Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
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.
|Study Design:||Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
|Official Title:||Clinical Assessment of Spinal Muscular Atrophy Type II and III|
- Change from baseline in total score of Motor Function Measure at one year [ Time Frame: at baseline and one year after ] [ Designated as safety issue: No ]
- Change from baseline of Pediatric Quality of Life Inventory Score at one year [ Time Frame: at baseline and one year after ] [ Designated as safety issue: No ]Pediatric Quality of Life Inventory - neuromuscular module
|Study Start Date:||May 2011|
|Estimated Study Completion Date:||December 2014|
|Estimated Primary Completion Date:||December 2013 (Final data collection date for primary outcome measure)|
Other: Functional tests
The ambulant patients will perform
Other: Functional tests
The non-ambulant patients will perform:
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.
|Contact: Laurent SERVAIS, PhD||01 42 16 58 firstname.lastname@example.org|
|Contact: Andreea TOTOESCU, MD||01 42 16 66 email@example.com|
|Paris, France, 75013|
|Contact: Laurent Servais, PhD 01 42 46 58 70 firstname.lastname@example.org|
|Contact: Andreea TOTOESCU, MD 01 42 16 66 49 email@example.com|
|Principal Investigator:||Laurent Servais, MD, PhD||Institut de Myologie|