Actively Recruiting
A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 Given by a Single Intravenous Infusion in Patients With Mucopolysaccharidosis Type I
Led by West China Hospital · Updated on 2024-07-25
6
Participants Needed
1
Research Sites
156 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to assess the safety, tolerability, and initial effectiveness of JWK008 injection in adults with Mucopolysaccharidosis Type I (MPS I), a rare genetic disorder caused by a deficiency of the IDUA gene enzyme. Current treatments have limitations, particularly in treating effects on the central nervous system. This study investigates a novel gene therapy designed to cross the blood-brain barrier and target liver tissue to deliver therapeutic effects to the brain and body. Participants will receive a single intravenous infusion of JWK008 at one of two doses: 5.0×10^12 or 2.0×10^13 vector genomes per kilogram of body weight. The study uses a dose-escalation design to monitor safety and responses in small groups of participants. This is an open-label trial without a placebo group, focusing on evaluating this gene therapy's effects over time. During the five-year follow-up, researchers will monitor adverse events and measure enzyme activity and glycosaminoglycan levels in blood, urine, and cerebrospinal fluid. Participants will also undergo physical tests like the Six-Minute Walk Test and joint motion assessments, as well as imaging studies to evaluate liver and spleen size. Vector shedding will be tracked to understand how the gene therapy is processed. This long-term monitoring aims to evaluate safety and biological effects comprehensively.
CONDITIONS
Brief 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 provide informed consent
- For females, sexual partner must agree to use reliable contraception until two consecutive negative blood tests for vector sequence
- For males, agreement to use reliable contraception until two consecutive negative semen tests for vector sequence
- Negative liver mass MRI as read by a radiologist
You will not qualify if you...
- Known unresponsiveness to enzyme replacement therapy or serious adverse reactions to laronidase
- Serum AAV5 neutralizing antibody titer greater than 1:100
- Contraindications for corticosteroids
- Current use of systemic immunomodulators or steroids (topical allowed)
- Contraindications for lumbar puncture
- Positive hepatitis B surface antigen with high HBV-DNA copy number
- Positive hepatitis C antibody with high HCV-RNA copy number
- Receiving antiviral treatment for hepatitis B or C
- Positive HIV test with CD4+ count ≤ 200/mm3
- Clinically significant abnormal lab values (ALT/AST >3× ULN, bilirubin >1.5× ULN, creatinine >1.5× ULN)
- History of chronic infections or diseases posing unacceptable risk
- Active severe infection or uncontrolled medical conditions aside from MPS I
- History of active malignancy in past 5 years (except certain skin or cervical cancers)
- Elevated or abnormal circulating alpha-fetoprotein
- Previous gene therapy or interventional clinical study participation within 12 weeks
- Pregnant or lactating females
- Other major concurrent diseases or conditions deemed unsuitable by the researcher
- Inability or unwillingness to comply with study visits and evaluations
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single administration with ongoing monitoring
Participants receive a single intravenous infusion of JWK008 to evaluate safety, tolerability, and initial efficacy.
1 infusion visit and multiple follow-up visits over 5 years
Duration - Up to 5 years
Participants are followed for safety and efficacy assessments including enzyme activity, GAG levels, physical tests, imaging, and vector shedding monitoring.
Regular visits for assessments over 5 years
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
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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