JR-471-NHX
Research type
Research Study
Full title
A Retrospective and Prospective Natural History Study of Patients with Fucosidosis
IRAS ID
363635
Contact name
Sarah O'Mahony
Contact email
Sponsor organisation
JCR Pharmaceuticals Co. Ltd.
Duration of Study in the UK
4 years, 5 months, 26 days
Research summary
The purpose of this research study is to learn more about fucosidosis, its symptoms, and how it progresses over time. As fucosidosis is a rare disease, it is not well understood, and there are no approved treatments to cure it or arrest the progression of the disease. The information collected in this study will be used to help understand the disease and could help design future studies and treatments.
This study is divided into 2 parts, Part A and Part B.
• Part A of the study involves retrospective data collection (collecting data from the past) from participant medical records.
• Part B of the study involves prospective data collection (collecting data in the future) from participant study visits for up to 4 years.REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
25/YH/0254
Date of REC Opinion
26 Jan 2026
REC opinion
Further Information Favourable Opinion