ASC618 Gene Therapy in Hemophilia A Patients

January 30, 2023 updated by: ASC Therapeutics

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

12

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

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.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: ASC618
Experimental Arm
Biological: ASC618
ASC618 will be given as a single IV infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of adverse events, and serious AEs
Time Frame: 12 months post-infusion
12 months post-infusion

Secondary Outcome Measures

Outcome Measure
Time Frame
Changes in FVIII activity levels from baseline
Time Frame: 12 months post-infusion
12 months post-infusion
Annualized FVIII consumption
Time Frame: 12 months post-infusion
12 months post-infusion
Annualized bleeding rate (ABR)
Time Frame: 12 months post-infusion
12 months post-infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

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 Major Dates

Study Start (ACTUAL)

August 3, 2022

Primary Completion (ANTICIPATED)

December 1, 2023

Study Completion (ANTICIPATED)

December 1, 2026

Study Registration Dates

First Submitted

December 15, 2020

First Submitted That Met QC Criteria

December 17, 2020

First Posted (ACTUAL)

December 19, 2020

Study Record Updates

Last Update Posted (ACTUAL)

February 1, 2023

Last Update Submitted That Met QC Criteria

January 30, 2023

Last Verified

August 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ASC-HA-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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