A Long-Term Observational Study of Patients With Fucosidosis
ConditionFucosidosis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorJCR Pharmaceuticals Co., Ltd.
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
Confirmed diagnosis of fucosidosis
Patient either with or without previous allogeneic hematopoietic stem cell transplant (HSCT) for treatment of fucosidosis
Disqualifiers
Patient/parent/caregiver not willing to consent to participate
Patient deceased with no availability of appropriate historical consent, and patient's family/caregivers are either unable to be contacted, or refuse consent to data sharing
Patient is living
Confirmed diagnosis of fucosidosis
Trial population
Individuals of any age diagnosed with fucosidosis. Part A: living and deceased participants. Part B: living participants.
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Evaluate the course of disease progression in individuals with fucosidosis who were/are untreated with any investigational products
These observations are intended to inform possible future treatment studies.
Time frame
Baseline to up to 4 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov