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Restore CFTR Function Enrolling

Phase 3 study of ivacaftor in babies who have a CFTR gating mutation (Vertex VX15-770-124)

This two-part, open-label study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of ivacaftor, as well as how the body processes the drug, in babies who have a CFTR gating mutation.

 

This is a two-part study. Both parts will look at the safety of ivacaftor (Kalydeco) in babies with CF. Part A will last for 10 weeks and look at how the body processes the drug. Part B will last 6 months and will look at the effectiveness of the drug. This study is open-label, meaning that all study participants will receive the study drug. Researchers will study how the body processes ivacaftor (Kalydeco) by measuring the amount of the drug in the bloodstream. They will study the drug’s safety by tracking adverse events and other measures. They will also study the drug's effectiveness by measuring sweat chloride concentration.

This study is currently enrolling babies less than 12 months old who have a CFTR gating mutation. It may require blood draws, sweat tests, electrocardiograms (ECGs), eye exams and/or other measurements.

Learn More

Email a research coordinator to express interest in participating in this study.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    Less than 13 Months

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

For a more comprehensive list of eligibility criteria and details on this study, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. Subjects who have an R117H-CFTR mutation will be eligible in regions where ivacaftor is approved for use in subjects 2 through 5 years of age with an R117H-CFTR mutation. Subjects who have an ivacaftor-responsive CFTR mutation on at least 1 allele will be
considered eligible for enrollment in Part A/B Cohort 8 (consistent with the approved
mutations in the region).

Study Design

  • Study Type: ?more info

    Interventional

  • Randomized Study: ?more info

    No

  • Placebo Controlled: ?more info

    No

  • Length of Participation:

    24 weeks

  • Number of Study Visits:

    8

Additional Information

Study Sites

  • Enrolling

    Alabama

    The Children's Hospital Alabama, University of Alabama at Birmingham, Birmingham, AL 35233

    Contact

    Heather Hathorne

    Phone: +1 (205) 638-9568

    Email:

  • Enrolling

    California

    Stanford University Medical Center, Palo Alto, CA 94304

    Contact

    Colleen Dunn

    Phone: +1 (650) 736-0388

    Email:

  • Enrolling

    Georgia

    Children’s Healthcare of Atlanta and Emory University, Atlanta, GA 30322

    Contact

    Ashleigh Streby

    Phone: +1 (404) 727-0305

    Email:

  • Enrolling

    Illinois

    Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL 60611

    Contact

    Larissa Rugg

    Phone: +1 (312) 227-6865

    Email:

  • Enrolling

    Indiana

    Riley Hospital for Children, Indianapolis, IN 46202

    Contact

    Lisa Bendy

    Phone: +1 (317) 278-7152

    Email:

  • Enrolling

    Maryland

    Johns Hopkins University, Baltimore, MD 21287

    Contact

    Britany Zeglin

    Phone: +1 (443) 287-8983

    Email:

  • Enrolling

    Massachusetts

    Boston Children's Hospital, Boston, MA 02115

    Contact

    Robert Fowler

    Phone: +1 (617) 355-1834

    Email:

  • Enrolling

    Missouri

    Children's Mercy Kansas City, KCMO, MO 64108

    Contact

    Candy Schmoll

    Phone: +1 (816) 302-3538

    Email:

  • Enrolling

    Ohio

    Nationwide Children's Hospital, Columbus, OH 43205

    Contact

    Diana Gilmore

    Phone: +1 (614) 722-4752

    Email:

  • Enrolling

    Pennsylvania

    Children's Hospital of Philadelphia, Philadelphia, PA 19104

    Contact

    Erin Donnelly

    Phone: +1 (267) 426-9626

    Email:

  • Enrolling

    Texas

    Baylor College of Medicine, Houston, TX 77030

    Contact

    Katie Howe

    Phone: +1 (832) 822-4252

    Email:

  • Enrolling

    Washington

    Seattle Children's Hospital, Seattle, WA 98105

    Contact

    Sharon McNamara

    Phone: +1 (206) 987-3921

    Email:

  • Enrolling

    Wisconsin

    University of Wisconsin, Madison, WI 53792

    Contact

    Melanie Nelson

    Phone: +1 (608) 265-4617

    Email:

Learn More

Email a research coordinator to express interest in participating in this study.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    Less than 13 Months

  • Mutation(s):

    One Copy F508del or No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

For a more comprehensive list of eligibility criteria and details on this study, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. Subjects who have an R117H-CFTR mutation will be eligible in regions where ivacaftor is approved for use in subjects 2 through 5 years of age with an R117H-CFTR mutation. Subjects who have an ivacaftor-responsive CFTR mutation on at least 1 allele will be
considered eligible for enrollment in Part A/B Cohort 8 (consistent with the approved
mutations in the region).

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