New hope for rare brain disease: Long-Term safety data collected

NCT ID NCT01887938

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed 24 people with metachromatic leukodystrophy (MLD), a rare and serious brain disease, who were already receiving an experimental drug called HGT-1110. The main goal was to check for side effects and immune reactions over a long period. Researchers wanted to see if the drug remained safe for continued use.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

metachromatic leukodystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • CHR Orleans - Hopital La Source

    Orléans, 45067, France

  • CHU de Nantes

    Nantes, 44093, France

  • Center for Pediatric Clinical Studies (CPCS)

    Tübingen, Baden-Wurttemberg, 72076, Germany

  • Detska Interni Klinika, Lf Mu A Fn Brno

    Brno, 61300, Czechia

  • Hopital Femme Mere Enfant

    Bron, 69 677, France

  • Hopital Gui de Chauliac - CHRU de Montpellier

    Montpellier, 34000, France

  • Hospital de Clinicas de Porto Alegre

    Porto Alegre, 90035-003, Brazil

  • Hôpital de Bicêtre

    Le Kremlin-Bicêtre, Île-de-France Region, 94275, France

  • Kitakyushu Municipal Yahata Hospital

    Fukuoka, 805-8534, Japan

  • Klinikum Oldenburg

    Oldenburg, 26133, Germany

  • Kurashiki Central Hospital

    Okayama Prefecture, 710-8602, Japan

  • Marien-Hospital Wesel gGmbh

    Wesel, 46483, Germany

  • Osaka University Hospital

    Osaka, 565-0871, Japan

  • Rigshospitalet

    Copenhagen, 2100, Denmark

  • The Children's Hospital at Westmead

    Westmead, 2145, Australia