A Long-Term Observational Study of Patients With Fucosidosis
Recruiting now
Conditions studied: Fucosidosis
In brief
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.
Key facts
- Study ID
- NCT07615400
- Run by
- JCR Pharmaceuticals Co., Ltd.
- People needed
- 57
- Starts
- 2026-03-25
- Expected to finish
- 2031-01-01
- Last updated by the study team
- 2026-08-05
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Children's Hospital of Orange County — Orange, California, United States (enrolling)
- Barnes Clinical Research Facility, Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust — Salford, United Kingdom (enrolling)
- 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
- University of Minnesota Health — Minneapolis, Minnesota, United States
Full record on ClinicalTrials.gov
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