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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Clinical Assessment of Spinal Muscular Atrophy Type II and III

Brief 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 Summary: 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.
Sponsor: Institut de Myologie, France

Current Primary Outcome: Change from baseline in total score of Motor Function Measure at one year [ Time Frame: at baseline and one year after ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Change from baseline of Pediatric Quality of Life Inventory Score at one year [ Time Frame: at baseline and one year after ]

Pediatric Quality of Life Inventory - neuromuscular module


Original Secondary Outcome: Same as current

Information By: Institut de Myologie, France

Dates:
Date Received: May 30, 2012
Date Started: May 2011
Date Completion:
Last Updated: March 4, 2014
Last Verified: March 2014