Study design (if review, criteria of inclusion for studies)
phase II, placebo-controlled, double-blinded, single-center.
Participants
24 patients with CF and stable lung disease
Interventions
multiple (5 consecutive days), rising-dose (daily dose, 0.5, 1.5, or 2.5 mg of Moli1901) study was conducted to investigate the safety and tolerability of multiple doses of aerosolized inhaled Moli1901
Outcome measures
investigate the safety and tolerability of multiple doses of aerosolized inhaled Moli1901
Main results
Moli1901 was well tolerated in all but one CF patient, in whom a transient significant decrease in FEV(1) developed following inhalation, which resolved spontaneously, and in a second patient in whom transient throat numbness developed during drug inhalation. A significant improvement of FEV(1) was observed in the group receiving treatment with 2.5 mg/d Moli1901 compared to the group receiving placebo (p = 0.01 [Wilcoxon test]). Moli1901 was not detected in the plasma of the highest dose group.
Authors' conclusions
The inhalation of Moli1901 up to a total cumulative dose of 12.5 mg appears to be safe in adult patients with CF. In addition, Moli1901 had a sustained beneficial effect on pulmonary function, which supports further studies of its efficacy in CF patients.
Keywords: Adolescent; Adult; Airway clearance drugs -expectorants- mucolytic- mucociliary-; Inhalation OR nebulised; Moli1901; pharmacological_intervention; Respiratory System Agents; Lancovutide; Duramycin;