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A Long-Term Observational Study of Patients With Fucosidosis

NCT07615400 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 57

Last updated 2026-05-29

No results posted yet for this study

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.

Conditions

  • Fucosidosis

Sponsors & Collaborators

  • JCR Pharmaceuticals Co., Ltd.

    lead INDUSTRY

Principal Investigators

  • Karolina M Stepien, MD · Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-03-25
Primary Completion
2031-01-31
Completion
2031-01-31

Countries

  • United States
  • India
  • Netherlands
  • Tunisia
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07615400 on ClinicalTrials.gov