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NCT03810508 · ClinicalTrials.gov registry record
A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
A clinical trial, sponsored by Neurogene.
- Terminated
- Registry status
- 21
- Enrollment target
NCT03810508: Clinical Trial study, sponsored by Neurogene.
NCT03810508 is a clinical trial that was terminated before completion, run by Neurogene. The registered enrollment target is 21 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03810508 was terminated before completion, sponsored by Neurogene.
- TERMINATED
- Registry status
- 21 participants
- Enrollment target
Study Summary
Charcot-Marie-Tooth 4J (CMT4J) is a rare inherited peripheral neuropathy often characterized by rapidly progressive, asymmetrical upper and lower extremity weakness, muscle atrophy leading to loss of ambulation, respiratory compromise and premature death with no available treatment. The purpose of this study is to investigate the clinical characteristics and natural clinical progression of symptoms in individuals with CMT4J. 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
This an 11 item scale is used in patients younger than 18 yrs of age and generates a linear score of disability.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 21 participants |
| Start Date | 2019-07-18 |
| Est. Completion | 2022-02-15 |
Why NCT03810508 stopped before completion
NCT03810508 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 21 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT03810508 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03810508 about?
NCT03810508 is a clinical study titled "A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)". Charcot-Marie-Tooth 4J (CMT4J) is a rare inherited peripheral neuropathy often characterized by rapidly progressive, asymmetrical upper and lower extremity weakness, muscle atrophy leading to loss of ambulation, respiratory compromise and premature death with no available treatment. The purpose of ...
What is the current status of trial NCT03810508?
This trial is currently terminated. The enrollment target is 21 participants. The study started on 2019-07-18. Estimated completion is 2022-02-15.
Who is sponsoring clinical trial NCT03810508?
This trial is sponsored by Neurogene, which has 6 total clinical trials registered on ClinicalTrials.gov.
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