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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 Active
Indication Hemophilia A
Disease Ontology Term DOID:12134
Compound Name ASC618
Compound 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
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2/8
Editor Type none
Dose 1 Undisclosed dose 1

Study Record Dates


Current Stage 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 First patient dosed January 2024

Resources/Links