Pediatric SMA China Registry

study id #: NCT05042921

condition: Muscular Atrophy, Spinal

status: Not yet recruiting

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).

results: https://clinicaltrials.gov/ct2/show/results/NCT05042921

last updated: February 04, 2022

start date: December 31, 2021

estimated completion: June 30, 2024

last updated: September 13, 2021

size / enrollment: 300

primary outcomes:

• Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA)
  
• Up to 21 months
  

inclusion criteria:

exclusion criteria: Criteria:

Unable or unwilling to provide informed consent.
Other types of SMA (non 5q-SMA).

NOTE: Other protocol defined inclusion/ exclusion criteria may apply.

sponsor: Biogen

contacts: US Biogen Clinical Trial Center, 866-633-4636, [email protected]

investigators: Medical Director,Biogen

trial center locations: