Neuromuscular DiseasesNCT03824223
'Fitness To Fly' Tests In Children With Type II Respiratory Failure
A study testing Hypoxic challenge test in people with Neuromuscular Diseases and Central Hypoventilation, run by Great Ormond Street Hospital for Children NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
The conventional hypoxic challenge test might not predict 'fitness-to-fly' equally well in all patients. Patients at risk of hypercapnia with supplemental oxygen are not well studied. Read more about this study
- Who is it for?
- Ages 0 to 18 years
- Conditions the study is for
- Neuromuscular Diseases, Central Hypoventilation
- What is being tested?
- Hypoxic challenge test
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 1 Feb 2019to 31 Aug 2021
- 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 0 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- ≤18 years
- Neuromuscular weakness or central hypoventilation
- Established nocturnal or intermittent daytime ventilatory support
Who cannot join
- Unable to sit in the plethysmograph
- Cardiac anomaly with right-to-left shunt
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH
About this study
Official title: 'Fitness To Fly' Testing In Children With Neuromuscular Weakness Or Central Hypoventilation: A Feasibility Study
The conventional hypoxic challenge test might not predict 'fitness-to-fly' equally well in all patients. Patients at risk of hypercapnia with supplemental oxygen are not well studied. Th investigators will study children who have either neuromuscular weakness or central hypoventilation and who require nocturnal or intermittent daytime ventilator support.
The study also aims to assess feasibility of a randomised controlled trial (RCT) comparing the standard hypoxic standard test to a modified test designed to better suit children with type two respiratory failure.
Read the detailed description
This study will assess feasibility of pulse oximetry (SpO2), end tidal carbon dioxide (ETCO2) and transcutaneous carbon dioxide partial pressure (TcCO2) measurement during a modified Hypoxic challenge test (HCT) in 12 children with neuromuscular weakness and 12 with central hypoventilation.
- Conditions
- Neuromuscular Diseases, Central Hypoventilation
- Being tested
- Hypoxic challenge test
- Medical subject headings
- Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hypoxic challenge testDiagnostic test
Physiological response to 15% ambient oxygen monitored.
| Study group | Role | What they receive |
|---|---|---|
| new/old HCT component order | Other | new/old hypoxic challenge test (HCT) component order
Where the new test uses pulse oximetry and transcutaneous CO2 monitoring to guide use of ventilatory support and if necessary supplementary oxygen and the old test uses pulse oximetry to titrate supplementary oxygen. Receives: Diagnostic Test: Hypoxic challenge test |
| old/new HCT component order | Other | old/new hypoxic challenge test (HCT) component order
Where the old test uses pulse oximetry to titrate supplementary oxygen and the new test uses pulse oximetry and transcutaneous CO2 monitoring to guide use of ventilatory support and if necessary supplementary oxygen. Receives: Diagnostic Test: Hypoxic challenge test |
What is being measured
The primary outcome is the main question the study is designed to answer.
proof of concept outcome 1
number of children/infants with SpO2 <90/85%
Measured: during hypoxic challenge test
proof of concept outcome 2
number of children with >15 mmHg rise in TcCO2/EtCO2
Measured: during hypoxic challenge test
Secondary outcomes (2)
Feasibility outcome 1: recruitment rate
recruitment rate
Measured: one year
Feasibility outcome 2: protocol completion rate
protocol completion rate
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2019
- Study start1 Feb 2019
- Primary completion31 Aug 2021
- Study completion31 Aug 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseasesStudies 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
- Crossover
- Model description
- Participants randomised to uniform balanced crossover (new/old or old/new hypoxic challenge test components).
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- University College, London
- Responsible party
- Sponsor
- Investigators
- Katharine C Pike, PhD, Principal investigator, Great Ormond Street Hospital for Children NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03824223
- Sponsor's protocol ID
- 17IA16
- Status last verified
- Dec 2021
- First submitted
- 29 Jan 2019
- Last updated
- 8 Dec 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 'Fitness To Fly' Testing In Children With Neuromuscular Weakness Or Central Hypoventilation: A Feasibility Study
- ClinicalTrials.gov ID
- NCT03824223
- Acronym / sponsor ID
- 17IA16
- Conditions
- Neuromuscular Diseases, Central Hypoventilation
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Great Ormond Street Hospital for Children NHS Foundation TrustUniversity, hospital or other
- Collaborators
- University College, London
- Study start
- 1 Feb 2019 (actual)
- Primary completion
- 31 Aug 2021 (actual)
- Study completion
- 31 Aug 2021 (actual)
- First posted
- 31 Jan 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 conventional hypoxic challenge test might not predict 'fitness-to-fly' equally well in all patients. Patients at risk of hypercapnia with supplemental oxygen are not well studied. Th investigators will study children who have either neuromuscular weakness or central hypoventilation and who require nocturnal or intermittent daytime ventilator support.
The study also aims to assess feasibility of a randomised controlled trial (RCT) comparing the standard hypoxic standard test to a modified test designed to better suit children with type two respiratory failure.
Detailed description
This study will assess feasibility of pulse oximetry (SpO2), end tidal carbon dioxide (ETCO2) and transcutaneous carbon dioxide partial pressure (TcCO2) measurement during a modified Hypoxic challenge test (HCT) in 12 children with neuromuscular weakness and 12 with central hypoventilation.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Participants randomised to uniform balanced crossover (new/old or old/new hypoxic challenge test components).
| Arm | Type | Receives |
|---|---|---|
| new/old HCT component order | Other | Diagnostic Test: Hypoxic challenge testnew/old hypoxic challenge test (HCT) component order Where the new test uses pulse oximetry and transcutaneous CO2 monitoring to guide use of ventilatory support and if necessary supplementary oxygen and the old test uses pulse oximetry to titrate supplementary oxygen. |
| old/new HCT component order | Other | Diagnostic Test: Hypoxic challenge testold/new hypoxic challenge test (HCT) component order Where the old test uses pulse oximetry to titrate supplementary oxygen and the new test uses pulse oximetry and transcutaneous CO2 monitoring to guide use of ventilatory support and if necessary supplementary oxygen. |
Who may qualify
- Ages
- 0 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- ≤18 years
- Neuromuscular weakness or central hypoventilation
- Established nocturnal or intermittent daytime ventilatory support
Key exclusion criteria (2 listed)
- Unable to sit in the plethysmograph
- Cardiac anomaly with right-to-left shunt
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 |
|---|---|---|
| Hypoxic challenge testPhysiological response to 15% ambient oxygen monitored. | Diagnostic test | new/old HCT component order, old/new HCT component order |
Primary outcome
proof of concept outcome 1
Time frame: during hypoxic challenge test
number of children/infants with SpO2 <90/85%
proof of concept outcome 2
Time frame: during hypoxic challenge test
number of children with >15 mmHg rise in TcCO2/EtCO2
2 secondary outcomes
Feasibility outcome 1: recruitment rate
Time frame: one year
Feasibility outcome 2: protocol completion rate
Time frame: one year
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 8 Dec 2021.
- Individual participant dataSharing planned: Undecided
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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.
Katharine C Pike, PhD principal investigator
Great Ormond Street Hospital for Children NHS Foundation Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Hypoxic challenge test in people with Neuromuscular Diseases and Central Hypoventilation, run by Great Ormond Street Hospital for Children NHS Foundation Trust at 1 site in United Kingdom.
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 3 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: finished, as of the sponsor's last update on 8 Dec 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 United Kingdom. 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?
proof of concept outcome 1 (time frame: during hypoxic challenge test). The study lists 2 primary outcomes in all. 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 8 Dec 2021.