Cure My Disease

Don't miss your cure!

 Log in / Sign up

New oral drug shows promise for kids with gaucher disease

NCT ID NCT03485677

Completed Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for GAUCHER'S DISEASE TYPE I are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Gaucher disease Gaucher disease type I Gaucher disease type III

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GAUCHER'S DISEASE TYPE I GAUCHER'S DISEASE TYPE III

Contacts and locations

Locations

  • Investigational Site Number : 0320001

    Buenos Aires, 1428, Argentina

  • Investigational Site Number : 1240001

    Toronto, Ontario, M5G 1X8, Canada

  • Investigational Site Number : 1240002

    Calgary, Alberta, T3B 6A9, Canada

  • Investigational Site Number : 1240003

    Vancouver, British Columbia, V6H 3N1, Canada

  • Investigational Site Number : 2500002

    Bron, 69500, France

  • Investigational Site Number : 3800002

    Rome, Roma, 00165, Italy

  • Investigational Site Number : 3920001

    Tokyo, 105-8461, Japan

  • Investigational Site Number : 3920002

    Koshigaya, Saitama, 343-0845, Japan

  • Investigational Site Number : 6430001

    Moscow, 119049, Russia

  • Investigational Site Number : 6430002

    Tomsk, 634050, Russia

  • Investigational Site Number : 6430004

    Moscow, 119991, Russia

  • Investigational Site Number : 6430005

    Saint Petersburg, 197341, Russia

  • Investigational Site Number : 7240001

    Esplugues de Llobregat, Barcelona [Barcelona], 08950, Spain

  • Investigational Site Number : 7240002

    Barakaldo, Basque Country, 48903, Spain

  • Investigational Site Number : 7240003

    Zaragoza, 50012, Spain

  • Investigational Site Number : 7520001

    Luleå, 971 80, Sweden

  • Investigational Site Number : 7520002

    Gothenburg, 416 85, Sweden

  • Investigational Site Number : 7920002

    Izmir, 35040, Turkey (Türkiye)

  • Investigational Site Number : 7920003

    Istanbul, 34093, Turkey (Türkiye)

  • Investigational Site Number : 7920004

    Adana, 01300, Turkey (Türkiye)

  • Investigational Site Number : 8260002

    Birmingham, England, B4 6NH, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.