Actively Recruiting
ASC618 Gene Therapy in Hemophilia A Patients
Led by ASC Therapeutics · Updated on 2023-02-01
12
Participants Needed
1
Research Sites
225 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
ASC618 Gene Therapy in Hemophilia A Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male aged 18 years or older
- Severe or moderately severe hemophilia A with factor VIII activity ≤ 2 IU/dL
- Medical history confirming hemophilia A diagnosis
- Received at least 150 days of factor VIII prophylactic or on-demand replacement therapy
- At least 12 bleeding episodes in the past 12 months if on on-demand therapy
- Body mass index (BMI) of 30 or less
- Agree to use double-barrier contraception until three consecutive semen samples test negative after ASC618 infusion
You will not qualify if you...
- Pre-existing immunity to AAV8 vector as shown by antibody tests
- Current inhibitors or history of high titer factor VIII inhibitors
- Liver fibrosis greater than Grade 2 based on imaging tests
- History of chronic kidney disease
- Active infection or immunosuppressive disorder
- History of heart surgery and need for anticoagulant therapy
- Cardiovascular or genetic risk factors for blood clots
- Active infections with Hepatitis B, Hepatitis C, HIV-1/2, or syphilis
- Previous treatment with any vector or gene transfer agent
- Current antiviral treatment for Hepatitis B or C
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
Research Team
C
Clinical Trial Manager, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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