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Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA) (ExerASI)

Primary Purpose

Infantile Spinal Muscular Atrophy of Type 2 or 3

Status

Completed

Phase

Not Applicable

Locations

France

Study Type

Interventional

Intervention

Physical exercise in a swimming pool during 6 months

About this trial

This is an interventional treatment trial for Infantile Spinal Muscular Atrophy of Type 2 or 3 focused on measuring neuromuscular disease, Spinal Muscular Atrophy, Infantile,, motor function measure, Hammersmith scale,, rehabilitation program, physical exercise

Eligibility Criteria

5 Years - 10 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patient with type II SMA who can sustain a sitting position without orthopedic corset or type III SMA, with genetic confirmation of the disease.
Age from 5 to 10 years old.
Patient able to understand and execute training instructions, and perform predetermined movements.
Patient able to achieve physical exercises in a swimming pool.
MFM score > 35.
Patient having any contraindication to physical rehabilitation in a swimming pool.
Consent signed by the holders of parental responsibility for children participating in the study.
Patient with social security insurance.

Exclusion Criteria:

Pneumonia: the inclusion is to be delayed for at least 3 months after recovery of infection.
Heart, renal or hepatic failure.
Patient undergoing a major surgery during the study: spinal fusion.
Patient under the care of a guardian.
Inability to cooperate.
Medication potentially interfering with the SMA (i.e., riluzole, salbutamol, phenylbutyrate, sodium valproate) started less than 6 months before inclusion.

Sites / Locations

Raymond Poincaré Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Swimming pool training group

Control group

Arm Description

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. M0, M6, M12 and M18 or M0, M6, M12, M18 and M24 or M0, M6, M12, M18, M24 and M30 assessments: Medical examination + MFM + Hammersmith scale + Non-invasive motor capacity analysis. M12 to M18 or M18 to M24 or M24 to M30: Physical exercise in a swimming pool (3 times per week). M24 or M30 or M36: Medical examination + MFM + Hammersmith scale + Non-invasive motor capacity analysis.

20 patients with same assessments at M0, M6, M12 and M18, but: without swimming pool training. without M24 assessment.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Hammersmith scale

Motor Assessments will be performed in the same order : MFM first, then Hammersmith scale.

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.

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.

Full Information

NCT ID

NCT02061189

First Posted

January 10, 2014

Last Updated

April 30, 2021

Sponsor

Assistance Publique - Hôpitaux de Paris

1. Study Identification

Unique Protocol Identification Number

NCT02061189

Brief Title

Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)

Acronym

ExerASI

Official Title

Physical Exercise and Neuromuscular Diseases: Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy

Study Type

Interventional

2. Study Status

Record Verification Date

April 2021

Overall Recruitment Status

Completed

Study Start Date

March 2014 (Actual)

Primary Completion Date

June 19, 2020 (Actual)

Study Completion Date

June 19, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Assistance Publique - Hôpitaux de Paris

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

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.

Detailed Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Infantile Spinal Muscular Atrophy of Type 2 or 3

Keywords

neuromuscular disease, Spinal Muscular Atrophy, Infantile,, motor function measure, Hammersmith scale,, rehabilitation program, physical exercise

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

19 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Swimming pool training group

Arm Type

Experimental

Arm Description

Arm Title

Control group

Arm Type

No Intervention

Arm Description

20 patients with same assessments at M0, M6, M12 and M18, but: without swimming pool training. without M24 assessment.

Intervention Type

Other

Intervention Name(s)

Physical exercise in a swimming pool during 6 months

Intervention Description

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.

Primary Outcome Measure Information:

Title

Motor function measurement (MFM)

Description

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.

Time Frame

18 months (control group) to 36 months maximum (swimming pool group)

Secondary Outcome Measure Information:

Title

Hammersmith scale

Description

Motor Assessments will be performed in the same order : MFM first, then Hammersmith scale.

Time Frame

M18 or M24 or M30 or M36

Title

Questionnaire on post-exercise physical well-being

Description

Force and pain felt assessment by questionnaire just before and after each physical training session for 6 months.

Time Frame

M18 or M24 or M30 or M36

Title

Non invasive motor capacity assessment

Description

Time Frame

M18 or M24 or M30 or M36

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Patient with type II SMA who can sustain a sitting position without orthopedic corset or type III SMA, with genetic confirmation of the disease. Age from 5 to 10 years old. Patient able to understand and execute training instructions, and perform predetermined movements. Patient able to achieve physical exercises in a swimming pool. MFM score > 35. Patient having any contraindication to physical rehabilitation in a swimming pool. Consent signed by the holders of parental responsibility for children participating in the study. Patient with social security insurance. Exclusion Criteria: Pneumonia: the inclusion is to be delayed for at least 3 months after recovery of infection. Heart, renal or hepatic failure. Patient undergoing a major surgery during the study: spinal fusion. Patient under the care of a guardian. Inability to cooperate. Medication potentially interfering with the SMA (i.e., riluzole, salbutamol, phenylbutyrate, sodium valproate) started less than 6 months before inclusion.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Susana QUIJANO-ROY, MD, PhD

Organizational Affiliation

Raymond Poincaré Hospital, Garches, FRANCE

Official's Role

Principal Investigator

Facility Information:

Facility Name

Raymond Poincaré Hospital

City

Garches

State/Province

Hauts-de-Seine

ZIP/Postal Code

92380

Country

France

12. IPD Sharing Statement

Learn more about this trial

Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)

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