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New MRI study maps rare brain disease in children

NCT ID NCT01325025

Knowledge-focused Sponsor: Assistance Publique - Hôpitaux de Paris 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 used powerful MRI scans to track how a rare genetic disease called metachromatic leukodystrophy (MLD) damages the brain's white matter in young children. Researchers studied 29 children aged 1 to 6 years with MLD, along with a control group, to better understand how the disease progresses. The goal was to find new ways to measure the disease that could help select patients for future treatments.

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

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

29 people

The number who actually took part.

Start date

Nov 2010

Finished

Jul 2016

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Ages

1 year to 6 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 (patients): * Children with proven metachromatic leukodystrophy (MLD) with decreased activity of arylsulfatase A enzyme in leukocytes and abnormal excretion of urinary sulfatides * Age ≥ 1 year and ≤ 6 years * Recently diagnosed (within \< 18 months) Inclusion Criteria (control): * Children with partial cryptogenic epilepsy or with a suspected brain lesion on conventional MRI, who should have high-field MRI to detect structural abnormalities that could benefit from surgical resection * Age ≥ 1 year and ≤ 6 years Exclusion Criteria: * Evolutive heart, pulmonary, renal or gastrointestinal disease * Contra-indication to sedation * Contra-indication to MRI (implanted magnetic material)

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Conditions

The condition(s) this trial relates to.

demyelinating disease metachromatic leukodystrophy metachromatic leukodystrophy, late infantile form

As listed by the trial registrant

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

Late infantile metachromatic leukodystrophy

Contacts and locations

Locations

  • Bâtiment Lavoisier - Unité INSERM U 663,Hôpital Necker Enfants Malades

    Paris, France

  • Service de Neurologie Pédiatrique, Hôpital Bicêtre

    Paris, 94275, France

  • Unité de recherche biomédicale, Neurospin, I2BM / DSV / SAC/ CEA,

    Gif-sur-Yvette, France

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