A Long-Term Observational Study of Patients With Fucosidosis

Conditions

• Fucosidosis

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Study Details

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.

Key Dates

Start date

Mar 25, 2026

Status verified

May 2026

Primary completion

Jan 31, 2031

Completion

Jan 31, 2031

Study Design

Enrollment

57 participants (estimated)

Primary Outcome Measure

Evaluate the course of disease progression in individuals with fucosidosis who were/are untreated with any investigational products [ Time Frame: Baseline to up to 4 years ]

Central Contacts

• JCR Pharmaceuticals Co., Ltd.
  +81 797 328582
  [email protected]

Locations (2)

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