EPS3.05 GLPG2222 in subjects with cystic fibrosis homozygous for F508del: results from a phase II study (FLAMINGO)

Ent, K.C. van der; Minic, P.; Verhulst, Stijn; Braeckel, Eva Van; Flume, Patrick A.; Boas, Steven R.; Geller, David E.; Kock, Herman de; Loyau, S.; Muller, Karine; Kanters, D.; Conrath, Katja; Steen, Olivier Van de

doi:10.1016/s1569-1993(18)30250-9

Cited by 3 publications

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“…The results of the sensitivity analysis were similar to the results of the primary ANCOVA analysis (Table S6). Trends in data for change in FEV 1 from baseline were similar to those for ppFEV 1 in both studies (absolute FEV 1 data are shown in Figs. S4 and S5).…”

Section: Pulmonary Functionsupporting

confidence: 66%

CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials

Bell

Barry

Boeck

et al. 2019

Journal of Cystic Fibrosis

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a b s t r a c tBackground: Several treatment approaches in cystic fibrosis (CF) aim to correct CF transmembrane conductance regulator (CFTR) function; the efficacy of each approach is dependent on the mutation(s) present. A need remains for more effective treatments to correct functional deficits caused by the F508del mutation. Methods: Two placebo-controlled, phase 2a studies evaluated GLPG2222, given orally once daily for 29 days, in subjects homozygous for F508del (FLAMINGO) or heterozygous for F508del and a gating mutation, receiving ivacaftor (ALBATROSS). The primary objective of both studies was to assess safety and tolerability. Secondary objectives included assessment of pharmacokinetics, and of the effect of GLPG2222 on sweat chloride concentrations, pulmonary function and respiratory symptoms. Results: Fifty-nine and 37 subjects were enrolled into FLAMINGO and ALBATROSS, respectively. Treatment-related treatment-emergent adverse events (TEAEs) were reported by 29.2% (14/48) of subjects in FLAMINGO and 40.0% (12/30) in ALBATROSS; most were mild to moderate in severity and comprised primarily respiratory, gastrointestinal, and infection events. There were no deaths or discontinuations due to TEAEs. Dose-dependent decreases in sweat chloride concentrations were seen in GLPG2222-treated subjects (maximum decrease in FLAMINGO: -17.6 mmol/L [GLPG2222 200 mg], p b 0.0001; ALBATROSS: -7.4 mmol/L [GLPG2222 300 mg], p b 0.05). No significant effects on pulmonary function or respiratory symptoms were reported. Plasma GLPG2222

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Section: Pulmonary Functionsupporting

confidence: 66%