A Study to Assess the Safety, Tolerability, and Pharmacokinetics of Brensocatib Tablets in Adults With Cystic Fibrosis

NCT05090904 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: INS1007-211
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 14

Brief 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.

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; Brensocatib

Outcome Measures

Primary Outcomes (8)

• Maximum Plasma Concentration (Cmax) of Brensocatib on Day 1

  Predose and 0.5, 1, 2, 4, 6, 8, and 24 hours postdose on Day 1

• Cmax of Brensocatib on Day 28

  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

  Predose and 0.5, 1, 2, 4, 6, 8, and 24 hours postdose on Day 1

• Tmax of Brensocatib on Day 28

  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

  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

  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

  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.

  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

  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

  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

  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

  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

Study Arms (4)

Brensocatib 10 mg

EXPERIMENTAL

Participants received brensocatib at a dose of 10 mg once per day for 28 days.

Drug: Brensocatib

Brensocatib 25 mg

EXPERIMENTAL

Participants received brensocatib at a dose of 25 mg once per day for 28 days.

Drug: Brensocatib

Brensocatib 40 mg

EXPERIMENTAL

Participants received brensocatib at a dose of 40 mg once per day for 28 days.

Drug: Brensocatib

Placebo

PLACEBO COMPARATOR

Participants received a placebo matching brensocatib once per day for 28 days.

Drug: Placebo

Interventions

Brensocatib DRUG

Oral tablet

Also known as: INS1007

Brensocatib 10 mg; Brensocatib 25 mg; Brensocatib 40 mg

Placebo DRUG

Oral tablet

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Insmed Incorporated: lead

Study Sites (14)

USA001

Gainesville, Florida, 32610, United States

Location

USA016

Augusta, Georgia, 30912, United States

Location

USA025

Glenview, Illinois, 60025, United States

Location

USA011

Boston, Massachusetts, 02115, United States

Location

USA023

Ann Arbor, Michigan, 48109, United States

Location

USA002

St Louis, Missouri, 63101, United States

Location

USA022

New York, New York, 10032, United States

Location

USA008

Cleveland, Ohio, 44106, United States

Location

USA006

Cleveland, Ohio, 44195, United States

Location

USA018

Portland, Oregon, 97239, United States

Location

USA009

Charleston, South Carolina, 29425, United States

Location

USA017

Nashville, Tennessee, 37232, United States

Location

USA004

Dallas, Texas, 75390, United States

Location

USA003

Tyler, Texas, 75708, United States

Location

Related Publications (1)

• 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.

  PMID: 40753522 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

brensocatib

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Insmed Medical Information
• Organization: Insmed Incorporated

medicalinformation@insmed.com

1-844-446-7633

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 14, 2021
• First Posted: October 25, 2021
• Study Start: November 30, 2021
• Primary Completion: November 1, 2022
• Study Completion: November 1, 2022
• Last Updated: March 12, 2026
• Results First Posted: March 12, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (14)