Clinical trial NCT00157183: Non-invasive Ventilation and Oxygen Therapy in Cystic Fibrosis Patients With Nocturnal Oxygen Desaturation
Official title: Non-invasive Ventilation and Oxygen Therapy in Cystic Fibrosis Patients With Nocturnal Oxygen Desaturation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Cystic Fibrosis
Keywords: cystic fibrosis
hypoxia
oxygen
noninvasive ventilation
quality of life
Lead sponsor: Bayside Health
Sponsor class: OTHER_GOV
Collaborators: name: National Health and Medical Research Council, Australia
name: Monash University
name: Cystic Fibrosis Federation Australia
First posted: 2005-09-12
Enrollment: 59
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine whether correction of low nighttime oxygen (O2) levels and/ or high carbon dioxide levels in patients with cystic fibrosis improves their quality of life. The treatments being used overnight are (1)O2 (2)pressurised air which assists breathing (non-invasive positive pressure ventilation, NIPPV)

Detailed description: Cystic fibrosis is the commonest life-limiting genetic disorder in the Caucasian population with a median survival of 31 years. Lung disease is responsible for the majority of morbidity and mortality and correlates with declining quality of life. Respiratory failure is the primary cause of death. Daytime respiratory failure (hypoxia with pO2\<55 and/or hypercapnia with pCO2\>50) is associated with a worse prognosis with a 2-year survival of 50%. Nocturnal respiratory failure (greater than 5% of the night spent with SpO2\<90% and/or rise in PtcCO2\>10mmHg overnight) is a precursor to the development of daytime respiratory failure. It has been postulated that earlier treatment of respiratory failure may improve outcome and quality of life.

Intervention: Nocturnal O2 and bilevel NIPPV in CF patients with nocturnal respiratory failure, compared to nocturnal placebo (air). Crossover trial utilising patients as their own control.

Aims: (1) To assess the effects of non-invasive ventilation (NIV) and oxygen (O2) therapy on quality of life, hospital admission rate, sleep quality and exercise tolerance in CF patients with NRF (2) To identify a level of severity of NRF where treatment with NIV is effective

Eligibility criteria: Inclusion Criteria:

proven diagnosis cystic fibrosis, age 18 years or older, FEV1\< 70% predicted normal, clinically stable (no admission or antibiotics last 2 weeks, OR end of admission where further clinical improvement not expected), nocturnal respiratory failure (SpO2\<90% for \> 10% of night or rise in PtcCO2 \> 5 mmHg in REM), daytime hypercapnia (PaCO2\> 45 mmHg)

Exclusion Criteria:

Previous home O2 or NIV use, Sedative medications, Cardiac/renal/endocrine/neurological disease likely to compromise ventilatory control

Primary outcomes:
measure: Quality of life questionnaires:
measure: CFQoL questionnaire (Gee,Thorax,2000)(a priori chest, physical function, treatment, emotion domains)
measure: Epworth Sleepiness Scale
measure: Pittsburgh Sleep Quality Index
measure: CF Subjective Symptoms Sleep disturbance Questionnaire (CSQ-in house)
measure: Medical Research Council Dyspnea Scale
measure: Baseline Dyspnea Index, Transitional Dyspnea Index
measure: Work or Study status
measure: Physiological:
measure: Nocturnal SpO2, nocturnal rise in transcutaneous CO2
measure: Daytime arterial blood gases (PaCO2, PaO2)
Secondary outcomes:
measure: Admission rate
measure: Lung function tests (FEV1, FVC, RV/ TLC)
measure: Modified CF shuttle walk test
measure: Neurocognitive testing (psychomotor vigilance task, Stroop, Controlled Oral Word Association Test, Trails A and B, digit recall forwards backwards)
measure: PSG (sleep efficiency, arousal index, % REM sleep, urinary catecholamines)
measure: Serum cytokines (IL-6, TNF alpha, IL-1 beta)
Other outcomes:
Locations:
city: Melbourne
country: Australia
facility: The Alfred
state: Victoria
Study officials:
affiliation: The Alfred
name: Matthew T Naughton, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Young AC, Wilson JW, Kotsimbos TC, Naughton MT. The impact of nocturnal oxygen desaturation on quality of life in cystic fibrosis. J Cyst Fibros. 2011 Mar;10(2):100-6. doi: 10.1016/j.jcf.2010.11.001. Epub 2010 Dec 30.