaging methodology; Receipt of any vector or gene transfer agent.