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Active, not recruiting No phase listed Observational

Mucolipidosis Type IV Natural History Study

ClinicalTrials.gov ID: NCT05782387

Public ClinicalTrials.gov record NCT05782387. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 2, 2026, 10:02 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A New Retrospective Natural History Study of Mucolipidosis Type IV

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT05782387
Recruitment status
Active, not recruiting
Study type
Observational
Phase
Not listed
Enrollment
50 participants

Conditions and interventions

Interventions

Not listed

Eligibility (public fields only)

Age range
Not listed
Sex
All
Healthy volunteers
Not listed

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Mar 14, 2023
Primary completion
Aug 31, 2028
Completion
Aug 31, 2028
Last update posted
Apr 22, 2026

2023 – 2028

United States locations

U.S. sites
1
U.S. states
1
U.S. cities
1
Facility City State ZIP Site status
Massachusetts General Hospital Boston Massachusetts 02144

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT05782387, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Apr 22, 2026 · Synced Sep 2, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT05782387 live on ClinicalTrials.gov.

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