Spinal Muscular AtrophyNCT02235090
Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting
A study testing Direct current stimulation of cervical spinal cord in people with Spinal Muscular Atrophy and Neuromuscular Disorders, run by Charitable Foundation Children with Spinal Muscular Atrophy at 1 site in Ukraine.
Trial summary
Clinical trials organization in several neuromuscular disorders (NMD) has some specific issues. Nonambulant status and difficulties with transportation are among them. Moreover a lot of patients with NMD have so poor condition that even short transportation is able to worse it. Read more about this study
- Who is it for?
- Ages 5 years and olderHealthy volunteers can take part
- Conditions the study is for
- Spinal Muscular Atrophy, Neuromuscular Disorders
- What is being tested?
- Direct current stimulation of cervical spinal cord
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUkraine
- Study dates
- Oct 2016to 30 Oct 2017
- 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 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent
- 5q SMA confirmed by molecular testing
Who cannot join
- Need for ventilation
- Hypersensitivity (pain or allergic reaction) to current stimulation
Trial locations and contacts
1 site in 1 country.
Ukraine1 site
SI "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"
Kharkiv, 61068
About this study
Official title: Assessment of Feasibility and Statistical Reliability of Functional Outcomes Measurement in Neuromuscular Patients Without Trial Site Visiting by Standard Functional Scales and by Special Autonomic Device in Double-blind, Placebo Controlled Study of Cervical Spinal Cord Transdermal Direct Current Stimulation in Patients With Spinal Muscular Atrophy
Clinical trials organization in several neuromuscular disorders (NMD) has some specific issues. Nonambulant status and difficulties with transportation are among them. Moreover a lot of patients with NMD have so poor condition that even short transportation is able to worse it. Such situation forces researchers to limit a region of recruitment for clinical trials and to exclude from trials more severe subgroup of patients, which cause additional issues especially for rare diseases.
The purpose of this study is to prove hypothesis about possibility to reliably monitor patient condition remotely, without trial site visiting. Visit-free study design is potentially able to widen eligible patient population and to decrease patient dropout rate as well as burden of numerous assessments. Meanwhile assessment frequency could be increased enabling monitoring of short fluctuations in patients' condition.
Spinal muscular atrophy (SMA) is a rare neuromuscular condition to which all mentioned above issues are completely applicable. Direct current stimulation (DCS) of neural structures is well studied and safe intervention, however, its effects on SMA patients' strength and durability has not been reported for today. The investigators suppose that investigation of DCS action in SMA patient population is an adequate model for visit-free design feasibility, reliability and sensitivity evaluation.
- Conditions
- Spinal Muscular Atrophy, Neuromuscular Disorders
- Being tested
- Direct current stimulation of cervical spinal cord
- Keywords
- Spinal muscular atrophy, Neuromuscular disorders, Outcome measures, Visit-free clinical trial design
- Medical subject headings
- Muscular Atrophy, Spinal, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Direct current stimulation of cervical spinal cordOther
10 minutes direct current stimulation of 0, 100 microamperes, 1 milliampere strengths applied through dermal electrodes to cervical spinal cord
| Study group | Role | What they receive |
|---|---|---|
| Zero-strength of direct current stimulation | Sham comparator | Sham transdermal direct current stimulation of cervical spinal cord Receives: Other: Direct current stimulation of cervical spinal cord |
| 100 microamperes direct current stimulation | Experimental | Transdermal direct current stimulation of cervical spinal cord Receives: Other: Direct current stimulation of cervical spinal cord |
| 1 milliampere direct current stimulation | Experimental | Transdermal direct current stimulation of cervical spinal cord Receives: Other: Direct current stimulation of cervical spinal cord |
What is being measured
The primary outcome is the main question the study is designed to answer.
Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity
Measured: Before and 0, 15, 30 minutes after spinal cord direct current stimulation
Secondary outcomes (1)
Short time fluctuations of Hammersmith Functional Motor Scale indexes
Measured: Three times, three days consecutive measurement, every two months, assessed up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2014 (estimated)
- Study startOct 2016
- Primary completion30 Sep 2017
- Study completion30 Oct 2017
Why it stopped: Trial was not started due to lack of funding and nobody received an intervention.
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
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Andriy V. Shatillo, MD, PhD, Principal investigator, State Institution "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02235090
- Sponsor's protocol ID
- CSMA-INPN-1
- Status last verified
- Feb 2019
- First submitted
- 3 Sep 2014
- Last updated
- 15 Feb 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Feasibility and Statistical Reliability of Functional Outcomes Measurement in Neuromuscular Patients Without Trial Site Visiting by Standard Functional Scales and by Special Autonomic Device in Double-blind, Placebo Controlled Study of Cervical Spinal Cord Transdermal Direct Current Stimulation in Patients With Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT02235090
- Acronym / sponsor ID
- CSMA-INPN-1
- Conditions
- Spinal Muscular Atrophy, Neuromuscular Disorders
- Keywords
- Spinal muscular atrophy, Neuromuscular disorders, Outcome measures, Visit-free clinical trial design
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Trial was not started due to lack of funding and nobody received an intervention.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Charitable Foundation Children with Spinal Muscular AtrophyUniversity, hospital or other
- Study start
- Oct 2016 (actual)
- Primary completion
- 30 Sep 2017 (actual)
- Study completion
- 30 Oct 2017 (actual)
- First posted
- 9 Sep 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.
Clinical trials organization in several neuromuscular disorders (NMD) has some specific issues. Nonambulant status and difficulties with transportation are among them. Moreover a lot of patients with NMD have so poor condition that even short transportation is able to worse it. Such situation forces researchers to limit a region of recruitment for clinical trials and to exclude from trials more severe subgroup of patients, which cause additional issues especially for rare diseases.
The purpose of this study is to prove hypothesis about possibility to reliably monitor patient condition remotely, without trial site visiting. Visit-free study design is potentially able to widen eligible patient population and to decrease patient dropout rate as well as burden of numerous assessments. Meanwhile assessment frequency could be increased enabling monitoring of short fluctuations in patients' condition.
Spinal muscular atrophy (SMA) is a rare neuromuscular condition to which all mentioned above issues are completely applicable. Direct current stimulation (DCS) of neural structures is well studied and safe intervention, however, its effects on SMA patients' strength and durability has not been reported for today. The investigators suppose that investigation of DCS action in SMA patient population is an adequate model for visit-free design feasibility, reliability and sensitivity evaluation.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Zero-strength of direct current stimulation | Sham comparator | Other: Direct current stimulation of cervical spinal cordSham transdermal direct current stimulation of cervical spinal cord |
| 100 microamperes direct current stimulation | Experimental | Other: Direct current stimulation of cervical spinal cordTransdermal direct current stimulation of cervical spinal cord |
| 1 milliampere direct current stimulation | Experimental | Other: Direct current stimulation of cervical spinal cordTransdermal direct current stimulation of cervical spinal cord |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Signed informed consent
- 5q SMA confirmed by molecular testing
Key exclusion criteria (2 listed)
- Need for ventilation
- Hypersensitivity (pain or allergic reaction) to current stimulation
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 |
|---|---|---|
| Direct current stimulation of cervical spinal cord10 minutes direct current stimulation of 0, 100 microamperes, 1 milliampere strengths applied through dermal electrodes to cervical spinal cord | Other | 1 milliampere direct current stimulation, 100 microamperes direct current stimulation, Zero-strength of direct current stimulation |
Primary outcome
Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity
Time frame: Before and 0, 15, 30 minutes after spinal cord direct current stimulation
1 secondary outcome
Short time fluctuations of Hammersmith Functional Motor Scale indexes
Time frame: Three times, three days consecutive measurement, every two months, assessed up to 6 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2019.
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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.
Andriy V. Shatillo, MD, PhD principal investigator
State Institution "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Direct current stimulation of cervical spinal cord in people with Spinal Muscular Atrophy and Neuromuscular Disorders, run by Charitable Foundation Children with Spinal Muscular Atrophy at 1 site in Ukraine.
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 2 inclusion and 2 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: withdrawn, as of the sponsor's last update on 15 Feb 2019. 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 Ukraine. 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. Some participants in this study may receive a placebo. 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 was withdrawn before enrolling participants, so there are no results.
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?
Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity (time frame: Before and 0, 15, 30 minutes after spinal cord direct current stimulation). 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 15 Feb 2019.