Pulmonary Arterial HypertensionPhase 2NCT02939599
Long-term Extension Study of the Safety and Pharmacokinetics of QCC374 in PAH Patients
A Phase 2 study testing QCC374 in people with Pulmonary Arterial Hypertension, run by Novartis Pharmaceuticals at 4 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- QCC374
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 1 Feb 2018to 6 Nov 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent must be obtained before any assessment is performed.
- Subject was enrolled in the QCC374X2201 study and completed per protocol
Who cannot join
- 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
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
Germany2 sites
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Heidelberg, 69120
United States1 site
Novartis Investigative Site
Pittsburgh, Pennsylvania, 15261
United Kingdom1 site
Novartis Investigative Site
Cambridge, Cambridgeshire, CB23 3RE
About this study
Official title: Long-term, Open Label, Multicenter, Extension Study to Evaluate the Safety and Tolerability of QCC374 in Patients With Pulmonary Arterial Hypertension (PAH)
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.
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- QCC374
- 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
- Medical subject headings
- Pulmonary Arterial Hypertension, Hypertension, Pulmonary, Dyspnea, Dizziness, Syncope, Cough
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QCC374Drug
0.015mg and 0.06mg
| Study group | Role | What they receive |
|---|---|---|
| QCC374 | Experimental | 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 Receives: Drug: QCC374 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Two years
Secondary outcomes (8)
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
Measured: 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.
Measured: 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.
Measured: 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
Measured: 16 Weeks
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2016 (estimated)
- Study start1 Feb 2018
- Primary completion6 Nov 2018
- Study completion6 Nov 2018
Why it stopped: Study was terminated early for strategic reasons. Only Part I of the study was completed.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02939599
- Sponsor's protocol ID
- CQCC374X2201E1
- Status last verified
- Feb 2020
- First submitted
- 12 Oct 2016
- Last updated
- 5 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term, Open Label, Multicenter, Extension Study to Evaluate the Safety and Tolerability of QCC374 in Patients With Pulmonary Arterial Hypertension (PAH)
- ClinicalTrials.gov ID
- NCT02939599
- Acronym / sponsor ID
- CQCC374X2201E1
- Conditions
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was terminated early for strategic reasons. Only Part I of the study was completed.
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 1 Feb 2018 (actual)
- Primary completion
- 6 Nov 2018 (actual)
- Study completion
- 6 Nov 2018 (actual)
- First posted
- 20 Oct 2016 (estimated)
- Results
- Posted 28 Feb 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| QCC374 | Experimental | Drug: QCC374placebo 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Written informed consent must be obtained before any assessment is performed.
- Subject was enrolled in the QCC374X2201 study and completed per protocol
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| QCC3740.015mg and 0.06mg | Drug | QCC374 |
Primary outcome
Number of Participants Who Experienced Adverse Events (AEs), Serious Adverse Events (SAEs) in Patients With PAH Over a Two Year Period
Time frame: Two years
Patients with all (serious and non-serious) adverse events, serious adverse events and death were reported
8 secondary outcomes
Maximum Observed Plasma Concentration (Cmax)
Time frame: 16 weeks
Time to Reach the Maximum Plasma Concentration (Tmax)
Time frame: 16 Weeks
Area Under the Plasma Concentration-time Curve From 0 to the Last Measurable Concentration (AUClast)
Time frame: 16 weeks
Area Under the Plasma Concentration Time Curve From 0 to the End of a Dosing Interval (AUCtau)
Time frame: 16 Weeks
Change From Baseline in Six Minute Walk Distance (6MWD)
Time frame: 16 weeks
Change in Tricuspid Annular Peak Systolic Velocity (TA S') at Week 16 (Day 112) Using Echocardiography
Time frame: Two Years
Change From Baseline in RV Tei Index at Week 16 (Day 112) Using Echocardiography
Time frame: 16 weeks
Change From Baseline in RV Fractional Area Change at Week 16 (Day 112) Using Echocardiography
Time frame: 16 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Trial Summary is available on novartisclinicatrials.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing QCC374 in people with Pulmonary Arterial Hypertension, run by Novartis Pharmaceuticals at 4 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Germany, United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Feb 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Experienced Adverse Events (AEs), Serious Adverse Events (SAEs) in Patients With PAH Over a Two Year Period (time frame: Two years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2021.