A Retrospective and Prospective Natural History Study of Patients With Fucosidosis
Status: Recruiting
Location: See all (16) locations...
Study Type: Observational
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.
Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
⁃ \- Confirmed diagnosis of fucosidosis
• Patient is alive
• Confirmed diagnosis of fucosidosis
Locations
United States
California
Children's Hospital of Orange County
RECRUITING
Orange
Minnesota
University of Minnesota Health
NOT_YET_RECRUITING
Minneapolis
Other Locations
India
Indira Gandhi Institute of Child Health, Department of Pediatric Neurology
NOT_YET_RECRUITING
Bengaluru
Netherlands
Erasmus University Medical Center
NOT_YET_RECRUITING
Rotterdam
Tunisia
La Rabta Hospital
NOT_YET_RECRUITING
Tunis
National Institute Mongi-Ben Hamida of Neurology of Tunis
NOT_YET_RECRUITING
Tunis
Turkey
Cukurova University, Faculty of Medicine
NOT_YET_RECRUITING
Adana
Istanbul University Cerrahpasa Medical Faculty Hospital
NOT_YET_RECRUITING
Istanbul
Marmara University Pendik Training and Research Hospital
NOT_YET_RECRUITING
Istanbul
Dokuz Eylül University Hospital
NOT_YET_RECRUITING
Izmir
Ege University, Faculty of Medicine
NOT_YET_RECRUITING
Izmir
United Kingdom
Royal Belfast Hospital for Sick Children
NOT_YET_RECRUITING
Belfast
Birmingham Women's and Children's NHS Foundation Trust
NOT_YET_RECRUITING
Birmingham
National Institute for Health and Care Research Clinical Research Facility, Great Ormond Street Hospital
NOT_YET_RECRUITING
London
St. Mary's Hospital, Manchester University NHS Foundation Trust
NOT_YET_RECRUITING
Manchester
Barnes Clinical Research Facility, Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust