Hope for young FA patients: groundbreaking trial tests first approved drug in kids
NCT ID NCT06953583
Summary
This study is testing a drug called omaveloxolone (SKYCLARYS®) in children and teenagers aged 2 to 15 with Friedreich's ataxia (FA), a rare genetic disease that affects movement and coordination. The drug is already approved for adults, but its safety and effects in younger patients are unknown. Researchers will measure if the drug improves symptoms like balance and stability, and closely monitor the children's overall health, heart function, and development over a period of up to three years.
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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Study contacts
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Contact
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AP-HP - Hôpital Armand Trousseau
RECRUITINGParis, 75012, France
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All India Institute of Medical Sciences (AIIMS) - New Delhi
NOT_YET_RECRUITINGNew Delhi, National Capital Territory of Delhi, 110029, India
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CHI at Temple Street
RECRUITINGDublin, D01 XD99, Ireland
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CHKD's Health Center - South Campus - PIN
RECRUITINGNorfolk, Virginia, 23507-1910, United States
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CHU de Montpellier- Hôpital Gui De Chauliac
RECRUITINGMontpellier, Hérault, 34090, France
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CHU de Quebec -Universite Laval
RECRUITINGQuébec, Quebec, G1V 4G2, Canada
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Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITINGMilan, 20133, Italy
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Hospital Sant Joan de Deu - PIN
RECRUITINGEspluges de Llobregat, Barcelona, 8950, Spain
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Hospital Universitario La Paz - PPDS
RECRUITINGMadrid, 28046, Spain
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IRCCS Eugenio Medea - Polo. Scientifico Veneto
NOT_YET_RECRUITINGConegliano, Veneto, 31015, Italy
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Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
NOT_YET_RECRUITINGIstanbul, 34093, Turkey (Türkiye)
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John Radcliffe Hospital
RECRUITINGOxford, Oxfordshire, OX3 9DU, United Kingdom
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King Faisal Specialist Hospital & Research Centre
NOT_YET_RECRUITINGRiyadh, Ar Riya, 12875, Saudi Arabia
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L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME
RECRUITINGBrasília, Federal District, 70200-730, Brazil
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McGill University
RECRUITINGMontreal, Quebec, H3H 2R9, Canada
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Murdoch Childrens Research Institute (MCRI)
RECRUITINGParkville, Victoria, 3052, Australia
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Norman Fixel Institute for Neurological Diseases UF Health
RECRUITINGGainesville, Florida, 32610-3010, United States
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Ospedale Pediatrico Bambino Gesù IRCCS
NOT_YET_RECRUITINGRome, Lazio, 165, Italy
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PSEG Centro de Pesquisa Clinica
RECRUITINGSão Paulo, São Paulo, 04024-002, Brazil
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Radboud Universitair Medisch Centrum
NOT_YET_RECRUITINGNijmegen, 6525 GA, Netherlands
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Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen
NOT_YET_RECRUITINGCopenhagen, 2100, Denmark
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105-3901, United States
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Sheffield Children's Hospital - PPDS
RECRUITINGSheffield, South Yorkshire, S10 5DD, United Kingdom
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St. Jude Children's Research Hospital - PIN
RECRUITINGMemphis, Tennessee, 38105-3678, United States
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Sydney Children's Hospital
NOT_YET_RECRUITINGRandwick, New South Wales, 2031, Australia
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UCLA Neurology Outpatient Clinic at Westwood
NOT_YET_RECRUITINGLos Angeles, California, 90095, United States
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UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen
RECRUITINGGiessen, 35392, Germany
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USF Health Morsani College of Medicine Department of Neurology
RECRUITINGTampa, Florida, 33612, United States
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University College Hospital - PPDS
RECRUITINGLondon, Lincolnshire, NW1 2BU, United Kingdom
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University of Campinas (UNICAMP) School of Medical Sciences
NOT_YET_RECRUITINGCampinas, São Paulo, 13083-970, Brazil
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Universitätsklinikum Aachen
RECRUITINGAachen, North Rhine-Westphalia, 52074, Germany
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Universitätsklinikum Hamburg Eppendorf
RECRUITINGHamburg, 20246, Germany
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Universitätsklinikum Innsbruck
RECRUITINGInnsbruck, 6020, Austria
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Conditions
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