Recruiting
Phase 1
Phase 2

ASC618

Sponsor:

ASC Therapeutics

Code:

NCT04676048

Conditions

Hemophilia A

Eligibility Criteria

Sex: Male

Age: 18+

Healthy Volunteers: Not accepted

Interventions

ASC618

Study Details

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

Study ID

NCT04676048

Start date

Aug 3, 2022

Status verified date

Aug, 2022

Completion date

Dec, 2026

Anticipated

Primary completion date

Dec, 2023

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: ASC618

Experimental Arm

Interventions

ASC618

ASC618 will be given as a single IV infusion

Primary outcome measure

  • Number of adverse events, and serious AEs [ Time Frame: 12 months post-infusion ]

Central Contacts and Locations

Central contacts

Clinical Trial Manager, PhD

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

Locations

Arkansas Children's Hospital

Recruiting

Little Rock, Arkansas, United States, 72202

More Information

Sponsor

ASC Therapeutics

Last update posted

Feb 1, 2023

Last verified

Aug, 2022

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by ASC Therapeutics on 2023-02-01.