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ASC618 Gene Therapy in Hemophilia A Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04676048
Recruitment Status : Recruiting
First Posted : December 19, 2020
Last Update Posted : February 1, 2023
Sponsor:
Information provided by (Responsible Party):
ASC Therapeutics

Study Description
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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
Hemophilia A Biological: ASC618 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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
Actual Study Start Date : August 3, 2022
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arms and Interventions
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Arm Intervention/treatment
Experimental: ASC618
Experimental Arm
 Biological: ASC618
ASC618 will be given as a single IV infusion


Outcome Measures
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Primary Outcome Measures :
  1. Number of adverse events, and serious AEs [ Time Frame: 12 months post-infusion ]

Secondary Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04676048


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

Locations
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United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Sponsors and Collaborators
ASC Therapeutics
More Information
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Responsible Party: ASC Therapeutics
ClinicalTrials.gov Identifier: NCT04676048    
Other Study ID Numbers: ASC-HA-001
First Posted: December 19, 2020    Key Record Dates
Last Update Posted: February 1, 2023
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn