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

NCT ID NCT03587961

Recruiting now Disease control Sponsor: George Solomon Source: ClinicalTrials.gov ↗

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.

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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

Contacts and locations

Locations

University of Alabama at Birmingham
RECRUITING

Birmingham, Alabama, 35233, United States

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