Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA) (ExerASI)
|ClinicalTrials.gov Identifier: NCT02061189|
Recruitment Status : Active, not recruiting
First Posted : February 12, 2014
Last Update Posted : October 12, 2018
This clinical trial aims to test a new physio-therapeutic approach tailored to type 2 and 3 Spinal Muscular Atrophy patients, based on physical training in swimming-pool. This specific exercise should promote motor skills of trained patients, as we have observed in different mouse models. Patient's motor skills will be assessed using different scales including MFM and Hammersmith.
This clinical trial attempts to develop a new non-invasive motor scale with sophisticated instruments.
This scale will be useful in future clinical trials on SMA, given the lack of sensitivity of currently available scales.
In addition, the study attempts to validate a questionnaire on post-exercise physical well-being.
|Condition or disease||Intervention/treatment||Phase|
|Infantile Spinal Muscular Atrophy of Type 2 or 3||Other: Physical exercise in a swimming pool during 6 months||Not Applicable|
This multicenter study is based on a longitudinal assessment of the natural disease progression and on the effects of exercise protocol in a swimming pool. This study will compare patients to themselves after determining motor skills and the disease natural course for 12 months.
The study will compare different scales (MFM, Hammersmith) to our new innovative scale based on non-invasive analysis of motor skills.
30 patients will be enrolled and tested with MFM, Hammersmith and our non-invasive scale every 6 months for a total of 18 to 36 months due to the slow natural progression of the disease.
Among these 30 patients, 10 patients will be selected to perform a 6 months training in a swimming pool, from 12 to 18 months or 18 to 24 months or 24 to 30 months after enrollment, in defined and reproducible conditions.
These trained patients will finally be reassessed at 18 and 24 months or 24 and 30 months or 30 and 36 months to test the therapeutic effect of the training compared to the slope of the natural disease progression, and other parameters.
Only patients who have undergone training in the swimming pool will be reevaluated at 24 or 30 or 36 months (6 months post-training) to assess long term exercise effects.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Physical Exercise and Neuromuscular Diseases: Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy|
|Actual Study Start Date :||March 2014|
|Estimated Primary Completion Date :||September 2019|
|Estimated Study Completion Date :||March 2020|
Experimental: Swimming pool training group
10 patients will be selected to perform a 6 months training in a swimming pool, from M12 to M18 or M18 to M24 or M24 to M36, in defined and reproducible conditions.
Other: Physical exercise in a swimming pool during 6 months
10 patients will undergo a training program of physical exercise in a swimming pool, 3 days per week during 6 months. Each session will last for 20 minutes.
No Intervention: Control group
20 patients with same assessments at M0, M6, M12 and M18, but:
- Motor function measurement (MFM) [ Time Frame: 18 months (control group) to 36 months maximum (swimming pool group) ]The motor ability of all patients will be followed for 18 or 24 months, for a total of four or five functional assessments, 1 every 6 months. Each patient will be assessed with the MFM.
- Hammersmith scale [ Time Frame: M18 or M24 or M30 or M36 ]Motor Assessments will be performed in the same order : MFM first, then Hammersmith scale.
- Questionnaire on post-exercise physical well-being [ Time Frame: M18 or M24 or M30 or M36 ]Force and pain felt assessment by questionnaire just before and after each physical training session for 6 months.
- Non invasive motor capacity assessment [ Time Frame: M18 or M24 or M30 or M36 ]Monitoring motor capacity with a non-invasive sophisticated instrument operating in the Paris Descartes University platform. This monitoring will be performed for 18 or 24 months, for a total of four or five functional assessments, 1 every 6 months.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02061189
|Raymond Poincaré Hospital|
|Garches, Hauts-de-Seine, France, 92380|
|Principal Investigator:||Susana QUIJANO-ROY, MD, PhD||Raymond Poincaré Hospital, Garches, FRANCE|