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Gene Therapy Trial Report

Summary

ASC618 Gene Therapy in Hemophilia A Patients


NCTID NCT04676048 (View at clinicaltrials.gov)
Description
Development Status 🔄 Inactive
Indication Hemophilia A
Disease Ontology Term DOID:12134
Drug Product Name ASC618
Drug Product Description HCB-ET3-LCO
Sponsor ASC Therapeutics
Funder Type Industry
Recruitment Status
Enrollment Count 12 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant F8
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type AAV2/8
Dose 1 🔄 Undisclosed dose

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2020-12-15
Completion Date 2026-12
Last Update 2023-02-01

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates ASC Therapeutics, Inc. filed for bankruptcy liquidation in late 2025

Resources/Links