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Rare disease study seeks clues for future treatments

NCT ID NCT07615400

Recruiting now Knowledge-focused Sponsor: JCR Pharmaceuticals Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 06, 2026 · Updated 2 times

Summary

This study tracks the natural course of fucosidosis, a rare genetic disorder, in up to 57 people. It collects medical history and ongoing health data without giving any experimental treatment. The goal is to better understand the disease and help design future therapies.

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Conditions

The condition(s) this trial relates to.

Brain Diseases, Metabolic, Inborn Congenital, Hereditary, and Neonatal Diseases and Abnormalities fucosidosis hereditary disease inborn carbohydrate metabolic disorder inborn errors of metabolism lysosomal storage disease Lysosomal Storage Diseases, Nervous System

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FUCOSIDOSIS

Contacts and locations

Locations

  • Barnes Clinical Research Facility, Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust

    RECRUITING

    Salford, M6 8HD, United Kingdom

  • Birmingham Women's and Children's NHS Foundation Trust

    NOT_YET_RECRUITING

    Birmingham, B4 6NH, United Kingdom

  • Children's Hospital of Orange County

    RECRUITING

    Orange, California, 92868, United States

  • Cukurova University, Faculty of Medicine

    NOT_YET_RECRUITING

    Adana, 01339, Turkey (Türkiye)

  • Dokuz Eylül University Hospital

    NOT_YET_RECRUITING

    Izmir, 35330, Turkey (Türkiye)

  • Ege University, Faculty of Medicine

    NOT_YET_RECRUITING

    Izmir, 35100, Turkey (Türkiye)

  • Erasmus University Medical Center

    NOT_YET_RECRUITING

    Rotterdam, 3015 AA, Netherlands

  • Indira Gandhi Institute of Child Health, Department of Pediatric Neurology

    NOT_YET_RECRUITING

    Bengaluru, 560029, India

  • Istanbul University Cerrahpasa Medical Faculty Hospital

    NOT_YET_RECRUITING

    Istanbul, 34098, Turkey (Türkiye)

  • La Rabta Hospital

    NOT_YET_RECRUITING

    Tunis, 1007, Tunisia

  • Marmara University Pendik Training and Research Hospital

    NOT_YET_RECRUITING

    Istanbul, 34899, Turkey (Türkiye)

  • National Institute Mongi-Ben Hamida of Neurology of Tunis

    NOT_YET_RECRUITING

    Tunis, 1007, Tunisia

  • National Institute for Health and Care Research Clinical Research Facility, Great Ormond Street Hospital

    NOT_YET_RECRUITING

    London, WC1N 3JH, United Kingdom

  • Royal Belfast Hospital for Sick Children

    NOT_YET_RECRUITING

    Belfast, BT12 6BA, United Kingdom

  • St. Mary's Hospital, Manchester University NHS Foundation Trust

    NOT_YET_RECRUITING

    Manchester, M13 9WL, United Kingdom

  • University of Minnesota Health

    NOT_YET_RECRUITING

    Minneapolis, Minnesota, 55455, United States

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