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NCT03834987 · ClinicalTrials.gov registry record
NGLY1 Deficiency: A Prospective Natural History Study
A clinical trial, sponsored by Stanford University.
- Terminated
- Registry status
- 29
- Enrollment target
NCT03834987: Clinical Trial study, sponsored by Stanford University.
NCT03834987 is a clinical trial that was terminated before completion, run by Stanford University. The registered enrollment target is 29 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03834987 was terminated before completion, sponsored by Stanford University.
- TERMINATED
- Registry status
- 29 participants
- Enrollment target
Study Summary
NGLY1 deficiency is a rare genetic disorder that is characterized by: global developmental delay and/or intellectual disability, hypo- or alacrima, transient elevation of transaminases, and a hyperkinetic movement disorder. Significant phenotypic variability has been observed in the small number of affected individuals described in the medical literature. The purpose of this study is to describe the natural history of NGLY1 deficiency in a prospective, detailed, and highly uniform manner. Study participants will be closely monitored over the course of five years in order to: * understand the clinical spectrum and progression of NGLY1 deficiency using standardized clinical and neurodevelopmental assessments * identify clinical and biomarker endpoints for use in therapeutic trials, and * identify genotype-phenotype correlations Close clinical follow-up will allow for generation of a rich dataset and detailed understanding of the natural history of NGLY1 deficiency.
Interventions
- OTHER Neurodevelopmental Assessment
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 29 participants |
| Start Date | 2019-02-01 |
| Est. Completion | 2021-11-19 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03834987
The ClinicalTrials.gov registry entry for NCT03834987 describes a study currently listed as terminated, categorized as an unspecified phase. The registered enrollment target is 29 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Stanford University, which has 1,744 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Neurodevelopmental Assessment is the first listed.
NCT03834987 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03834987 about?
NCT03834987 is a clinical study titled "NGLY1 Deficiency: A Prospective Natural History Study". NGLY1 deficiency is a rare genetic disorder that is characterized by: global developmental delay and/or intellectual disability, hypo- or alacrima, transient elevation of transaminases, and a hyperkinetic movement disorder. Significant phenotypic variability has been observed in the small number of ...
What is the current status of trial NCT03834987?
This trial is currently terminated. The enrollment target is 29 participants. The study started on 2019-02-01. Estimated completion is 2021-11-19.
What interventions are being tested in trial NCT03834987?
The interventions under investigation include: Neurodevelopmental Assessment (OTHER).
Who is sponsoring clinical trial NCT03834987?
This trial is sponsored by Stanford University, which has 1,744 total clinical trials registered on ClinicalTrials.gov.
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