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

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ClinicalTrials.gov Identifier: NCT01611610
Recruitment Status : Completed
First Posted : June 5, 2012
Last Update Posted : March 5, 2014
Sponsor:
Information provided by (Responsible Party):
Institut de Myologie, France

May 30, 2012
June 5, 2012
March 5, 2014
May 2011
May 2013   (Final data collection date for primary outcome measure)
Change from baseline in total score of Motor Function Measure at one year [ Time Frame: at baseline and one year after ]
Same as current
Complete list of historical versions of study NCT01611610 on ClinicalTrials.gov Archive Site
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
Same as current
Not Provided
Not Provided
 
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
Clinical Assessment of Spinal Muscular Atrophy Type II and III

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.

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.
Interventional
Not Applicable
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Spinal Muscular Atrophy
  • 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
  • Ambulant SMA
    Intervention: Other: Functional tests
  • Non-ambulant SMA
    Intervention: Other: Functional tests
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
4
16
May 2013
May 2013   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
30 Months to 18 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
Not Provided
France
 
NCT01611610
SMA Europe
No
Not Provided
Not Provided
Institut de Myologie, France
Institut de Myologie, France
Not Provided
Principal Investigator: Laurent Servais, MD, PhD Institut de Myologie
Institut de Myologie, France
March 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP