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NCT03853876 · ClinicalTrials.gov registry record
A Natural History Study of Aspartylglucosaminuria
A clinical trial, sponsored by Neurogene.
- Terminated
- Registry status
- 8
- Enrollment target
NCT03853876: Clinical Trial study, sponsored by Neurogene.
NCT03853876 is a clinical trial that was terminated before completion, run by Neurogene. The registered enrollment target is 8 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03853876 was terminated before completion, sponsored by Neurogene.
- TERMINATED
- Registry status
- 8 participants
- Enrollment target
Study Summary
Aspartylglucosaminuria (AGU) is a rare neurodegenerative lysosomal storage disease (LSD) characterized by developmental delay, psychomotor regression, worsening intellectual disability, gait disturbance and, ultimately, 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 AGU. This natural history study is important to better understand disease course to be able to determine clinically meaningful outcome measures for use in future clinical trials.
Primary Outcome
Participants will undergo a standardized neuropsychological evaluation every 6-12 months, depending upon the assessments as follows: Global Cognitive: Leiter International Performance Scale, 3rd Ed, Reynolds Intellectual Assessment Scales, 2nd Ed, Mullen Scales of Early Learning Emotional: Aberrant Behavior Checklist, 2nd Ed, Behavior Assessment System for Children, 3rd Ed Behavioral functioning: Aberrant Behavior Checklist, 2nd Ed, Behavior Assessment System for Children, 3rd Ed
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2019-04-18 |
| Est. Completion | 2022-03-17 |
Why NCT03853876 stopped before completion
NCT03853876 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 8 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT03853876 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03853876 about?
NCT03853876 is a clinical study titled "A Natural History Study of Aspartylglucosaminuria". Aspartylglucosaminuria (AGU) is a rare neurodegenerative lysosomal storage disease (LSD) characterized by developmental delay, psychomotor regression, worsening intellectual disability, gait disturbance and, ultimately, premature death, and has no available treatments. The purpose of this study is ...
What is the current status of trial NCT03853876?
This trial is currently terminated. The enrollment target is 8 participants. The study started on 2019-04-18. Estimated completion is 2022-03-17.
Who is sponsoring clinical trial NCT03853876?
This trial is sponsored by Neurogene, which has 6 total clinical trials registered on ClinicalTrials.gov.
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