Cystic FibrosisPhase 2NCT05090904
A Study to Assess the Safety, Tolerability, and Pharmacokinetics of Brensocatib Tablets in Adults With Cystic Fibrosis
A Phase 2 study testing Brensocatib in people with Cystic Fibrosis, run by Insmed Incorporated at 14 sites in United States.
Trial summary
The main objective of the study is to evaluate the pharmacokinetics of brensocatib in participants with cystic fibrosis following once daily oral administration of study drug and to evaluate the safety of brensocatib compared to placebo in participants with cystic fibrosis (CF) over the 4-week treatment period. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Brensocatib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 29 participants
- Where
- 14 sitesUnited States
- Study dates
- 30 Nov 2021to 1 Nov 2022
- Phase
- Phase 2
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
- Participants must be ≥18 years of age at the time of signing the informed consent.
- Male or female participants with a confirmed diagnosis of CF related lung disease:
- Percent predicted forced expiratory volume in 1 second (ppFEV1) between 40% to 90% (inclusive) at Screening Visit and at Baseline.
- Stable CF treatment for at least 30 days before screening and willing to remain on a stable regimen throughout the treatment period.
- Has a body mass index ≥18 kg/m^2.
- Male and female participants must use contraceptives that are consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
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- Male participants, who are not sterile, with female partners of childbearing potential must be using effective contraception from Day 1 to at least 90 days after the last dose.
- Women must be postmenopausal (defined as no menses for 12 months without an alternative medical cause), surgically sterile, or using highly effective contraception methods (i.e., methods that alone or in combination achieve <1% unintended pregnancy rates per year when used consistently and correctly) from Day 1 to at least 90 days after the last dose.
- Female participants of childbearing potential must have a negative serum pregnancy test at Screening.
- Male participants with pregnant or nonpregnant women of childbearing potential partners must use a condom.
Who cannot join
- Severe or unstable CF, per Investigator's judgement.
- Currently being treated for allergic bronchopulmonary aspergillosis or nontuberculous mycobacteria or tuberculosis.
- Active and current infection by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).
- History of malignancy in the past 5 years, except completely treated in situ carcinoma of the cervix and completely treated non-metastatic squamous or basal cell carcinoma of the skin.
- Established diagnosis of hepatitis B viral infection or positive for hepatitis B surface antigen (HBsAg) at Screening.
- Established diagnosis of hepatitis C virus (HCV) infection at Screening. Participants positive for hepatitis C antibody are eligible only if HCV RNA is negative.
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- History of human immunodeficiency virus (HIV) infection.
- Acute upper or lower respiratory tract infection, pulmonary exacerbation, or changes in therapy (including intravenous and oral antibiotics) for pulmonary disease within 4 weeks prior to Day 1 (administration of the first dose of study drug). Participants meeting this criterion could be rescreened 4 weeks after resolution of symptoms.
- History of prolonged QT/QTc interval with QTcF >480 millisecond (msec) at Screening.
- History of solid organ or hematological transplantation.
- Have diagnosed periodontal disease and are either:
- Currently treated by a dentist for this condition or
- Expected to have periodontal disease-related procedures within the study period.
- Received any live attenuated vaccine within 4 weeks prior Screening.
- Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 90 days prior to Screening.
- Known history of hypersensitivity to brensocatib or any of its excipients.
- Use of any immunomodulatory agents within 4 weeks before the Screening Visit is prohibited during the study through end of study (including, but not limited to: bortezomib, ixazomib, thalidomide, cyclophosphamide, mycophenolate, Janus kinase inhibitors, interferon gamma (IFN-γ], and azathioprine).
- Continuous use of high-dose non-steroidal anti-inflammatory drugs (NSAIDs) is prohibited during the study through end of study.
- History of alcohol, medication, or illicit drug abuse.
- Current smoker, as defined by Centers for Disease Control and Prevention: An adult who has smoked 100 cigarettes in his or her lifetime and who currently smokes cigarettes.
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
USA023
Ann Arbor, Michigan, 48109
USA016
Augusta, Georgia, 30912
USA011
Boston, Massachusetts, 02115
USA009
Charleston, South Carolina, 29425
USA008
Cleveland, Ohio, 44106
USA006
Cleveland, Ohio, 44195
USA004
Dallas, Texas, 75390
USA001
Gainesville, Florida, 32610
USA025
Glenview, Illinois, 60025
USA017
Nashville, Tennessee, 37232
USA022
New York, New York, 10032
USA018
Portland, Oregon, 97239
USA002
St Louis, Missouri, 63101
USA003
Tyler, Texas, 75708
About this study
Official title: A Phase 2a, Single-Blind, Placebo-Controlled, Parallel-Group Study to Assess Safety, Tolerability, and Pharmacokinetics of Brensocatib Tablets in Adults With Cystic Fibrosis
The main objective of the study is to evaluate the pharmacokinetics of brensocatib in participants with cystic fibrosis following once daily oral administration of study drug and to evaluate the safety of brensocatib compared to placebo in participants with cystic fibrosis (CF) over the 4-week treatment period.
- Conditions
- Cystic Fibrosis
- Being tested
- Brensocatib, Placebo
- Keywords
- Cystic Fibrosis, Brensocatib
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BrensocatibDrug
Also called INS1007
Oral tablet
PlaceboDrug
Oral tablet
| Study group | Role | What they receive |
|---|---|---|
| Brensocatib 10 mg | Experimental | Participants received brensocatib at a dose of 10 mg once per day for 28 days. Receives: Drug: Brensocatib |
| Brensocatib 25 mg | Experimental | Participants received brensocatib at a dose of 25 mg once per day for 28 days. Receives: Drug: Brensocatib |
| Brensocatib 40 mg | Experimental | Participants received brensocatib at a dose of 40 mg once per day for 28 days. Receives: Drug: Brensocatib |
| Placebo | Placebo comparator | Participants received a placebo matching brensocatib once per day for 28 days. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Plasma Concentration (Cmax) of Brensocatib on Day 1
Measured: Predose and 0.5, 1, 2, 4, 6, 8, and 24 hours postdose on Day 1
Cmax of Brensocatib on Day 28
Measured: Predose and at 0.5, 1, 2, 4, 6, 8, 24, and 168 hours postdose on Day 28
Time to Maximum Plasma Concentration (Tmax) of Brensocatib on Day 1
Measured: Predose and 0.5, 1, 2, 4, 6, 8, and 24 hours postdose on Day 1
Tmax of Brensocatib on Day 28
Measured: Predose and at 0.5, 1, 2, 4, 6, 8, 24, and 168 hours postdose on Day 28
Area Under the Concentration-time Curve From Time 0 to 24 Hours Postdose (AUC0-24) of Brensocatib in Plasma on Day 1
Measured: Predose and 0.5, 1, 2, 4, 6, 8, and 24 hours postdose on Day 1
AUC0-24 of Brensocatib in Plasma on Day 28
Measured: Predose and at 0.5, 1, 2, 4, 6, 8, and 24 hours postdose on Day 28
Elimination Half-life (t1/2) of Brensocatib in Plasma on Day 28
Measured: Predose and at 0.5, 1, 2, 4, 6, 8, 24, and 168 hours postdose on Day 28
Number of Participants Who Experienced at Least One Treatment-emergent Adverse Event (TEAE)
A TEAE is defined as any adverse event (AE) that occurred after the first dose of study investigational medicinal product (IMP) and within 28 days after the last dose of study IMP.
Measured: Up to Day 56
Secondary outcomes (4)
Dose Proportionality of Brensocatib for Dose Levels 10mg to 40mg Using Brensocatib Cmax After Administration of Brensocatib on Days 1 and 28
Analysis of dose dependency for brensocatib Cmax was performed using a power law model. The logarithm of the Cmax was linearly related to the logarithm of the dose using a linear regression analysis. Slope of the regression and corresponding 90% CIs are presented. Slope (90% CI): log(PK parameter) = intercept + slope * log(dose) + error term, for relationship between each of the dose levels. All 3 dose levels are included in the same model for the dose-exposure relationship.
Measured: Day 1: Predose and 0.5, 1, 2, 4, 6, 8, and 24 hours postdose; Day 28: Predose and at 0.5, 1, 2, 4, 6, 8, 24 and 168 hours postdose
Dose Proportionality of Brensocatib for Dose Levels 10mg to 40mg Using Brensocatib AUC0-24 After Administration of Brensocatib on Days 1 and 28
Analysis of dose dependency for brensocatib AUC0-24 was performed using a power law model. The logarithm of the AUC0-24 was linearly related to the logarithm of the dose using a linear regression analysis. Slope of the regression and corresponding 90% CIs are presented. Slope (90% CI): log(PK parameter) = intercept + slope * log(dose) + error term, for relationship between each of the dose levels. All 3 dose levels are included in the same model for the dose-exposure relationship.
Measured: Day 1: Predose and 0.5, 1, 2, 4, 6, 8, and 24 hours postdose; Day 28: Predose and at 0.5, 1, 2, 4, 6, 8, and 24 hours postdose
AUClast of Brensocatib in Plasma on Days 1 and 28
Measured: Day 1: Predose and 0.5, 1, 2, 4, 6, 8, and 24 hours postdose; Day 28: Predose and at 0.5, 1, 2, 4, 6, 8, 24 and 168 hours postdose
Dose Proportionality of Brensocatib for Dose Levels 10mg to 40mg Using Brensocatib AUClast, After Administration of Brensocatib on Days 1 and 28
Analysis of dose dependency for brensocatib AUClast was performed using a power law model. The logarithm of the AUClast was linearly related to the logarithm of the dose using a linear regression analysis. Slope of the regression and corresponding 90% CIs are presented. Slope (90% CI): log(PK parameter) = intercept + slope * log(dose) + error term, for relationship between each of the dose levels. All 3 dose levels are included in the same model for the dose-exposure relationship.
Measured: Day 1: Predose and 0.5, 1, 2, 4, 6, 8, and 24 hours postdose; Day 28: Predose and at 0.5, 1, 2, 4, 6, 8, 24 and 168 hours postdose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2021
- Study start30 Nov 2021
- Primary completion1 Nov 2022
- Study completion1 Nov 2022
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Insmed Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05090904
- Sponsor's protocol ID
- INS1007-211
- Status last verified
- Feb 2026
- First submitted
- 14 Oct 2021
- Last updated
- 12 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Konstan MW, Tolle JJ, DiMango E, Flume PA, Usansky H, Teper A, Ramirez CN, Flarakos J, Basso J, Li S, Vergara M. A Phase IIa, Single-Blind, Placebo-Controlled, Parallel-Group Study to Assess Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Brensocatib in Adults with Cystic Fibrosis. Clin Pharmacokinet. 2025 Oct;64(10):1561-1574. doi: 10.1007/s40262-025-01550-z. Epub 2025 Aug 3.
PubMed 40753522