ASC618 Gene Therapy in Hemophilia A Patients

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04676048
• Other study ID #: ASC-HA-001
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: August 3, 2022
• Est. completion date: December 2026

Study information

• Verified date: August 2022
• Source: ASC Therapeutics
• Contact: Clinical Trial Manager, PhD
• Phone: (408) 495-3891
• Email: gil.gonen[@]asctherapeutics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: December 2026
• Est. primary completion date: December 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• ASC618
ASC618 will be given as a single IV infusion

Locations

Country	Name	City	State
United States	Arkansas Children's Hospital	Little Rock	Arkansas

Sponsors (1)

Lead Sponsor	Collaborator
ASC Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of adverse events, and serious AEs		12 months post-infusion
Secondary	Changes in FVIII activity levels from baseline		12 months post-infusion
Secondary	Annualized FVIII consumption		12 months post-infusion
Secondary	Annualized bleeding rate (ABR)		12 months post-infusion