Spinal Muscular AtrophyNCT01611610
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
A study testing Functional tests in people with Spinal Muscular Atrophy, run by Institut de Myologie, France.
Trial summary
The aim of this project is to establish a network of clinical teams including the major neuromuscular centers in Europe. We plan to work together to find the best common outcome measures for the following multicenter therapeutic trials. Read more about this study
- Who is it for?
- Ages 30 months to 18 years
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- Functional tests
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- No sites listed yet
- Study dates
- May 2011to May 2013
- 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 30 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- age between 30 months and 24 years
- documented SMA diagnosis by genetic tests; Each should also have the determination of the SMN2 copy number
- able to walk 10 meters without support
- subject who signed an informed consent- subject affiliated to a social security system
- documented diagnosis of SMA by genetic tests
- not able to walk 10 meters without support
- subject affiliated to a social security system
- subject who signed an informed consent
Who cannot join
- Patient who are currently involved in other clinical trials
- severe intellectual impairment limiting the comprehension of the demanded tasks
- acute neurologic, inflammatory, infectious, endocrine, orthopedic disease in the month preceding the inclusion
- chronic neurologic (besides SMA), inflammatory, infectious, endocrine, orthopedic disease which are not a natural consequence of SMA
- spinal surgery scheduled 6 months before or within 12 months after enrollment
- pregnant women
About this study
Official title: Clinical Assessment of Spinal Muscular Atrophy Type II and III
The aim of this project is to establish a network of clinical teams including the major neuromuscular centers in Europe.
We plan to work together to find the best common outcome measures for the following multicenter therapeutic trials.
Read the detailed description
16 patients (6 ambulant and 10 non ambulant), affected by type II and type III SMA will be enrolled and assessed at baseline and 6 and 12 months later. Non ambulant patients will be assessed using the modified version of the Hammersmith Motor Functional Scale while ambulant patients will be assessed using the extended module of the Hammersmith Motor Functional Scale and timed items, the 6 minute walk and a step activity monitor. All patients will also be assessed using the Motor Function Measure (MFM), that covers the whole range of activities for both ambulant and non ambulant patients. All measures will undergo a process of validation including inter observer reliability. This information will be most valuable for any future trial and will make the groups involved ready to participate to future collaborative studies saving a lot of time on the preliminary aspects (validation, reliability, training) that will be fulfilled by the present study. The study will also provide natural history data for a 12 month period on patients with SMA II and III.
- Conditions
- Spinal Muscular Atrophy
- Being tested
- Functional tests
- Keywords
- outcome measures, spinal muscular atrophy
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Functional testsOther
The ambulant patients will perform
- MFM (20 items)
- Hammersmith modified module - MHMFS
- Timed tests (time to rise from the floor,...)
- 6 minute walk test
- PedsQL - neuromuscular module
Functional testsOther
The non-ambulant patients will perform:
- MFM (20 items)
- Hammersmith modified module - MHMFS
- Upper Limb Functional Items
- Egen Klassifikation 2 questionnaire - EK2
- PedsQL questionnaire - neuromuscular module
| Study group | Role | What they receive |
|---|---|---|
| Ambulant SMA | Other | Not described Receives: Other: Functional tests |
| Non-ambulant SMA | Other | Not described Receives: Other: Functional tests |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in total score of Motor Function Measure at one year
Measured: at baseline and one year after
Secondary outcomes (1)
Change from baseline of Pediatric Quality of Life Inventory Score at one year
Pediatric Quality of Life Inventory - neuromuscular module
Measured: at baseline and one year after
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2012 (estimated)
- Study startMay 2011
- Primary completionMay 2013
- Study completionMay 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies 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 N/A
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut de Myologie, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Laurent Servais, MD, PhD, Principal investigator, Institut de Myologie
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01611610
- Sponsor's protocol ID
- SMA Europe
- Status last verified
- Mar 2014
- First submitted
- 30 May 2012
- Last updated
- 5 Mar 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Assessment of Spinal Muscular Atrophy Type II and III
- ClinicalTrials.gov ID
- NCT01611610
- Acronym / sponsor ID
- SMA Europe
- Conditions
- Spinal Muscular Atrophy
- Keywords
- outcome measures, spinal muscular atrophy
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Institut de Myologie, FranceUniversity, hospital or other
- Study start
- May 2011
- Primary completion
- May 2013 (actual)
- Study completion
- May 2013 (actual)
- First posted
- 5 Jun 2012 (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 aim of this project is to establish a network of clinical teams including the major neuromuscular centers in Europe.
We plan to work together to find the best common outcome measures for the following multicenter therapeutic trials.
Detailed description
16 patients (6 ambulant and 10 non ambulant), affected by type II and type III SMA will be enrolled and assessed at baseline and 6 and 12 months later. Non ambulant patients will be assessed using the modified version of the Hammersmith Motor Functional Scale while ambulant patients will be assessed using the extended module of the Hammersmith Motor Functional Scale and timed items, the 6 minute walk and a step activity monitor. All patients will also be assessed using the Motor Function Measure (MFM), that covers the whole range of activities for both ambulant and non ambulant patients. All measures will undergo a process of validation including inter observer reliability. This information will be most valuable for any future trial and will make the groups involved ready to participate to future collaborative studies saving a lot of time on the preliminary aspects (validation, reliability, training) that will be fulfilled by the present study. The study will also provide natural history data for a 12 month period on patients with SMA II and III.
Study design
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ambulant SMA | Other | Other: Functional tests |
| Non-ambulant SMA | Other | Other: Functional tests |
Who may qualify
- Ages
- 30 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- age between 30 months and 24 years
- documented SMA diagnosis by genetic tests; Each should also have the determination of the SMN2 copy number
- able to walk 10 meters without support
- subject who signed an informed consent- subject affiliated to a social security system
- documented diagnosis of SMA by genetic tests
Key exclusion criteria (6 listed)
- Patient who are currently involved in other clinical trials
- severe intellectual impairment limiting the comprehension of the demanded tasks
- acute neurologic, inflammatory, infectious, endocrine, orthopedic disease in the month preceding the inclusion
- chronic neurologic (besides SMA), inflammatory, infectious, endocrine, orthopedic disease which are not a natural consequence of SMA
- spinal surgery scheduled 6 months before or within 12 months after enrollment
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 |
|---|---|---|
| Functional testsThe ambulant patients will perform * MFM (20 items) * Hammersmith modified module - MHMFS * Timed tests (time to rise from the floor,...) * 6 minute walk test * PedsQL - neuromuscular module | Other | Ambulant SMA |
| Functional testsThe non-ambulant patients will perform: * MFM (20 items) * Hammersmith modified module - MHMFS * Upper Limb Functional Items * Egen Klassifikation 2 questionnaire - EK2 * PedsQL questionnaire - neuromuscular module | Other | Non-ambulant SMA |
Primary outcome
Change from baseline in total score of Motor Function Measure at one year
Time frame: at baseline and one year after
1 secondary outcome
Change from baseline of Pediatric Quality of Life Inventory Score at one year
Time frame: at baseline and one year after
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 5 Mar 2014.
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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.
Laurent Servais, MD, PhD principal investigator
Institut de Myologie
Questions about this trial
What is this clinical trial studying?
A study testing Functional tests in people with Spinal Muscular Atrophy, run by Institut de Myologie, 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 5 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.
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?
Change from baseline in total score of Motor Function Measure at one year (time frame: at baseline and one year after). 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 5 Mar 2014; AzTrials last refreshed its copy on 8 Nov 2026.