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NCT Number: NCT04676048

ASC618 Gene Therapy in Hemophilia A Patients

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

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location status: Recruiting

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ASC618

Biological

ASC618 will be given as a single IV infusion

Primary outcomes

  1. Number of adverse events, and serious AEs

    Time frame: 12 months post-infusion

Secondary outcomes

  1. Changes in FVIII activity levels from baseline

    Time frame: 12 months post-infusion

  2. Annualized FVIII consumption

    Time frame: 12 months post-infusion

  3. Annualized bleeding rate (ABR)

    Time frame: 12 months post-infusion

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trial Manager, PhD

CONTACT

[email protected]

(408) 495-3891

Sponsors and collaborators

Lead sponsor

ASC Therapeutics

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2023
Study completion
2026
First posted
Dec 19, 2020
Registry last updated
Feb 1, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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