Spinal Muscular Atrophy Clinical Trial
Official title:
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.
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 |
Allocation: Non-Randomized, Intervention Model: Parallel Assignment, Masking: Open Label
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
Institut de Myologie, France |
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 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
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|
Phase 3 | |
Completed |
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N/A | |
Completed |
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N/A | |
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Phase 2 | |
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N/A | |
Completed |
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N/A | |
Not yet recruiting |
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Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)
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N/A | |
Completed |
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Phase 2 | |
Completed |
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Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
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Phase 1 | |
Enrolling by invitation |
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||
Recruiting |
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||
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||
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||
Completed |
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Phase 1/Phase 2 | |
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N/A | |
Completed |
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N/A | |
Completed |
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A Pilot Study of Biomarkers for Spinal Muscular Atrophy
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N/A | |
Completed |
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Phase 2 | |
Recruiting |
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||
Recruiting |
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