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Trial record 1 of 119 for:    "Progressive Muscular Atrophy"
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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

Tracking Information
First Submitted Date  ICMJE May 30, 2012
First Posted Date  ICMJE June 5, 2012
Last Update Posted Date March 5, 2014
Study Start Date  ICMJE May 2011
Actual Primary Completion Date May 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 1, 2012)
Change from baseline in total score of Motor Function Measure at one year [ Time Frame: at baseline and one year after ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01611610 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 1, 2012)
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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
Official Title  ICMJE 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 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Condition  ICMJE Spinal Muscular Atrophy
Intervention  ICMJE
  • 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
Study Arms  ICMJE
  • Ambulant SMA
    Intervention: Other: Functional tests
  • Non-ambulant SMA
    Intervention: Other: Functional tests
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 4, 2014)
4
Original Estimated Enrollment  ICMJE
 (submitted: June 1, 2012)
16
Actual Study Completion Date  ICMJE May 2013
Actual Primary Completion Date May 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 30 Months to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries France
 
Administrative Information
NCT Number  ICMJE NCT01611610
Other Study ID Numbers  ICMJE SMA Europe
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Institut de Myologie, France
Study Sponsor  ICMJE Institut de Myologie, France
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Laurent Servais, MD, PhD Institut de Myologie
PRS Account Institut de Myologie, France
Verification Date March 2014

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