Noncystic Fibrosis Bronchiectasis (NCFB)Phase 1NCT04643587
Study to Assess CSL787 in Non-cystic Fibrosis Bronchiectasis (NCFB)
A Phase 1 study testing CSL787 in people with Noncystic Fibrosis Bronchiectasis (NCFB), run by CSL Behring at 3 sites in 2 countries.
Trial summary
This study is a prospective, multicenter, randomized, double-blind, placebo-controlled study to investigate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and exploratory efficacy of nebulized CSL787 after administrations of single (SAD) ascending doses in healthy subjects and multiple (MAD)… Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Noncystic Fibrosis Bronchiectasis (NCFB)
- What is being tested?
- CSL787
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 7 Dec 2020to 12 Mar 2023
- 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
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Male or female, aged ≥ 18 years at the time of providing written informed consent
- For Part A (SAD) Only:
- Healthy and free of medical conditions that could in the opinion of the investigator affect's the subject's participation in the study or the interpretation of results.
- For Part B (MAD) Only:
- Diagnosis of NCFB made by a respiratory physician, confirmed per CT showing bronchial wall dilatation with or without bronchial wall thickening, with a FEV1 ≥ 40% of the predicted value regarding age, height, gender, ethnicity, and FEV1 ≥ 1 L (pre-bronchodilator values) at the Screening Visit.
- No antibiotic use within 1 month before the Screening Visit.
- Presence of one or more of the following bacteria (H. influenzae, P. aeruginosa, M. catarrhalis, S. pneumoniae, members of Enterobacterales family or S. aureus) in the sputum culture at the Screening Visit.
- Has been fully vaccinated against COVID-19 (as per country recommendations) at least 7 days prior to Day 1
Who cannot join
- Evidence of a clinically significant medical condition, disorder, or disease, including but not limited to any of the following: hepatic (hepatitis, cirrhosis); biliary; renal; cardiac; bronchopulmonary; vascular; hematologic; gastrointestinal; allergy; endocrine / metabolic (diabetes, thyroid disorders, adrenal disease); neurologic; psychiatric; immunodeficiency; cancer.
- History of chronic respiratory disease (eg, COPD or bronchiectasis) or current asthma with regular treatment including occasional use of an inhaler for exercise induced asthma.
- Current moderate-severe allergic disease (eg, allergic rhinitis) with regular treatment.
- Diagnosis of cystic fibrosis, mycobacterial disease, connective tissue disease, or alpha-1 antitrypsin deficiency as underlying disease for bronchiectasis.
- Oral/parenteral corticosteroid 28 days before the Screening Visit until EOS Visit. Use of long acting bronchodilators (long acting muscarinic antagonists (LAMA) and / or long acting beta2 agonists (LABA) and/or inhaled corticosteroids that have been at a stable dose for at least 3 months before the Screening Visit is permitted; inhalation with hypertonic saline solution is permitted up to and including Day -1.
- Any systemic or inhaled antibiotic for acute pulmonary exacerbation within 1 month before the Screening Visit until EOS Visit.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United Kingdom2 sites
Celerion
Belfast, Northern Ireland, BT9 6AD
Medicines Evaluation Unit (MEU)
Manchester, England, M23 9QZ
About this study
Official title: A Phase 1, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Nebulized CSL787 in Healthy Subjects and Subjects With Non-Cystic Fibrosis Bronchiectasis (NCFB)
This study is a prospective, multicenter, randomized, double-blind, placebo-controlled study to investigate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and exploratory efficacy of nebulized CSL787 after administrations of single (SAD) ascending doses in healthy subjects and multiple (MAD) ascending doses in subjects with NCFB.
- Conditions
- Noncystic Fibrosis Bronchiectasis (NCFB)
- Being tested
- CSL787, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CSL787Biological
Human plasma-derived polyvalent immunoglobulin G (IgG) administered via inhalation of an aerosol produced using a nebulizer
PlaceboDrug
Normal saline (0.9% NaCl)
| Study group | Role | What they receive |
|---|---|---|
| CSL787 (SAD dose 1) | Experimental | Inhalation by mouth of a nebulized aerosol in healthy subjects Receives: Biological: CSL787 |
| CSL787 (SAD dose 2) | Experimental | Inhalation by mouth of a nebulized aerosol in healthy subjects Receives: Biological: CSL787 |
| CSL787 (SAD dose 3) | Experimental | Inhalation by mouth of a nebulized aerosol in healthy subjects Receives: Biological: CSL787 |
| CSL787 (SAD dose 4) | Experimental | Inhalation by mouth of a nebulized aerosol in healthy subjects Receives: Biological: CSL787 |
| CSL787 (MAD dose 1) | Experimental | Inhalation by mouth of a nebulized aerosol in NCFB subjects Receives: Biological: CSL787 |
| CSL787 (MAD dose 2) | Experimental | Inhalation by mouth of a nebulized aerosol in NCFB subjects Receives: Biological: CSL787 |
| CSL787 (MAD dose 3) | Experimental | Inhalation by mouth of a nebulized aerosol in NCFB subjects Receives: Biological: CSL787 |
| Placebo | Placebo comparator | Inhalation by mouth of a nebulized aerosol Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with treatment emergent adverse events (TEAEs) - overall, severity and causality
Measured: Up to 8 days (healthy volunteers); Up to 21 days (NCFB patients)
Percent of subjects with TEAEs - overall, severity and causality
Measured: Up to 8 days (healthy volunteers); Up to 21 days (NCFB patients)
Secondary outcomes (22)
Maximum concentration (Cmax) of CSL787 in sputum and serum in healthy subjects
Measured: Up to 8 days from inhalation
Time of maximum concentration (Tmax) of CSL787 in sputum and serum in healthy subjects
Measured: Up to 8 days from inhalation
Area under the concentration-time curve from time 0 to 24 hours (AUC0-24h) of CSL787 in sputum and serum in healthy subjects
Measured: Up to 8 days from inhalation
Area under the concentration-time curve from time 0 to last quantifiable time point (AUC0-last) of CSL787 in sputum and serum in healthy subjects
Measured: Up to 8 days from inhalation
Area under the concentration-time curve from time 0 extrapolated to infinity (AUC0-inf) of CSL787 in sputum and serum in healthy subjects
Measured: Up to 8 days from inhalation
Apparent total clearance of the drug (CL/F) of CSL787 in sputum and serum in healthy subjects
Measured: Up to 8 days from inhalation
Apparent volume of distribution during the elimination phase (V/F) of CSL787 in sputum and serum in healthy subjects
Measured: Up to 8 days from inhalation
Terminal elimination half-life (T1/2) of CSL787 in sputum and serum in healthy subjects
Measured: Up to 8 days from inhalation
Cmax of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 1, after dosing
Tmax of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 1, after dosing
Ctrough of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 1, after dosing
AUCtau of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 1, after dosing
Cmax of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
Tmax of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
Ctrough of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
AUCtau of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
T1/2 of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
CL/F of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
V/F of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
Accumulation Ratio (AR) for Cmax of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
AR for Ctrough of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
AR for AUCtau of CSL787 in sputum and serum of NCFB subjects
Measured: On Day 14, after last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2020
- Study start7 Dec 2020
- Primary completion12 Mar 2023
- Study completion12 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Noncystic Fibrosis Bronchiectasis (NCFB)Studies of the same condition, open and under way.
- More trials run by CSL BehringThe 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
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04643587
- Sponsor's protocol ID
- CSL787_1001
- Other IDs
- 2020-002684-66
- Status last verified
- Dec 2023
- First submitted
- 19 Nov 2020
- Last updated
- 15 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
Study at a glance
From the registry record, last updated by the sponsor on 15 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Nebulized CSL787 in Healthy Subjects and Subjects With Non-Cystic Fibrosis Bronchiectasis (NCFB)
- ClinicalTrials.gov ID
- NCT04643587
- Acronym / sponsor ID
- CSL787_1001
- Conditions
- Noncystic Fibrosis Bronchiectasis (NCFB)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 7 Dec 2020 (actual)
- Primary completion
- 12 Mar 2023 (actual)
- Study completion
- 12 Mar 2023 (actual)
- First posted
- 25 Nov 2020 (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.
This study is a prospective, multicenter, randomized, double-blind, placebo-controlled study to investigate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and exploratory efficacy of nebulized CSL787 after administrations of single (SAD) ascending doses in healthy subjects and multiple (MAD) ascending doses in subjects with NCFB.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| CSL787 (SAD dose 1) | Experimental | Biological: CSL787Inhalation by mouth of a nebulized aerosol in healthy subjects |
| CSL787 (SAD dose 2) | Experimental | Biological: CSL787Inhalation by mouth of a nebulized aerosol in healthy subjects |
| CSL787 (SAD dose 3) | Experimental | Biological: CSL787Inhalation by mouth of a nebulized aerosol in healthy subjects |
| CSL787 (SAD dose 4) | Experimental | Biological: CSL787Inhalation by mouth of a nebulized aerosol in healthy subjects |
| CSL787 (MAD dose 1) | Experimental | Biological: CSL787Inhalation by mouth of a nebulized aerosol in NCFB subjects |
| CSL787 (MAD dose 2) | Experimental | Biological: CSL787Inhalation by mouth of a nebulized aerosol in NCFB subjects |
| CSL787 (MAD dose 3) | Experimental | Biological: CSL787Inhalation by mouth of a nebulized aerosol in NCFB subjects |
| Placebo | Placebo comparator | Drug: PlaceboInhalation by mouth of a nebulized aerosol |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female, aged ≥ 18 years at the time of providing written informed consent
- Healthy and free of medical conditions that could in the opinion of the investigator affect's the subject's participation in the study or the interpretation of results.
- Diagnosis of NCFB made by a respiratory physician, confirmed per CT showing bronchial wall dilatation with or without bronchial wall thickening, with a FEV1 ≥ 40% of the predicted value regarding age, height, gender, ethnicity, and FEV1 ≥ 1 L (pre-bronchodilator values) at the Screening Visit.
- No antibiotic use within 1 month before the Screening Visit.
- Presence of one or more of the following bacteria (H. influenzae, P. aeruginosa, M. catarrhalis, S. pneumoniae, members of Enterobacterales family or S. aureus) in the sputum culture at the Screening Visit.
Key exclusion criteria (6 listed)
- Evidence of a clinically significant medical condition, disorder, or disease, including but not limited to any of the following: hepatic (hepatitis, cirrhosis); biliary; renal; cardiac; bronchopulmonary; vascular; hematologic; gastrointestinal; allergy; endocrine / metabolic (diabetes, thyroid disorders, adrenal disease); neurologic; psychiatric; immunodeficiency; cancer.
- History of chronic respiratory disease (eg, COPD or bronchiectasis) or current asthma with regular treatment including occasional use of an inhaler for exercise induced asthma.
- Current moderate-severe allergic disease (eg, allergic rhinitis) with regular treatment.
- Diagnosis of cystic fibrosis, mycobacterial disease, connective tissue disease, or alpha-1 antitrypsin deficiency as underlying disease for bronchiectasis.
- Oral/parenteral corticosteroid 28 days before the Screening Visit until EOS Visit. Use of long acting bronchodilators (long acting muscarinic antagonists (LAMA) and / or long acting beta2 agonists (LABA) and/or inhaled corticosteroids that have been at a stable dose for at least 3 months before the Screening Visit is permitted; inhalation with hypertonic saline solution is permitted up to and including Day -1.
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 |
|---|---|---|
| CSL787Human plasma-derived polyvalent immunoglobulin G (IgG) administered via inhalation of an aerosol produced using a nebulizer | Biological | CSL787 (MAD dose 1), CSL787 (MAD dose 2), CSL787 (MAD dose 3), CSL787 (SAD dose 1), CSL787 (SAD dose 2), CSL787 (SAD dose 3), CSL787 (SAD dose 4) |
| PlaceboNormal saline (0.9% NaCl) | Drug | Placebo |
Primary outcome
Number of subjects with treatment emergent adverse events (TEAEs) - overall, severity and causality
Time frame: Up to 8 days (healthy volunteers); Up to 21 days (NCFB patients)
Percent of subjects with TEAEs - overall, severity and causality
Time frame: Up to 8 days (healthy volunteers); Up to 21 days (NCFB patients)
22 secondary outcomes
Maximum concentration (Cmax) of CSL787 in sputum and serum in healthy subjects
Time frame: Up to 8 days from inhalation
Time of maximum concentration (Tmax) of CSL787 in sputum and serum in healthy subjects
Time frame: Up to 8 days from inhalation
Area under the concentration-time curve from time 0 to 24 hours (AUC0-24h) of CSL787 in sputum and serum in healthy subjects
Time frame: Up to 8 days from inhalation
Area under the concentration-time curve from time 0 to last quantifiable time point (AUC0-last) of CSL787 in sputum and serum in healthy subjects
Time frame: Up to 8 days from inhalation
Area under the concentration-time curve from time 0 extrapolated to infinity (AUC0-inf) of CSL787 in sputum and serum in healthy subjects
Time frame: Up to 8 days from inhalation
Apparent total clearance of the drug (CL/F) of CSL787 in sputum and serum in healthy subjects
Time frame: Up to 8 days from inhalation
Apparent volume of distribution during the elimination phase (V/F) of CSL787 in sputum and serum in healthy subjects
Time frame: Up to 8 days from inhalation
Terminal elimination half-life (T1/2) of CSL787 in sputum and serum in healthy subjects
Time frame: Up to 8 days from inhalation
Cmax of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 1, after dosing
Tmax of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 1, after dosing
Ctrough of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 1, after dosing
AUCtau of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 1, after dosing
Cmax of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
Tmax of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
Ctrough of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
AUCtau of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
T1/2 of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
CL/F of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
V/F of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
Accumulation Ratio (AR) for Cmax of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
AR for Ctrough of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
AR for AUCtau of CSL787 in sputum and serum of NCFB subjects
Time frame: On Day 14, after last dose
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 15 Dec 2023.
- Individual participant dataSharing planned: Yes. CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
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.
Study Director study director
CSL Behring
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CSL787 in people with Noncystic Fibrosis Bronchiectasis (NCFB), run by CSL Behring at 3 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 8 inclusion and 6 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 15 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United Kingdom, 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?
Number of subjects with treatment emergent adverse events (TEAEs) - overall, severity and causality (time frame: Up to 8 days (healthy volunteers); Up to 21 days (NCFB patients)). The study lists 2 primary outcomes in all. 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 15 Dec 2023.