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Personalized drug trial offers hope for cystic fibrosis patients with rare gene mutations

NCT ID NCT03587961

Disease control Sponsor: George Solomon 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial is testing whether certain cystic fibrosis drugs, called CFTR modulators, can help people with rare gene mutations that are not currently approved for these medications. The study will enroll 20 participants aged 6 and older and measure changes in lung function. The goal is to see if personalized treatment can improve breathing in patients who have few other options.

What this could mean

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

Active substance
Symdeko (also known as Orkambi, Ivacaftor)
What this could lead to
If it works, this could expand treatment options for people with cystic fibrosis who have rare mutations not currently covered by approved drugs.
What could go wrong
This is a very early, small trial with only 20 participants, so results may not apply widely. The drugs are used off-label and may not work for all mutations, and there are risks like side effects or no improvement in lung function.

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

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

About 20 people

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

Started

Aug 2019

Expected to finish

Sep 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.

Ages

6 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: * Diagnosis of CF * Age ≥6 y.o. * CFTR mutation that may respond to approved correctors/potentiators in the opinion of the study investigators * Informed Consent/Assent * Stable CF pulmonary regimen Exclusion Criteria: * Exacerbation requiring antibiotic or steroids for \>28 days before trial entry * Ongoing participation in a CFTR modulator study * Active smoking in the past 6 months * History of solid organ transplant * Any condition which precludes the use of CFTR modulators: e.g. advanced cirrhosis, End-stage Renal Disease (ESRD) * Any condition that precludes the patient from participation in the opinion of the investigator * Any meds that have significant drug-drug interactions or any other off label use of CFTR modulators

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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 places running it

    1 site. The list below names each one and where it is.

  2. 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 ↗

  3. 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

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35233, United States

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