Long-term Extension Study of the Safety and Pharmacokinetics of QCC374 in PAH Patients

Back to results

Primary Purpose

Status

Terminated

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

QCC374

About this trial

This is an interventional treatment trial for Pulmonary Arterial Hypertension focused on measuring Pulmonary hypertension (PH),, Increase blood pressure in the pulmonary artery, Increased blood pressure in the pulmonary vein, Increased blood pressure in the lung vasculature, Shortness of breath, Dizziness, Fainting, Leg swelling, Cough, Angina pector

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Written informed consent must be obtained before any assessment is performed.
Subject was enrolled in the QCC374X2201 study and completed per protocol

Exclusion Criteria:

Subjects who have started receiving prostacyclin (epoprostenol), prostacyclin analogs (i.e. trepostinil, iloprost, beraprost) or prostacyclin receptor agonists (i.e. selexipag) since the last study drug intake in the QCC374X2201 study.
Females who are pregnant, or who plan to become pregnant during the study, or who are breastfeeding
Any known factor or disease that may interfere with treatment compliance or study conduct (i.e. drug or alcohol dependence)
Subjects who withdrew consent from the study QCC374X2201

Sites / Locations

Novartis Investigative Site
Novartis Investigative Site
Novartis Investigative Site
Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

QCC374

Arm Description

placebo patients from QCC374X2201 rolled into extension study will start at 0.03mg b.i.d. or 0.06mg b.i.d. and have the opportunity to up-titrate 0.12mg -active patients will continue at the dose they finished on the QCC374X2201 study

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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 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.

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.

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

Change From Baseline in Six Minute Walk Distance (6MWD)

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.

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.

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.

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.

Full Information

NCT ID

NCT02939599

First Posted

October 12, 2016

Last Updated

December 9, 2020

Sponsor

Novartis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT02939599

Brief Title

Long-term Extension Study of the Safety and Pharmacokinetics of QCC374 in PAH Patients

Official Title

Long-term, Open Label, Multicenter, Extension Study to Evaluate the Safety and Tolerability of QCC374 in Patients With Pulmonary Arterial Hypertension (PAH)

Study Type

Interventional

2. Study Status

Record Verification Date

February 2020

Overall Recruitment Status

Terminated

Why Stopped

Study was terminated early for strategic reasons. Only Part I of the study was completed.

Study Start Date

February 1, 2018 (Actual)

Primary Completion Date

November 6, 2018 (Actual)

Study Completion Date

November 6, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

No

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pulmonary Arterial Hypertension

Keywords

Pulmonary hypertension (PH),, Increase blood pressure in the pulmonary artery, Increased blood pressure in the pulmonary vein, Increased blood pressure in the lung vasculature, Shortness of breath, Dizziness, Fainting, Leg swelling, Cough, Angina pector

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

5 (Actual)

8. Arms, Groups, and Interventions

Arm Title

QCC374

Arm Type

Experimental

Arm Description

placebo patients from QCC374X2201 rolled into extension study will start at 0.03mg b.i.d. or 0.06mg b.i.d. and have the opportunity to up-titrate 0.12mg -active patients will continue at the dose they finished on the QCC374X2201 study

Intervention Type

Drug

Intervention Name(s)

QCC374

Intervention Description

0.015mg and 0.06mg

Primary Outcome Measure Information:

Title

Number of Participants Who Experienced Adverse Events (AEs), Serious Adverse Events (SAEs) in Patients With PAH Over a Two Year Period

Description

Patients with all (serious and non-serious) adverse events, serious adverse events and death were reported

Time Frame

Two years

Secondary Outcome Measure Information:

Title

Maximum Observed Plasma Concentration (Cmax)

Description

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

Title

Time to Reach the Maximum Plasma Concentration (Tmax)

Description

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

Title

Area Under the Plasma Concentration-time Curve From 0 to the Last Measurable Concentration (AUClast)

Description

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

Title

Area Under the Plasma Concentration Time Curve From 0 to the End of a Dosing Interval (AUCtau)

Description

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

Title

Change From Baseline in Six Minute Walk Distance (6MWD)

Description

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

Title

Change in Tricuspid Annular Peak Systolic Velocity (TA S') at Week 16 (Day 112) Using Echocardiography

Description

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

Title

Change From Baseline in RV Tei Index at Week 16 (Day 112) Using Echocardiography

Description

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

Title

Change From Baseline in RV Fractional Area Change at Week 16 (Day 112) Using Echocardiography

Description

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

No

Eligibility Criteria

Inclusion Criteria: Written informed consent must be obtained before any assessment is performed. Subject was enrolled in the QCC374X2201 study and completed per protocol Exclusion Criteria: Subjects who have started receiving prostacyclin (epoprostenol), prostacyclin analogs (i.e. trepostinil, iloprost, beraprost) or prostacyclin receptor agonists (i.e. selexipag) since the last study drug intake in the QCC374X2201 study. Females who are pregnant, or who plan to become pregnant during the study, or who are breastfeeding Any known factor or disease that may interfere with treatment compliance or study conduct (i.e. drug or alcohol dependence) Subjects who withdrew consent from the study QCC374X2201

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Novartis Pharmaceuticals

Organizational Affiliation

Novartis Pharmaceuticals

Official's Role

Study Director

Facility Information:

Facility Name

Novartis Investigative Site

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15261

Country

United States

Facility Name

Novartis Investigative Site

City

Dresden

ZIP/Postal Code

01307

Country

Germany

Facility Name

Novartis Investigative Site

City

Heidelberg

ZIP/Postal Code

69120

Country

Germany

Facility Name

Novartis Investigative Site

City

Cambridge

State/Province

Cambridgeshire

ZIP/Postal Code

CB23 3RE

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

Links:

URL

https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=486

Description

A Plain Language Trial Summary is available on novartisclinicatrials.com

Pulmonary Arterial Hypertension

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial