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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 : Not yet recruiting
First Posted : December 19, 2020
Last Update Posted : November 18, 2021
Sponsor:
Information provided by (Responsible Party):
ASC Therapeutics

Tracking Information
First Submitted Date  ICMJE December 15, 2020
First Posted Date  ICMJE December 19, 2020
Last Update Posted Date November 18, 2021
Estimated Study Start Date  ICMJE April 2022
Estimated Primary Completion Date December 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 17, 2020)
Number of adverse events, and serious AEs [ Time Frame: 12 months post-infusion ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 17, 2020)
  • Changes in FVIII activity levels from baseline [ Time Frame: 12 months post-infusion ]
  • Annualized FVIII consumption [ Time Frame: 12 months post-infusion ]
  • Annualized bleeding rate (ABR) [ Time Frame: 12 months post-infusion ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE ASC618 Gene Therapy in Hemophilia A Patients
Official Title  ICMJE Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
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

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia A
Intervention  ICMJE Biological: ASC618
ASC618 will be given as a single IV infusion
Study Arms  ICMJE Experimental: ASC618
Experimental Arm
Intervention: Biological: ASC618
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: December 17, 2020)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2026
Estimated Primary Completion Date December 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Trial Manager, PhD (408) 495-3891 gil.gonen@asctherapeutics.com
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04676048
Other Study ID Numbers  ICMJE ASC-HA-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party ASC Therapeutics
Study Sponsor  ICMJE ASC Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account ASC Therapeutics
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP