Drug trial targets rare genetic overgrowth disorder

NCT ID NCT02443818

Summary

This study tested whether the drug sirolimus could stop the progression of abnormal, excessive growth in specific body parts of people with a genetic condition called PIK3CA-related overgrowth. Sixteen participants, aged 3 to 65, took sirolimus for six months after a six-month monitoring period. The main goal was to see if the drug could reduce or halt the growth of the overgrown tissue, measured by MRI scans.

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Contacts and locations

Locations

  • CHRU de Montpellier - Hôpital Saint-Eloi

    Montpellier, 34295, France

  • CHRU de Tours

    Tours, 37044, France

  • CHU Paris - Necker

    Paris, 75015, France

  • CHU d'Angers

    Angers, 49933, France

  • CHU de Bordeaux - GH Pellegrin

    Bordeaux, 33076, France

  • CHU de DIJON

    Dijon, 21079, France

  • HCL Lyon - CH Lyon Sud

    Pierre-Bénite, 69495, France

  • Hopitaux de Brabois Chu Nancy

    Nancy, 54511, France

  • Hôpital Jeanne de Flandre

    Lille, 59037, France

  • Hôpital mère-enfant de Nantes

    Nantes, 44093, France

  • Pôle Cardiovasculaire et Métabolique - Hôpital Larrey

    Toulouse, 31059, France

Conditions

Explore the condition pages connected to this study.