Spinal Muscular Atrophy Type 3Phase 2EMOTASNCT02227823
Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
A Phase 2 study testing Pyridostigmine Bromide in people with Spinal Muscular Atrophy Type 3, run by Centre Hospitalier Régional de la Citadelle at 1 site in Belgium.
Trial summary
The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Spinal Muscular Atrophy Type 3
- What is being tested?
- Pyridostigmine Bromide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteBelgium
- Study dates
- Jul 2014to Jul 2017
- Phase
- Phase 2
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Spinal muscular atrophy type 3, genetically confirmed
- Age higher than 6 years old
- Ambulatory patient
- Informed consent signed
- More than 100 meters of walking at 6-minute walk test at screening
- Value at screening and baseline in a range of 20% of the highest value at 6-minute walk test
Who cannot join
- Patient who had surgical intervention or suffer from a recent traumatism (less than 6 months)
- Associated pathology such as endocrinopathy, infectious disease, allergy, myopathy, chronic or acute inflammatory pathology, during 3 weeks preceding the inclusion.
- Other therapeutics than food supplements or those frequently prescribed in spinal muscular atrophy or its complications
- Non tolerance of electromyography
- Limited collaboration due to trouble in information comprehension
- Pathology inducing contra-indication for pyridostigmine treatment (allergy at molecule, asthma, Parkinson disease, mechanic obstruction of urinary or digestive tracts)
Trial locations and contacts
1 site in 1 country.
Belgium1 site
Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle
Liège, 4000
About this study
Official title: Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.
The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).
Read the detailed description
Spinal muscular atrophy (SMA) is the second neuromuscular disease meet in children. SMA is a genetically transmitted disease inducing muscular weakness predominating on shoulders and hips. Currently, there is no effective therapy to slow the progression of the disease. SMA is due to a neuron motor attempt of the spinal cord and recently it has been demonstrated a neuromuscular junction (NMJ) involvement, according to recent studies.
EMOTAS study aim to understand if NMJ abnormalities could have an impact on motor performance and fatigue in SMA type 3 ambulatory patients by electromyogram and to improve by non-invasive therapy quality of life of patients.
- Conditions
- Spinal Muscular Atrophy Type 3
- Being tested
- Pyridostigmine Bromide
- Keywords
- spinal muscular atrophy type 3, electromyography, fatigability, decrement
- Medical subject headings
- Spinal Muscular Atrophies of Childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pyridostigmine BromideDrug
Also called Mestinon
| Study group | Role | What they receive |
|---|---|---|
| significant decrement | Experimental | Patients with significant decrement at electromyogram will be treated by pyridostigmine bromide 60mg 3 times a day for patients older than 18 and 1.5mg/kg 3 times a day for children less than 40kg Receives: Drug: Pyridostigmine Bromide |
| no decrement | No intervention | Patient without significant decrement will not receive any treatment and will be the control group |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in the distance walked at 6-minute walk test at 6 months
Measured: 6 months
Secondary outcomes (4)
Change from baseline of decrement at 6 months
Measured: 6 months
Change from baseline of MFM-D1
Comparison of treated and control group values will be made
Measured: 6 months
Change from baseline of Moviplate values at 6 months
Comparison between treated and control group value will be made
Measured: 6 months
Change from baseline of the ratio at 6 minutes walk test at 6 months
It's the ratio between the number of meters during the last minute of the 6-minute walk test and the first minute of the 6-minute walk test.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2014 (estimated)
- Study startJul 2014
- Primary completionJul 2017
- Study completionJul 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular Atrophy Type 3Studies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Régional de la Citadelle
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr. Stéphanie Delstanche, Centre Hospitalier Régional de la Citadelle
- Investigators
- Stephanie Delstanche, Principal investigator, Centre de référence des maladies neuromusculaire de Liège
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02227823
- Sponsor's protocol ID
- 1376
- Acronym
- EMOTAS
- Status last verified
- Oct 2023
- First submitted
- 20 Aug 2014
- Last updated
- 11 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.
- ClinicalTrials.gov ID
- NCT02227823
- Acronym / sponsor ID
- EMOTAS / 1376
- Conditions
- Spinal Muscular Atrophy Type 3
- Keywords
- spinal muscular atrophy type 3, electromyography, fatigability, decrement
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Centre Hospitalier Régional de la CitadelleUniversity, hospital or other
- Study start
- Jul 2014
- Primary completion
- Jul 2017 (actual)
- Study completion
- Jul 2017 (actual)
- First posted
- 28 Aug 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.
The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).
Detailed description
Spinal muscular atrophy (SMA) is the second neuromuscular disease meet in children. SMA is a genetically transmitted disease inducing muscular weakness predominating on shoulders and hips. Currently, there is no effective therapy to slow the progression of the disease. SMA is due to a neuron motor attempt of the spinal cord and recently it has been demonstrated a neuromuscular junction (NMJ) involvement, according to recent studies.
EMOTAS study aim to understand if NMJ abnormalities could have an impact on motor performance and fatigue in SMA type 3 ambulatory patients by electromyogram and to improve by non-invasive therapy quality of life of patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| significant decrement | Experimental | Drug: Pyridostigmine BromidePatients with significant decrement at electromyogram will be treated by pyridostigmine bromide 60mg 3 times a day for patients older than 18 and 1.5mg/kg 3 times a day for children less than 40kg |
| no decrement | No intervention | –Patient without significant decrement will not receive any treatment and will be the control group |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Spinal muscular atrophy type 3, genetically confirmed
Key exclusion criteria (6 listed)
- Patient who had surgical intervention or suffer from a recent traumatism (less than 6 months)
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 |
|---|---|---|
| Pyridostigmine BromideAlso: Mestinon | Drug | significant decrement |
Primary outcome
Change from Baseline in the distance walked at 6-minute walk test at 6 months
Time frame: 6 months
4 secondary outcomes
Change from baseline of decrement at 6 months
Time frame: 6 months
Change from baseline of MFM-D1
Time frame: 6 months
Change from baseline of Moviplate values at 6 months
Time frame: 6 months
Change from baseline of the ratio at 6 minutes walk test at 6 months
Time frame: 6 months
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 11 Oct 2023.
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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.
Stephanie Delstanche principal investigator
Centre de référence des maladies neuromusculaire de Liège
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pyridostigmine Bromide in people with Spinal Muscular Atrophy Type 3, run by Centre Hospitalier Régional de la Citadelle at 1 site in Belgium.
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 6 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 11 Oct 2023. 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 Belgium. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Change from Baseline in the distance walked at 6-minute walk test at 6 months (time frame: 6 months). 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 11 Oct 2023.