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New pill for cystic fibrosis moves to human testing

NCT ID NCT05090904

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

29 people

The number who actually took part.

Started

Nov 2021

Finished

Nov 2022

Lead sponsor

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

Anyone

Healthy volunteers

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

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

  • USA001

    Gainesville, Florida, 32610, United States

  • USA002

    St Louis, Missouri, 63101, United States

  • USA003

    Tyler, Texas, 75708, United States

  • USA004

    Dallas, Texas, 75390, United States

  • USA006

    Cleveland, Ohio, 44195, United States

  • USA008

    Cleveland, Ohio, 44106, United States

  • USA009

    Charleston, South Carolina, 29425, United States

  • USA011

    Boston, Massachusetts, 02115, United States

  • USA016

    Augusta, Georgia, 30912, United States

  • USA017

    Nashville, Tennessee, 37232, United States

  • USA018

    Portland, Oregon, 97239, United States

  • USA022

    New York, New York, 10032, United States

  • USA023

    Ann Arbor, Michigan, 48109, United States

  • USA025

    Glenview, Illinois, 60025, United States

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