NCT02391831

Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 81 (actual)
• Sponsor: Institut de Myologie, France · Academic / Other
• Sex: All
• Age: 2 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

NatHis-SMA is a prospective, longitudinal and interventional study of the natural history of patients with type 2 and 3 Spinal Muscular Atrophy (SMA). The purpose of this study is to characterize the disease course over 2 years and identify prognostic variables of the disease and biomarkers of SMA progression, as well as determine the best outcome measures for further therapeutics approaches.

Conditions

• Type 2 Spinal Muscular Atrophy
• Type 3 Spinal Muscular Atrophy

Interventions

Type	Name	Description
OTHER	Strength, function and activity measurements
OTHER	Muscle MRI
OTHER	Electrophysiology measurements
OTHER	Blood sampling for biomarker analysis

Timeline

• Start date: 2015-05-01
• Primary completion: 2018-06-01
• Completion: 2018-06-01
• First posted: 2015-03-18
• Last updated: 2018-07-19

Locations

9 sites across 3 countries: Belgium, France, Germany