CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 20 enrolled
Drug / intervention
OTL-200 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.

Search/NCT01560182
NCT01560182Phase 2Completed
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A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy

Orchard Therapeutics·interventional·Posted Mar 22, 2012·Updated Dec 5, 2025

In Brief

A Phase 2 clinical trial evaluating OTL-200 Gene Therapy for Lysosomal Storage Disease and Metachromatic Leukodystrophy. Completed, enrolled 20 participants across 1 site.

Detailed Summary

This Phase I/II clinical trial consists of the application of lentiviral vector-based gene therapy to patients affected by Metachromatic Leukodystrophy (MLD), a rare inherited Lysosomal Storage Disorder (LSD) resulting from mutations in the gene encoding the Arylsulfatase A (ARSA) enzyme. The medicinal product consists of autologous CD34+ hematopoietic stem/progenitor cells in which a functional ARSA cDNA is introduced by means of 3rd generation VSV-G pseudotyped lentiviral vectors.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsLysosomal Storage Disease, Metachromatic Leukodystrophy
CountriesItaly
CollaboratorsOspedale San Raffaele

Timeline

Phase 2CompletedFinished
20102011201220132014201520162017201820192020202120222023202420252026
First PostedMar 22, 2012
Enrollment StartApr 9, 2010
Primary CompletionApr 9, 2018
Study CompletionSep 19, 2025
TodayJul 2, 2026
Enrollment to primary: 8 yearsPosted 14.3 years ago

Interventions

OTL-200 Gene Therapygenetic

Autologous hematopoietic stem/progenitor cells collected from the bone marrow and transduced ex vivo with a Lentiviral vector encoding the human ARSA cDNA