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New study tracks CF Drug's impact on Kids' lungs and lives

NCT ID NCT04613128

Disease control Sponsor: Nicole Hamblett Source: ClinicalTrials.gov ↗

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 followed 125 children aged 6-11 with cystic fibrosis who were prescribed a triple therapy (elexacaftor, tezacaftor, and ivacaftor). Researchers measured changes in sweat chloride, lung function, and weight over 24 months to see how well the treatment works in real life. The goal is to understand the therapy's benefits and help guide future care.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
elexacaftor, tezacaftor, and ivacaftor (triple combination therapy)
What this could lead to
If successful, this could show that the therapy improves lung function and quality of life in children with cystic fibrosis, guiding future treatments.
What could go wrong
This is an observational study, not a controlled trial, so results may be less definitive. The therapy is already FDA-approved for this age group, so the main goal is to understand real-world effects, not test a new cure.

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.

Participants

125 people

The number who actually took part.

Started

Jun 2021

Finished

Dec 2025

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Cystic Fibrosis, 6-11 years old prescribed ETI.

Ages

6 to 11 years

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: 1. Written parental informed consent and assent obtained from subject and the subject's legal guardian. 2. Be willing and able to adhere to the study visit schedule and other protocol requirements. 3. All genders 6-11 years old on Day 1. 4. Diagnosis of CF. 5. CFTR mutations consistent with the FDA labeled indication for the ETI. 6. Physician intent to prescribe the ETI. 7. Able to attempt the testing and procedures required for this study, as judged by the investigator. 8. Enrolled in the Cystic Fibrosis Foundation Patient Registry. 9. Clinically stable with no significant changes in health status within the 14 days prior to Visit 1 (and inclusive of Visit 1). Exclusion Criteria: 1. Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives. 2. Use of any ETI within the 180 days prior to Visit 1. 3. Any acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids within the 14 days prior to Visit 1 (inclusive of Visit 1) for lower respiratory tract symptoms. 4. Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, inhaled tobramycin, Cayston®, Kalydeco, Orkambi®, Symdeko®) within the 4 weeks prior to Visit 1 (inclusive of Visit 1). 5. Use of an investigational agent within the 28 days prior to Visit 1. 6. Use of chronic oral corticosteroids (equivalent to 10 mg. or more per day of prednisone) within the 28 days prior to Visit 1. 7. Treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two antibiotics (oral, IV, and/or inhaled) within the 28 days prior to Visit 1. 8. History of lung or liver transplantation,or listing for organ transplantation.

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Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cystic fibrosis Cystic fibrosis in children

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Boston Children's Hospital, Brigham & Women's Hospital

    Boston, Massachusetts, 02115, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Children's Mercy Kansas City

    Kansas City, Missouri, 64108, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • John Hopkins University

    Baltimore, Maryland, 21287, United States

  • Oregon Health Sciences University

    Portland, Oregon, 97239, United States

  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44146, United States

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • The Minnesota Cystic Fibrosis Center

    Minneapolis, Minnesota, 55455, United States

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • University of Wisconsin

    Madison, Wisconsin, 53792, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23219, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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