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NCT03822650 · ClinicalTrials.gov registry record
A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)
A clinical trial of Ceroid Lipofuscinosis, Neuronal 5, sponsored by Neurogene.
- Completed
- Registry status
- 37
- Enrollment target
- 1
- Study location
NCT03822650: Completed study of Ceroid Lipofuscinosis, Neuronal 5, sponsored by Neurogene.
NCT03822650 is a study of Ceroid Lipofuscinosis, Neuronal 5 that has completed, run by Neurogene. The registered enrollment target is 37 participants. The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03822650, a study of Ceroid Lipofuscinosis, Neuronal 5, has completed, sponsored by Neurogene.
- COMPLETED
- Registry status
- 37 participants
- Enrollment target
- 1
- Study location
Study Summary
CLN5 is a form of Batten Disease, a neurodegenerative disorder in children causing psychomotor regression, seizures, blindness, loss of ambulation and premature death, and has no available treatments. The purpose of this study is to investigate the clinical characteristics and natural clinical progression of symptoms in individuals with CLN5. This natural history study is important to better understand disease course to be able to determine clinically relevant outcome measures for use in future clinical trials.
Primary Outcome
Disease-specific clinical assessment used to assess physical, seizure, behavioral and functional capabilities. For physical assessments scores range from 0 to 4 with the score of 4 being most severe.
Conditions Studied
Study Locations (1)
New York
- University of Rochester Medical Center - Rochester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 37 participants |
| Start Date | 2019-03-13 |
| Est. Completion | 2024-05-30 |
What the finished NCT03822650 record still lists
NCT03822650 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 37 participants, a relatively small participant target.
The record links to 1 condition, with Ceroid Lipofuscinosis, Neuronal 5 appearing as the primary indexed condition, and to 0 interventions.
NCT03822650 reports a single indexed study location in New York.
Frequently Asked Questions
What is clinical trial NCT03822650 about?
NCT03822650 is a clinical study titled "A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)". CLN5 is a form of Batten Disease, a neurodegenerative disorder in children causing psychomotor regression, seizures, blindness, loss of ambulation and premature death, and has no available treatments. The purpose of this study is to investigate the clinical characteristics and natural clinical prog...
What is the current status of trial NCT03822650?
This trial is currently completed. The enrollment target is 37 participants. The study started on 2019-03-13. Estimated completion is 2024-05-30.
What conditions does trial NCT03822650 study?
This clinical trial studies the following conditions: Ceroid Lipofuscinosis, Neuronal 5.
Who is sponsoring clinical trial NCT03822650?
This trial is sponsored by Neurogene, which has 6 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03822650 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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