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NCT01971957 · ClinicalTrials.gov registry record
Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers
A clinical trial, sponsored by University of Nebraska.
- Completed
- Registry status
- 20
- Enrollment target
NCT01971957: Completed study, sponsored by University of Nebraska.
NCT01971957 is a clinical trial that has completed, run by University of Nebraska. The registered enrollment target is 20 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01971957 has completed, sponsored by University of Nebraska.
- COMPLETED
- Registry status
- 20 participants
- Enrollment target
Study Summary
Sjogren-Larsson syndrome (SLS) is a rare genetic disease in which patients typically exhibit ichthyosis (dry, scaly skin), intellectual disability, spasticity, seizures and a distinctive maculopathy. The purpose of this study is to define the clinical spectrum and natural history of Sjogren-Larsson syndrome, and identify biomarkers that correlate with disease phenotype while establishing a registry for future investigations of biochemical pathogenesis and therapy.
Primary Outcome
Determine the spectrum of clinical disease severity and changes in severity of symptoms over time. Each organ system will be evaluated using validated clinical exams (for example, Modified Ashworth Spasticity Score for neurologic severity) or categorical tests (such as EEG normal or abnormal). The clinical data will be used to develop a quantitative SLS severity score whereby patients will be described (for example, overall severity 1 to 5 with score 1 being the mildest phenotype and score 5 bei
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2013-04-01 |
| Est. Completion | 2023-09-30 |
What the finished NCT01971957 record still lists
NCT01971957 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 20 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT01971957 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01971957 about?
NCT01971957 is a clinical study titled "Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers". Sjogren-Larsson syndrome (SLS) is a rare genetic disease in which patients typically exhibit ichthyosis (dry, scaly skin), intellectual disability, spasticity, seizures and a distinctive maculopathy. The purpose of this study is to define the clinical spectrum and natural history of Sjogren-Larsson ...
What is the current status of trial NCT01971957?
This trial is currently completed. The enrollment target is 20 participants. The study started on 2013-04-01. Estimated completion is 2023-09-30.
Who is sponsoring clinical trial NCT01971957?
This trial is sponsored by University of Nebraska, which has 407 total clinical trials registered on ClinicalTrials.gov.
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