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

NCT ID NCT01887938

Disease control Sponsor: Shire Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

24 people

The number who actually took part.

Started

May 2013

Finished

Feb 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 to 13 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Participant has participated in Study HGT-MLD-070 (NCT01510028) through Week 40. 2. Participant must have no safety or medical issues that contraindicate participation. 3. The participant, participant's parent(s), or legally authorized representative(s) must provide written informed consent and/or assent (if applicable) prior to performing any study-related activities. Exclusion Criteria: 1. The participant is unable to comply with the protocol (example, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the investigator. 2. Undergoes bone marrow transplant (BMT), hematopoietic stem cell transplantation (HSCT), or gene therapy at any point during the study. 3. The participant has any known or suspected hypersensitivity to agents used for anesthesia or is thought to be at an unacceptably high risk for associated potential complications of airway compromise or other conditions. 4. The participant is pregnant or breastfeeding. 5. The participant is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or drug delivery device) other than those used in HGT-MLD-070 (NCT01510028) within 6 months prior to study enrollment or at any time during the study. 6. The participant has a condition that is contraindicated as described in the SOPH-A-PORT Mini SIDDD Instructions for Use (IFU), including: 1. The participant has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device. 2. The participant's body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the investigator. 3. The participant has a known or suspected local or general infection. 4. The participant is at risk of abnormal bleeding due to a medical condition or therapy. 5. The participant has one or more spinal abnormalities that could complicate safe implantation or fixation. 6. The participant has a functioning CSF shunt device. 7. The participant has shown an intolerance to an implanted device.

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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.

Metachromatic leukodystrophy (MLD)

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.