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Neuromuscular DiseasesNCT03312361

Assessing Air Travel Safety in Neuromuscular Dystrophy: Standard Versus Prolonged High Altitude Simulation Tests

A study testing 15% oxygen in people with Neuromuscular Diseases and High Altitude Hypoxia, run by The Hospital for Sick Children at 1 site in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

To determine if a prolonged high altitude simulation test (HAST) lasting two hours, identifies more patients at risk of respiratory failure than the standard HAST lasting 20 minutes, in patients with neuromuscular disease (NMD) and severe pulmonary restriction. Read more about this study

Who is it for?
Ages 5 years and older
Conditions the study is for
Neuromuscular Diseases, High Altitude Hypoxia
What is being tested?
15% oxygen
Could I receive a placebo?
No, everyone receives an active treatment
How many people
23 participants
Where
1 siteCanada
Study dates
1 Apr 2016to 1 Apr 2018
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
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • individuals with a confirmed diagnosis of NMD
  • age ≥ 5 years of age
  • absence of hypoxia at rest, defined as oxygen saturation ≥ 92% for two consecutive minutes while at rest and breathing room air
  • absence of daytime hypercapnea at rest, defined by partial pressure of carbon dioxide (pCO2) less than 45 mmHg on capillary or arterial blood gas at rest
  • patient or authorized substitute decision maker provides consent to study participation.

Who cannot join

  • current respiratory tract infection
  • inability to comply with the study procedures
  • severe heart failure as defined by uncontrolled symptoms of heart failure, or left ventricular ejection fraction < 30%
  • presence of a tracheostomy
  • use of domiciliary bilevel positive airway pressure (BPAP) for more than 12 hours daily
  • recent hospital admission within the past 4 weeks.

Trial locations and contacts

1 site in 1 country.

Canada1 site
  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

Other trials in Canada

About this study

To determine if a prolonged high altitude simulation test (HAST) lasting two hours, identifies more patients at risk of respiratory failure than the standard HAST lasting 20 minutes, in patients with neuromuscular disease (NMD) and severe pulmonary restriction. To evaluate the safety of supplemental oxygen administered in those with a positive HAST in the NMD population.

Read the detailed description

Study Procedures

Patients were screened for eligibility during a regularly scheduled clinic visit in which spirometry was performed in accordance with the American Thoracic Society recommendations.4 If eligible, they were approached by a Research Coordinator and invited to participate in the study. The study visit was organized within 4 weeks of screening and included both diagnostic and (if applicable) therapeutic HCT trials. After enrolment, patients' local medical records were reviewed for baseline data.

Baseline Assessment:

On the day of the HCT, a brief focused history including current respiratory symptoms was performed. Study participants sat for a minimum of 5 minutes before baseline measurements were taken. Vital signs (heart rate, respiratory rate, blood pressure), capillary blood gas, transcutaneous capnography and pulse oximetry were performed immediately prior to the start of testing. The transcutaneous CO2 and SpO2 values obtained just prior to provision of the gas mixture were considered the baseline values.

Testing Procedures: Study participants underwent a standard 20 minute HCT and if eligible (i.e. not positive or test not terminated) then immediately continued on to complete the 120 minute extended HCT. A gas mixture of 15% oxygen in nitrogen from a specially prepared cylinder was administered via a non-rebreather mask sealed to the face. Flow was adjusted for patient comfort, and to match inspiratory flow by maintaining non-rebreather bag inflation thus ensuring no entrainment of room air (between 6-15 L/minute). The patient would breathe this mixture for up to 120 minutes, or until they met stopping criteria. Continuous pulse oximetry, transcutaneous capnography and respiratory rate were recorded throughout the HCT. Patients were asked to indicate the severity of dyspnea every 10 minutes using the modified Borg scale. Capillary blood gases were drawn at the end of the standard and prolonged HCTs (i.e. immediately after 20 and 120 minutes, respectively). The patient was observed for 20 minutes after completion of the HCT, or until oxygen saturation and transcutaneous pCO2 returned to baseline levels. The HCT was considered positive and the test was stopped if any of the following occurred: i) oxygen saturation <88% for two consecutive minutes (as per institutional cutoff for prescription of oxygen); ii) ≥ 10mmHg rise from baseline in transcutaneous CO2 sustained for 2 minutes and confirmed by capillary blood gas; iii) severe symptoms such as dyspnea or confusion; iv) or participant request to stop testing. Repeat complete vital signs were performed prior to the end of the study visit.

Being tested
15% oxygen
Medical subject headings
Neuromuscular Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 15% oxygenDrug

    Participants will inhale 15% oxygen for 2 hours to see how it affects them.

Study groups in NCT03312361
Study groupRoleWhat they receive
15% OxygenExperimentalAll participants will breath in 15% oxygen for 2 hours
Receives: Drug: 15% oxygen

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Prolonged high altitude simulation test

    To determine if a prolonged high altitude simulation test (HAST) lasting two hours, identifies more patients at risk of respiratory failure than the standard HAST lasting 20 minutes, in patients with NMD and severe pulmonary restriction

    Measured: 3 years

Secondary outcomes (1)
  1. Interventional high altitude simulation test

    In patients with a positive standard or prolonged HAST, we will evaluate the safety of supplemental oxygen administered during a HAST for this population

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Oct 2017
  2. Study start1 Apr 2016
  3. Primary completion1 Apr 2018
  4. Study completion1 Apr 2018

Resources

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
Diagnosis
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
23 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03312361
Sponsor's protocol ID
1000051539
Status last verified
Aug 2021
First submitted
12 Oct 2017
Last updated
19 Aug 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A study testing 15% oxygen in people with Neuromuscular Diseases and High Altitude Hypoxia, run by The Hospital for Sick Children 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 5 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 19 Aug 2021. 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. 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?

Prolonged high altitude simulation test (time frame: 3 years). 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 19 Aug 2021.

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