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

NCT06519552 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2024-07-25

No results posted yet for this study

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.

Conditions

  • Mucopolysaccharidosis Type I

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.

Sponsors & Collaborators

  • West China Hospital

    lead OTHER

Principal Investigators

  • Xingchen Peng, Ph.D · West China Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-07-22
Primary Completion
2026-06-22
Completion
2029-06-22

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06519552 on ClinicalTrials.gov