Pediatric Spinal Muscular Atrophy (SMA) China Registry

Launched by BIOGEN · Sep 8, 2021

Keywords

ClinConnect Summary

The Pediatric Spinal Muscular Atrophy (SMA) China Registry is a clinical trial aimed at understanding how spinal muscular atrophy, specifically the type linked to chromosome 5q (5q-SMA), affects children in China. The study will look at how the disease progresses over time and how effective different treatments are in real-life situations. Researchers will gather information both from the past and ongoing cases to get a comprehensive view of the condition.

To participate in this trial, children must be under 18 years old and have a confirmed diagnosis of 5q-SMA. They need to be able to understand the study's purpose and risks, and a parent or guardian must provide consent. The trial is currently recruiting participants, and it welcomes all genders. Those who cannot provide consent or have a different type of SMA will not be eligible. Participants can expect to share their health information and experiences related to the disease, which will help improve understanding and treatment options for SMA in the future.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • Ability of the participant and/or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations.
• • Genetically confirmed 5q-SMA.
• • Age \< 18 years at enrollment.
• Key Exclusion Criteria:
• • Unable or unwilling to provide informed consent.
• • Other types of SMA (non 5q-SMA).
• • NOTE: Other protocol defined inclusion/ exclusion criteria may apply.