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Chinese Regional Spinal Muscular Atrophy Patient Registration Study

NCT05631418 · Status: WITHDRAWN · Type: OBSERVATIONAL

Last updated 2023-02-23

No results posted yet for this study

Summary

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.

Conditions

  • Recruitment

Sponsors & Collaborators

  • The Children's Hospital of Zhejiang University School of Medicine

    lead OTHER

Principal Investigators

  • Shanshan Mao, MD · The Children's Hospital of Zhejiang University School of Medicine

Eligibility

Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-11-30
Primary Completion
2025-12-31
Completion
2026-12-31

Countries

  • China

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 NCT05631418 on ClinicalTrials.gov