deltatrials
Completed PHASE1/PHASE2 INTERVENTIONAL 1-arm NCT01560182

Gene Therapy for Metachromatic Leukodystrophy (MLD)

A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy

Sponsor: Orchard Therapeutics

Conditions Lysosomal Storage Disease Metachromatic Leukodystrophy
Interventions OTL-200 Gene Therapy
Updated 17 times since 2017 Last updated: Dec 1, 2025 Started: Apr 9, 2010 Primary completion: Apr 9, 2018 Completion: Sep 19, 2025
This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

Listed as NCT01560182, this PHASE1/PHASE2 trial focuses on Lysosomal Storage Disease and Metachromatic Leukodystrophy and remains completed. Sponsored by Orchard Therapeutics, it has been updated 17 times since 2010, reflecting substantial change activity. This study adds to the evidence base for this therapeutic area through structured, versioned documentation.

Status Flow

~Jan 2017 – ~Apr 2017 · 3 months · monthly snapshot~Apr 2017 – ~Jun 2018 · 14 months · monthly snapshot~Jun 2018 – ~Nov 2018 · 5 months · monthly snapshot~Nov 2018 – ~Jan 2019 · 2 months · monthly snapshot~Jan 2019 – ~Mar 2019 · 59 days · monthly snapshot~Mar 2019 – ~Jun 2019 · 3 months · monthly snapshot~Jun 2019 – ~Jun 2020 · 12 months · monthly snapshot~Jun 2020 – ~Jan 2021 · 7 months · monthly snapshot~Jan 2021 – ~Oct 2021 · 9 months · monthly snapshot~Oct 2021 – ~Nov 2021 · 31 days · monthly snapshot~Nov 2021 – ~Dec 2022 · 13 months · monthly snapshot~Dec 2022 – ~Dec 2023 · 12 months · monthly snapshot~Dec 2023 – ~Jul 2024 · 7 months · monthly snapshot~Jul 2024 – ~Sep 2024 · 2 months · monthly snapshot~Sep 2024 – ~Oct 2025 · 13 months · monthly snapshot~Oct 2025 – ~Jan 2026 · 3 months · monthly snapshot~Jan 2026 – present · 6 months · monthly snapshot

Change History

17 versions recorded

  1. Jan 2026 — Present [monthly]

    Completed PHASE1/PHASE2

    Status: Active Not Recruiting → Completed

  2. Oct 2025 — Jan 2026 [monthly]

    Active Not Recruiting PHASE1/PHASE2

  3. Sep 2024 — Oct 2025 [monthly]

    Active Not Recruiting PHASE1/PHASE2

  4. Jul 2024 — Sep 2024 [monthly]

    Active Not Recruiting PHASE1/PHASE2

    Phase: PHASE1_PHASE2 → PHASE1/PHASE2

  5. Dec 2023 — Jul 2024 [monthly]

    Active Not Recruiting PHASE1_PHASE2

▶ Show 12 earlier versions

  1. Dec 2022 — Dec 2023 [monthly]

    Active Not Recruiting PHASE1_PHASE2

  2. Nov 2021 — Dec 2022 [monthly]

    Active Not Recruiting PHASE1_PHASE2

  3. Oct 2021 — Nov 2021 [monthly]

    Active Not Recruiting PHASE1_PHASE2

  4. Jan 2021 — Oct 2021 [monthly]

    Active Not Recruiting PHASE1_PHASE2

  5. Jun 2020 — Jan 2021 [monthly]

    Active Not Recruiting PHASE1_PHASE2

  6. Jun 2019 — Jun 2020 [monthly]

    Active Not Recruiting PHASE1_PHASE2

  7. Mar 2019 — Jun 2019 [monthly]

    Active Not Recruiting PHASE1_PHASE2

  8. Jan 2019 — Mar 2019 [monthly]

    Active Not Recruiting PHASE1_PHASE2

    Phase: PHASE2 → PHASE1_PHASE2

  9. Nov 2018 — Jan 2019 [monthly]

    Active Not Recruiting PHASE2

  10. Jun 2018 — Nov 2018 [monthly]

    Active Not Recruiting PHASE2

  11. Apr 2017 — Jun 2018 [monthly]

    Active Not Recruiting PHASE2

    Phase: PHASE1_PHASE2 → PHASE2

  12. Jan 2017 — Apr 2017 [monthly]

    Active Not Recruiting PHASE1_PHASE2

    First recorded

Apr 2010

Trial started

Per CT.gov start date — pre-dates our first snapshot

Eligibility Summary

No eligibility information available.

Contact Information

Sponsor contact:
  • Orchard Therapeutics
  • Ospedale San Raffaele
Data source: Orchard Therapeutics

For direct contact, visit the study record on ClinicalTrials.gov .

Study Locations