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Rare disease study seeks clues for future treatments

NCT ID NCT07615400

Knowledge-focused Sponsor: JCR Pharmaceuticals Co., Ltd. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 18, 2026 · Updated 4 times

Summary

This study tracks the natural course of fucosidosis, a rare genetic disorder, in up to 57 people. It collects medical history and ongoing health data without giving any experimental treatment. The goal is to better understand the disease and help design future therapies.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 57 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2026

Expected to finish

Jan 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Individuals of any age diagnosed with fucosidosis. Part A: living and deceased participants. Part B: living participants.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 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 Inclusion Criteria (Part B): * Patient is living * Confirmed diagnosis of fucosidosis * Patient either with or without previous allogeneic HSCT for treatment of fucosidosis Exclusion Criteria (Part B): * Patient/parent/caregiver not willing to consent to participate * Current participation in any other interventional or therapeutic study (exception: patients who have previously received bone marrow / HSCT as treatment for fucosidosis are not excluded) * Patients who, in the opinion of the site investigator, would be unable or unsuitable to participate in the demands of the study

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Conditions

The condition(s) this trial relates to.

Brain Diseases, Metabolic, Inborn Congenital, Hereditary, and Neonatal Diseases and Abnormalities fucosidosis hereditary disease inborn carbohydrate metabolic disorder inborn errors of metabolism lysosomal storage disease Lysosomal Storage Diseases, Nervous System

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fucosidosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    16 sites in 6 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Barnes Clinical Research Facility, Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust

    RECRUITING

    Salford, M6 8HD, United Kingdom

  • Birmingham Women's and Children's NHS Foundation Trust

    RECRUITING

    Birmingham, B4 6NH, United Kingdom

  • Children's Hospital of Orange County

    RECRUITING

    Orange, California, 92868, United States

  • Cukurova University, Faculty of Medicine

    NOT_YET_RECRUITING

    Adana, 01339, Turkey (Türkiye)

  • Dokuz Eylül University Hospital

    RECRUITING

    Izmir, 35330, Turkey (Türkiye)

  • Ege University, Faculty of Medicine

    NOT_YET_RECRUITING

    Izmir, 35100, Turkey (Türkiye)

  • Erasmus University Medical Center

    RECRUITING

    Rotterdam, 3015 AA, Netherlands

  • Indira Gandhi Institute of Child Health, Department of Pediatric Neurology

    NOT_YET_RECRUITING

    Bengaluru, 560029, India

  • Istanbul University Cerrahpasa Medical Faculty Hospital

    RECRUITING

    Istanbul, 34098, Turkey (Türkiye)

  • La Rabta Hospital

    NOT_YET_RECRUITING

    Tunis, 1007, Tunisia

  • Marmara University Pendik Training and Research Hospital

    RECRUITING

    Istanbul, 34899, Turkey (Türkiye)

  • National Institute Mongi-Ben Hamida of Neurology of Tunis

    NOT_YET_RECRUITING

    Tunis, 1007, Tunisia

  • National Institute for Health and Care Research Clinical Research Facility, Great Ormond Street Hospital

    NOT_YET_RECRUITING

    London, WC1N 3JH, United Kingdom

  • Royal Belfast Hospital for Sick Children

    NOT_YET_RECRUITING

    Belfast, BT12 6BA, United Kingdom

  • St. Mary's Hospital, Manchester University NHS Foundation Trust

    NOT_YET_RECRUITING

    Manchester, M13 9WL, United Kingdom

  • University of Minnesota Health

    NOT_YET_RECRUITING

    Minneapolis, Minnesota, 55455, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.