Status:
RECRUITING
ASC618 Gene Therapy in Hemophilia A Patients
Lead Sponsor:
ASC Therapeutics
Conditions:
Hemophilia A
Eligibility:
MALE
18+ years
Phase:
PHASE1
PHASE2
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, ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
August 3 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2026
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT04676048
Start Date
August 3 2022
End Date
December 1 2026
Last Update
February 1 2023
Active Locations (1)
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1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202