NCT07615400 A Long-Term Observational Study of Patients With Fucosidosis
| NCT ID | NCT07615400 |
| Status | Recruiting |
| Phase | — |
| Sponsor | JCR Pharmaceuticals Co., Ltd. |
| Condition | Fucosidosis |
| Study Type | OBSERVATIONAL |
| Enrollment | 57 participants |
| Start Date | 2026-03-25 |
| Primary Completion | 2031-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 57 participants in total. It began in 2026-03-25 with a primary completion date of 2031-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
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 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
Contact & Investigator
Karolina M Stepien, MD
PRINCIPAL INVESTIGATOR
Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust
Frequently Asked Questions
Who can join the NCT07615400 clinical trial?
This trial is open to participants of all sexes, studying Fucosidosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07615400 currently recruiting?
Yes, NCT07615400 is actively recruiting participants. Contact the research team at clinical_development@jp.jcrpharm.com for enrollment information.
Where is the NCT07615400 trial being conducted?
This trial is being conducted at Orange, United States, Minneapolis, United States, Bengaluru, India, Rotterdam, Netherlands and 11 additional locations.
Who is sponsoring the NCT07615400 clinical trial?
NCT07615400 is sponsored by JCR Pharmaceuticals Co., Ltd.. The principal investigator is Karolina M Stepien, MD at Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust. The trial plans to enroll 57 participants.