Pulmonary Cystic FibrosisPhase 1/2NCT02566044

Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis

A Phase 1/2 study testing QBW276 in people with Pulmonary Cystic Fibrosis, run by Novartis Pharmaceuticals at 4 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This is a study of multiple doses of inhaled QBW276 in patients with cystic fibrosis on top of standard of care. The study was divided into 3 Cohorts. Cohorts 1 and 2 are designed to be a randomized, double-blind, placebo-controlled, parallel arm, multiple dose study to assess the safety, tolerability… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Pulmonary Cystic Fibrosis
What is being tested?: QBW276
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 16 participants
Where: 4 sitesin 2 countries
Study dates: 27 Sep 2017to 24 Apr 2018
Phase: Phase 1/2
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

Cohorts 1 and 2 = any genotype on any standard of care treatment
Cohort 3 = F508del homozygotes on standard of care at that time
FEV₁between 40 and 100%
LCI2.5 ≥ 8 if FEV₁is more than 80%

Who cannot join

Adrenal or electrolyte abnormalities
Lung transplant
Autonomic dysfunction (e.g. recurrent episodes of fainting, palpitations, etc.)

Trial locations and contacts

4 sites in 2 countries.

Country

City, postcode or hospital

4 sites in 2 countries

United States2 sites

Novartis Investigative Site
Dallas, Texas, 75390-9034
Novartis Investigative Site
New York, New York, 10032

Other trials in United States

Germany2 sites

Novartis Investigative Site
Cologne, 50924
Novartis Investigative Site
Essen, 45147

Other trials in Germany

About this study

Official title: A Randomized, Double-blind, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Doses of Inhaled QBW276 in Patients With Cystic Fibrosis

The study was terminated after Cohort 2 due to the resource issues.

Conditions: Pulmonary Cystic Fibrosis
Being tested: Placebo, QBW276
Keywords: Cystic Fibrosis
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PlaceboOther
Placebo
QBW276Drug
0.3 mg and 1.5 mg strengths

Study groups in NCT02566044
Study group	Role	What they receive
CQBW276	Experimental	Cohorts 1 and 2 will enroll 8 patients each (6:2 QBW276 and placebo, respectively). Cohort 1: dose is 3 mg bid (6 mg daily) QBW276 or placebo for 7 days. Cohort 2: dose and frequency will be confirmed after cohort 1 is complete. The duration is 14 days. Cohort 3: dose and frequency will be confirmed after cohort 2 is complete. The duration is approximately 4 months. Patients will be randomized to one of two treatment sequences: QBW276 in Period 1 and Placebo in Period 2 or Placebo in Period 1 and QBW276 in Period 2. Twenty four patients are required to complete cohort 3. Receives: Drug: QBW276
Placebo	Placebo comparator	Cohorts 1 and 2 will enroll 8 patients each (6:2 QBW276 and placebo, respectively). Cohort 1: dose is 3 mg bid (6 mg daily) QBW276 or placebo for 7 days. Cohort 2: dose and frequency will be confirmed after cohort 1 is complete. The duration is 14 days. Cohort 3: dose and frequency will be confirmed after cohort 2 is complete. The duration is approximately 4 months. Patients will be randomized to one of two treatment sequences: QBW276 in Period 1 and Placebo in Period 2 or Placebo in Period 1 and QBW276 in Period 2. Twenty four patients are required to complete cohort 3. Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Cohorts 1 and 2: Safety Assessments, Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]).
Adverse events were summarized by the number of patients having any adverse event overall and presented in the safety section. Study was prematurely terminated
Measured: Cohort 1: day 1-7; Cohort 2: day 1-14
Cohorts 1 and 2: Pharmacokinetics (Cmax) of QBW276, QBP545, and QBV697 in Plasma
Blood collection will be used to observe the maximum plasma concentration (Cmax) following administration of QBW276. Pharmacokinetic blood samples were collected at the time points. In Cohorts 1 and 2 this consisted of multiple samples through 6 hours after inhalation on Days 1 and 7 in Cohort 1 and Days 1 and 14 in Cohort 2 with predose samples on selected days. The Cmax, was determined using the actual recorded sampling times and noncompartmental methods. Since steady state was likely reached by Day 7, Cmax on Days 7 or 14 correspond to Cmax,ss
Measured: Cohort 1: day 1, 7; Cohort 2: day 1, 14
Cohorts 1 and 2: Pharmacokinetics (Tmax) of QBW276, QBP545, and QBV697 in Plasma
Blood collection will be used to observe the maximum plasma concentration (Tmax) following administration of QBW276. Pharmacokinetic blood samples were collected at the time points. In Cohorts 1 and 2 this consisted of multiple samples through 6 hours after inhalation on Days 1 and 7 in Cohort 1 and Days 1 and 14 in Cohort 2 with predose samples on selected days. The Tmax, was determined using the actual recorded sampling times and noncompartmental methods. Since steady state was likely reached by Day 7, Tmax on Days 7 or 14 correspond to Tmax,ss
Measured: Day 1, 7 and 14
Cohorts 1 and 2: Pharmacokinetics (AUCtau) of QBW276, QBP545, and QBV697 in Plasma
Blood collection will be used to observe the maximum plasma concentration (AUCtau) following administration of QBW276. Pharmacokinetic blood samples were collected at the time points. In Cohorts 1 and 2 this consisted of multiple samples through 6 hours after inhalation on Days 1 and 7 in Cohort 1 and Days 1 and 14 in Cohort 2 with predose samples on selected days. The AUCtau, was determined using the actual recorded sampling times and noncompartmental methods. Since steady state was likely reached by Day 7, AUCtau on Days 7 or 14 correspond to AUClast,ss
Measured: Day 1, 7 and 14
Cohorts 1 and 2: Pharmacokinetics Accumulation Ratio (Racc) of QBW276, QBP545, and QBV697 in Plasma
The accumulation ratio (Racc) will be reported using blood samples taken on days 1 -7 in cohort 1 and days 1-14 in cohort 2. Accumulation ratio (Racc) for QBW276 and metabolites was not calculated by PK software for patients where BLOQ values were observed for all blood samples in their PK profile.
Measured: Cohort 1: 7 days; Cohort 2: 14 days

Secondary outcomes (2)

Cohorts 1 and 2: Change From Baseline in Percent Predicted Forced Expiratory Volume in the First Second by Spirometry (% Predicted FEV1)
To evaluate the response to multiple doses of inhaled QBW276 in percent predicted forced expiratory volume in the first second by spirometry according to international standards over 1 or 2 weeks of treatment compared with placebo in patients with cystic fibrosis.
Measured: Baseline to End of study (EOS)
Cohorts 1, 2: Change From Baseline in Lung Clearance Index (LCI) From Baseline to Day 7 for Cohort 1, Day 14 for Cohort 2.
Change in Lung Clearance Index (LCI) will be conducted by multiple breath nitrogen washout according to international standards
Measured: Baseline to EOS

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

27 Sep 201724 Apr 2018

First posted1 Oct 2015 (estimated)
Study start27 Sep 2017
Primary completion24 Apr 2018
Study completion24 Apr 2018

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 Cystic FibrosisStudies 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 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Crossover
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 16 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02566044
Sponsor's protocol ID: CQBW276X2201
Other IDs: 2014-004915-35, 2014
Status last verified: Jun 2019
First submitted: 18 Sep 2015
Last updated: 30 Dec 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Pulmonary Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-blind, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Doses of Inhaled QBW276 in Patients With Cystic Fibrosis
ClinicalTrials.gov ID: NCT02566044
Acronym / sponsor ID: CQBW276X2201
Conditions: Pulmonary Cystic Fibrosis
Keywords: Cystic Fibrosis
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 16 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 27 Sep 2017 (actual)
Primary completion: 24 Apr 2018 (actual)
Study completion: 24 Apr 2018 (actual)
First posted: 1 Oct 2015 (estimated)
Results: Posted 17 Jul 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The study was terminated after Cohort 2 due to the resource issues.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Crossover
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
CQBW276	Experimental	Drug: QBW276Cohorts 1 and 2 will enroll 8 patients each (6:2 QBW276 and placebo, respectively). Cohort 1: dose is 3 mg bid (6 mg daily) QBW276 or placebo for 7 days. Cohort 2: dose and frequency will be confirmed after cohort 1 is complete. The duration is 14 days. Cohort 3: dose and frequency will be confirmed after cohort 2 is complete. The duration is approximately 4 months. Patients will be randomized to one of two treatment sequences: QBW276 in Period 1 and Placebo in Period 2 or Placebo in Period 1 and QBW276 in Period 2. Twenty four patients are required to complete cohort 3.
Placebo	Placebo comparator	Other: PlaceboCohorts 1 and 2 will enroll 8 patients each (6:2 QBW276 and placebo, respectively). Cohort 1: dose is 3 mg bid (6 mg daily) QBW276 or placebo for 7 days. Cohort 2: dose and frequency will be confirmed after cohort 1 is complete. The duration is 14 days. Cohort 3: dose and frequency will be confirmed after cohort 2 is complete. The duration is approximately 4 months. Patients will be randomized to one of two treatment sequences: QBW276 in Period 1 and Placebo in Period 2 or Placebo in Period 1 and QBW276 in Period 2. Twenty four patients are required to complete cohort 3.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Cohorts 1 and 2 = any genotype on any standard of care treatment
Cohort 3 = F508del homozygotes on standard of care at that time
FEV₁between 40 and 100%
LCI2.5 ≥ 8 if FEV₁is more than 80%

Key exclusion criteria (3 listed)

Adrenal or electrolyte abnormalities
Lung transplant
Autonomic dysfunction (e.g. recurrent episodes of fainting, palpitations, etc.)

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
PlaceboPlacebo	Other	Placebo
QBW2760.3 mg and 1.5 mg strengths	Drug	CQBW276

Primary outcome

Cohorts 1 and 2: Safety Assessments, Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]).
Time frame: Cohort 1: day 1-7; Cohort 2: day 1-14
Adverse events were summarized by the number of patients having any adverse event overall and presented in the safety section. Study was prematurely terminated
Cohorts 1 and 2: Pharmacokinetics (Cmax) of QBW276, QBP545, and QBV697 in Plasma
Time frame: Cohort 1: day 1, 7; Cohort 2: day 1, 14
Blood collection will be used to observe the maximum plasma concentration (Cmax) following administration of QBW276. Pharmacokinetic blood samples were collected at the time points. In Cohorts 1 and 2 this consisted of multiple samples through 6 hours after inhalation on Days 1 and 7 in Cohort 1 and Days 1 and 14 in Cohort 2 with predose samples on selected days. The Cmax, was determined using the actual recorded sampling times and noncompartmental methods. Since steady state was likely reached by Day 7, Cmax on Days 7 or 14 correspond to Cmax,ss
Cohorts 1 and 2: Pharmacokinetics (Tmax) of QBW276, QBP545, and QBV697 in Plasma
Time frame: Day 1, 7 and 14
Blood collection will be used to observe the maximum plasma concentration (Tmax) following administration of QBW276. Pharmacokinetic blood samples were collected at the time points. In Cohorts 1 and 2 this consisted of multiple samples through 6 hours after inhalation on Days 1 and 7 in Cohort 1 and Days 1 and 14 in Cohort 2 with predose samples on selected days. The Tmax, was determined using the actual recorded sampling times and noncompartmental methods. Since steady state was likely reached by Day 7, Tmax on Days 7 or 14 correspond to Tmax,ss
Cohorts 1 and 2: Pharmacokinetics (AUCtau) of QBW276, QBP545, and QBV697 in Plasma
Time frame: Day 1, 7 and 14
Blood collection will be used to observe the maximum plasma concentration (AUCtau) following administration of QBW276. Pharmacokinetic blood samples were collected at the time points. In Cohorts 1 and 2 this consisted of multiple samples through 6 hours after inhalation on Days 1 and 7 in Cohort 1 and Days 1 and 14 in Cohort 2 with predose samples on selected days. The AUCtau, was determined using the actual recorded sampling times and noncompartmental methods. Since steady state was likely reached by Day 7, AUCtau on Days 7 or 14 correspond to AUClast,ss
Cohorts 1 and 2: Pharmacokinetics Accumulation Ratio (Racc) of QBW276, QBP545, and QBV697 in Plasma
Time frame: Cohort 1: 7 days; Cohort 2: 14 days
The accumulation ratio (Racc) will be reported using blood samples taken on days 1 -7 in cohort 1 and days 1-14 in cohort 2. Accumulation ratio (Racc) for QBW276 and metabolites was not calculated by PK software for patients where BLOQ values were observed for all blood samples in their PK profile.

2 secondary outcomes

Cohorts 1 and 2: Change From Baseline in Percent Predicted Forced Expiratory Volume in the First Second by Spirometry (% Predicted FEV1)
Time frame: Baseline to End of study (EOS)
Cohorts 1, 2: Change From Baseline in Lung Clearance Index (LCI) From Baseline to Day 7 for Cohort 1, Day 14 for Cohort 2.
Time frame: Baseline to EOS

Results and safety

Results posted to the registry on 17 Jul 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cohort 1 QBW276	6	6
Cohort 2 QBW276	6	6
Placebo	4	4

Primary outcome: Cohorts 1 and 2: Safety Assessments, Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]).

Time frame: Cohort 1: day 1-7; Cohort 2: day 1-14. Number. Unit: Participants

Measure	Cohort 1 QBW276 n = 6	Cohort 2 QBW276 n = 6
Death	0	0
Serious AE	0	0
Subjects with at least one AE	2	6

Population analysed: Safety set: The safety analysis set included all patients that received any study drug

Primary outcome: Cohorts 1 and 2: Pharmacokinetics (Cmax) of QBW276, QBP545, and QBV697 in Plasma

Time frame: Cohort 1: day 1, 7; Cohort 2: day 1, 14. Mean (Standard Deviation). Unit: ng/mL

Measure	Cohort 1 QBW276 n = 6	Cohort 1 QBP545 n = 6	Cohort 1 QBV697 n = 6	Cohort 2 QBW276 n = 6	Cohort 2 QBP545 n = 6	Cohort 2 QBV697 n = 6
Day 1	0.159 (NA)	3.88 (1.38)	1.21 (0.415)	0.174 (0.0716)	5.98 (3.72)	1.63 (0.931)
Day 7	0.145 (0.114)	5.46 (1.26)	1.45 (0.504)	–	–	–
Day 14	–	–	–	0.267 (0.109)	7.80 (4.34)	3.07 (1.72)

Population analysed: Pharmacokinetic set: The PK analysis set included all patients with at least one available valid (i.e., not flagged for exclusion) PK concentration measurement, who received study drug, and experienced no protocol deviations with relevant impact on PK data.

Primary outcome: Cohorts 1 and 2: Pharmacokinetics (Tmax) of QBW276, QBP545, and QBV697 in Plasma

Time frame: Day 1, 7 and 14. Median (Full Range). Unit: hr

Measure	Cohort 1 QBW276 n = 6	Cohort 1 QBP545 n = 6	Cohort 1 QBV697 n = 6	Cohort 2 QBW276 n = 6	Cohort 2 QBP545 n = 6	Cohort 2 QBV697 n = 6
Day 1	0.183 (0.167 to 0.200)	0.375 (0.233 to 0.933)	0.433 (0.233 to 0.500)	0.250 (0.167 to 0.250)	0.250 (0.167 to 2.03)	0.250 (0.167 to 1.03)
Day 7	0.183 (0.167 to 0.183)	0.500 (0.250 to 0.967)	0.375 (0.167 to 2.0)	–	–	–
Day 14	–	–	–	0.258 (0.233 to 0.533)	0.500 (0.267 to 0.533)	0.500 (0.233 to 0.533)

Primary outcome: Cohorts 1 and 2: Pharmacokinetics (AUCtau) of QBW276, QBP545, and QBV697 in Plasma

Time frame: Day 1, 7 and 14. Mean (Standard Deviation). Unit: hr*ng/mL

Measure	Cohort 1 QBW276 n = 6	Cohort 1 QBP545 n = 6	Cohort 1 QBV697 n = 6	Cohort 2 QBW276 n = 6	Cohort 2 QBP545 n = 6	Cohort 2 QBV697 n = 6
Day 1	0.0332 (NA)	14.3 (4.09)	1.35 (0.827)	0.0634 (0.0473)	13.9 (6.57)	1.79 (1.54)
Day 7	0.0278 (0.0362)	18 (4.21)	1.80 (0.797)	–	–	–
Day 14	–	–	–	0.0918 (0.0689)	30.6 (6.59)	2.94 (1.59)

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 18 days. Other adverse events reported at 2% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cohort 1 QBW276	0 of 6 (0%)	2 of 6 (33.3%)	0 of 6
Cohort 2 QBW276	0 of 6 (0%)	6 of 6 (100%)	0 of 6
Placebo	0 of 4 (0%)	0 of 4 (0%)	0 of 4

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Dec 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
A Plain Language Trial Summary is available on novartisclinicatrials.comLink provided by the sponsor.

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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 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing QBW276 in people with Pulmonary Cystic Fibrosis, run by Novartis Pharmaceuticals at 4 sites in 2 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 4 inclusion and 3 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: finished, as of the sponsor's last update on 30 Dec 2020. 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 United States, Germany. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 17 Jul 2019). 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?

Cohorts 1 and 2: Safety Assessments, Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]). (time frame: Cohort 1: day 1-7; Cohort 2: day 1-14). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02566044.

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 30 Dec 2020.