Rare disease study aims to map MLIV's natural course

NCT ID NCT01067742

Terminated Knowledge-focused Sponsor: Baylor Research Institute Source: ClinicalTrials.gov ↗

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

Summary

This study followed 7 people with Mucolipidosis Type IV (MLIV) to learn how the disease naturally progresses. Researchers used tests like neuropsychological exams, blood and urine tests, and brain MRIs to find better ways to measure the disease. The goal was to improve future clinical trials, not to test a treatment.

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Conditions

The condition(s) this trial relates to.

Corneal Opacity corpus callosum, agenesis of demyelinating disease inherited retinal dystrophy intellectual disability lysosomal storage disease mucolipidosis mucolipidosis type IV

As listed by the trial registrant

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

MUCOLIPIDOSIS TYPE IV

Contacts and locations

Locations

Baylor Institute of Metabolic Disease

Dallas, Texas, 75226, United States

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