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New hope for kids with rare genetic disorder

NCT ID NCT03485677

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

Summary

This study tested a daily pill called eliglustat in children aged 2 to 18 with Gaucher disease, a rare genetic condition. It aimed to see if the drug was safe, how the body processed it, and if it could help control the disease by improving blood counts, organ size, and bone health. The trial included children already doing well on standard therapy and those with more severe symptoms who also received an additional medication.

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

Locations

  • Investigational Site Number : 0320001

    Capital Federal, Buenos Aires, C1425DUC, Argentina

  • Investigational Site Number : 1240001

    Toronto, Ontario, M5G 1X8, Canada

  • Investigational Site Number : 1240002

    Calgary, Alberta, T3B 6A8, Canada

  • Investigational Site Number : 1240003

    Vancouver, British Columbia, V6H 3V4, Canada

  • Investigational Site Number : 2500002

    Bron, 69677, France

  • Investigational Site Number : 3800002

    Roma, 00165, Italy

  • Investigational Site Number : 3920001

    Minato-ku, Tokyo, 105-8471, Japan

  • Investigational Site Number : 3920002

    Koshigaya-shi, Saitama, 343-8555, 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, Catalunya [Cataluña], 08950, Spain

  • Investigational Site Number : 7240002

    Barakaldo, Bizkaia, 48903, Spain

  • Investigational Site Number : 7240003

    Zaragoza, 50006, Spain

  • Investigational Site Number : 7520001

    Luleå, 97180, 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, B4 6NH, United Kingdom

Conditions

Explore the condition pages connected to this study.

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