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A Long-Term Observational Study of Patients With Fucosidosis
Sponsor: JCR Pharmaceuticals Co., Ltd.
Summary
The purpose of this observational research study is to learn more about the natural history of fucosidosis, its symptoms, and how it develops over time. This study intends to collect information from participants diagnosed with fucosidosis; however, this study does not include any medication or treatment other than the usual medical care provided to study participants. The information collected in this study will be used to help understand the disease characteristics of fucosidosis; with this information potentially being able to help design future studies and treatments for this disease. There is currently no approved treatment for patients with fucosidosis. The study consists of 2 parts: a) Part A - retrospective data collection, and b) Part B - prospective data collection.
Official title: A Retrospective and Prospective Natural History Study of Patients With Fucosidosis
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
57
Start Date
2026-03-25
Completion Date
2031-01
Last Updated
2026-08-05
Healthy Volunteers
No
Conditions
Locations (16)
Children's Hospital of Orange County
Orange, California, United States
University of Minnesota Health
Minneapolis, Minnesota, United States
Indira Gandhi Institute of Child Health, Department of Pediatric Neurology
Bengaluru, India
Erasmus University Medical Center
Rotterdam, Netherlands
La Rabta Hospital
Tunis, Tunisia
National Institute Mongi-Ben Hamida of Neurology of Tunis
Tunis, Tunisia
Cukurova University, Faculty of Medicine
Adana, Turkey (Türkiye)
Istanbul University Cerrahpasa Medical Faculty Hospital
Istanbul, Turkey (Türkiye)
Marmara University Pendik Training and Research Hospital
Istanbul, Turkey (Türkiye)
Ege University, Faculty of Medicine
Izmir, Turkey (Türkiye)
Dokuz Eylül University Hospital
Izmir, Turkey (Türkiye)
Royal Belfast Hospital for Sick Children
Belfast, United Kingdom
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
National Institute for Health and Care Research Clinical Research Facility, Great Ormond Street Hospital
London, United Kingdom
St. Mary's Hospital, Manchester University NHS Foundation Trust
Manchester, United Kingdom
Barnes Clinical Research Facility, Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust
Salford, United Kingdom