Actively Recruiting
A Retrospective and Prospective Natural History Study of Patients With Fucosidosis
Led by JCR Pharmaceuticals Co., Ltd. · Updated on 2026-05-29
57
Participants Needed
16
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to better understand fucosidosis, a rare disease, by studying its natural history, symptoms, and how it progresses over time. The study collects information from people diagnosed with fucosidosis without providing any new treatments, relying only on the usual medical care. The findings may help design future studies and treatments for this condition. The study includes two parts: Part A gathers past medical data from up to 57 participants, while Part B involves ongoing data collection from up to 31 participants, most of whom may have participated in Part A. No investigational drugs or therapies are given during the study. Participants will provide information through medical records and ongoing observations, helping researchers evaluate how fucosidosis progresses over up to four years. The study team will monitor disease changes and collect data to improve understanding, without altering the participants’ usual care. The study is expected to continue until January 2031.
CONDITIONS
Brief Title
A Long-Term Observational Study of Patients With Fucosidosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of fucosidosis (Part A)
- Patient is alive (Part B)
- Confirmed diagnosis of fucosidosis (Part B)
You will not qualify if you...
- Patient, parent, or caregiver not willing to consent to participate (Parts A and B)
- Patient deceased with no available historical consent and family/caregivers cannot be contacted or refuse consent (Part A)
- Current participation in an interventional or therapeutic study (Part B)
- Considered unable or unsuitable to participate in study demands by site investigator (Part B)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 4 years
Participants are observed over time to evaluate the progression of fucosidosis without receiving investigational treatments.
Trial Site Locations
Total: 16 locations
1
Children's Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
2
University of Minnesota Health
Minneapolis, Minnesota, United States, 55455
Not Yet Recruiting
3
Indira Gandhi Institute of Child Health, Department of Pediatric Neurology
Bengaluru, India, 560029
Not Yet Recruiting
4
Erasmus University Medical Center
Rotterdam, Netherlands, 3015 AA
Not Yet Recruiting
5
La Rabta Hospital
Tunis, Tunisia, 1007
Not Yet Recruiting
6
National Institute Mongi-Ben Hamida of Neurology of Tunis
Tunis, Tunisia, 1007
Not Yet Recruiting
7
Cukurova University, Faculty of Medicine
Adana, Turkey (Türkiye), 01339
Not Yet Recruiting
8
Istanbul University Cerrahpasa Medical Faculty Hospital
Istanbul, Turkey (Türkiye), 34098
Not Yet Recruiting
9
Marmara University Pendik Training and Research Hospital
Istanbul, Turkey (Türkiye), 34899
Not Yet Recruiting
10
Ege University, Faculty of Medicine
Izmir, Turkey (Türkiye), 35100
Not Yet Recruiting
11
Dokuz Eylül University Hospital
Izmir, Turkey (Türkiye), 35330
Not Yet Recruiting
12
Royal Belfast Hospital for Sick Children
Belfast, United Kingdom, BT12 6BA
Not Yet Recruiting
13
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom, B4 6NH
Not Yet Recruiting
14
National Institute for Health and Care Research Clinical Research Facility, Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Not Yet Recruiting
15
St. Mary's Hospital, Manchester University NHS Foundation Trust
Manchester, United Kingdom, M13 9WL
Not Yet Recruiting
16
Barnes Clinical Research Facility, Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust
Salford, United Kingdom, M6 8HD
Actively Recruiting
Research Team
J
JCR Pharmaceuticals Co., Ltd.
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
Frequently Asked Questions
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