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ASC618 Gene Therapy in Hemophilia A Patients

Sponsor
ASC Therapeutics (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04676048
Collaborator
(none)
12
Enrollment
1
Location
1
Arm
52
Anticipated Duration (Months)
0.2
Patients Per Site Per Month

Study Details

Study Description

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

Condition or Disease Intervention/Treatment Phase
  • Biological: ASC618
 Phase 1/Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
12 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
Anticipated Study Start Date :
Aug 1, 2022
Anticipated Primary Completion Date :
Dec 1, 2023
Anticipated Study Completion Date :
Dec 1, 2026

Arms and Interventions

Arm Intervention/Treatment
Experimental: ASC618

Experimental Arm

Biological: ASC618
ASC618 will be given as a single IV infusion

Outcome Measures

Primary Outcome Measures

  1. Number of adverse events, and serious AEs [12 months post-infusion]

Secondary Outcome Measures

  1. Changes in FVIII activity levels from baseline [12 months post-infusion]

  2. Annualized FVIII consumption [12 months post-infusion]

  3. Annualized bleeding rate (ABR) [12 months post-infusion]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
Male
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Arkansas Children's Hospital Little Rock Arkansas United States 72202

Sponsors and Collaborators

  • ASC Therapeutics

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
ASC Therapeutics
ClinicalTrials.gov Identifier:
NCT04676048
Other Study ID Numbers:
  • ASC-HA-001
First Posted:
Dec 19, 2020
Last Update Posted:
Aug 19, 2022
Last Verified:
Aug 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Hemophilia A

Study Results

No Results Posted as of Aug 19, 2022