Brief Title
ASC618 Gene Therapy in Hemophilia A Patients
Official Title
Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
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
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Number of adverse events, and serious AEs
Secondary Outcome
Changes in FVIII activity levels from baseline
Condition
Hemophilia A
Intervention
ASC618
Study Arms / Comparison Groups
ASC618
Description: Experimental Arm
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
12
Start Date
April 2022
Completion Date
December 2026
Primary Completion Date
December 2023
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
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
, (408) 495-3891, [email protected]
Administrative Informations
NCT ID
NCT04676048
Organization ID
ASC-HA-001
Responsible Party
Sponsor
Study Sponsor
ASC Therapeutics
Study Sponsor
, ,
Verification Date
March 2021