An observational study in Fucosidosis, sponsored by JCR Pharmaceuticals Co., Ltd.. Recruiting at 16 sites in 6 countries. Per ClinicalTrials.gov, last updated 2026-09-17.
Sponsored by JCR Pharmaceuticals Co., Ltd. · Observational
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.
Time Perspective: Both retrospective (Part A) and prospective (Part B).
Enrollment: Part A is anticipated to enroll up to 57 participants for retrospective data collection. Part B is anticipated to enroll up to 31 participants for prospective data collection, the majority of whom are expected to be participants also enrolled in Part A.
123 studies on the registry are indexed under Lysosomal Storage Diseases; 16 are open to participants now.
This study's planned enrollment of 57 is close to the median of 57 across 38 observational studies indexed under Lysosomal Storage Diseases.
Browse Lysosomal Storage Diseases studies →JCR Pharmaceuticals Co., Ltd. is the lead sponsor of 13 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Individuals of any age diagnosed with fucosidosis. Part A: living and deceased participants. Part B: living participants.
Inclusion Criteria (Part A):
Exclusion Criteria (Part A):
Inclusion Criteria (Part B):
Exclusion Criteria (Part B):
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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 years
Plan to share: No
No publications or documents are linked to this record.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
JCR Pharmaceuticals Co., Ltd.