Clinical trial: A Clinical Study to Investigate Safety, Tolerability and Distribution of CHF 6333 After One or After Repeated Inhalation in Patients With Cystic Fibrosis (CF) and in Patients With Non Cystic Fibrosis (NCFB) Bronchiectasis (NCT04010799)

Eligibility criteria:
INCLUSION CRITERIA:

        CF patients:

          -  Patient's written informed consent obtained prior to any study-related procedure;

          -  Male or female patient ≥ 18 years old with a confirmed historical diagnosis of cystic
             fibrosis;

          -  Ability to provide a spontaneous sputum sample at screening;

          -  Non- or ex-smokers who smoked < 10 pack years and stopped smoking > 1 year before
             screening visit;

          -  Patient in stable clinical condition and free from exacerbation for at least 4 weeks
             prior to screening and/or prior to randomisation;

          -  Patient on stable concomitant treatment regimen within 4 weeks prior to screening
             and/or prior to randomisation;

          -  Patient with pre-bronchodilator FEV1 ≥ 50% of predicted normal at screening and/or
             prior to randomisation;

          -  Vital signs within normal limits at screening and prior to randomisation;

        NCFB patients:

          -  Patient's written informed consent obtained prior to any study-related procedure;

          -  Male or female patient ≥ 18 years old with a diagnosis of Bronchiectasis confirmed by
             a historical Chest CT;

          -  Presence of clinically significant symptoms related to Bronchiectasis, such as daily
             cough that occurs over months or years, daily production of large amount of sputum,
             shortness of breath, wheezing chest pain;

          -  Ability to provide a spontaneous sputum sample at screening;

          -  Non- or ex-smokers who smoked < 10 pack years and stopped smoking > 1 year before
             screening visit;

          -  Patients in stable clinical condition and free from exacerbation since at least 4
             weeks before screening and/or prior to randomisation;

          -  Patients on stable concomitant treatment regimen within 4 weeks prior to screening
             and/or prior to randomisation

          -  Patient with pre- bronchodilator FEV1 ≥ 50% of predicted normal at screening and/or
             prior randomization visit;

          -  Vital signs within normal limits at screening and prior to randomisation

        EXCLUSION CRITERIA CF Patients

          -  Patient with BMI ≤ 17

          -  History of a clinically meaningful unstable or uncontrolled chronic comorbidity in the
             opinion of the Investigator;

          -  Unstable pulmonary status or symptomatic respiratory tract infection and related
             changes in therapy for pulmonary disease as per Investigator's judgment within 4 weeks
             before screening or prior to randomisation;

          -  Abnormal and clinically significant 12-lead ECG at screening or prior to
             randomisation;

          -  History of asthma based on objective evidence;

          -  History of malignancy, solid organ/haematological transplantation;

          -  Patient with evidence of active Nontuberculous Mycobacteria (NTM) and Tuberculous
             Mycobacteria (TM) infection or related bronchiectasis in the past 12 months;

          -  Patient with a positive test for active Allergic Bronchopulmonary Aspergillosis (ABPA)
             infection confirmed at screening or patient withABPA related bronchiectasis.

          -  Pregnant or lactating women.

          -  Patient on non-steroidal anti-inflammatory drugs (NSAIDs) within 4 weeks prior to
             screening or prior to randomization visit.

          -  Patient on cystic fibrosis transmembrane conductance regulator (CFTR) modulators and
             correctors if not on stable treatment regimen for at least 3 months prior to screening
             or prior to randomization.

          -  Positive HIV1 or HIV2 serology at screening; Positive results from the Hepatitis
             serology which indicates acute or chronic Hepatitis B or Hepatitis C at screening
             (i.e. positive HB surface antigen (HBsAg), HB core antibody (anti-HBc), HC antibody);

        NCFB Patients

          -  Patient with BMI ≤ 17

          -  History of a clinically meaningful unstable or uncontrolled chronic comorbidity in the
             opinion of the Investigator;

          -  Unstable pulmonary status or symptomatic respiratory tract infection and related
             changes in therapy for pulmonary disease as per Investigator's judgment within 4 weeks
             before screening or prior to randomisation.

          -  Abnormal and clinically significant 12-lead ECG at screening or prior to randomisation
             that results in active medical problem which may impact the safety of the patients as
             per Investigator's judgment.

          -  History of malignancy, solid organ/haematological transplantation;

          -  Known diagnosis of cystic fibrosis. A negative sweat test is required at screening
             (sweat chloride should be < 40 mmol/L);

          -  History of asthma based on objective evidence of the condition;

          -  Patient with primary diagnosis of COPD in the opinion of theInvestigator;

          -  Patient with rheumatoid factor positivity;

          -  Patient with evidence of active Nontuberculous Mycobacteria (NTM) and Tuberculous
             Mycobacteria (TM) infection or related bronchiectasis in the past 12 months;

          -  Patient with a positive test for active Allergic Bronchopulmonary Aspergillosis (ABPA)
             infection confirmed at screening or patient with ABPA related bronchiectasis;

          -  Patient with Connective Tissue Disease (CTD) related bronchiectasis;

          -  Diagnosis of common variable immunodeficiency (CVID);

          -  Patient on any antibiotics (except for stable macrolides treatment),oral, inhaled and
             IV, within 4 weeks prior to screening or prior to randomisation;

          -  Patient on oral corticosteroids within 4 weeks prior to screening visit or prior to
             randomization.

          -  Patient on non-steroidal anti-inflammatory drugs (NSAIDs) within 4 weeks prior to
             screening or randomization visit.

          -  Patient on Carbocysteine and Mannitol treatment within 4 weeks before the screening or
             randomization visit.

          -  Patient with traction bronchiectasis;

          -  Patient with any condition that prevent them to use inhaledantibiotics (including
             patients who previously experienced adverse reaction to inhaled antibiotics;

          -  Patient treated with monoclonal antibodies (mAb);

          -  Pregnant or lactating women.

          -  Positive HIV1 or HIV2 serology at screening; Positive results from the Hepatitis
             serology which indicates acute or chronic Hepatitis B or Hepatitis C at screening
             (i.e. positive HB surface antigen (HBsAg), HBcore antibody (anti-HBc), HC antibody).
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.


Patient description (medical language): A 30-year-old man who is a computer scientist came to the clinic with the lab result stating azoospermia.  The patient is sexually active with his wife and does not use any contraception methods.  They have been trying to conceive for the past year with no success.  The patient has a past medical history of recurrent pneumonia, shortness of breath, and  persistent cough that produces large amounts of thick sputum. The patient had multiple lung infections during childhood. He does not smoke, use illicit drugs or alcohol.  The patient has no history of other medical conditions including allergies or any kind of surgery.  On physical examination, the digits show clubbing.  An ultrasound shows bilateral absence of the vas deferens, and FEV1 was 75% on the respiratory function test.

Patient description (patient's own words): I'm a 30-year-old computer scientist. I did some lab tests that came back quite alarming, stating that I have azoospermia?! I'm sexually active with my wife and we don't use contraception. We've been trying to have a child for the past year. I have a medical history of pneumonia and shortness of breath and regular wet cough. I had several lung infections when I was a kid. That's why I'm very careful and don't smoke, do drugs, or drink. I don't have other medical conditions including allergies or any kind of surgery. The doctor did the clubbing test on my fingers, which was positive. I also did an ultrasound and it showed that I'm missing the tubes that carry sperm around. I also had a 75% for my respiratory function test.

Does the patient match the trial criteria? Entailment