Neuromuscular DiseaseHSTNMDNCT00695591
Home Sleep Testing in Neuromuscular Disease Patients
An observational study of Neuromuscular Disease, run by Landon Pediatric Foundation at 1 site in United States.
Trial summary
Lay Summary Patients with severe neuromuscular develop hypoventilation, which leads to elevated carbon dioxide levels. Measuring only oxygen saturation levels with pulse oximetry may be inadequate. Read more about this study
- Who is it for?
- Ages 8 to 50 years
- Conditions the study is for
- Neuromuscular Disease
- What is being tested?
- Evaluation for Nocturnal non invasive ventilation
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2008to Jul 2009
- Phase
- Observational
- 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 8 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Study Population Description Patients from the Neuromuscular Disease Clinic at Ventura County Medical Center Non-Probability Sample: invitation to volunteer Neuromuscular Disease
Who may be able to join
- Clinical diagnosis of neuromuscular disease
- Must have home caregivers
Who cannot join
- No home caregiver
Trial locations and contacts
1 site in 1 country.
United States1 site
Pediatric Diagnostic Center
Ventura, California, 93003
About this study
Lay Summary Patients with severe neuromuscular develop hypoventilation, which leads to elevated carbon dioxide levels. Measuring only oxygen saturation levels with pulse oximetry may be inadequate. End tidal carbon dioxide levels or arterial blood gases should be measured periodically, depending on the clinical condition of the patient. A thorough review of systems will help define any problems. Patients who are hypoventilating often have elevated carbon dioxide levels at night and complain of a morning headache, restlessness or nightmares, and poor quality sleep. This may cause daytime sleepiness. Insufficient respiration with hypoxia may occur later, especially if the lung is damaged by chronic aspiration.
We propose to evaluate the use of the Nonin LifeSense monitor in home evaluation of respiration, oxygen level, heart rate, and carbon dioxide level and to develop interpretation of the results that will lead to appropriate interventions for apnea, and insufficient respiration.
Read more
Relevance to MDA Fewer than one per cent of the Muscular Dystrophy Association have pulmonologists as co-directors.Late referral of progressive restrictive lung disease leads to invasive support of respiratory failure. Early initiation of non invasive ventilation techniques requires patience on the part of the caregiver and exploration of mask interfaces and ventilation techniques. In addition, the development of new therapies, currently manifested through enhanced diagnostic accuracy, will require new signal for initiation and in the assessment of success or failure.
Aims Aim 1. To assess the utility of a small portable device (LifeSense Monitor Nonin Medical Inc. Plymouth Minnesota) with extended recording capabilty to provide accurate diagnosis of hypoventilation.
Aim 2. To provide an easily interpretable report defining sleep hypoxemia, hypercapnea, and apnea.
Aim 3. To promote early evaluation and treatment of the respiratory problems in centers that do not have pumonologists as these are essential to prognosis, whether of survival or of quality of life, in neuromuscular diseases.
- Conditions
- Neuromuscular Disease
- Being tested
- Evaluation for Nocturnal non invasive ventilation
- Keywords
- Home sleep testing neuromuscular disease, Neuromuscular disease patients with FEV1<50% of predicted
- Medical subject headings
- Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Evaluation for Nocturnal non invasive ventilationOther
Patients will be referred for evaluation for NNIV is indicated in patients with diurnal Pa,CO2 >6.0 Pa; and 2) nocturnal Sa,O2 <88% for 5 consecutive min. End tidal CO2 representing equivalent values will be referred for NNIV
| Study group | Role | What they receive |
|---|---|---|
| 1SevereRLD,NMD | Patients with FEV1<40% Receives: Other: Evaluation for Nocturnal non invasive ventilation | |
| 2VerySevereRLD,NMD | FEV1<30% Receives: Other: Evaluation for Nocturnal non invasive ventilation | |
| 3NINV | FEV1<25%,on non invasive ventilation Receives: Other: Evaluation for Nocturnal non invasive ventilation | |
| ModerateRLD,NMD | Patients with FEV1 40-50% of predicted Receives: Other: Evaluation for Nocturnal non invasive ventilation |
What is being measured
The primary outcome is the main question the study is designed to answer.
1. End tidal CO2 catheter is tolerated for six hours or more
Measured: 3 months
Secondary outcomes (3)
1. Interpretable data are captured for oximetry, heart rate, and end-tidal CO2
Measured: 3 months
2. An interpretive report is generated that will guide the non-pulmonary physician in potential interventions
Measured: 6 months
2. Patients and parents are able to apply the monitors at home and a six hour data set is collected
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2008 (estimated)
- Study startJul 2008
- Primary completionSep 2008
- Study completionJul 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Neuromuscular DiseaseStudies 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Landon Pediatric Foundation
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Chris Landon, MD, Principal investigator, Landon Pediatric Foundation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00695591
- Sponsor's protocol ID
- LS01
- Acronym
- HSTNMD
- Status last verified
- Aug 2014
- First submitted
- 3 Jun 2008
- Last updated
- 13 Aug 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Shneerson JM, Simonds AK. Noninvasive ventilation for chest wall and neuromuscular disorders. Eur Respir J. 2002 Aug;20(2):480-7. doi: 10.1183/09031936.02.00404002.
PubMed 12212984Kumar SP, Sword D, Petty RK, Banham SW, Patel KR. Assessment of sleep studies in myotonic dystrophy. Chron Respir Dis. 2007;4(1):15-8. doi: 10.1177/1479972306074480.
PubMed 17416148Kirk VG, Flemons WW, Adams C, Rimmer KP, Montgomery MD. Sleep-disordered breathing in Duchenne muscular dystrophy: a preliminary study of the role of portable monitoring. Pediatr Pulmonol. 2000 Feb;29(2):135-40. doi: 10.1002/(sici)1099-0496(200002)29:23.0.co;2-#.
PubMed 10639204Birnkrant DJ, Panitch HB, Benditt JO, Boitano LJ, Carter ER, Cwik VA, Finder JD, Iannaccone ST, Jacobson LE, Kohn GL, Motoyama EK, Moxley RT, Schroth MK, Sharma GD, Sussman MD. American College of Chest Physicians consensus statement on the respiratory and related management of patients with Duchenne muscular dystrophy undergoing anesthesia or sedation. Chest. 2007 Dec;132(6):1977-86. doi: 10.1378/chest.07-0458.
PubMed 18079231
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Home Sleep Testing in Neuromuscular Disease Patients
- ClinicalTrials.gov ID
- NCT00695591
- Acronym / sponsor ID
- HSTNMD / LS01
- Conditions
- Neuromuscular Disease
- Keywords
- Home sleep testing neuromuscular disease, Neuromuscular disease patients with FEV1<50% of predicted
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Landon Pediatric FoundationUniversity, hospital or other
- Study start
- Jul 2008
- Primary completion
- Sep 2008 (actual)
- Study completion
- Jul 2009 (actual)
- First posted
- 12 Jun 2008 (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.
Lay Summary Patients with severe neuromuscular develop hypoventilation, which leads to elevated carbon dioxide levels. Measuring only oxygen saturation levels with pulse oximetry may be inadequate. End tidal carbon dioxide levels or arterial blood gases should be measured periodically, depending on the clinical condition of the patient. A thorough review of systems will help define any problems. Patients who are hypoventilating often have elevated carbon dioxide levels at night and complain of a morning headache, restlessness or nightmares, and poor quality sleep. This may cause daytime sleepiness. Insufficient respiration with hypoxia may occur later, especially if the lung is damaged by chronic aspiration.
We propose to evaluate the use of the Nonin LifeSense monitor in home evaluation of respiration, oxygen level, heart rate, and carbon dioxide level and to develop interpretation of the results that will lead to appropriate interventions for apnea, and insufficient respiration.
Relevance to MDA Fewer than one per cent of the Muscular Dystrophy Association have pulmonologists as co-directors.Late referral of progressive restrictive lung disease leads to invasive support of respiratory failure. Early initiation of non invasive ventilation techniques requires patience on the part of the caregiver and exploration of mask interfaces and ventilation techniques. In addition, the development of new therapies, currently manifested through enhanced diagnostic accuracy, will require new signal for initiation and in the assessment of success or failure.
Aims Aim 1. To assess the utility of a small portable device (LifeSense Monitor Nonin Medical Inc. Plymouth Minnesota) with extended recording capabilty to provide accurate diagnosis of hypoventilation.
Aim 2. To provide an easily interpretable report defining sleep hypoxemia, hypercapnea, and apnea.
Aim 3. To promote early evaluation and treatment of the respiratory problems in centers that do not have pumonologists as these are essential to prognosis, whether of survival or of quality of life, in neuromuscular diseases.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 1SevereRLD,NMD | – | Other: Evaluation for Nocturnal non invasive ventilationPatients with FEV1<40% |
| 2VerySevereRLD,NMD | – | Other: Evaluation for Nocturnal non invasive ventilationFEV1<30% |
| 3NINV | – | Other: Evaluation for Nocturnal non invasive ventilationFEV1<25%,on non invasive ventilation |
| ModerateRLD,NMD | – | Other: Evaluation for Nocturnal non invasive ventilationPatients with FEV1 40-50% of predicted |
Who may qualify
- Ages
- 8 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Study Population Description Patients from the Neuromuscular Disease Clinic at Ventura County Medical Center Non-Probability Sample: invitation to volunteer Neuromuscular Disease
Key inclusion criteria (2 listed)
- Clinical diagnosis of neuromuscular disease
- Must have home caregivers
Key exclusion criteria (1 listed)
- No home caregiver
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 |
|---|---|---|
| Evaluation for Nocturnal non invasive ventilationPatients will be referred for evaluation for NNIV is indicated in patients with diurnal Pa,CO2 >6.0 Pa; and 2) nocturnal Sa,O2 <88% for 5 consecutive min. End tidal CO2 representing equivalent values will be referred for NNIV | Other | 1SevereRLD,NMD, 2VerySevereRLD,NMD, 3NINV, ModerateRLD,NMD |
Primary outcome
1. End tidal CO2 catheter is tolerated for six hours or more
Time frame: 3 months
3 secondary outcomes
1. Interpretable data are captured for oximetry, heart rate, and end-tidal CO2
Time frame: 3 months
2. An interpretive report is generated that will guide the non-pulmonary physician in potential interventions
Time frame: 6 months
2. Patients and parents are able to apply the monitors at home and a six hour data set is collected
Time frame: 3 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 13 Aug 2014.
- Background: PubMed 12212984Shneerson JM, Simonds AK. Noninvasive ventilation for chest wall and neuromuscular disorders. Eur Respir J. 2002 Aug;20(2):480-7. doi: 10.1183/09031936.02.00404002.
- Background: PubMed 17416148Kumar SP, Sword D, Petty RK, Banham SW, Patel KR. Assessment of sleep studies in myotonic dystrophy. Chron Respir Dis. 2007;4(1):15-8. doi: 10.1177/1479972306074480.
- Background: PubMed 10639204Kirk VG, Flemons WW, Adams C, Rimmer KP, Montgomery MD. Sleep-disordered breathing in Duchenne muscular dystrophy: a preliminary study of the role of portable monitoring. Pediatr Pulmonol. 2000 Feb;29(2):135-40. doi: 10.1002/(sici)1099-0496(200002)29:23.0.co;2-#.
- Background: PubMed 18079231Birnkrant DJ, Panitch HB, Benditt JO, Boitano LJ, Carter ER, Cwik VA, Finder JD, Iannaccone ST, Jacobson LE, Kohn GL, Motoyama EK, Moxley RT, Schroth MK, Sharma GD, Sussman MD. American College of Chest Physicians consensus statement on the respiratory and related management of patients with Duchenne muscular dystrophy undergoing anesthesia or sedation. Chest. 2007 Dec;132(6):1977-86. doi: 10.1378/chest.07-0458.
Related trials for Neuromuscular Disease
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Enrolling by invitation
Online Exercise Program for Transplant Patients: A Pilot Study
Kidney Transplantation, Physical Function
Active, not recruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
RecruitingObservational study
Pain Phenotypes in Individuals With Multiple Sclerosis
Multiple Sclerosis, Chronic Pain
Not yet recruitingPhase 2
A Study of Bimagrumab in Participants With HFpEF
Heart Failure, Diastolic, Diastolic Heart Failure
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.
Chris Landon, MD principal investigator
Landon Pediatric Foundation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neuromuscular Disease, run by Landon Pediatric Foundation at 1 site in United States.
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 1 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 13 Aug 2014. 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 United States. 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?
1. End tidal CO2 catheter is tolerated for six hours or more (time frame: 3 months). 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 NCT00695591, which also lists 4 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 13 Aug 2014.