CI

At a glance

ClinicalIndex Comparison Record
N/ARecruiting· 10 target
Drug / intervention
Intrathecal and intravenous LV gene therapygenetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

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NCT03725670N/ARecruitingOn Track
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Direct Lentiviral TYF-ARSA Injection Gene Therapy for Metachromatic Leukodystrophy (MLD)

Shenzhen Geno-Immune Medical Institute·interventional·Posted Oct 31, 2018·Updated Jun 23, 2026

In Brief

A clinical study evaluating Intrathecal and intravenous LV gene therapy for Metachromatic Leukodystrophy (MLD). Currently recruiting, targeting 10 participants across 1 site.

Detailed Summary

This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical protocol.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMetachromatic Leukodystrophy (MLD)
CountriesChina
Collaborators--

Timeline

N/ARecruiting
2019202020212022202320242025202620272028202920302031
First PostedOct 31, 2018
Enrollment StartMay 31, 2025
Primary CompletionDec 31, 2029
Study CompletionDec 31, 2030
TodayJul 1, 2026
Enrollment to primary: 4.6 yearsPosted 7.7 years agoPrimary completion in 3.5 years

Arms & Interventions

Lentivirus-mediated delivery of ARSA to the CNS and the bodyexperimental

Intrathecal and intravenous injections with lentiviral TYF-ARSA vector carrying the functional gene

Genetic: Intrathecal and intravenous LV gene therapy

Interventions

Intrathecal and intravenous LV gene therapygenetic

Direct IT and IV LV gene therapy to deliver high levels of LVs at 1-2×10\^9 transduction units/ml which carry a normal ARSA gene