Infantile Spinal Muscular Atrophy of Type 2 or 3 Clinical Trial
— ExerASIOfficial title:
Physical Exercise and Neuromuscular Diseases: Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy
Verified date | April 2021 |
Source | Assistance Publique - Hôpitaux de Paris |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 19 |
Est. completion date | June 19, 2020 |
Est. primary completion date | June 19, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 5 Years to 10 Years |
Eligibility | Inclusion Criteria: 1. Patient with type II SMA who can sustain a sitting position without orthopedic corset or type III SMA, with genetic confirmation of the disease. 2. Age from 5 to 10 years old. 3. Patient able to understand and execute training instructions, and perform predetermined movements. 4. Patient able to achieve physical exercises in a swimming pool. 5. MFM score > 35. 6. Patient having any contraindication to physical rehabilitation in a swimming pool. 7. Consent signed by the holders of parental responsibility for children participating in the study. 8. Patient with social security insurance. Exclusion Criteria: 1. Pneumonia: the inclusion is to be delayed for at least 3 months after recovery of infection. 2. Heart, renal or hepatic failure. 3. Patient undergoing a major surgery during the study: spinal fusion. 4. Patient under the care of a guardian. 5. Inability to cooperate. 6. Medication potentially interfering with the SMA (i.e., riluzole, salbutamol, phenylbutyrate, sodium valproate) started less than 6 months before inclusion. |
Country | Name | City | State |
---|---|---|---|
France | Raymond Poincaré Hospital | Garches | Hauts-de-Seine |
Lead Sponsor | Collaborator |
---|---|
Assistance Publique - Hôpitaux de Paris |
France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Motor function measurement (MFM) | 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. | 18 months (control group) to 36 months maximum (swimming pool group) | |
Secondary | Hammersmith scale | Motor Assessments will be performed in the same order : MFM first, then Hammersmith scale. | M18 or M24 or M30 or M36 | |
Secondary | Questionnaire on post-exercise physical well-being | Force and pain felt assessment by questionnaire just before and after each physical training session for 6 months. | M18 or M24 or M30 or M36 | |
Secondary | Non invasive motor capacity assessment | 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. | M18 or M24 or M30 or M36 |