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OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

StatusActive, Not Recruiting
PhasePhase 3
Started
View on ClinicalTrials.gov ↗
Record status
This record was last updated September 5, 2025 (before its estimated January 31, 2026 completion). Its status may not reflect the trial's current state.

Record History

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  1. Primary completion date: Mar 31, 2025 to Jan 31, 2026v11
  2. Trial status: Recruiting to Active, Not Recruiting
    • Site details revised
    v10
  3. Re-verified, no change to tracked fieldsv9
  4. Primary completion date: Jan 17, 2032 to Mar 31, 2025v8 Show 4 more changesShow less
    • Primary endpoints revised (2)
    • Study completion date: Jan 17, 2032 to Mar 31, 2031
    • Eligibility criteria expanded, about 311 words
    • Secondary endpoints expanded, about 192 words
  5. Start date: Dec 31, 2020 to Jan 17, 2022
    • Primary and study completion dates: Jan 2032 to Jan 17, 2032
    v7
Show 5 earlier versionsHide 5 earlier versions
  1. Start date changed from estimate of Dec 2020 to Dec 31, 2020v6
  2. Start date: Sep 2020 to Dec 2020v5
  3. Start date: Jun 2020 to Sep 2020v4
  4. Start date: Mar 2020 to Jun 2020v3
  5. Primary endpoints revised (2)
    • Start date: Feb 2020 to Mar 2020
    v2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination affecting the central and peripheral nervous system. The aim of this clinical study is to assess the pharmacodynamic effect and long-term clinical efficacy and safety of OTL-200 in Late Juvenile MLD patients.
Trial Details
NCT Number NCT04283227
Lead Sponsor Orchard Therapeutics
Collaborators: Ospedale San Raffaele
Conditions
(from sponsor)		 Lysosomal Storage Diseases, Metachromatic Leukodystrophy
Related terms Leukodystrophy, Metachromatic, Lysosomal Storage Diseases
Enrollment 6 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov