New shot aims to boost energy in rare Muscle-Wasting diseases

NCT ID NCT05162768

Summary

This study tested whether a daily injection called elamipretide could help adults with a specific type of genetic mitochondrial disease that causes muscle weakness and fatigue. About 102 participants received either the drug or a placebo for 48 weeks. Researchers measured changes in how far people could walk in six minutes and how quickly they could perform simple strength and mobility tasks.

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Contacts and locations

Locations

  • Akron Children's Hospital

    Akron, Ohio, 44308, United States

  • Azienda Ospedaliero Universitaria Policlinico G. Martino

    Messina, 98125, Italy

  • Azienda Ospedaliero Universitario Pisana, Dipartimento Ambientale di Neuroscienze

    Pisa, 56126, Italy

  • Calvary Health Care Bethlehem

    Parkdale, Victoria, 3162, Australia

  • Columbia University Medical Center College of Physician and Surgeon

    New York, New York, 10032, United States

  • Department of Neurology, University Clinics Munich

    Munich, Bavaria, 80336, Germany

  • Helse Bergen HF

    Bergen, 5021, Norway

  • Hospital Clinic de Barcelona

    Barcelona, 8036, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Hospital de la Sta Creu i Sant Pau

    Barcelona, 8025, Spain

  • Hospital la Fe de Valencia

    Valencia, 46026, Spain

  • IRCCS Institute of Neorological Sciences of Bologna Bellaria Hospital

    Bologna, Italy

  • Istituto Nazionale Neurologico Carlo Besta

    Milan, 20133, Italy

  • Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli, Università Cattolica del Sacro Cuore

    Rome, Lazio, 00168, Italy

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Newcastle upon Tyne Hospitals Freeman Hospital

    Newcastle, NE77DN, United Kingdom

  • Queen Square Centre for Neuromuscular Diseases The National Hospital for Neurology and Neurosurgery

    London, WC1N 3BG, United Kingdom

  • Radboud University Medical Center

    Nijmegen, 6525, Netherlands

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30329, United States

  • Royal North Shore Hospital Neurology

    Sydney, New South Wales, 2065, Australia

  • Semmelweis Egyetem Genomikai Medicina es Ritka Betegsegek

    Budapest, 1083, Hungary

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • UT Health,Center for the Treatment of Pediatric Neurodegenerative Disease

    Houston, Texas, 77030, United States

  • Univ of Tubingen, Hertie Institute for Clinical Brain Research

    Tübingen, Germany

  • Universitaetsklinikum Carl Gustav Carus Dresden Neurologie

    Dresden, Germany

  • University of Auckland - Auckland City Hospital, Neurology Department

    Auckland, 1023, New Zealand

  • University of Brescia, NeMO Clinical Center for Neuromuscular Diseases

    Gussago, Brescia, 25064, Italy

  • University of California, San Diego

    San Diego, California, 92093, United States

  • University of Cambridge, Department of Clinical Neurosciences

    Cambridge, CB2 0QQ, United Kingdom

  • University of Pittsburgh School of Medicine Children's Hospital of Pittsburgh of UPMC Department of Genetics

    Pittsburgh, Pennsylvania, 15224, United States

  • University of Pécs, Department of Neurology Klinikai Kozpont - neurologiai Klinika

    Pécs, 7624, Hungary

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.