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NCT05782387 · ClinicalTrials.gov registry record
Mucolipidosis Type IV Natural History Study
A clinical trial of Mucolipidosis Type IV, sponsored by Massachusetts General Hospital.
- Recruiting
- Registry status
- 50
- Enrollment target
- 1
- Study location
NCT05782387: Recruiting study of Mucolipidosis Type IV, sponsored by Massachusetts General Hospital.
NCT05782387 is a study of Mucolipidosis Type IV that is actively recruiting participants, run by Massachusetts General Hospital. The registered enrollment target is 50 participants. The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05782387, a study of Mucolipidosis Type IV, is actively recruiting participants, sponsored by Massachusetts General Hospital.
- RECRUITING
- Registry status
- 50 participants
- Enrollment target
- 1
- Study location
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.
Primary Outcome
Describe the characteristics of the current international MLIV population.
Conditions Studied
Study Locations (1)
Massachusetts
- Massachusetts General Hospital - Boston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 50 participants |
| Start Date | 2023-03-15 |
| Est. Completion | 2026-03 |
What NCT05782387 shows while recruiting
NCT05782387 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 50 participants, a relatively small participant target.
The record links to 1 condition, with Mucolipidosis Type IV appearing as the primary indexed condition, and to 0 interventions.
NCT05782387 reports a single indexed study location in Massachusetts.
Frequently Asked Questions
What is clinical trial NCT05782387 about?
NCT05782387 is a clinical study titled "Mucolipidosis Type IV Natural History Study". 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 Syst...
What is the current status of trial NCT05782387?
This trial is currently recruiting. The enrollment target is 50 participants. The study started on 2023-03-15. Estimated completion is 2026-03.
What conditions does trial NCT05782387 study?
This clinical trial studies the following conditions: Mucolipidosis Type IV.
Who is sponsoring clinical trial NCT05782387?
This trial is sponsored by Massachusetts General Hospital, which has 2,032 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05782387 being conducted?
This trial has 1 study location across Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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