A Retrospective and Prospective Natural History Study of Patients With Fucosidosis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 57
- 试验地点
- 21
研究概览
简要总结
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.
详细描述
Time Perspective: Both retrospective (Part A) and prospective (Part B).
Enrollment: Part A is anticipated to enroll up to 57 participants for retrospective data collection. Part B is anticipated to enroll up to 31 participants for prospective data collection, the majority of whom are expected to be participants also enrolled in Part A.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of fucosidosis
- •Patient either with or without previous allogeneic hematopoietic stem cell transplant (HSCT) for treatment of fucosidosis
排除标准
- •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