NCT01611610

Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)

Clinical Assessment of Spinal Muscular Atrophy Type II and III

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Institut de Myologie, France · Academic / Other
• Sex: All
• Age: 30 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

The aim of this project is to establish a network of clinical teams including the major neuromuscular centers in Europe. We plan to work together to find the best common outcome measures for the following multicenter therapeutic trials.

Detailed description

16 patients (6 ambulant and 10 non ambulant), affected by type II and type III SMA will be enrolled and assessed at baseline and 6 and 12 months later. Non ambulant patients will be assessed using the modified version of the Hammersmith Motor Functional Scale while ambulant patients will be assessed using the extended module of the Hammersmith Motor Functional Scale and timed items, the 6 minute walk and a step activity monitor. All patients will also be assessed using the Motor Function Measure (MFM), that covers the whole range of activities for both ambulant and non ambulant patients. All measures will undergo a process of validation including inter observer reliability. This information will be most valuable for any future trial and will make the groups involved ready to participate to future collaborative studies saving a lot of time on the preliminary aspects (validation, reliability, training) that will be fulfilled by the present study. The study will also provide natural history data for a 12 month period on patients with SMA II and III.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
OTHER	Functional tests	The ambulant patients will perform * MFM (20 items) * Hammersmith modified module - MHMFS * Timed tests (time to rise from the floor,...) * 6 minute walk test * PedsQL - neuromuscular module
OTHER	Functional tests	The non-ambulant patients will perform: * MFM (20 items) * Hammersmith modified module - MHMFS * Upper Limb Functional Items * Egen Klassifikation 2 questionnaire - EK2 * PedsQL questionnaire - neuromuscular module

Timeline

• Start date: 2011-05-01
• Primary completion: 2013-05-01
• Completion: 2013-05-01
• First posted: 2012-06-05
• Last updated: 2014-03-05