A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
Trial Parameters
Brief Summary
This study is a single-center, single-arm, non-randomized, open-label, non controlled, dose-escalation, prospective clinical trial designed to assess the safety, tolerability, and preliminary efficacy of JWK008 injection in patients with MPS I.
Eligibility Criteria
Inclusion Criteria: 1. Age≥ 18 years old; 2. Diagnosis of MPS type I; 3. Be able to understand the purpose, content, and possible risks of this clinical study, voluntarily participate in and sign the informed consent form; 4. If the subject is female, her sexual partner must agree to use reliable contraception until 2 consecutive tests of vector sequence in the blood are negative; If the subject is male, the subject agrees to use reliable contraception until the semen sample is negative for 2 consecutive tests of the vector sequence. 5. Magnetic resonance imaging (MRI) of the liver mass read by the radiologist is negative. Exclusion Criteria: 1. Known to be unresponsive to ERT; or those who have been treated with intrathecal or intravenous laronidase and have serious adverse reactions, such as significant infusion-related reactions (IARs) or anaphylactic shock. 2. Serum AAV5 neutralizing antibody titer is greater than 1:100. 3. Has contraindications for Corticosteroids. 4. Current trea