New pill for cystic fibrosis moves to human testing
NCT ID NCT05090904
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested an experimental pill called brensocatib in 29 adults with cystic fibrosis. The main goals were to see how the drug behaves in the body and whether it is safe compared to a placebo over 4 weeks. Participants took the pill once daily, and researchers measured drug levels and side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- brensocatib (also called INS1007) oral tablet
- What this could lead to
- If successful, this could point toward a new treatment option to help control cystic fibrosis lung disease.
- What could go wrong
- This is a small, early-phase trial focused on safety and drug levels, not on whether the drug improves symptoms. Many drugs fail in later, larger studies.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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29 people
The number who actually took part.
- Started
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Nov 2021
- Finished
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Nov 2022
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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: 1. Percent predicted forced expiratory volume in 1 second (ppFEV1) between 40% to 90% (inclusive) at Screening Visit and at Baseline. 2. 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. 1. 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. 2. 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. Exclusion Criteria: * 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: 1. Currently treated by a dentist for this condition or 2. 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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USA001
Gainesville, Florida, 32610, United States
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USA002
St Louis, Missouri, 63101, United States
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USA003
Tyler, Texas, 75708, United States
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USA004
Dallas, Texas, 75390, United States
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USA006
Cleveland, Ohio, 44195, United States
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USA008
Cleveland, Ohio, 44106, United States
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USA009
Charleston, South Carolina, 29425, United States
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USA011
Boston, Massachusetts, 02115, United States
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USA016
Augusta, Georgia, 30912, United States
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USA017
Nashville, Tennessee, 37232, United States
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USA018
Portland, Oregon, 97239, United States
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USA022
New York, New York, 10032, United States
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USA023
Ann Arbor, Michigan, 48109, United States
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USA025
Glenview, Illinois, 60025, United States
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