Experimental drug for lung disease fails to reach goal
NCT ID NCT04396366
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested an experimental drug called QBW251 in 42 adults with bronchiectasis, a lung condition that causes mucus buildup and infections. The goal was to see if the drug could reduce bacteria in the lungs and improve symptoms like coughing and breathing. However, the study was stopped early, so results are limited.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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42 people
The number who actually took part.
- Started
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Feb 2021
- Finished
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Jun 2023
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 100 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Male or female patients aged ≥18 years at screening. * Proven diagnosis of bronchiectasis by chest CT at screening as determined by investigator. * Evidence of sputum bacterial load of ≥106 CFU/mL with at least one potentially pathogenic microorganism (H. Influenzae, M catarrhalis, S aureus, S pneumoniae, Enterobacteriaceae, P aeruginosa, Stenotrophomonous maltophilia, or any potential pathogenic non-fermenting Gram-negative bacteria measured by dilution/outgrowth). * Documented history of at least one bronchiectasis exacerbation between January 2019 and study screening. * Patients with bronchial hypersecretion, defined as productive cough that occurred on most days (defined as \>50% days) for at least three consecutive months within 12 months prior to screening, as assessed by documentation of patient recollection (anamnesis) or documented in patients' record. * Patients were allowed to stay on fixed or free combinations of LABA/LAMA or LABA/ICS or LABA/LAMA/ICS as maintenance therapy if they were treated with them at a stable dose for the last 3 months prior to screening. Patients were also allowed to stay on macrolides as maintenance therapy if they were treated with them at a stable dose, 3 months before screening. Patients were allowed to use mucolytics or hyperosmolar agents if they were treated with them before study start. * If prescribed, patients were included in the study with unchanged chest physiotherapy for at least 4 weeks prior to screening. * Clinically stable pulmonary status in the opinion of the investigator and unlikely to require any change in the standard regimen of care during the course of the study. Exclusion Criteria: * Patients with a history of long-QT syndrome or the QTcF interval at screening and baseline was prolonged (QTcF \> 450 ms in males, \> 460 ms in females). * Patients with a history or current treatment for hepatic disease including but not limited to acute or chronic hepatitis, cirrhosis or hepatic failure. A history of resolved Hepatitis A was not exclusionary. Patients with a prothrombin time international normalized ratio (PT/INR) of more than 1.5 × ULN at screening. Patients excluded for the PT/INR of more than 1.5 x ULN could be re-screened when the values returned to normal. * History of lung transplant or malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there was evidence of local recurrence or metastases. Patients with segmentectomy for other reasons than cancer were allowed to be included in the study. Patients with a history of cancer and 5 years or more disease free survival time might be included in the study by agreement with Novartis Medical Monitor on a case-by-case basis. * Patients requiring long-term oxygen therapy for chronic hypoxemia. This was typically patients requiring oxygen therapy \>12 h per day delivered by home oxygen cylinder or concentrator. Note: Nocturnal oxygen therapy for transient oxygen desaturations during sleep was allowed. * Patients with bronchiectasis who had a pulmonary exacerbation with a deterioration in three or more of the following key symptoms for at least 48 h: * cough; * sputum volume and/or consistency; * sputum purulence; * breathlessness and/or exercise tolerance; * fatigue and/or malaise; * hemoptysis And A clinician determined that a change in bronchiectasis treatment was required (e.g., requiring systemic glucocorticosteroid treatment and/or systemic or inhaled antibiotics) within 4 weeks prior to screening. In the event of an exacerbation occurring 4 weeks before screening, or between the screening and baseline (please see definition above), the participant was not to be enrolled. The participant might be rescreened once, 4 weeks after the resolution of exacerbation. * Participants with bronchiectasis requiring therapy that might interfere with the assessment of QBW251 efficiency or who were unlikely to respond to QBW251 as follows: * Participants with suspected active pulmonary tuberculosis or currently being treated for active pulmonary tuberculosis were not allowed. Note: Participants with a history of pulmonary tuberculosis could be enrolled if they met the following requirements: history of appropriate drug treatment followed by negative imaging results within 12 months prior to baseline visit suggesting low probability of recurrent active tuberculosis * Patients with active allergic bronchopulmonary aspergillosis and asthma as primary diagnosis. * Patients with cystic fibrosis * Current or ex-smokers with severe emphysema. * Participants with another concomitant pulmonary disease according to the definition of the International ERS/ATS guidelines, including but not limited to idiopathic pulmonary fibrosis (IPF), sarcoidosis or other granulomatous or infectious process. Concomitant COPD and asthma with characteristics of airway hyperresponsiveness as well as COPD-Asthma overlap syndrome were allowed as long as it was not the main, primary diagnosis in the opinion of the investigator. Primary ciliary dyskinesia (PCD) was allowed. * Participants currently receiving treatment for nontuberculous mycobacterial (NTM) pulmonary disease. If performed, patients with one or more positive cultures in the last 12 months for M. avium complex, M. abscessus complex, M. kansasii, M. malmoense, M. xenopi, M. simiae or M. chelonae, unless all subsequent NTM cultures (at least two) were negative and in the opinion of the investigator the patient did not met ATS criteria for NTM-pulmonary disease. * Patients receiving any medication that might influence the response to treatment within 4 weeks prior to screening including systemic or inhaled steroids (ICS alone), or other systemic immunomodulators, recombinant human DNAse, any systemic or inhaled antibiotics. * Patients with a body mass index (BMI) of more than 40 kg/m\^2
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Novartis Investigative Site
Guangzhou, Guangdong, 510120, China
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Novartis Investigative Site
Shanghai, 200433, China
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Novartis Investigative Site
Frankfurt, 60596, Germany
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Novartis Investigative Site
Hanover, 30625, Germany
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Novartis Investigative Site
Mainz, 55128, Germany
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Novartis Investigative Site
Barcelona, Catalonia, 08035, Spain
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Novartis Investigative Site
Girona, Catalonia, 17007, Spain
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Novartis Investigative Site
Leeds, West Yorkshire, LS9 7TF, United Kingdom
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Novartis Investigative Site
Cambridge, CB2 0AY, United Kingdom
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Novartis Investigative Site
Edinburgh, EH10 5HF, United Kingdom
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Novartis Investigative Site
Liverpool, L7 8XP, United Kingdom
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Novartis Investigative Site
Liverpool, L9 7AL, United Kingdom
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Novartis Investigative Site
London, SW3 6PH, United Kingdom
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Novartis Investigative Site
Manchester, M23 9LT, United Kingdom
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