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Mechanisms of Cell Death in Spinal Muscular Atrophy
NCT01754441 · View on ClinicalTrials.gov ↗
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 |
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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. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 25 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Nemours Children's Clinic, which has 96 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT01754441 reports 2 study locations spanning 2 distinct geographic areas — top geographies include Delaware, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT01754441?
This trial is sponsored by Nemours Children's Clinic, which has 96 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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