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NCT06776341 · ClinicalTrials.gov registry record
Natural History Study of GEMIN-5 Related Neurodevelopmental Disorder
A clinical trial of Neurodevelopmental Disorders and SMN Complex Proteins, sponsored by University of Pittsburgh.
- Recruiting
- Registry status
- 500
- Enrollment target
- 1
- Study location
NCT06776341: Recruiting study of Neurodevelopmental Disorders and SMN Complex Proteins, sponsored by University of Pittsburgh.
NCT06776341 is a study of Neurodevelopmental Disorders and SMN Complex Proteins that is actively recruiting participants, run by University of Pittsburgh. The registered enrollment target is 500 participants, below the 563-participant average among 27 other Neurodevelopmental Disorders trials with a reported enrollment target (11% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06776341, a study of Neurodevelopmental Disorders and SMN Complex Proteins, is actively recruiting participants, sponsored by University of Pittsburgh.
- RECRUITING
- Registry status
- 500 participants
- Enrollment target
- 1
- Study location
Study Summary
This study will include a comprehensive retrospective chart review and a longitudinal prospective observational natural history study to characterize the phenotypic spectrum of GEMIN5-Related Neurodevelopmental Disorder. We aim to define the trajectory of this ultra-rare disease, core clinical features, characteristics at disease onset and diagnosis, neurological symptomatology, and neuroimaging findings over time. In this study, biological specimens (serum) will also be collected in a biorepository for translational research purposes.
Primary Outcome
time to acquisition and/or loss of milestones
Conditions Studied
Interventions
- OTHER GEMIN5-Related Neurodevelopmental Disorder
Study Locations (1)
Pennsylvania
- Children's Hospital of Pittsburgh of UPMC - Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 500 participants |
| Start Date | 2025-07-07 |
| Est. Completion | 2050-12 |
What NCT06776341 shows while recruiting
NCT06776341 is an observational study that tracks outcomes without assigning an intervention. The registered 500 participants enrollment target is mid-sized for trials with a published cap, below the 563-participant average among 27 other Neurodevelopmental Disorders trials with a reported enrollment target (11% lower).
The record links to 3 conditions, with Neurodevelopmental Disorders appearing as the primary indexed condition, and to 1 intervention - of which GEMIN5-Related Neurodevelopmental Disorder is the first listed.
NCT06776341 reports a single indexed study location in Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT06776341 about?
NCT06776341 is a clinical study titled "Natural History Study of GEMIN-5 Related Neurodevelopmental Disorder". This study will include a comprehensive retrospective chart review and a longitudinal prospective observational natural history study to characterize the phenotypic spectrum of GEMIN5-Related Neurodevelopmental Disorder. We aim to define the trajectory of this ultra-rare disease, core clinical featu...
What is the current status of trial NCT06776341?
This trial is currently recruiting. The enrollment target is 500 participants. The study started on 2025-07-07. Estimated completion is 2050-12.
What conditions does trial NCT06776341 study?
This clinical trial studies the following conditions: Neurodevelopmental Disorders, SMN Complex Proteins, GEMIN5 Protein, Human.
What interventions are being tested in trial NCT06776341?
The interventions under investigation include: GEMIN5-Related Neurodevelopmental Disorder (OTHER).
Who is sponsoring clinical trial NCT06776341?
This trial is sponsored by University of Pittsburgh, which has 1,238 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06776341 being conducted?
This trial has 1 study location across Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT06776341's enrollment target sits among peer trials
500 6th of 27 higher than 21 of 27 other Neurodevelopmental Disorders trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Neurodevelopmental Disorders trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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