Experimental drug trial aims to slow rare neurological disease
NCT ID NCT03759678
Summary
This study tested an oral drug called N-Acetyl-L-Leucine (IB1001) to see if it could help people with Ataxia-Telangiectasia (A-T), a rare genetic disorder that affects coordination and movement. The trial involved 17 participants aged 6 and older who took the drug for 6 weeks, followed by a washout period, to measure changes in symptoms, walking, hand function, and quality of life. The goal was to see if the treatment could ease symptoms and potentially slow the progression of the disease.
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Contacts and locations
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Locations
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Hospital Universitario La Paz
Madrid, Spain, Spain
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Royal Papworth Hospital NHS Foundation Trust
Cambridge, Cambridgeshire, CB2 0AY, United Kingdom
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University of California - Los Angeles
Los Angeles, California, 90095, United States
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University of Giessen
Giessen, Germany
Conditions
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