Oculopharyngeal Muscular DystrophyNCT04009408
Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders
A study testing Expiratory muscle strength therapy (EMST150, Aspire LLC) in people with Oculopharyngeal Muscular Dystrophy and Muscular Dystrophies, run by University of Calgary at 1 site in Canada.
Trial summary
The purpose of this study is to investigate the impact of expiratory muscle strength training (EMST) on the swallowing, breathing, oral intake, quality of life and cough function of people with oculopharyngeal muscular dystrophy (OPMD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Oculopharyngeal Muscular Dystrophy, Muscular Dystrophies, Myopathy; Hereditary
- What is being tested?
- Expiratory muscle strength therapy (EMST150, Aspire LLC)
- Type of treatment
- Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteCanada
- Study dates
- May 2022to 1 Dec 2022
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of oculopharyngeal muscular dystrophy (OPMD)
- 18 years of age or older
- Must be capable of providing informed consent
- Must be able to undergo respiratory function testing and swallowing studies
- Must have a forced vital capacity (FVC) greater than 60%
- A score of 3 or greater on the Eating Assessment Tool-10 (EAT-10; self-administered, symptom-specific outcome instrument for dysphagia. A score of 3 or greater indicates increased stress around eating)
- A score of 26 or greater on the Montreal Cognitive Assessment (MoCA; 30-point screening assessment used for detecting cognitive impairment. A score of 26 or greater is considered to be within functional limits.)
Who cannot join
- Severe coronary artery disease
- Acute myocardial infarction
- Moderate to severe hypovolemia
- Acute neurological events
- Unstable cardiac status
- Recent hernia
Show all 10Show fewer
- Severe chronic obstructive pulmonary disease (COPD)
- Uncontrolled reflux issues
- Women who are pregnant, or who suspect they may be pregnant
- Cognitive impairment that would prevent comprehension of instructions and adherence to intervention guidelines (a score of less than 26 points on the MoCA)
Trial locations and contacts
1 site in 1 country.
Canada1 site
Neuromuscular Clinic, South Health Campus
Calgary, Alberta, T3M 1M4
About this study
Official title: Interventional Study of Expiratory Muscle Strength Training as a Treatment in Neuromuscular Disorders
The purpose of this study is to investigate the impact of expiratory muscle strength training (EMST) on the swallowing, breathing, oral intake, quality of life and cough function of people with oculopharyngeal muscular dystrophy (OPMD).
Read the detailed description
Outline:
Twenty participants with OPMD, with dysphagia, will be recruited from Neuromuscular clinics within Calgary. The investigators will enrol patients in a parallel group, sham-controlled, randomized clinical trial, with 10 participants in each group (active EMST and sham EMST).
Participants will have baseline measurements of: (i) global swallowing function via modified barium swallow study, (ii) maximum expiratory pressure, (iii) voluntary cough spirometry, (iv) forced vital capacity, (v) functional oral intake, (vi) patient report of self-perceived swallowing impairment (EAT-10 Questionnaire), and (vii) biomarker analyses.
Participants will undergo 5-weeks of EMST (active or sham). All baseline measurements will be repeated after 5-weeks of EMST and 10-weeks post-EMST to measure durability of effect.
Outcomes:
The end-goal of the current research is to obtain preliminary data for the benefit of EMST in a new study population, and direct future studies that may provide evidence for a new standard of care in treating neuromuscular diagnoses.
- Being tested
- Expiratory muscle strength therapy (EMST150, Aspire LLC)
- Keywords
- Dysphagia, Myopathy, Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Oculopharyngeal, Muscular Dystrophies, Muscular Diseases, Deglutition Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Expiratory muscle strength therapy (EMST150, Aspire LLC)Device
Active therapy calibrated to the participant's maximum expiratory pressure
| Study group | Role | What they receive |
|---|---|---|
| EMST therapy | Experimental | Participants use the EMST device as per study protocol, set to 50% of the patient's maximal expiratory pressure, as measured by handheld manometer. Receives: Device: Expiratory muscle strength therapy (EMST150, Aspire LLC) |
| Sham EMST therapy | Sham comparator | Participants use a sham EMST device that has the spring removed as per study protocol, with no significant airflow resistance. Receives: Device: Expiratory muscle strength therapy (EMST150, Aspire LLC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Global Swallowing Function
Global swallowing function is rated from videofluoroscopy swallowing studies (VFSS), using the Dynamic Imaging Grade of Swallowing Toxicity (DIGEST), a validated 5-point scale. Global swallowing function is rated from 0-4: 0 = no pharyngeal dysphagia; 1 = mild; 2 = moderate; 3 = severe; 4 = life-threatening. A lower score is a better outcome.
Measured: Change in score from week 0 to week 5
Secondary outcomes (7)
Global Swallowing Function
Global swallowing function is rated from videofluoroscopy swallowing studies (VFSS), using the Dynamic Imaging Grade of Swallowing Toxicity (DIGEST), a validated 5-point scale. Global swallowing function is rated from 0-4: 0 = no pharyngeal dysphagia; 1 = mild; 2 = moderate; 3 = severe; 4 = life-threatening.
Measured: Change in score from week 0 to week 15; change in score from week 5 to week 15.
Maximum expiratory pressure (MEP)
MEP is a measure of respiratory muscle strength and is assessed with a handheld manometer, measured in centimetres of water (cmH2O). A higher score is a better outcome.
Measured: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Volitional cough strength (peak cough flow)
Measure of cough strength that is assessed using a spirometer, measured in litres per minute (L/min). A higher score is a better outcome.
Measured: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Forced vital capacity (FVC)
Measure of how much air is exhaled during forced exhalation and is assessed with a spirometer, measured in litres. A higher score is a better outcome.
Measured: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Oral Intake
A measure daily nutritional and hydration consumption. Oral intake is assessed using the Functional Oral Intake Scale (FOIS), a validated 7-point ordinal scale (1 = no oral intake; 2 = tube dependent with minimal/inconsistent oral intake; 3 = tube supplements with consistent oral intake; 4 = total oral intake in single consistency; 5 = total oral intake of multiple consistencies requiring special preparation; 6 = total oral intake with no special preparation, but must avoid specific foods or liquid items; 7 = total oral intake with no restrictions). A higher score is a better outcome.
Measured: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Self-perceived swallowing impairment
Will be measured using the Eating Assessment Tool-10 (EAT-10), a self-administered, symptom-specific outcome instrument for dysphagia. The EAT-10 allows patients to rate their swallowing symptoms on scale of 0 = no problem to 4 = severe problem. A lower score is a better outcome.
Measured: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Biomarker analyses
An optional blood sample will be collected for biomarker analysis, to identify correlations with clinical response. We will measure genetic biomarkers associated with swallowing function including rs6265, rs165599, rs10835211, rs17601696, and APOE4 genotype status. For these 5 genetic biomarkers, participants will be scored as having zero, one, or two alleles. This information will be used in subgroup analyses for the primary and secondary outcomes.
Measured: Baseline measurement (week 0)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2019
- Study startMay 2022 (estimated)
- Primary completion1 Dec 2022 (estimated)
- Study completion1 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (23)Papers the sponsor has linked to this study.
- Other trials for Oculopharyngeal Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of CalgaryThe 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Parallel group, double blind, sham controlled study
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Calgary
- Sponsor type
- University, hospital or other
- Collaborators
- Muscular Dystrophy Canada
- Responsible party
- Sponsor
- Investigators
- Gerald Pfeffer, MD, PhD, Principal investigator, University of Calgary
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04009408
- Sponsor's protocol ID
- REB18-1121
- Status last verified
- Apr 2022
- First submitted
- 6 Jun 2019
- Last updated
- 21 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No
Publications
Ertekin C, Yuceyar N, Aydogdu, Karasoy H. Electrophysiological evaluation of oropharyngeal swallowing in myotonic dystrophy. J Neurol Neurosurg Psychiatry. 2001 Mar;70(3):363-71. doi: 10.1136/jnnp.70.3.363.
PubMed 11181860Luchesi KF, Kitamua S, Mourao LF. Amyotrophic Lateral Sclerosis survival analysis: Swallowing and non-oral feeding. NeuroRehabilitation. 2014;35(3):535-42. doi: 10.3233/NRE-141149.
PubMed 25238863Chio A, Logroscino G, Hardiman O, Swingler R, Mitchell D, Beghi E, Traynor BG; Eurals Consortium. Prognostic factors in ALS: A critical review. Amyotroph Lateral Scler. 2009 Oct-Dec;10(5-6):310-23. doi: 10.3109/17482960802566824.
PubMed 19922118Paris G, Martinaud O, Petit A, Cuvelier A, Hannequin D, Roppeneck P, Verin E. Oropharyngeal dysphagia in amyotrophic lateral sclerosis alters quality of life. J Oral Rehabil. 2013 Mar;40(3):199-204. doi: 10.1111/joor.12019. Epub 2012 Dec 27.
PubMed 23278936Yang R, Huang R, Chen D, Song W, Zeng Y, Zhao B, Zhou D, Shang HF. Causes and places of death of patients with amyotrophic lateral sclerosis in south-west China. Amyotroph Lateral Scler. 2011 May;12(3):206-9. doi: 10.3109/17482968.2011.572979. Epub 2011 Apr 21.
PubMed 21506897Abu-Baker A, Rouleau GA. Oculopharyngeal muscular dystrophy: recent advances in the understanding of the molecular pathogenic mechanisms and treatment strategies. Biochim Biophys Acta. 2007 Feb;1772(2):173-85. doi: 10.1016/j.bbadis.2006.10.003. Epub 2006 Oct 11.
PubMed 17110089Duranceau CA, Letendre J, Clermont RJ, Levesque HP, Barbeau A. Oropharyngeal dysphagia in patients with oculopharyngeal muscular dystrophy. Can J Surg. 1978 Jul;21(4):326-9.
PubMed 679079Pitts T, Bolser D, Rosenbek J, Troche M, Okun MS, Sapienza C. Impact of expiratory muscle strength training on voluntary cough and swallow function in Parkinson disease. Chest. 2009 May;135(5):1301-1308. doi: 10.1378/chest.08-1389. Epub 2008 Nov 24.
PubMed 19029430Troche MS, Okun MS, Rosenbek JC, Musson N, Fernandez HH, Rodriguez R, Romrell J, Pitts T, Wheeler-Hegland KM, Sapienza CM. Aspiration and swallowing in Parkinson disease and rehabilitation with EMST: a randomized trial. Neurology. 2010 Nov 23;75(21):1912-9. doi: 10.1212/WNL.0b013e3181fef115.
PubMed 21098406Silverman EP, Miller S, Zhang Y, Hoffman-Ruddy B, Yeager J, Daly JJ. Effects of expiratory muscle strength training on maximal respiratory pressure and swallow-related quality of life in individuals with multiple sclerosis. Mult Scler J Exp Transl Clin. 2017 May 29;3(2):2055217317710829. doi: 10.1177/2055217317710829. eCollection 2017 Apr-Jun.
PubMed 28607760Jones HN, Crisp KD, Robey RR, Case LE, Kravitz RM, Kishnani PS. Respiratory muscle training (RMT) in late-onset Pompe disease (LOPD): Effects of training and detraining. Mol Genet Metab. 2016 Feb;117(2):120-8. doi: 10.1016/j.ymgme.2015.09.003. Epub 2015 Sep 8.
PubMed 26381077Plowman EK, Tabor-Gray L, Rosado KM, Vasilopoulos T, Robison R, Chapin JL, Gaziano J, Vu T, Gooch C. Impact of expiratory strength training in amyotrophic lateral sclerosis: Results of a randomized, sham-controlled trial. Muscle Nerve. 2019 Jan;59(1):40-46. doi: 10.1002/mus.26292. Epub 2018 Nov 29.
PubMed 29981250Plowman EK, Watts SA, Tabor L, Robison R, Gaziano J, Domer AS, Richter J, Vu T, Gooch C. Impact of expiratory strength training in amyotrophic lateral sclerosis. Muscle Nerve. 2016 Jun;54(1):48-53. doi: 10.1002/mus.24990. Epub 2016 Mar 3.
PubMed 26599236Rosenbek JC, Robbins JA, Roecker EB, Coyle JL, Wood JL. A penetration-aspiration scale. Dysphagia. 1996 Spring;11(2):93-8. doi: 10.1007/BF00417897.
PubMed 8721066Nimmons D, Pendleton N, Payton A, Ollier W, Horan M, Wilkinson J, Hamdy S. A novel association between COMT and BDNF gene polymorphisms and likelihood of symptomatic dysphagia in older people. Neurogastroenterol Motil. 2015 Sep;27(9):1223-31. doi: 10.1111/nmo.12609. Epub 2015 Jun 14.
PubMed 26073434
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Interventional Study of Expiratory Muscle Strength Training as a Treatment in Neuromuscular Disorders
- ClinicalTrials.gov ID
- NCT04009408
- Acronym / sponsor ID
- REB18-1121
- Keywords
- Dysphagia, Myopathy, Muscular Dystrophy
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of CalgaryUniversity, hospital or other
- Collaborators
- Muscular Dystrophy Canada
- Study start
- May 2022 (estimated)
- Primary completion
- 1 Dec 2022 (estimated)
- Study completion
- 1 Dec 2022 (estimated)
- First posted
- 5 Jul 2019 (actual)
- 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 investigate the impact of expiratory muscle strength training (EMST) on the swallowing, breathing, oral intake, quality of life and cough function of people with oculopharyngeal muscular dystrophy (OPMD).
Detailed description
Outline:
Twenty participants with OPMD, with dysphagia, will be recruited from Neuromuscular clinics within Calgary. The investigators will enrol patients in a parallel group, sham-controlled, randomized clinical trial, with 10 participants in each group (active EMST and sham EMST).
Participants will have baseline measurements of: (i) global swallowing function via modified barium swallow study, (ii) maximum expiratory pressure, (iii) voluntary cough spirometry, (iv) forced vital capacity, (v) functional oral intake, (vi) patient report of self-perceived swallowing impairment (EAT-10 Questionnaire), and (vii) biomarker analyses.
Participants will undergo 5-weeks of EMST (active or sham). All baseline measurements will be repeated after 5-weeks of EMST and 10-weeks post-EMST to measure durability of effect.
Outcomes:
The end-goal of the current research is to obtain preliminary data for the benefit of EMST in a new study population, and direct future studies that may provide evidence for a new standard of care in treating neuromuscular diagnoses.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
- Model description
- Parallel group, double blind, sham controlled study
| Arm | Type | Receives |
|---|---|---|
| EMST therapy | Experimental | Device: Expiratory muscle strength therapy (EMST150, Aspire LLC)Participants use the EMST device as per study protocol, set to 50% of the patient's maximal expiratory pressure, as measured by handheld manometer. |
| Sham EMST therapy | Sham comparator | Device: Expiratory muscle strength therapy (EMST150, Aspire LLC)Participants use a sham EMST device that has the spring removed as per study protocol, with no significant airflow resistance. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of oculopharyngeal muscular dystrophy (OPMD)
- 18 years of age or older
- Must be capable of providing informed consent
- Must be able to undergo respiratory function testing and swallowing studies
- Must have a forced vital capacity (FVC) greater than 60%
Key exclusion criteria (10 listed)
- Severe coronary artery disease
- Acute myocardial infarction
- Moderate to severe hypovolemia
- Acute neurological events
- Unstable cardiac status
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 |
|---|---|---|
| Expiratory muscle strength therapy (EMST150, Aspire LLC)Active therapy calibrated to the participant's maximum expiratory pressure | Device | EMST therapy, Sham EMST therapy |
Primary outcome
Global Swallowing Function
Time frame: Change in score from week 0 to week 5
Global swallowing function is rated from videofluoroscopy swallowing studies (VFSS), using the Dynamic Imaging Grade of Swallowing Toxicity (DIGEST), a validated 5-point scale. Global swallowing function is rated from 0-4: 0 = no pharyngeal dysphagia; 1 = mild; 2 = moderate; 3 = severe; 4 = life-threatening. A lower score is a better outcome.
7 secondary outcomes
Global Swallowing Function
Time frame: Change in score from week 0 to week 15; change in score from week 5 to week 15.
Maximum expiratory pressure (MEP)
Time frame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Volitional cough strength (peak cough flow)
Time frame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Forced vital capacity (FVC)
Time frame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Oral Intake
Time frame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Self-perceived swallowing impairment
Time frame: Change in score from week 0 to week 5; change in score from week 0 to week 15; change in score from week 5 to week 15.
Biomarker analyses
Time frame: Baseline measurement (week 0)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2022.
- Background: PubMed 11181860Ertekin C, Yuceyar N, Aydogdu, Karasoy H. Electrophysiological evaluation of oropharyngeal swallowing in myotonic dystrophy. J Neurol Neurosurg Psychiatry. 2001 Mar;70(3):363-71. doi: 10.1136/jnnp.70.3.363.
- Background: PubMed 25238863Luchesi KF, Kitamua S, Mourao LF. Amyotrophic Lateral Sclerosis survival analysis: Swallowing and non-oral feeding. NeuroRehabilitation. 2014;35(3):535-42. doi: 10.3233/NRE-141149.
- Background: PubMed 19922118Chio A, Logroscino G, Hardiman O, Swingler R, Mitchell D, Beghi E, Traynor BG; Eurals Consortium. Prognostic factors in ALS: A critical review. Amyotroph Lateral Scler. 2009 Oct-Dec;10(5-6):310-23. doi: 10.3109/17482960802566824.
- Background: PubMed 23278936Paris G, Martinaud O, Petit A, Cuvelier A, Hannequin D, Roppeneck P, Verin E. Oropharyngeal dysphagia in amyotrophic lateral sclerosis alters quality of life. J Oral Rehabil. 2013 Mar;40(3):199-204. doi: 10.1111/joor.12019. Epub 2012 Dec 27.
- Background: PubMed 21506897Yang R, Huang R, Chen D, Song W, Zeng Y, Zhao B, Zhou D, Shang HF. Causes and places of death of patients with amyotrophic lateral sclerosis in south-west China. Amyotroph Lateral Scler. 2011 May;12(3):206-9. doi: 10.3109/17482968.2011.572979. Epub 2011 Apr 21.
- Background: PubMed 17110089Abu-Baker A, Rouleau GA. Oculopharyngeal muscular dystrophy: recent advances in the understanding of the molecular pathogenic mechanisms and treatment strategies. Biochim Biophys Acta. 2007 Feb;1772(2):173-85. doi: 10.1016/j.bbadis.2006.10.003. Epub 2006 Oct 11.
- Background: PubMed 679079Duranceau CA, Letendre J, Clermont RJ, Levesque HP, Barbeau A. Oropharyngeal dysphagia in patients with oculopharyngeal muscular dystrophy. Can J Surg. 1978 Jul;21(4):326-9.
- Background: PubMed 19029430Pitts T, Bolser D, Rosenbek J, Troche M, Okun MS, Sapienza C. Impact of expiratory muscle strength training on voluntary cough and swallow function in Parkinson disease. Chest. 2009 May;135(5):1301-1308. doi: 10.1378/chest.08-1389. Epub 2008 Nov 24.
- Background: PubMed 21098406Troche MS, Okun MS, Rosenbek JC, Musson N, Fernandez HH, Rodriguez R, Romrell J, Pitts T, Wheeler-Hegland KM, Sapienza CM. Aspiration and swallowing in Parkinson disease and rehabilitation with EMST: a randomized trial. Neurology. 2010 Nov 23;75(21):1912-9. doi: 10.1212/WNL.0b013e3181fef115.
- Background: PubMed 28607760Silverman EP, Miller S, Zhang Y, Hoffman-Ruddy B, Yeager J, Daly JJ. Effects of expiratory muscle strength training on maximal respiratory pressure and swallow-related quality of life in individuals with multiple sclerosis. Mult Scler J Exp Transl Clin. 2017 May 29;3(2):2055217317710829. doi: 10.1177/2055217317710829. eCollection 2017 Apr-Jun.
- Background: PubMed 26381077Jones HN, Crisp KD, Robey RR, Case LE, Kravitz RM, Kishnani PS. Respiratory muscle training (RMT) in late-onset Pompe disease (LOPD): Effects of training and detraining. Mol Genet Metab. 2016 Feb;117(2):120-8. doi: 10.1016/j.ymgme.2015.09.003. Epub 2015 Sep 8.
- Background: PubMed 29981250Plowman EK, Tabor-Gray L, Rosado KM, Vasilopoulos T, Robison R, Chapin JL, Gaziano J, Vu T, Gooch C. Impact of expiratory strength training in amyotrophic lateral sclerosis: Results of a randomized, sham-controlled trial. Muscle Nerve. 2019 Jan;59(1):40-46. doi: 10.1002/mus.26292. Epub 2018 Nov 29.
- Individual participant dataSharing planned: No
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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.
Gerald Pfeffer, MD, PhD principal investigator
University of Calgary
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Expiratory muscle strength therapy (EMST150, Aspire LLC) in people with Oculopharyngeal Muscular Dystrophy and Muscular Dystrophies, run by University of Calgary at 1 site in Canada.
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 7 inclusion and 10 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: status not confirmed, as of the sponsor's last update on 21 Apr 2022. 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 Canada. 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?
Results are not available in the source record.
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?
Global Swallowing Function (time frame: Change in score from week 0 to week 5). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04009408, which also lists 23 linked publications.
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 21 Apr 2022.