The present study is designed to assess the safety and tolerability of escalating, multiple ascending doses of Cavosonstat (N91115) in healthy subjects.
This study will assess the safety and tolerability of escalating, multiple ascending doses of Cavosonstat (N91115) in healthy subjects. Approximately 5 ascending cohorts are planned with approximately 8 subjects per cohort (6 active, 2 placebo). Each subject will undergo screening (Day -28 to Day -2) and, if eligible, return to the unit on Day -1 when eligibility will be reconfirmed. Eligible subjects will be randomized in a 3:1 ratio to receive investigational medicinal product (IMP) N91115 (daily \[QD\] or every 12 hours \[Q12H\]) or matching placebo (QD or Q12H) for 7 days and will be followed for safety while housed in the clinical research unit (CRU) until discharge on Day 8. Pharmacokinetics will be followed from Study Day 1 through the morning of Study Day 8. The subjects will be discharged from the CRU and complete the post treatment withdrawal phase, including a follow-up phone call on Day 15.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
BASIC_SCIENCE
Masking
QUADRUPLE
Enrollment
32
CFTR modulator that stabilizes CFTR
Matched placebo
DaVita Clinical Research
Lakewood, Colorado, United States
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Safety assessments based on clinical evaluations, laboratory assessments, and adverse events
Time frame: 14 days
Pharmacokinetic parameters of N91115 and metabolites (Amount of analyte excreted in the urine [Ae])
Pharmacokinetic parameter of N91115 and metabolites will be measured in urine.
Time frame: 7 days
Pharmacokinetic parameters of N91115 and metabolites (% analyte excreted in the urine [Fe])
Pharmacokinetic parameter of N91115 and metabolites will be measured in urine.
Time frame: 7 days
Pharmacokinetic parameters of N91115 and metabolites (area under the curve [AUC])
Pharmacokinetic parameter of N91115 and metabolites will be measured in plasma.
Time frame: 7 days
Pharmacokinetic parameters of N91115 and metabolites (maximum concentration [Cmax])
Pharmacokinetic parameter of N91115 and metabolites will be measured in plasma.
Time frame: 7 days
Pharmacokinetic parameters of N91115 and metabolites (clearance [CL])
Pharmacokinetic parameter of N91115 and metabolites will be measured in urine and plasma.
Time frame: 7 days
Pharmacokinetic parameters of N91115 and metabolites (accumulation index [Racc])
Pharmacokinetic parameter of N91115 and metabolites will be measured in plasma.
Time frame: 7 days
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Pharmacokinetic parameters of N91115 and metabolites (Terminal elimination half-life [t1/2])
Pharmacokinetic parameter of N91115 and metabolites will be measured in plasma.
Time frame: 7 days
Pharmacokinetic parameters of N91115 and metabolites (time of maximum concentration [Tmax])
Pharmacokinetic parameter of N91115 and metabolites will be measured in plasma.
Time frame: 7 days
Pharmacokinetic parameters of N91115 and metabolites (metabolite to parent exposure ratio [M/P ratio])
Pharmacokinetic parameter of N91115 and metabolites will be measured in plasma.
Time frame: 7 days
Pharmacokinetic parameters of N91115 and metabolites (terminal elimination rate constant [lambda z])
Pharmacokinetic parameter of N91115 and metabolites will be measured in plasma.
Time frame: 7 days