NCT01611610

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.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
4

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started May 2011

Typical duration for not_applicable

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2011

Completed
1.1 years until next milestone

First Submitted

Initial submission to the registry

May 30, 2012

Completed
6 days until next milestone

First Posted

Study publicly available on registry

June 5, 2012

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2013

Completed
Last Updated

March 5, 2014

Status Verified

March 1, 2014

Enrollment Period

2 years

First QC Date

May 30, 2012

Last Update Submit

March 4, 2014

Conditions

Spinal Muscular Atrophy

Keywords

outcome measuresspinal muscular atrophy

Outcome Measures

Primary Outcomes (1)

  • Change from baseline in total score of Motor Function Measure at one year

    at baseline and one year after

Secondary Outcomes (1)

  • Change from baseline of Pediatric Quality of Life Inventory Score at one year

    at baseline and one year after

Study Arms (2)

Ambulant SMA

OTHER
Other: Functional tests

Non-ambulant SMA

OTHER
Other: Functional tests

Interventions

Functional testsOTHER

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

Ambulant SMA

Eligibility Criteria

Age30 Months - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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
  • 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

You may not qualify if:

  • Patient who are currently involved in other clinical trials
  • severe intellectual impairment limiting the comprehension of the demanded tasks
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Laurent Servais, MD, PhD

    Institut de Myologie

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 30, 2012

First Posted

June 5, 2012

Study Start

May 1, 2011

Primary Completion

May 1, 2013

Study Completion

May 1, 2013

Last Updated

March 5, 2014

Record last verified: 2014-03