Back to resultsASC618 Gene Therapy in Hemophilia A Patients
Primary Purpose
Hemophilia A
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
ASC618
Sponsored by
About this trial
This is an interventional treatment trial for Hemophilia A
Eligibility 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
Sites / Locations
- Arkansas Children's HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
ASC618
Arm Description
Experimental Arm
Outcomes
Primary Outcome Measures
Number of adverse events, and serious AEs
Secondary Outcome Measures
Changes in FVIII activity levels from baseline
Annualized FVIII consumption
Annualized bleeding rate (ABR)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04676048
Brief Title
ASC618 Gene Therapy in Hemophilia A Patients
Official Title
Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
Study Type
Interventional
2. Study Status
Record Verification Date
August 2022
Overall Recruitment Status
Recruiting
Study Start Date
August 3, 2022 (Actual)
Primary Completion Date
December 2023 (Anticipated)
Study Completion Date
December 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ASC Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. 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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
ASC618
Arm Type
Experimental
Arm Description
Experimental Arm
Intervention Type
Biological
Intervention Name(s)
ASC618
Intervention Description
ASC618 will be given as a single IV infusion
Primary Outcome Measure Information:
Title
Number of adverse events, and serious AEs
Time Frame
12 months post-infusion
Secondary Outcome Measure Information:
Title
Changes in FVIII activity levels from baseline
Time Frame
12 months post-infusion
Title
Annualized FVIII consumption
Time Frame
12 months post-infusion
Title
Annualized bleeding rate (ABR)
Time Frame
12 months post-infusion
10. Eligibility
Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Clinical Trial Manager, PhD
Phone
(408) 495-3891
Email
gil.gonen@asctherapeutics.com
Facility Information:
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
ASC618 Gene Therapy in Hemophilia A Patients
We'll reach out to this number within 24 hrs