RECRUITING

ASC618 Gene Therapy in Hemophilia A Patients

Conditions

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

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

Quick Facts

Study Start:2022-08-03
Study Completion:2026-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04676048

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Male ≥18 years of age
  2. * Severe or moderately severe hemophilia A (FVIII activity ≤ 2 IU/dL) as evidenced by
  3. * medical history
  4. * Received FVIII prophylactic or on-demand replacement therapy for ≥ 150 accumulated
  5. * days (exposure days)
  6. * ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months
  7. * BMI ≤ 30
  8. * Agree to use double-barrier contraceptive until at least 3 consecutive semen samples are negative after ASC-618 infusion
  1. * Pre-existing immunity to AAV8 vector as defined by AAV8 total antibodies and neutralizing antibodies qualified tests.
  2. * Current inhibitors, or history of high titer FVIII inhibitors
  3. * Presence of \> Grade 2 liver fibrosis on elastography/Fibroscan or comparable imaging methodology
  4. * History of chronic renal disease
  5. * Active infection or any immunosuppressive disorder
  6. * History of cardiac surgery and need anticoagulant therapy
  7. * Any cardiovascular / genetic risk factors for thromboembolic disorders
  8. * Evidence of active Hepatitis B, Hepatitis C, Human Immunodeficiency Virus (HIV)-1/2 or syphilis infection.
  9. * Receipt of any vector or gene transfer agent
  10. * Current antiviral therapy for hepatitis B or C

Contacts and Locations

Study Contact

Clinical Trial Manager, PhD
CONTACT
(408) 495-3891
gil.gonen@asctherapeutics.com

Study Locations (Sites)

Arkansas Children's Hospital
Little Rock, Arkansas, 72202
United States

Collaborators and Investigators

Sponsor: ASC Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-08-03
Study Completion Date2026-12

Study Record Updates

Study Start Date2022-08-03
Study Completion Date2026-12

Terms related to this study

Additional Relevant MeSH Terms

  • Hemophilia A