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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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01754441
The ClinicalTrials.gov registry entry for NCT01754441 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 25 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 108-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (77% lower). The listed sponsor is Nemours Children's Clinic, which has 108 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 0 interventions.
NCT01754441 reports 2 study locations spanning 2 distinct geographic areas - top geographies include 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.
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Read our methodology - how this data is sourced, computed, and verified.
Related
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