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

Spinal Muscular Atrophy Clinical Trial

Official title:

Clinical Assessment of Spinal Muscular Atrophy Type II and III

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01611610
• Other study ID #: SMA Europe
• Status: Completed
• Phase: N/A
• First received: May 30, 2012
• Last updated: March 4, 2014
• Start date: May 2011
• Est. completion date: May 2013

Study information

• Verified date: March 2014
• Source: Institut de Myologie, France
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: May 2013
• Est. primary completion date: May 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 30 Months to 18 Years

Eligibility

Inclusion Criteria for ambulant patients:

• age between 30 months and 24 years

• documented SMA diagnosis by genetic tests; Each should also have the determination of the SMN2 copy number

• able to walk 10 meters without support

• subject who signed an informed consent- subject affiliated to a social security system

Inclusion Criteria non ambulant patients:

• documented diagnosis of SMA by genetic tests

• not able to walk 10 meters without support

• subject affiliated to a social security system

• subject who signed an informed consent

Exclusion Criteria:

• Patient who are currently involved in other clinical trials

• severe intellectual impairment limiting the comprehension of the demanded tasks

• acute neurologic, inflammatory, infectious, endocrine, orthopedic disease in the month preceding the inclusion

• chronic neurologic (besides SMA), inflammatory, infectious, endocrine, orthopedic disease which are not a natural consequence of SMA

• spinal surgery scheduled 6 months before or within 12 months after enrollment

• pregnant women

Study Design

Allocation: Non-Randomized, Intervention Model: Parallel Assignment, Masking: Open Label

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Intervention

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
• 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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Institut de Myologie, France

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in total score of Motor Function Measure at one year		at baseline and one year after	No
Secondary	Change from baseline of Pediatric Quality of Life Inventory Score at one year	Pediatric Quality of Life Inventory - neuromuscular module	at baseline and one year after	No