New oral drug shows promise for kids with gaucher disease
NCT ID NCT03485677
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested the safety and effectiveness of eliglustat, an oral medication, in 57 children aged 2 to 17 with Gaucher disease types 1 and 3. Some children also received the standard enzyme therapy imiglucerase. Researchers measured how the drug moves through the body, side effects, and changes in blood counts and organ sizes. The goal was to see if eliglustat could be a good treatment option for young patients.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
eliglustat (Cerdelga) and imiglucerase (Cerezyme)
What this could lead to
If successful, this could provide a safe and effective oral treatment option for children with Gaucher disease, potentially replacing or reducing the need for intravenous enzyme therapy.
What could go wrong
This is a completed phase 3 trial, but results are not yet widely published. The study is relatively small (57 participants), and long-term benefits or rare side effects may not be fully captured.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Investigational Site Number : 0320001
Buenos Aires, 1428, 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 6A9, Canada
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Investigational Site Number : 1240003
Vancouver, British Columbia, V6H 3N1, Canada
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Investigational Site Number : 2500002
Bron, 69500, France
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Investigational Site Number : 3800002
Rome, Roma, 00165, Italy
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Investigational Site Number : 3920001
Tokyo, 105-8461, Japan
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Investigational Site Number : 3920002
Koshigaya, Saitama, 343-0845, 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, Barcelona [Barcelona], 08950, Spain
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Investigational Site Number : 7240002
Barakaldo, Basque Country, 48903, Spain
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Investigational Site Number : 7240003
Zaragoza, 50012, Spain
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Investigational Site Number : 7520001
Luleå, 971 80, 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, England, B4 6NH, United Kingdom