Clinical Trial: Macitentan Use in an Idiopathic Pulmonary Fibrosis Clinical Study

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Double-blind, Randomized, Placebo-controlled, Multicenter, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Macitentan in Patients With Idiopathic Pul

Brief Summary:

The AC-055B201/MUSIC study is a Phase II study, comparing one dose of ACT-064922 (macitentan) 10 mg with placebo in patients with idiopathic pulmonary fibrosis (IPF). The main study objective is to demonstrate that macitentan positively affects the forced vital capacity (FVC) in comparison with placebo in patients with idiopathic pulmonary fibrosis (IPF).

The secondary objectives are to evaluate the effect of macitentan on the time to disease worsening or death in patients with IPF, and to evaluate the benefit/risk profile of macitentan in the treatment of patients with IPF.


Detailed Summary: The study included two treatment periods: Period 1 (fixed duration) from randomization up to the primary endpoint evaluation (Month 12 or earlier in case of premature discontinuation of study drug) and Period 2 (variable duration) from the primary endpoint evaluation visit up to the end of study (EOS). EOS occurred when the last patient randomized and not prematurely discontinued completed Period 1.
Sponsor: Actelion

Current Primary Outcome: Forced Vital Capacity (FVC) at Baseline and End of Period 1 [ Time Frame: 12 months ]

FVC was measured at baseline and at the end of Period 1. The same equipment and tester were used during the course of the study. The equipment was calibrated and the calibration documented prior to each patient's measurement. The person responsible for conducting the pulmonary function tests was required to comply with the study guidelines and the American Thoracic Society/European Respiratory Society joint criteria on lung function testing.


Original Primary Outcome: To demonstrate that macitentan positively affects FVC compared with placebo in patients with IPF. [ Time Frame: Baseline - 12 months ]

Current Secondary Outcome: Number of Patients at Risk of Event of Disease Worsening or Death up to the End of Study [ Time Frame: Up to end of study (Up to 24 months) ]

Disease worsening was indicated by pulmonary function test/idiopathic pulmonary fibrosis worsening (PFT/IPF) or acute respiratory decompensation of IPF.

PFT/IPF worsening was indicated by the occurrence of both of the following: confirmed by two tests at least 4 weeks apart, as defined by the occurrence of both of the following: decrease from baseline ≥ 10% in forced vital capacity and decrease from baseline ≥ 15% in corrected diffusing capacity of the lung for carbon monoxide.

Acute respiratory decompensation of IPF was defined as an unexplained rapid deterioration (over a period of less than 4 weeks) of the patient's condition with increasing shortness of breath requiring oxygen supplementation ≥ 5 L/min to maintain a resting oxygen saturation ≥ 90% or arterial oxygen pressure ≥ 55 mmHg (sea level) or 50 mmHg (high altitude).



Original Secondary Outcome: To evaluate the effect of macitentan on the time to disease worsening or death in patients with IPF and to evaluate the safety and tolerability of macitentan in this patient population. [ Time Frame: 3 years ]

Information By: Actelion

Dates:
Date Received: May 14, 2009
Date Started: May 2009
Date Completion:
Last Updated: January 2, 2014
Last Verified: January 2014