Study of JWK008 Gene Therapy Given by One Intravenous Dose to Adults With Mucopolysaccharidosis Type I to Evaluate Safety and Early Effects

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 5.01012 or 2.01013 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 therapys 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.

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I