A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function

Completed

Conditions studied: Cystic Fibrosis

In brief

This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor (ETI) triple combination therapy in people with one or more copies of the F508del mutation, study the effects of ETI across a number of CF disease manifestations, and collect specimens for future research. Subjects in the study will have one "before TCT" visit within 30 days before initiation of the therapy and five "after TCT" visits over a 30-month follow-up period. Participants who have participated in the original PROMISE cohort have the option of participating in a long-term extension with annual visits performed at the 42- and 54-month timepoints. The durability of the clinical and biological changes in PROMISE can be assessed with extended follow-up, which would enable the sub-studies to consider potential clinical consequences of the biological or physiological effects being studied. This work will help to inform long term prognosis and feasibility of certain clinical trials outcomes for interventional studies and may be useful when considering research priorities in drug development. Most participating sites have been divided into sub-study groups; each sub-study group has specific non-optional procedures conducted in addition to the "Core" procedures. Finally, there is one optional procedure (transient elastography) that will be offered to subjects at certain sites. The duration of participation for each subject is 30 months (with an additional 24 months if participants agree to the optional long-term extension). NOTE: FDA has reviewed the New Drug Application (NDA) for elexacaftor, tezacaftor and ivacaftor and has granted approval.

Key facts

Study ID: NCT04038047
Run by: Nicole Hamblett
People needed: 490
Starts: 2019-10-22
Expected to finish: 2024-12-23
Last updated by the study team: 2025-01-28

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

All genders within the age limit of the FDA approved indication for elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT) at Day 1.
Diagnosis of CF.
CFTR mutations consistent with the FDA approved indication for elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT).
Physician intent to prescribe elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT).
Willing to fast for 8 hours prior to all study visits (for subjects on overnight enteric tube feedings, willing to hold the feeding for at least 8 hours).
Able to perform the testing and procedures required for this study, as judged by the investigator.
Enrolled in the Cystic Fibrosis Foundation Patient Registry.
Clinically stable with no significant changes in health status within the 14 days prior to Visit 1.

You may not qualify if…

Use of any TCT within the 180 days prior to Visit 1.
Any acute use of antibiotics (oral, inhaled or IV) or systemic corticosteroids within the 2 weeks prior to Visit 1 for lower respiratory tract symptoms.
Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, inhaled tobramycin, Cayston®, Kalydeco, Orkambi®, Symdeko®) within the 4 weeks prior to Visit 1.
Use of an investigational agent within the 28 days prior to Visit 1.
Use of chronic oral corticosteroids (equivalent to 10 mg. or more per day of prednisone) within the 28 days prior to Visit 1.
Treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two antibiotics (oral, IV, and/or inhaled) within the 28 days prior to Visit 1.
History of lung or liver transplantation, or listing for organ transplantation.

Where it is running

Providence Alaska Medical Center — Anchorage, Alaska, United States
University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States
Stanford University Medical Center — Palo Alto, California, United States
Children's Hospital Colorado — Aurora, Colorado, United States
National Jewish Health — Denver, Colorado, United States
Yale University School of Medicine — New Haven, Connecticut, United States
Children's National Medical Center — Washington D.C., District of Columbia, United States
University of Florida — Gainesville, Florida, United States
Children's Healthcare of Atlanta and Emory University — Atlanta, Georgia, United States
Emory University — Atlanta, Georgia, United States
Augusta University — Augusta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
Saint Francis Medical Center — Peoria, Illinois, United States
Riley Hospital for Children — Indianapolis, Indiana, United States
University of Iowa — Iowa City, Iowa, United States
University of Kansas Medical Center — Kansas City, Kansas, United States
University of Kentucky — Lexington, Kentucky, United States
John Hopkins Hospital — Baltimore, Maryland, United States
Massachusetts General Hospital — Boston, Massachusetts, United States
Boston Children's Hospital, Brigham & Women's Hospital — Boston, Massachusetts, United States
University of Michigan Health System — Ann Arbor, Michigan, United States
Wayne State University Harper University Hospital — Detroit, Michigan, United States
Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States
The Minnesota Cystic Fibrosis Center — Minneapolis, Minnesota, United States
University of Alabama at Birmingham — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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