New pill combo shows promise for common CF mutation

NCT ID NCT02070744

First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 30 times

Summary

This study tested a combination of two oral drugs, VX-661 and ivacaftor, in 40 adults with cystic fibrosis who have two copies of the F508del mutation. The goal was to see if the combo is safe and improves lung function over 12 weeks. Participants were randomly assigned to receive the drugs or a placebo, with an option to continue in an open-label extension.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Study site

    Birmingham, Alabama, United States

  • Study site

    Palo Alto, California, United States

  • Study site

    Stanford, California, United States

  • Study site

    Altamonte Springs, Florida, United States

  • Study site

    Miami, Florida, United States

  • Study site

    Orlando, Florida, United States

  • Study site

    Tampa, Florida, United States

  • Study site

    Boise, Idaho, United States

  • Study site

    Chicago, Illinois, United States

  • Study site

    Boston, Massachusetts, United States

  • Study site

    New Brunswick, New Jersey, United States

  • Study site

    New York, New York, United States

  • Study site

    Durham, North Carolina, United States

  • Study site

    Cincinnati, Ohio, United States

  • Study site

    Oklahoma City, Oklahoma, United States

  • Study site

    Pittsburgh, Pennsylvania, United States

  • Study site

    Charleston, South Carolina, United States

  • Study site

    Memphis, Tennessee, United States

  • Study site

    Houston, Texas, United States

  • Study site

    Burlington, Vermont, United States

  • Study site

    Colchester, Vermont, United States

  • Study site

    Seattle, Washington, United States

  • Study site

    Milwaukee, Wisconsin, United States

What this could mean

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

Active substance

VX-661 and ivacaftor (oral tablets)

What this could lead to

If successful, this combination could improve lung function and reduce symptoms for people with cystic fibrosis who have the F508del mutation.

What could go wrong

This is an early Phase 2 trial with only 40 participants, so results may not apply to everyone. Side effects are possible, and the drug may not work as well in real-world settings.

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.