- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01586728
Oxygen Therapy in Cystic Fibrosis (MUCOXY)
Indication and Benefits of Nocturnal Oxygen Therapy in Cystic Fibrosis
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Rationale Oxygen therapy may be proposed to patients with cystic fibrosis (CF) with nocturnal hypoxemia but the indication and benefits of oxygen therapy have not been validated in CF.
Aim of the study and main objective The aim of the study is to evaluate the improvement in nocturnal hypoxemia by the measurement of nocturnal pulse oximetry (SpO2) after 6 weeks of nocturnal oxygen therapy in stable patients with CF.
An improvement is defined by a nocturnal SpO2 > 90% during the whole night.
Objectives and secondary aims
- Comparison of subjective sleep quality during oxygen therapy and room air after a period of 6 weeks by means of 4 validated sleep questionnaires
- Comparison of quality of life during oxygen therapy and room air after a period of 6 weeks by means of a validated questionnaire for CF patients (CFQ).
- Detection of the appearance or increase in nocturnal hypercapnia during oxygen therapy and room air after a period of 6 weeks
- Comparison of a possible arterial pulmonary hypertension during oxygen therapy and room air after a period of 6 weeks by means of an echocardiography
Study design This is an open, multicentre, prospective, randomised and cross over study, comparing in individual patient two periods of 6 weeks: one period with nocturnal oxygen therapy and one period in room air, separated by a wash out period of 2 to 6 weeks.
Inclusion criteria
- Children ≥ 6 years and adults ≥ 18 years with CF in a stable state
- With a forced expiratory volume in one second (FEV1) ≤ 50% of predicted value
- Having a pulse oximetry (SpO2) in room air ≤ 90% for ≥ 10% of the night and/or a SpO2 ≤ 90% for ≥ 10 minutes during the night
- Any patient with prior noninvasive positive pressure ventilation (NPPV) but who has stopped NPPV at least 15 days before the start of the study.
- Written approval by the patient and by the parents in case of a pediatric patient
- Patient having the French social security coverage Non-inclusion criteria
- Patients with a respiratory exacerbation during the last 15 days
- Patients with NPPV or long term oxygen therapy prior to the start of the study and unable to stop this treatment
- Impossibility of a medical examination
- Pregnant patient or breastfeeding patient
Study protocol The study is proposed to the patients during the V0 visit, corresponding to the screening monitoring showing a nocturnal SpO2 in room air ≤ 90% for ≥ 10% of the night and/or a SpO2 ≤ 90% for ≥ 10 minutes during the night. After a period of 2 to 30 days, the written approval is obtained during a routine visit.
Thereafter, the patient will be randomised to receive either:
- nocturnal oxygen therapy with a stable flow to obtain a nocturnal SpO2 >90% (Oxygen period) while continuing his routine long term treatment
- his routine long term treatment without nocturnal oxygen therapy (Air period) During the Oxygen period, the oxygen flow will be at least 1.5 L/min. This flow may be increased up to 2L/min in case of the presence of nocturnal periods with a SpO2 ≤ 90%.
The two periods Oxygen and Air will be separated by a wash out period of 2 to 6 weeks to rule out any long lasting effect of nocturnal oxygen therapy.
At the end of the 2 periods of 6 weeks oxygen or air, the following investigations will be performed:
- A nocturnal evaluation of gas exchange by the Sentec™ monitor (which measures SpO2 and transcutaneous carbon dioxide (PCO2)) and objective sleep quality by means of actigraphy
- an evaluation of subjective sleep quality by means of 4 validates sleep questionnaires
- an evaluation of quality of life by a validated CF questionnaire (CFQ)
- spirometry with blood gases in room air
- an echocardiography
Number of subjects Only 3 studies have evaluated the benefit of oxygen therapy on nocturnal gas exchange during one night and only one study has evaluated the benefit of oxygen therapy after a period of 26 months. This last study is limited by the small number of patients and the low compliance with oxygen therapy.
No study has evaluated the effect of oxygen therapy during a 6 weeks period. This limits the calculation of the number of subjects.
As the study is a cross over study, a number of 30 analysable patients is estimated to be sufficient for the evaluation of the primary outcome measure and the secondary outcomes. Because of the possibility of premature withdrawal, it is planned to include 50 patients.
Total duration of the study: maximum 22.5 months
Inclusion period: 18 months
Duration of participation for one patient: maximum 4.5 months
Statistical analysis and final report: 3 months
Number of participating centres: 8
Estimation of the mean number of patients included per centre: 3 patients / 3 months / centre
Expected results The results of the present study should help to define the criteria to start long term oxygen therapy and to assess the potential benefits of this treatment in stable patients with CF.
Study Type
Enrollment (Actual)
Phase
- Not Applicable
Contacts and Locations
Study Locations
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Paris, France, 75012
- Service de pneumologie pédiatrique, Hôpital Armand Trousseau
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Children ≥ 6 years and adults ≥ 18 years with CF in a stable state
- With a forced expiratory volume in one second (FEV1) ≤ 50% of predicted value
- Having a pulse oximetry (SpO2) in room air ≤ 90% for ≥ 10% of the night and/or a SpO2 ≤ 90% for ≥ 10 minutes during the night
- Any patient with prior noninvasive positive pressure ventilation (NPPV) but who has stopped NPPV at least 15 days before the start of the study.
- Written approval by the patient and by the parents in case of a pediatric patient
- Patient having the French social security coverage
Exclusion Criteria:
- Patients with a respiratory exacerbation during the last 15 days
- Patients with NPPV or long term oxygen therapy prior to the start of the study and unable to stop this treatment
- Impossibility of a medical examination
- Pregnant patient or breastfeeding patient
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: CROSSOVER
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
OTHER: Air - oxygen
One period in room air and one period with nocturnal oxygen therapy, separated by a wash out period of 2 to 6 weeks.
|
During the oxygen period, the oxygen flow will be at least 1.5l/min. This flow may be increased up to 2L/min in case of the presence of nocturnal periods with SpO2 <= 90%. |
OTHER: Oxygen - Air
One period with nocturnal oxygen therapy and one period in room air, separated by a wash out period of 2 to 6 weeks.
|
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
the improvement in nocturnal hypoxemia by the measurement of nocturnal pulse oximetry (SpO2) after 6 weeks of nocturnal oxygen therapy in stable patients with CF
Time Frame: after a 6 weeks period
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after a 6 weeks period
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Comparison of subjective sleep quality during oxygen therapy and room air after a period of 6 weeks by means of 4 validated sleep questionnaires
Time Frame: after a 6 weeks period
|
after a 6 weeks period
|
Comparison of quality of life during oxygen therapy and room air after a period of 6 weeks by means of a validated questionnaire for CF patients (CFQ).
Time Frame: after a 6 weeks period
|
after a 6 weeks period
|
Detection of the appearance or increase in nocturnal hypercapnia during oxygen therapy and room air after a period of 6 weeks
Time Frame: after a 6 weeks period
|
after a 6 weeks period
|
Comparison of a possible arterial pulmonary hypertension during oxygen therapy and room air after a period of 6 weeks by means of an echocardiography
Time Frame: after a 6 weeks period
|
after a 6 weeks period
|
Collaborators and Investigators
Collaborators
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ESTIMATE)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- P 100801
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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