Infantile Spinal Muscular Atrophy of Type 2 or 3ExerASINCT02061189
Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)
A study testing Physical exercise in a swimming pool during 6 months in people with Infantile Spinal Muscular Atrophy of Type 2 or 3, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 10 years
- Conditions the study is for
- Infantile Spinal Muscular Atrophy of Type 2 or 3
- What is being tested?
- Physical exercise in a swimming pool during 6 months
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteFrance
- Study dates
- Mar 2014to 19 Jun 2020
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Physical Exercise and Neuromuscular Diseases: Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy
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.
Read more
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.
Read the 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.
- Being tested
- Physical exercise in a swimming pool during 6 months
- Keywords
- neuromuscular disease, Spinal Muscular Atrophy, Infantile,, motor function measure, Hammersmith scale,, rehabilitation program, physical exercise
- Medical subject headings
- Neuromuscular Diseases, Muscular Atrophy, Spinal, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Physical exercise in a swimming pool during 6 monthsOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Swimming pool training group | Experimental | 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. Receives: Other: Physical exercise in a swimming pool during 6 months |
| Control group | No intervention | 20 patients with same assessments at M0, M6, M12 and M18, but: * without swimming pool training. * without M24 assessment. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 18 months (control group) to 36 months maximum (swimming pool group)
Secondary outcomes (3)
Hammersmith scale
Motor Assessments will be performed in the same order : MFM first, then Hammersmith scale.
Measured: M18 or M24 or M30 or M36
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.
Measured: M18 or M24 or M30 or M36
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.
Measured: M18 or M24 or M30 or M36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2014 (estimated)
- Study startMar 2014
- Primary completion19 Jun 2020
- Study completion19 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile Spinal Muscular Atrophy of Type 2 or 3Studies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Susana QUIJANO-ROY, MD, PhD, Principal investigator, Raymond Poincaré Hospital, Garches, FRANCE
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02061189
- Sponsor's protocol ID
- P120124
- Other IDs
- 2013-A01331-44
- Acronym
- ExerASI
- Status last verified
- Apr 2021
- First submitted
- 10 Jan 2014
- Last updated
- 3 May 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Physical Exercise and Neuromuscular Diseases: Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT02061189
- Acronym / sponsor ID
- ExerASI / P120124
- Keywords
- neuromuscular disease, Spinal Muscular Atrophy, Infantile,, motor function measure, Hammersmith scale,, rehabilitation program, physical exercise
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Mar 2014 (actual)
- Primary completion
- 19 Jun 2020 (actual)
- Study completion
- 19 Jun 2020 (actual)
- First posted
- 12 Feb 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Swimming pool training group | Experimental | Other: Physical exercise in a swimming pool during 6 months10 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. |
| Control group | No intervention | –20 patients with same assessments at M0, M6, M12 and M18, but: * without swimming pool training. * without M24 assessment. |
Who may qualify
- Ages
- 5 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Physical exercise in a swimming pool during 6 months10 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. | Other | Swimming pool training group |
Primary outcome
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.
3 secondary outcomes
Hammersmith scale
Time frame: M18 or M24 or M30 or M36
Questionnaire on post-exercise physical well-being
Time frame: M18 or M24 or M30 or M36
Non invasive motor capacity assessment
Time frame: M18 or M24 or M30 or M36
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Susana QUIJANO-ROY, MD, PhD principal investigator
Raymond Poincaré Hospital, Garches, FRANCE
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Physical exercise in a swimming pool during 6 months in people with Infantile Spinal Muscular Atrophy of Type 2 or 3, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Motor function measurement (MFM) (time frame: 18 months (control group) to 36 months maximum (swimming pool group)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 May 2021.