Table 3

Overview of assessments by stage of study

Stage 0Stage 1Stage 2Stage 3Stage 4End
Time pointEligibilityBaselineEnd of week
1*		End of week
2		End of week
3		End of 3 monthsWithdrawal or completion
History and demographicsDemographics✓
 Physical examination✓
 Medicines Hx and LTOT✓✓
 Smoking Hx✓
 Height/Weight✓
 6-month weight Hx✓
InvestigationsLFT, FBC, Elec, CC✓
 Serum testosterone✓
 FEV1/FVC✓
 End-tidal CO2 ✓✓✓✓
 Pulse oximetry✓✓✓✓
 Vital signs✓✓✓✓✓
 Activity Fitbit monitor✓✓✓
Participant mediatedDiary entries✓✓✓✓✓
 HADS✓✓✓
 CAT✓✓✓✓
 CRQ-DS and CRQ-M✓✓✓
 EQ-5D-5L✓✓✓✓
 Epworth SS✓✓✓
 LSQ✓✓✓
 KSS✓✓
 ESAS✓✓✓
 Blinded preference✓✓✓✓✓
 SOWS✓
Research team mediatedAKPS✓✓✓✓✓
 mMRC✓✓✓✓
 GIC✓✓✓✓✓
 Barthel Index✓✓
 Blinded preference✓✓✓
 Side effects✓✓✓✓✓✓
 CCI✓
 SLUMS✓
 Compliance✓✓✓✓
EconomicInpatient stay days✓✓✓✓
 ED visits✓✓✓✓
 Med/health visits✓✓✓✓
 Date of death✓
SubstudiesZCBS✓✓✓✓✓
 PG blood sample✓
 PK and PD blood sample✓✓
 Home sleep study✓✓
 Laboratory sleep study✓✓
 Driving simulation✓✓
 Qualitative interview✓
 Serum testosterone✓✓

  • *Primary endpoint.

  • AKPS, Australian-modified Karnofsky Performance Status; CAT, COPD Assessment Test; CC, creatinine clearance; CCI, Charlson Comorbidity Index; COPD, chronic obstructive pulmonary disease; CO2, carbon dioxide; CRQ-DS, Chronic Respiratory Questionnaire—Dyspnoea Subscale; CRQ-M, Chronic Respiratory Questionnaire—Mastery; ED, emergency department; Elec, electrolytes; Epworth SS, Epworth Sleepiness Scale; ESAS, Edmonton Symptom Assessment Scale; EQ-5D-5L, Five-Level EuroQol five dimensions questionnaire; FEV1/FVC, forced expiratory volume in 1 s over forced expiratory volume; FBC, full blood count; GIC, Global Impression of Change; HADS, Hospital Anxiety and Depression Scale; Hx, history; KSS, Karolinska Sleepiness Scale; LFT, Liver Function Tests; LSQ, Leeds Sleep Questionnaire; LTOT, long-term oxygen therapy; Med, medical; mMRC, Modified Medical Research Council Dyspnoea Scale; PD, pharmacodynamic; PG, pharmacogenetic; PK, pharmacokinetic; SLUMS, St Louis University Mental State Examination; SOWS, Subjective Opioid Withdrawal Scale; ZCBS, Zarit Caregiver Burden Scale (short form).