A Long-Term Observational Study of Patients With Fucosidosis
Recruiting
Observational Study
Fucosidosis
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 57 (estimated)
- Sponsor
- JCR Pharmaceuticals Co., Ltd. · Industry
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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 …
Trial Locations
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Eligibility Criteria
Inclusion Criteria (Part A):
* Confirmed diagnosis of fucosidosis
* Patient either with or without previous allogeneic hematopoietic stem cell transplant (HSCT) for treatment of fucosidosis
Exclusion Criteria (Part A):
* Patient/parent/caregiver not willing to consent to participate
* Patient dec…
Contacts