NCT05631418Withdrawn
Chinese Regional Spinal Muscular Atrophy Patient Registration Study
The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health
Source: ClinicalTrials.gov · Last updated 23 Feb 2023
Reason given: Due to the lack of application materials, the normal research can not be carried out for the time being.
Brief summary (registry, in English)
The primary objectives of this study are to obtain clinically meaningful data on the survival, outcomes, prognosis and treatment effect of all the patients with spinal muscular atrophy (SMA) 5q types 1 to 3 (according to international classification), being followed in the Children's Hospital, Zhejiang University School of Medicine since October 2019. The registry will collect retrospectively and prospectively the longitudinal data of the long-term follow-up for children patients, under real life conditions of current medical practice, in order to document the clinical evolution of patients (survival, motor, respiratory, orthopedic and nutritional and so on), the conditions of use of the treatments, the mortality rates of treated and untreated patients, the tolerance of the treatments, adverse events.
- Status (registry)
- Withdrawn
- Study type
- Observational
- Enrollment
- 0 (actual)
- First submitted
- 23 Oct 2022
- Start date
- Nov 2022
- Primary completion date
- Dec 2025
- Lead sponsor
- The Children's Hospital of Zhejiang University School of Medicine
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT05631418
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- Other type of SMA (not 5q).
- Under guardianship or curatorship.
- Unable to understand or cooperate with the study.
Who the study is looking for
- Genetically confirmed 5qSMA patients through 1 to 3 types younger than 18 years old followed in our hospital since October 2019;
- For prospective study: inform consent form signed by patient;
- Not combined with any other genetic and metabolic diseases;
Age in the registry: - to 18 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The Children's Hospital, Zhejiang University School of Medicine | Hangzhou | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.