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Mucolipidosis Type IV Natural History Study

NCT05782387 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2026-04-23

No results posted yet for this study

Summary

The primary objectives of the study are: to describe the characteristics of the current international MLIV population; to define the median age at which patients with MLIV achieve or lose developmental milestones; to define the natural history of MLIV for the Gross Motor Function Classification System (GMFCS) (Morris and Bartlett, 2004) and the MLIV specific scale and test the validity of retrospectively applying these scales to medical record data; to define the rate of visual decline in patients with MLIV.

Conditions

  • Mucolipidosis Type IV

Sponsors & Collaborators

Principal Investigators

  • Patricia Musolino, MD, PHD · Neurologist

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-03-15
Primary Completion
2028-09-30
Completion
2028-09-30

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05782387 on ClinicalTrials.gov