A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function
Public ClinicalTrials.gov record NCT04038047. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (The PROMISE Study)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04038047
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 490 participants
Conditions and interventions
Conditions
Interventions
Not listed
Eligibility (public fields only)
- Age range
- 12 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 22, 2019 (Actual)
- Primary completion
- Dec 23, 2024 (Actual)
- Completion
- Dec 23, 2024 (Actual)
- Last update posted
- Jan 28, 2025
2019 – 2024
United States locations
- U.S. sites
- 56
- U.S. states
- 32
- U.S. cities
- 50
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294 | — |
| Providence Alaska Medical Center | Anchorage | Alaska | 99508 | — |
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72205 | — |
| Stanford University Medical Center | Palo Alto | California | 94025 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| National Jewish Health | Denver | Colorado | 80206 | — |
| Yale University School of Medicine | New Haven | Connecticut | 06520 | — |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | — |
| University of Florida | Gainesville | Florida | 32610 | — |
| Children's Healthcare of Atlanta and Emory University | Atlanta | Georgia | 30322 | — |
| Emory University | Atlanta | Georgia | 30327 | — |
| Augusta University | Augusta | Georgia | 30912 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Saint Francis Medical Center | Peoria | Illinois | 61637 | — |
| Riley Hospital for Children | Indianapolis | Indiana | 46202 | — |
| University of Iowa | Iowa City | Iowa | 52242 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| University of Kentucky | Lexington | Kentucky | 40506 | — |
| John Hopkins Hospital | Baltimore | Maryland | 21287 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Boston Children's Hospital, Brigham & Women's Hospital | Boston | Massachusetts | 02115 | — |
| University of Michigan Health System | Ann Arbor | Michigan | 48109 | — |
| Wayne State University Harper University Hospital | Detroit | Michigan | 48201 | — |
| Helen DeVos Children's Hospital | Grand Rapids | Michigan | 49503 | — |
| The Minnesota Cystic Fibrosis Center | Minneapolis | Minnesota | 55455 | — |
| Children's Mercy Kansas City | Kansas City | Missouri | 64108 | — |
| Washington University School of Medicine | St Louis | Missouri | 63110 | — |
| University of Nebraska Medical Center | Omaha | Nebraska | 69198 | — |
| Rutgers Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08901 | — |
| The Cystic Fibrosis Center of Western New York | Buffalo | New York | 14203 | — |
| Cohen Children's Medical Center of New York | Lake Success | New York | 11042 | — |
| Northwell CF Center | New York | New York | 10021 | — |
| Children's Hospital of New York | New York | New York | 10032 | — |
| University of Rochester Medical Center Strong Memorial | Rochester | New York | 14642 | — |
| New York Medical College at Westchester Medical Center | Valhalla | New York | 10595 | — |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | — |
| Children's Hospital Medical Center of Akron | Akron | Ohio | 44308 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| University Hospitals Case Medical Center/Rainbow Babies and Children's Hospital | Cleveland | Ohio | 44106 | — |
| Cleveland Clinic Cystic Fibrosis Program | Cleveland | Ohio | 44195 | — |
| Oklahoma Cystic Fibrosis Center | Oklahoma City | Oklahoma | 73104 | — |
| Oregon Health Sciences University | Portland | Oregon | 97239 | — |
| Hershey Medical Center Pennsylvania State University | Hershey | Pennsylvania | 17033 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | — |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | — |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| Intermountain Cystic Fibrosis Center | Salt Lake City | Utah | 84132 | — |
| University of Virginia | Charlottesville | Virginia | 22904 | — |
| Virginia Commonwealth University | Richmond | Virginia | 23219 | — |
| Seattle Children's Hospital | Seattle | Washington | 98105 | — |
| University of Washington Medical Center | Seattle | Washington | 98195 | — |
| University of Wisconsin | Madison | Wisconsin | 53792 | — |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04038047, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 28, 2025 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04038047 live on ClinicalTrials.gov.