Clinical Trial

Primordial Dwarfism Registry

Recruiting
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Summary
The goal of this registry is to collect information on individuals with forms of microcephalic primordial dwarfism as well as related conditions. The study team hopes to learn more about these conditions and improve the care of people with them by establishing this registry.
Protocol Amendment History 4 amendments
This ClinicalTrials.gov record has been amended 4 times since 2020-09-23; most recent amendment 2025-09-25.
Trial Details
NCT Number NCT04569149
Lead Sponsor Nemours Children's Clinic
Collaborators: Potentials Foundation, Walking with Giants Foundation
Conditions MOPDII, Meier-Gorlin Syndrome, Saul-Wilson Syndrome, Microcephalic Primordial Dwarfism, IMAGe Syndrome, RNU4atac-opathy (e.g MOPDI, Lowry-Wood Syndrome, and Roifman Syndrome), LIG4 Syndrome
Enrollment 200 participants
Start Date 2008-03-11
Primary Completion 2030-01-01 (estimated)
Study Completion 2030-01-01 (estimated)
Updated on ClinicalTrials.gov 2025-10-01