New drug shows promise in controlling rare fat disorder

NCT ID NCT05130450

First seen Jan 05, 2026 · Last updated May 16, 2026 · Updated 25 times

Summary

This study looks at how well the drug olezarsen works over time to lower fat levels in the blood of people with familial chylomicronemia syndrome (FCS), a rare genetic condition. About 60 participants who completed a prior study will receive the drug for up to 3 years. The main goal is to see if fasting triglycerides drop significantly and stay lower.

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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

  • Academisch Medisch Centrum Goederenontvangst Apotheek t.a.v. Kenniscentrum

    Amsterdam, Noor-Holland, 1105 AZ, Netherlands

  • Apokteket AB

    Malmö, 211 24, Sweden

  • Azienda Ospedaliero Universitaria Policlinico Umberto I

    Rome, 00161, Italy

  • CTSI Investigational Pharmacy

    New York, New York, 10016, United States

  • Clinique des Maladies Lipidiques de Quebec Inc.

    Québec, Quebec, G1V 4W2, Canada

  • Department of Pharmacy

    Park Ridge, Illinois, 60068, United States

  • Diabetes/Lipid Management & Research Center

    Huntington Beach, California, 92648, United States

  • Dra Margarida Falcao Centro Hospitalar Lisboa Ocidental Hospital Egas Moniz

    Lisbon, 1340-019, Portugal

  • Ecogene-21

    Chicoutimi, Quebec, G7H 7K9, Canada

  • Excel Medical Clinical Trials, LLC

    Boca Raton, Florida, 33434, United States

  • Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique

    Bron, 69677, France

  • Hospital Clínic Barcelona C/ Villarroel

    Barcelona, 08036, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Hospital Universitario Virgen del Rocío

    Seville, 41013, Spain

  • Hospital da Senhora da Oliveira Guimaraes

    Creixomil, 4835-044, Portugal

  • Hôpital Bicêtre

    Le Kremlin-Bicêtre, 94270, France

  • IDS Central

    Philadelphia, Pennsylvania, 19104, United States

  • Institute de Recherches Cliniques de Montreal

    Montreal, Quebec, H2W 1R7, Canada

  • Metabolicke centrum MU

    Bratislava, 83101, Slovakia

  • Milstein Hospital

    New York, New York, 10032, United States

  • Nathalie Saint-Pierre

    Montreal, Quebec, H2W 1R7, Canada

  • Oslo Hospital Pharmacy Rikshospitalet

    Oslo, 0372, Norway

  • Pharmacie Hopital de la Conception

    Marseille, 05 13385, France

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • UOC di Farmacia AOUP PAOLO GIACCONE

    Palermo, 90127, Italy

  • University of Kansas Medical Center (KUMC)

    Kansas City, Kansas, 66160, United States

  • Via Sergio Pansini 5

    Naples, 80131, Italy

Conditions

Explore the condition pages connected to this study.