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RECRUITING
NCT06519552
PHASE1

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

Sponsor: West China Hospital

View on ClinicalTrials.gov

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.

Official title: A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 Given by a Single Intravenous Infusion in Patients With Mucopolysaccharidosis Type I

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

6

Start Date

2024-07-22

Completion Date

2029-06-22

Last Updated

2024-07-25

Healthy Volunteers

No

Interventions

GENETIC

JWK008 Single intravenous infusion administration

Six participants with MPS I will be enrolled in the study. The participantss will be divided into two different dose groups, and a "3+3" dose escalation design is used. The low dose is 5.0×10\^12vg/kg, and the high dose is 2.0×10\^13vg/kg. Only one intravenous infusion of JWK008 will be administered to each participant.

Locations (1)

West China Hospital, Sichuan Universit

Chengdu, Sichuan, China