This is a long-term open-label safety extension to the Phase 2a study of inhaled QCC374 in adult patients with PAH. This study provides the patients who completed the QCC374X2201 study with the option to continue receiving QCC374. The study will monitor the long-term safety, tolerability and efficacy of QCC374 in patients with PAH.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
0.015mg and 0.06mg
Novartis Investigative Site
Pittsburgh, Pennsylvania, United States
Novartis Investigative Site
Dresden, Germany
Novartis Investigative Site
Heidelberg, Germany
Novartis Investigative Site
Cambridge, Cambridgeshire, United Kingdom
Number of Participants Who Experienced Adverse Events (AEs), Serious Adverse Events (SAEs) in Patients With PAH Over a Two Year Period
Patients with all (serious and non-serious) adverse events, serious adverse events and death were reported
Time frame: Two years
Maximum Observed Plasma Concentration (Cmax)
Cmax is the maximum (peak) observed plasma drug concentration after single dose administration. PK parameters were calculated from plasma concentration-time data using non-compartmental methods. Only descriptive analysis performed
Time frame: 16 weeks
Time to Reach the Maximum Plasma Concentration (Tmax)
Tmax is the time to reach maximum plasma concentration after single dose administration. PK parameters were calculated from plasma concentration-time data using non-compartmental methods. Only descriptive analysis performed.
Time frame: 16 Weeks
Area Under the Plasma Concentration-time Curve From 0 to the Last Measurable Concentration (AUClast)
AUClast is the area under the plasma concentration-time curve from time zero to the last measurable concentration sampling time. PK parameters were calculated from plasma concentration-time data using non-compartmental methods. Only descriptive analysis performed.
Time frame: 16 weeks
Area Under the Plasma Concentration Time Curve From 0 to the End of a Dosing Interval (AUCtau)
AUCtau is the area under the plasma concentration-time curve from time zero to the end of the dosing interval. PK parameters were calculated from plasma concentration-time data using non-compartmental methods. Only descriptive analysis performed
Time frame: 16 Weeks
Change From Baseline in Six Minute Walk Distance (6MWD)
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The Six Minute Walk Test measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface. The goal is for the individual to walk as far as possible in six minutes. The individual is able to self-pace and rest as needed as they traverse back and forth along a marked walkway. Only descriptive analysis performed.
Time frame: 16 weeks
Change in Tricuspid Annular Peak Systolic Velocity (TA S') at Week 16 (Day 112) Using Echocardiography
Key Right Ventricular (RV) function endpoints such as Tricuspid Annular Peak Systolic Velocity (TA S') were assessed with echocardiography. Only descriptive analysis performed.
Time frame: Two Years
Change From Baseline in RV Tei Index at Week 16 (Day 112) Using Echocardiography
Key Right Ventricular (RV) function endpoints such as Tei Index were assessed with echocardiography. The RV Tei index is using both systolic and diastolic time intervals to evaluate the overall global dysfunction of the right ventricle in PAH patients. A lower number in RV Tei Index indicates an improvement. Only descriptive analysis performed.
Time frame: 16 weeks
Change From Baseline in RV Fractional Area Change at Week 16 (Day 112) Using Echocardiography
Key Right Ventricular (RV) function endpoints such as Tei Index were assessed with echocardiography. The RV Tei index is using both systolic and diastolic time intervals to evaluate the overall global dysfunction of the right ventricle in PAH patients. A lower number in RV Tei Index indicates an improvement. Only descriptive analysis performed.
Time frame: 16 weeks