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Early use of powerful CF drugs may boost growth in young children

NCT ID NCT04509050

Disease control Sponsor: Sonya Heltshe Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 08, 2026 · Last updated Jul 09, 2026 · Updated 1 time

Summary

This study looks at how highly effective CFTR modulators—medicines like Kalydeco and Trikafta—affect growth and health in infants and young children with cystic fibrosis. Researchers will track changes in weight and height over time in children under 10 years old. The goal is to understand whether starting these drugs early can improve long-term outcomes.

What this could mean

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

Active substance
ivacaftor (Kalydeco) or elexacaftor/tezacaftor/ivacaftor (Trikafta)
What this could lead to
If successful, this could show that early, highly effective treatment improves growth and long-term health in children with cystic fibrosis.
What could go wrong
This is an observational study, not a controlled trial, so results may not prove cause and effect. Side effects of the drugs are possible, and benefits may vary.

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

About 210 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2020

Expected to finish

Dec 2027

An estimate. End dates often move.

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

Part A - children with confirmed diagnosis of cystic fibrosis who are less than 10 years of age and not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy Part B - children with confirmed diagnosis of cystic fibrosis who are less than 7 years of age (or participated in Part A of the study) with an intention to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy

Ages

Up to 10 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: * Part A: * Less than 10 years of age at the first study visit. * Documentation of a CF diagnosis. Part B: * Participated in Part A OR less than 7 years of age at the first study visit. * Documentation of a CF diagnosis. * CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor). * Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor. Exclusion Criteria: * Part A and Part B: * Use of an investigational drug within 28 days prior to and including the first study visit. * Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days prior to and including the first study visit. * Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    35 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Baylor College of Medicine

    RECRUITING

    Houston, Texas, 77030, United States

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital of New York

    RECRUITING

    New York, New York, 10032, United States

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of Pittsburgh of UPMC

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

  • Children's Hospitals and Clinics of Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55404, United States

  • Children's Mercy Kansas City

    RECRUITING

    Kansas City, Missouri, 64108, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Cook Children's Medical Center

    RECRUITING

    Fort Worth, Texas, 76104, United States

  • Helen DeVos Children's Hospital

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

  • Hershey Medical Center Pennsylvania State University

    RECRUITING

    Hershey, Pennsylvania, 17033, United States

  • Nationwide Children's Hospital

    RECRUITING

    Columbus, Ohio, 43205, United States

  • Nemours Children's Clinic

    RECRUITING

    Jacksonville, Florida, 32207, United States

  • New York Medical College at Westchester Medical Center

    RECRUITING

    Valhalla, New York, 10595, United States

  • Oklahoma Cystic Fibrosis Center

    RECRUITING

    Oklahoma City, Oklahoma, 73104, United States

    Contact Email: •••••@•••••

  • Oregon Health Sciences University

    RECRUITING

    Portland, Oregon, 97239, United States

  • Primary Children's Cystic Fibrosis Center

    RECRUITING

    Salt Lake City, Utah, 84113, United States

  • Riley Hospital for Children

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • SUNY Upstate Medical University

    RECRUITING

    Syracuse, New York, 13210, United States

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • St. Louis Children's Hospital

    RECRUITING

    St Louis, Missouri, 63110, United States

  • Stanford University Medical Center

    RECRUITING

    Palo Alto, California, 94304, United States

  • The Children's Hospital Alabama, University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • The Cystic Fibrosis Center of Western New York

    RECRUITING

    Buffalo, New York, 14203, United States

  • The Minnesota Cystic Fibrosis Center

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

  • The Nemours Children's Clinic - Orlando

    RECRUITING

    Orlando, Florida, 32827, United States

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Kansas Medical Center

    RECRUITING

    Kansas City, Kansas, 66160, United States

  • University of Miami

    RECRUITING

    Miami, Florida, 33136, United States

  • University of Michigan, Michigan Medicine

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Texas Southwestern / Children's Health

    RECRUITING

    Dallas, Texas, 75207, United States

  • University of Virginia

    RECRUITING

    Charlottesville, Virginia, 22903, United States

  • University of Wisconsin

    RECRUITING

    Madison, Wisconsin, 53792, United States

  • Vermont Children's Hospital

    RECRUITING

    Burlington, Vermont, 05401, United States

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