New pill shows promise for kids with rare enzyme disorder
NCT ID NCT03485677
First seen Jan 21, 2026 · Last updated May 20, 2026 · Updated 18 times
Summary
This study tested an oral medication called eliglustat in 57 children aged 2 to 18 with Gaucher disease types 1 and 3. The goal was to see if the drug is safe and works well, either alone or with another treatment (imiglucerase). Researchers measured drug levels in the blood, side effects, and improvements in blood counts and organ sizes.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Investigational Site Number : 0320001
Capital Federal, Buenos Aires, C1425DUC, Argentina
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Investigational Site Number : 1240001
Toronto, Ontario, M5G 1X8, Canada
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Investigational Site Number : 1240002
Calgary, Alberta, T3B 6A8, Canada
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Investigational Site Number : 1240003
Vancouver, British Columbia, V6H 3V4, Canada
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Investigational Site Number : 2500002
Bron, 69677, France
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Investigational Site Number : 3800002
Roma, 00165, Italy
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Investigational Site Number : 3920001
Minato-ku, Tokyo, 105-8471, Japan
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Investigational Site Number : 3920002
Koshigaya-shi, Saitama, 343-8555, Japan
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Investigational Site Number : 6430001
Moscow, 119049, Russia
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Investigational Site Number : 6430002
Tomsk, 634050, Russia
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Investigational Site Number : 6430004
Moscow, 119991, Russia
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Investigational Site Number : 6430005
Saint Petersburg, 197341, Russia
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Investigational Site Number : 7240001
Esplugues de Llobregat, Catalunya [Cataluña], 08950, Spain
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Investigational Site Number : 7240002
Barakaldo, Bizkaia, 48903, Spain
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Investigational Site Number : 7240003
Zaragoza, 50006, Spain
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Investigational Site Number : 7520001
Luleå, 97180, Sweden
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Investigational Site Number : 7520002
Gothenburg, 416 85, Sweden
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Investigational Site Number : 7920002
Izmir, 35040, Turkey (Türkiye)
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Investigational Site Number : 7920003
Istanbul, 34093, Turkey (Türkiye)
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Investigational Site Number : 7920004
Adana, 01300, Turkey (Türkiye)
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Investigational Site Number : 8260002
Birmingham, B4 6NH, United Kingdom
Conditions
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