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NCT01754441 · ClinicalTrials.gov registry record
Mechanisms of Cell Death in Spinal Muscular Atrophy
A clinical trial of Spinal Muscular Atrophy, sponsored by Nemours Children's Clinic.
- Completed
- Registry status
- 25
- Enrollment target
- 2
- Study locations
NCT01754441: Completed study of Spinal Muscular Atrophy, sponsored by Nemours Children's Clinic.
NCT01754441 is a study of Spinal Muscular Atrophy that has completed, run by Nemours Children's Clinic. The registered enrollment target is 25 participants, below the 108-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (77% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01754441, a study of Spinal Muscular Atrophy, has completed, sponsored by Nemours Children's Clinic.
- COMPLETED
- Registry status
- 25 participants
- Enrollment target
- 2
- Study locations
Study Summary
Spinal muscular atrophy is a genetically based disease that affects motor neurons in the spinal cord and leads to muscle wasting and weakness. The gene found to be responsible for the underlying disease is called the SMN or survival motor neuron gene. Individuals with SMA are either missing a copy of the gene or have a mutation in the gene. Although the gene has been identified, how it actually causes the motor neurons to die and leads to muscle wasting and weakness is not completely understood. The investigators have found that skin cells from children with SMA tend to be more susceptible to cell death when exposed to cell death inducing agents. In this protocol, The investigators wish to study the mechanisms by which these cells die when exposed to these agents and how this may be related to the gene defect and the disease.
Primary Outcome
Established fibroblast lines from SMA patients will be immunolabeled with antibodies directed against SMN and examined for changes in the nuclear localization of SMN in gems.
Conditions Studied
Study Locations (2)
Delaware
- Alfred I. duPont Hospital for Children - Wilmington
Florida
- Nemours Children's Specialty Care, Jacksonville - Jacksonville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2008-05 |
| Est. Completion | 2020-02 |
What the finished NCT01754441 record still lists
NCT01754441 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 25 participants, a relatively small participant target, below the 108-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (77% lower).
The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 0 interventions.
NCT01754441 reports a single indexed study location in Delaware, Florida.
Frequently Asked Questions
What is clinical trial NCT01754441 about?
NCT01754441 is a clinical study titled "Mechanisms of Cell Death in Spinal Muscular Atrophy". Spinal muscular atrophy is a genetically based disease that affects motor neurons in the spinal cord and leads to muscle wasting and weakness. The gene found to be responsible for the underlying disease is called the SMN or survival motor neuron gene. Individuals with SMA are either missing a copy o...
What is the current status of trial NCT01754441?
This trial is currently completed. The enrollment target is 25 participants. The study started on 2008-05. Estimated completion is 2020-02.
What conditions does trial NCT01754441 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy.
Who is sponsoring clinical trial NCT01754441?
This trial is sponsored by Nemours Children's Clinic, which has 108 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01754441 being conducted?
This trial has 2 study locations across Delaware, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Spinal Muscular Atrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01754441's enrollment target sits among peer trials
25 18th of 22 higher than 5 of 22 other Spinal Muscular Atrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Spinal Muscular Atrophy 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.
Source ClinicalTrials.gov registry export · 2026-08-08
-
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