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

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified October 2013 by Assistance Publique - Hôpitaux de Paris
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT02061189
First received: January 10, 2014
Last updated: March 4, 2014
Last verified: October 2013
  Purpose

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 Intervention
Infantile Spinal Muscular Atrophy of Type 2 or 3
Other: Physical exercise in a swimming pool during 6 months

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Physical Exercise and Neuromuscular Diseases: Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy

Resource links provided by NLM:


Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • Motor function measurement (MFM) [ Time Frame: 18 months (control group) or 24 months (swimming pool group) ] [ Designated as safety issue: No ]
    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 [ Time Frame: M18 or M24 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 30
Study Start Date: March 2014
Estimated Study Completion Date: February 2017
Estimated Primary Completion Date: February 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Swimming pool training group

10 patients will be selected to perform a 6 months training in a swimming pool, from M12 to M18, in defined and reproducible conditions.

  • M0, M6, M12 and M18 assessments: Medical examination + MFM + Hammersmith scale + Non-invasive motor capacity analysis.
  • M12 to M18 (26 weeks): Physical exercise in a swimming pool (3 times per week).
  • M24: Medical examination + MFM + Hammersmith scale + Non-invasive motor capacity analysis.
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:

  • without swimming pool training.
  • without M24 assessment.

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 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 after enrollment, in defined and reproducible conditions.

These trained patients will finally be reassessed at 18 and 24 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 months (6 months post-training) to assess long term exercise effects.

  Eligibility

Ages Eligible for Study:   5 Years to 10 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02061189

Contacts
Contact: Brigitte Estournet, MD +33 (1) 47 10 78 90 brigitte.estournet@rpc.aphp.fr
Contact: Olivier BIONDI, PhD +33 (1) 42 86 21 53 olivier.biondi@parisdescartes.fr

Locations
France
Raymond Poincaré Hospital Not yet recruiting
Garches, Hauts-de-Seine, France, 92380
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Brigitte Estournet, MD Raymond Poincaré Hospital, Garches, FRANCE
  More Information

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT02061189     History of Changes
Other Study ID Numbers: P120124, 2013-A01331-44
Study First Received: January 10, 2014
Last Updated: March 4, 2014
Health Authority: France: Agence Nationale de Sécurité du Médicament et des produits de santé

Keywords provided by Assistance Publique - Hôpitaux de Paris:
neuromuscular disease, Spinal Muscular Atrophy, Infantile,
motor function measure, Hammersmith scale,
rehabilitation program, physical exercise

Additional relevant MeSH terms:
Muscular Atrophy, Spinal
Spinal Muscular Atrophies of Childhood
Atrophy
Muscular Atrophy
Neuromuscular Diseases
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on September 18, 2014