Cystic FibrosisPhase 1NCT04010799

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

A Phase 1 study testing CHF 6333 in people with Cystic Fibrosis and Non-Cystic Fibrosis Bronchiectasis, run by Chiesi Farmaceutici S.p.A. at 1 site in Germany.

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

CHF 6333 is a medicinal product on development for the treatment of cystic fibrosis and non-CF bronchiectasis and undergoing clinical testing. It has not yet been approved by the authorities for the treatment of these diseases. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Cystic Fibrosis, Non-Cystic Fibrosis Bronchiectasis
What is being tested?: CHF 6333
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 68 participants
Where: 1 siteGermany
Study dates: 27 May 2019to 8 Mar 2021
Phase: Phase 1
Are there results?: None posted in the registry

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

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;

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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

Who cannot join

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;

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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).

Trial locations and contacts

1 site in 1 country.

Germany1 site

IKF Institut für klinische Forschung Pneumologie
Frankfurt am Main

Other trials in Germany

About this study

Official title: A Phase Ib, Randomised, Double-blind, Placebo-controlled Study to Investigate the Safety, Tolerability and Pharmacokinetics of Inhaled CHF 6333 After Single and Repeated Ascending Doses in Patients Affected by Cystic Fibrosis and Non Cystic Fibrosis Bronchiectasis

CHF6333 is an inhaled anti-inflammatory which mechanism of action is based on the inhibition of Human Neutrofil Elastase.

The safety and tolerability of single and repeated ascending doses of inhaled CHF 6333 was previously investigated in healthy subjects: information was gathered on the uptake, distribution and excretion of the medicinal product being tested (pharmacokinetics). In this current clinical trial CHF 6333 will be tested in patients(CF and NCFB) for the first time.

Three dose level will be tested during the first part of the study, as single administration. One repeated dose will be administered in the second part of the study.

Conditions: Cystic Fibrosis, Non-Cystic Fibrosis Bronchiectasis
Being tested: CHF 6333, Placebo
Medical subject headings: Cystic Fibrosis

What participants receive

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

CHF 6333Drug
CHF 6333 - Part I - SAD CHF 6333 - Part II - MD
PlaceboDrug
Placebo - Part I - SAD Placebo Part II - MAD

Study groups in NCT04010799
Study group	Role	What they receive
CHF6333	Experimental	CHF6333 Active (part I - SAD). Once daily inhaled single dose of CHF6333 at each period (three dose level). CHF6333 Active (part II -MD). Once daily inhaled multiple dose of CHF6333 for 7 consecutive days. Receives: Drug: CHF 6333
CHF6333 Placebo	Placebo comparator	Part I (SAD): Single dose of placebo matching CHF6333 at each period Part II (MD): Once daily multiple doses of placebo matching CHF6333 for 7 consecutive days Receives: Drug: Placebo

What is being measured

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

Adverse event
Occurrence and severity of adverse events
Measured: Part I: Baseline through end of treatment (up to a maximum of 30 days after last study drug intake) ; Part II Baseline through end of treatment (up to a maximum of 30 days after last study drug intake)
Change in Vital signs
Change in Systolic and Diastolic blood pressure
Measured: Part I: Day 1 pre-dose up to 6 hours post dose. Part II: Day 1 and Day 7 pre dose up to 6 hours post dose
Heart Rate
Change in Heart Rate
Measured: Part I: Day 1 pre dose up to 8 hours post dose. Part II: Day 1 and Day 7 pre dose up to 12 hours post dose
PR interval
Change in PR interval
Measured: Part I: Day 1 pre dose up to 8 hours post dose. Part II: Day 1 and Day 7 pre dose up to 12 hours post dose
QRS interval
Change in QRS interval
Measured: Part I: Day 1 pre dose up to 8 hours post dose. Part II: Day 1 and Day 7 pre dose up to 12 hours post dose
QTCf interval
Change in QTCf interval
Measured: Part I: Day 1 pre dose up to 8 hours post dose. Part II: Day 1 and Day 7 pre dose up to 12 hours post dose
FEV1
Change in FEV1
Measured: Part I: Day 1 pre dose up to 6 hours post dose. Part II: Day 1 and Day 7 pre dose up to 6 hours post dose. Day 2 -6: pre dose up to 2 hours post dose

Secondary outcomes (6)

AUC
Area under the plasma concentration curve
Measured: Part I: Day 1. Part II Day 1-7
Cmax
Peak plasma concentration
Measured: Part I: Day 1. Part II Day 1-7
T max
Time to reach the maximum plasma concentration
Measured: Part I: Day 1. Part II Day 1-7
C24h
Trough drug concentration 24 h post dose
Measured: Part II: Day 5 Day 6
Rac
Accumulation ratio
Measured: Part II: Day 7
NE activity
Change in neutrophil elastase activity in sputum
Measured: Part I: Day -1 Day 1. Part II: Day -1 - 7

Timeline

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

27 May 20198 Mar 2021

First posted8 Jul 2019
Study start27 May 2019
Primary completion8 Mar 2021
Study completion8 Mar 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
More trials run by Chiesi Farmaceutici S.p.A.The 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
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Model description: Part I: Randomised, double-blind, placebo-controlled, single-dose escalation, cross-over design in one cohort of CF patients and in one cohort of NCFB patients. Part II: Randomised, double-blind, placebo-controlled, repeated-dose, parallel-group design in one cohort of CF patients and in one cohort of NCFB patients.
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 68 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04010799
Sponsor's protocol ID: CLI-06333AA1-16
Other IDs: 2018-002508-15, 2018
Status last verified: Mar 2021
First submitted: 10 Jun 2019
Last updated: 30 Mar 2021
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: 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

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
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Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
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RecruitingObservational study
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RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
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All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase Ib, Randomised, Double-blind, Placebo-controlled Study to Investigate the Safety, Tolerability and Pharmacokinetics of Inhaled CHF 6333 After Single and Repeated Ascending Doses in Patients Affected by Cystic Fibrosis and Non Cystic Fibrosis Bronchiectasis
ClinicalTrials.gov ID: NCT04010799
Acronym / sponsor ID: CLI-06333AA1-16
Conditions: Cystic Fibrosis, Non-Cystic Fibrosis Bronchiectasis
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 68 participants (actual)
Lead sponsor: Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
Study start: 27 May 2019 (actual)
Primary completion: 8 Mar 2021 (actual)
Study completion: 8 Mar 2021 (actual)
First posted: 8 Jul 2019 (actual)
Results: Not posted in the source record

Clinical summary

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

CHF6333 is an inhaled anti-inflammatory which mechanism of action is based on the inhibition of Human Neutrofil Elastase.

Three dose level will be tested during the first part of the study, as single administration. One repeated dose will be administered in the second part of the study.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2
Model description: Part I: Randomised, double-blind, placebo-controlled, single-dose escalation, cross-over design in one cohort of CF patients and in one cohort of NCFB patients. Part II: Randomised, double-blind, placebo-controlled, repeated-dose, parallel-group design in one cohort of CF patients and in one cohort of NCFB patients.

Arm	Type	Receives
CHF6333	Experimental	Drug: CHF 6333CHF6333 Active (part I - SAD). Once daily inhaled single dose of CHF6333 at each period (three dose level). CHF6333 Active (part II -MD). Once daily inhaled multiple dose of CHF6333 for 7 consecutive days.
CHF6333 Placebo	Placebo comparator	Drug: PlaceboPart I (SAD): Single dose of placebo matching CHF6333 at each period Part II (MD): Once daily multiple doses of placebo matching CHF6333 for 7 consecutive days

Who may qualify

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

Key inclusion criteria (19 listed)

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;

Key exclusion criteria (35 listed)

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;

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
CHF 6333CHF 6333 - Part I - SAD CHF 6333 - Part II - MD	Drug	CHF6333
PlaceboPlacebo - Part I - SAD Placebo Part II - MAD	Drug	CHF6333 Placebo

Primary outcome

Adverse event
Time frame: Part I: Baseline through end of treatment (up to a maximum of 30 days after last study drug intake) ; Part II Baseline through end of treatment (up to a maximum of 30 days after last study drug intake)
Occurrence and severity of adverse events
Change in Vital signs
Time frame: Part I: Day 1 pre-dose up to 6 hours post dose. Part II: Day 1 and Day 7 pre dose up to 6 hours post dose
Change in Systolic and Diastolic blood pressure
Heart Rate
Time frame: Part I: Day 1 pre dose up to 8 hours post dose. Part II: Day 1 and Day 7 pre dose up to 12 hours post dose
Change in Heart Rate
PR interval
Time frame: Part I: Day 1 pre dose up to 8 hours post dose. Part II: Day 1 and Day 7 pre dose up to 12 hours post dose
Change in PR interval
QRS interval
Time frame: Part I: Day 1 pre dose up to 8 hours post dose. Part II: Day 1 and Day 7 pre dose up to 12 hours post dose
Change in QRS interval
QTCf interval
Time frame: Part I: Day 1 pre dose up to 8 hours post dose. Part II: Day 1 and Day 7 pre dose up to 12 hours post dose
Change in QTCf interval
FEV1
Time frame: Part I: Day 1 pre dose up to 6 hours post dose. Part II: Day 1 and Day 7 pre dose up to 6 hours post dose. Day 2 -6: pre dose up to 2 hours post dose
Change in FEV1

6 secondary outcomes

AUC
Time frame: Part I: Day 1. Part II Day 1-7
Cmax
Time frame: Part I: Day 1. Part II Day 1-7
T max
Time frame: Part I: Day 1. Part II Day 1-7
C24h
Time frame: Part II: Day 5 Day 6
Rac
Time frame: Part II: Day 7
NE activity
Time frame: Part I: Day -1 Day 1. Part II: Day -1 - 7

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2021.
Individual participant dataSharing planned: No

Related trials for Cystic Fibrosis

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
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Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
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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.

The record lists no contact. Use the official record to reach the sponsor.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CHF 6333 in people with Cystic Fibrosis and Non-Cystic Fibrosis Bronchiectasis, run by Chiesi Farmaceutici S.p.A. at 1 site in Germany.

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 19 inclusion and 35 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 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Adverse event (time frame: Part I: Baseline through end of treatment (up to a maximum of 30 days after last study drug intake) ; Part II Baseline through end of treatment (up to a maximum of 30 days after last study drug intake)). The study lists 7 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 NCT04010799.

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 Mar 2021.