Measuring Levels of SMN in Blood Samples of SMA Patients
Completed
Conditions studied: Spinal Muscular Atrophy
In brief
Spinal muscular atrophy (SMA) is a disorder that affects the motor neurons. SMA is caused by a mutation in a part of the DNA called the survival motor neuron (SMN1) gene, which normally produces a protein called SMN. Because of their gene mutation, people with SMA make less SMN protein, which results in the loss of motor neurons. SMA symptoms may be improved by increasing the levels of SMN protein. The purpose of this study is to determine whether a drug called a histone deacetylase inhibitor can increase SMN levels. After undergoing a general medical and neurological evaluation, study participants will donate a blood sample. Researchers will use this sample to measure SMN levels. They will also isolate cells from the blood and treat the cells with various drugs that may increase SMN levels.
Key facts
- Study ID
- NCT00061607
- Run by
- National Institute of Neurological Disorders and Stroke (NINDS)
- People needed
- 73
- Starts
- 2003-05-19
- Expected to finish
- 2017-04-04
- Last updated by the study team
- 2017-10-06
Who can join
Age: 2 and older. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- History of valproic acid or other HDAC inhibitor use within the past14 days.
- History of bleeding disorder, which would make a blood draw unsafe.
Where it is running
- National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States
Full record on ClinicalTrials.gov
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