May 15, 2007

Inhalation of Moli1901 in patients with cystic fibrosis

Hartmut GrasemannFelix Ratjen


In cystic fibrosis (CF) patients, the absence or dysfunction of the chloride channel CF transmembrane conductance regulator (CFTR) results in reduced chloride ion transport in respiratory epithelial cells. Moli1901 stimulates an alternative chloride channel and may thus compensate for the CFTR deficiency in the airway epithelium of CF patients. A phase II, placebo-controlled, double-blinded, single-center, 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 in 24 patients with CF and stable lung disease. 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. The inhalation of Moli1901 up to a total cumulative dose of 12.5 mg appears to be safe ...Continue Reading

  • References10
  • Citations69


Mentioned in this Paper

Cyclic Peptides
Inhalation of Drugs
Squamous Transitional Epithelial Cell Count
Structure of Respiratory Epithelium
Cystic Fibrosis
Integral to Membrane
Calcium-Activated Chloride Channels
Wilcoxon Signed Rank Test

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