New hope for kids with gaucher disease: drug trial shows promise
NCT ID NCT03485677
First seen Jan 21, 2026 · Last updated May 15, 2026 · Updated 17 times
Summary
This study tested the drug eliglustat in 57 children aged 2 to 18 with Gaucher disease types 1 and 3. The goal was to see how safe the drug is, how the body processes it, and whether it helps control the disease. Some children also took another drug called imiglucerase. The study measured side effects, blood counts, and organ sizes to see if eliglustat works well in kids.
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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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