ASC618 Gene Therapy in Hemophilia A Patients

NCT04676048 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: ASC-HA-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Number of adverse events, and serious AEs

  12 months post-infusion

Secondary Outcomes (3)

• Changes in FVIII activity levels from baseline

  12 months post-infusion

• Annualized FVIII consumption

  12 months post-infusion

• Annualized bleeding rate (ABR)

  12 months post-infusion

Study Arms (1)

ASC618

EXPERIMENTAL

Experimental Arm

Biological: ASC618

Interventions

ASC618 BIOLOGICAL

ASC618 will be given as a single IV infusion

ASC618

Eligibility Criteria

• Age: 18 Years+
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• ASC Therapeutics: lead

Study Sites (1)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Clinical Trial Manager, PhD

CONTACT

(408) 495-3891; gil.gonen@asctherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2020
• First Posted: December 19, 2020
• Study Start: August 3, 2022
• Primary Completion: December 1, 2023
• Study Completion (Estimated): December 1, 2026
• Last Updated: February 1, 2023

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)