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NCT Number: NCT05042921

Pediatric Spinal Muscular Atrophy (SMA) China Registry

The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA). The study will examine SMA natural history and DMT outcomes in a real-world setting both prospectively and retrospectively.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Muscular Atrophy, Spinal Central Nervous System Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Anhui Children's Hospital, Hefei, Anhui, China

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA)

    Time frame: Up to 60 months

Other outcomes

  1. Effectiveness of DMT Treatments

    Time frame: Up to 60 months

    Effectiveness will be assessed using following measures: time to mortality, motor function, motor measures, pulmonary function, scoliosis, hospitalizations and comorbidities, clinical observations, patient reported outcomes, wheelchair use, nutrition and electrophysiology and biomarkers.

  2. Safety of DMT Treatments

    Time frame: Up to 60 months

    Safety will be assessed by adverse events (AEs) and serious AEs.

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry

Important dates

Study start
2021
Primary completion
2027
Study completion
2027
First posted
Sep 13, 2021
Registry last updated
Sep 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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