200 Participants Needed

Registry for Primordial Dwarfism

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CB
EL
Overseen ByEmily Longenecker, BS
Age: Any Age
Sex: Any
Trial Phase: Academic
Sponsor: Nemours Children's Clinic
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to gather detailed information about individuals with various forms of microcephalic primordial dwarfism and similar conditions. By creating a registry—a database of patient information—researchers hope to better understand these conditions and improve care for those affected. Individuals diagnosed with conditions such as MOPDII, Meier-Gorlin syndrome, or similar types of dwarfism might be suitable candidates for this trial.

As an unphased study, this trial offers a unique opportunity to contribute to a deeper understanding of these rare conditions, potentially enhancing future care and treatment options.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications.

Why are researchers excited about this trial?

Researchers are excited about the Primordial Dwarfism Registry because it provides a comprehensive approach to understanding this rare condition, which currently lacks standardized treatments. By tracking various health data from individuals with primordial dwarfism, the registry aims to uncover patterns and insights that could lead to better management and care options. Unlike traditional treatments that only address symptoms, this registry could pave the way for more targeted and effective interventions in the future.

Who Is on the Research Team?

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Angela Duker, MS

Principal Investigator

Nemours

Are You a Good Fit for This Trial?

Inclusion Criteria

Individuals with MOPDII, MOPDI/III, Meier-Gorlin syndrome, or unclassified or closely related types of microcephalic primordial dwarfism as diagnosed by a medical provider are eligible for this registry.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Data Collection

Collection and storage of data extracted from medical records, including specialist evaluations, surgical reports, and imaging

5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 years

What Are the Treatments Tested in This Trial?

Interventions

  • Registry

Find a Clinic Near You

Who Is Running the Clinical Trial?

Nemours Children's Clinic

Lead Sponsor

Trials
128
Recruited
18,000+

Potentials Foundation

Collaborator

Trials
1
Recruited
150+

Walking with Giants Foundation

Collaborator

Trials
1
Recruited
150+