Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
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Purpose
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.
| Condition | Intervention |
|---|---|
|
Spinal Muscular Atrophy |
Other: Functional tests |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: Open Label |
| Official Title: | Clinical Assessment of Spinal Muscular Atrophy Type II and III |
- Change from baseline in total score of Motor Function Measure at one year [ Time Frame: at baseline and one year after ] [ Designated as safety issue: No ]
- Change from baseline of Pediatric Quality of Life Inventory Score at one year [ Time Frame: at baseline and one year after ] [ Designated as safety issue: No ]Pediatric Quality of Life Inventory - neuromuscular module
| Estimated Enrollment: | 16 |
| Study Start Date: | May 2011 |
| Estimated Study Completion Date: | December 2014 |
| Estimated Primary Completion Date: | December 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Ambulant SMA |
Other: Functional tests
The ambulant patients will perform
|
| Non-ambulant SMA |
Other: Functional tests
The non-ambulant patients will perform:
|
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.
Eligibility| Ages Eligible for Study: | 30 Months to 18 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations| Contact: Laurent SERVAIS, PhD | 01 42 16 58 70 | l.servais@institut-myologie.org |
| Contact: Andreea TOTOESCU, MD | 01 42 16 66 49 | a.totoescu@institut-myologie.org |
| France | |
| Institut Myology | Recruiting |
| Paris, France, 75013 | |
| Contact: Laurent Servais, PhD 01 42 46 58 70 l.servais@institut-myologie.org | |
| Contact: Andreea TOTOESCU, MD 01 42 16 66 49 a.totoescu@institut-myologie.org | |
| Principal Investigator: | Laurent Servais, MD, PhD | Institut de Myologie |
More Information
No publications provided
| Responsible Party: | Institut de Myologie, France |
| ClinicalTrials.gov Identifier: | NCT01611610 History of Changes |
| Other Study ID Numbers: | SMA Europe |
| Study First Received: | May 30, 2012 |
| Last Updated: | June 1, 2012 |
| Health Authority: | France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) |
Keywords provided by Institut de Myologie, France:
|
outcome measures spinal muscular atrophy |
Additional relevant MeSH terms:
|
Muscular Atrophy Muscular Atrophy, Spinal Atrophy Spinal Muscular Atrophies of Childhood Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Pathological Conditions, Anatomical |
Signs and Symptoms Spinal Cord Diseases Central Nervous System Diseases Motor Neuron Disease Neurodegenerative Diseases Neuromuscular Diseases Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on June 17, 2013