This clinical study is a phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel group study to evaluate two doses of orally administered GLPG2222 in adult subjects with a confirmed diagnosis of CF harbouring one F508del CFTR mutation and a second gating (class III) mutation and on stable treatment with ivacaftor. Up to 35 evaluable subjects are planned to be included in the study. Eligible subjects must be on stable treatment with physician prescribed ivacaftor (Kalydeco®) for at least 28 days at the baseline visit. They will be randomized in a 2:2:1 ratio to receive one of two active doses of GLPG2222 (150 mg q.d. or 300 mg q.d.) or placebo q.d. administered for 29 days. Subjects will be in the study for a minimum of 6 weeks and a maximum of 10 weeks, from screening until the follow-up visit.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
37
GLPG2222 150 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
GLPG2222 300 mg administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
Placebo administered as a ready-to-use oral suspension, once daily (q.d.) for 29 days
The Prince Charles Hospital
Chermside, Australia
The Alfred
Melbourne, Australia
Sir Charles Gairdner Hospital
Nedlands, Australia
Westmead Hospital
Westmead, Australia
UZ Brussel
Brussels, Belgium
UZ Gent
Ghent, Belgium
UZ Leuven
Leuven, Belgium
Fakultni nemocnice v Motole
Prague, Czechia
Universitaetsklinikum Carl Gustav Carus TU Dresden
Dresden, Germany
Universitätsklinikum Erlangen
Erlangen, Germany
...and 12 more locations
Changes in adverse events
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of adverse events
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
Changes in abnormal laboratory
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of laboratory
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
Changes in abnormal vital signs, ECG or physical examination
To evaluate the safety and tolerability of GLPG2222 as compared to placebo in terms of vital signs, ECG or physical examination
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
Change from baseline of Sweat chloride concentration
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
Change from baseline of FEV1 (L) and percent predicted FEV1 for age, gender and height as assessed by spirometry
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
Change from baseline on the respiratory domain of Revised Cystic Fibrosis Questionnaire (CFQ-R)
Time frame: at screening and at each study visit up to day 43 which is the final FU visit
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.