Actively Recruiting
A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
Led by West China Hospital · Updated on 2024-07-25
6
Participants Needed
1
Research Sites
256 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Diagnosis of Mucopolysaccharidosis Type I
- Ability to understand the study purpose, risks, and voluntarily sign informed consent
- Females must have sexual partners agree to use reliable contraception until 2 negative vector sequence blood tests; males must use reliable contraception until 2 negative semen vector sequence tests
- Liver MRI must be negative for liver masses as read by a radiologist
You will not qualify if you...
- Known unresponsiveness to enzyme replacement therapy or serious adverse reactions to laronidase treatments
- Serum AAV5 neutralizing antibody titer above 1:100
- Contraindications to corticosteroids
- Current systemic immunomodulator or steroid use (except topical treatments)
- Contraindications to lumbar puncture
- Positive hepatitis B or C markers with viral loads above normal limits, or receiving antiviral treatment for hepatitis B or C
- Positive HIV test with CD4+ T cell count ≤ 200/mm3
- Clinically significant abnormal liver or kidney laboratory values
- History of chronic infections or other chronic diseases posing unacceptable risk
- Active severe infection or uncontrolled significant medical conditions (excluding MPS I related)
- History of active cancer within past 5 years (except certain skin or cervical cancers)
- Elevated or abnormal alpha-fetoprotein levels
- Previous gene therapy or participation in interventional studies within past 12 weeks
- Pregnant or lactating females
- Any other major diseases or conditions judged unsuitable by the researcher
- Unable or unwilling to comply with study visits and evaluations
AI-Screening
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Trial Site Locations
Total: 1 location
1
West China Hospital, Sichuan Universit
Chengdu, Sichuan, China, 610041
Actively Recruiting
Research Team
X
Xingchen Peng, Ph.D
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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