Cystic FibrosisPROMISENCT04038047
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function
An observational study of Cystic Fibrosis, run by Nicole Hamblett at 56 sites in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 490 participants
- Where
- 56 sitesUnited States
- Study dates
- 22 Oct 2019to 23 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals with Cystic Fibrosis, prescribed the elexacaftor, tezacaftor and ivacaftor CFTR modulator therapy (TCT).
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
56 sites in 1 country.
56 sites in 1 country
United States56 sites
Children's Hospital Medical Center of Akron
Akron, Ohio, 44308
Providence Alaska Medical Center
Anchorage, Alaska, 99508
University of Michigan Health System
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta and Emory University
Atlanta, Georgia, 30322
Emory University
Atlanta, Georgia, 30327
Augusta University
Augusta, Georgia, 30912
Children's Hospital Colorado
Aurora, Colorado, 80045
John Hopkins Hospital
Baltimore, Maryland, 21287
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Massachusetts General Hospital
Boston, Massachusetts, 02114
Boston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts, 02115
The Cystic Fibrosis Center of Western New York
Buffalo, New York, 14203
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
University of Virginia
Charlottesville, Virginia, 22904
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
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
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
National Jewish Health
Denver, Colorado, 80206
Wayne State University Harper University Hospital
Detroit, Michigan, 48201
Cook Children's Medical Center
Fort Worth, Texas, 76104
University of Florida
Gainesville, Florida, 32610
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Hershey Medical Center Pennsylvania State University
Hershey, Pennsylvania, 17033
Baylor College of Medicine
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
University of Iowa
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
Children's Mercy Kansas City
Kansas City, Missouri, 64108
Cohen Children's Medical Center of New York
Lake Success, New York, 11042
University of Kentucky
Lexington, Kentucky, 40506
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
University of Wisconsin
Madison, Wisconsin, 53792
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, 55455
Rutgers Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
Yale University School of Medicine
New Haven, Connecticut, 06520
Northwell CF Center
New York, New York, 10021
Children's Hospital of New York
New York, New York, 10032
Oklahoma Cystic Fibrosis Center
Oklahoma City, Oklahoma, 73104
University of Nebraska Medical Center
Omaha, Nebraska, 69198
Stanford University Medical Center
Palo Alto, California, 94025
Saint Francis Medical Center
Peoria, Illinois, 61637
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Oregon Health Sciences University
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23219
University of Rochester Medical Center Strong Memorial
Rochester, New York, 14642
Intermountain Cystic Fibrosis Center
Salt Lake City, Utah, 84132
Seattle Children's Hospital
Seattle, Washington, 98105
University of Washington Medical Center
Seattle, Washington, 98195
Washington University School of Medicine
St Louis, Missouri, 63110
New York Medical College at Westchester Medical Center
Valhalla, New York, 10595
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (The PROMISE Study)
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.
Read the detailed description
Cystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In people with CF, this manifests as dysfunction in multiple organ systems including the lungs, pancreas, liver, intestines, skin and others.
While nearly 2000 mutations have been described, the most common disease-causing CFTR mutation is F508del, which is found in >85% of patients followed in the US CF Patient Registry. Two CFTR corrector drugs plus the potentiator ivacaftor have been developed as a triple combination therapy for CF patients with one or two copies of the F508del mutation. We predict that over 90% of CF patients (initially age 12 y/o and above) will be eligible for highly effective CFTR modulator therapy in the U.S.
The PROMISE study is designed to measure the direct and indirect CFTR-dependent anion secretion by collecting and analyzing clinical research outcomes and biomarkers on a large number of patients both before and after they begin treatment with elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT). This study will investigate the impact of TCT across a wide range of CF disease manifestations and organ systems. While specific biomarkers of special interest have been selected for detailed analysis in this study, an additional important goal is to collect blood, urine, stool, and airway epithelial cell specimens for long-term storage in a biorepository to enable future research. These samples can be made available for research beyond the current scope of work. The PROMISE study will provide a coordinated collection of clinical research outcomes data that can be linked with these specimens.
- Conditions
- Cystic Fibrosis
- Keywords
- CF, CFTR modulator, triple combination therapy
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Core | Cystic Fibrosis patients prescribed elexacaftor, tezacaftor and ivacaftor CFTR modulator therapy (TCT). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sweat Chloride at 6 months
Change in sweat chloride from Baseline to 6 months.
Measured: 6 months
Sweat Chloride at 30 months
Change sweat chloride from Baseline to 30 months.
Measured: 30 months
Forced expiratory volume at one second (FEV1) at 6 months
Change in FEV1 from Baseline to 6 months.
Measured: 6 months
Forced expiratory volume at one second (FEV1) at 30 months
Change in FEV1 from Baseline to 30 months.
Measured: 30 months
Secondary outcomes (6)
Weight at 6 Months
Change in weight from Baseline to 6 months.
Measured: 6 months
Weight at 30 Months
Change in weight from Baseline to 30 months.
Measured: 30 months
BMI at 6 Months
Change in BMI from Baseline to 6 months.
Measured: 6 months
BMI at 30 months
Change in BMI from Baseline to 30 months.
Measured: 30 months
Cystic Fibrosis Questionnaire Revised (CFQ-R) at 6 Months
Change in CFQ-R (respiratory domain) from Baseline to 6 months.
Measured: 6 months
Cystic Fibrosis Questionnaire Revised (CFQ-R) at 30 months
Change in CFQ-R (respiratory domain) from Baseline to 30 months.
Measured: 30 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2019
- Study start22 Oct 2019
- Primary completion23 Dec 2024
- Study completion23 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 490 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nicole Hamblett
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor-investigator, Nicole Hamblett, Seattle Children's Hospital
- Investigators
- Steven Rowe, MD, Principal investigator, University of Alabama at BirminghamDavid Nichols, MD, Principal investigator, University of Washington
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04038047
- Sponsor's protocol ID
- PROMISE-OB-18
- Acronym
- PROMISE
- Status last verified
- Jan 2025
- First submitted
- 25 Jul 2019
- Last updated
- 28 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Sagel SD, Poore TS, Wagner BD, Xie J, Heltshe SL, Cross M, Bratcher PE, Taylor-Cousar JL, Wilson A, McBennett K, Morgan SJ, Singh PK, Nichols DP, Kelly A, Solomon GM; PROMISE Study Group. Long-term reductions in inflammation in people with cystic fibrosis treated with elexacaftor/tezacaftor/ivacaftor. Ann Am Thorac Soc. 2026 Feb 1;23(2):241-251. doi: 10.1513/AnnalsATS.202507-817OC.
PubMed 40961158Pittman JE, Morgan S, McCreary M, Vu PT, Jorth P, Heltshe S, Hoffman LR, Kelly A, Sagel SD, Singh PK, Solomon GM, Rosenfeld M, Ratjen F; PROMISE Pediatric substudy Investigators. Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in 6- to 11-Year-Olds with Cystic Fibrosis: An Observational Study. Ann Am Thorac Soc. 2025 Nov;22(11):1696-1708. doi: 10.1513/AnnalsATS.202501-103OC.
PubMed 40540670Nichols DP, Morgan SJ, Skalland M, Vo AT, Van Dalfsen JM, Singh SB, Ni W, Hoffman LR, McGeer K, Heltshe SL, Clancy JP, Rowe SM, Jorth P, Singh PK; PROMISE-Micro Study Group. Pharmacologic improvement of CFTR function rapidly decreases sputum pathogen density, but lung infections generally persist. J Clin Invest. 2023 May 15;133(10):e167957. doi: 10.1172/JCI167957.
PubMed 36976651Schwarzenberg SJ, Vu PT, Skalland M, Hoffman LR, Pope C, Gelfond D, Narkewicz MR, Nichols DP, Heltshe SL, Donaldson SH, Frederick CA, Kelly A, Pittman JE, Ratjen F, Rosenfeld M, Sagel SD, Solomon GM, Stalvey MS, Clancy JP, Rowe SM, Freedman SD; Promise Study Group. Elexacaftor/tezacaftor/ivacaftor and gastrointestinal outcomes in cystic fibrosis: Report of promise-GI. J Cyst Fibros. 2023 Mar;22(2):282-289. doi: 10.1016/j.jcf.2022.10.003. Epub 2022 Oct 21.
PubMed 36280527Nichols DP, Paynter AC, Heltshe SL, Donaldson SH, Frederick CA, Freedman SD, Gelfond D, Hoffman LR, Kelly A, Narkewicz MR, Pittman JE, Ratjen F, Rosenfeld M, Sagel SD, Schwarzenberg SJ, Singh PK, Solomon GM, Stalvey MS, Clancy JP, Kirby S, Van Dalfsen JM, Kloster MH, Rowe SM; PROMISE Study group. Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial. Am J Respir Crit Care Med. 2022 Mar 1;205(5):529-539. doi: 10.1164/rccm.202108-1986OC.
PubMed 34784492
Study at a glance
From the registry record, last updated by the sponsor on 28 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (The PROMISE Study)
- ClinicalTrials.gov ID
- NCT04038047
- Acronym / sponsor ID
- PROMISE / PROMISE-OB-18
- Conditions
- Cystic Fibrosis
- Keywords
- CF, CFTR modulator, triple combination therapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 490 participants (actual)
- Lead sponsor
- Nicole HamblettUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- 22 Oct 2019 (actual)
- Primary completion
- 23 Dec 2024 (actual)
- Study completion
- 23 Dec 2024 (actual)
- First posted
- 30 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Cystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In people with CF, this manifests as dysfunction in multiple organ systems including the lungs, pancreas, liver, intestines, skin and others.
While nearly 2000 mutations have been described, the most common disease-causing CFTR mutation is F508del, which is found in >85% of patients followed in the US CF Patient Registry. Two CFTR corrector drugs plus the potentiator ivacaftor have been developed as a triple combination therapy for CF patients with one or two copies of the F508del mutation. We predict that over 90% of CF patients (initially age 12 y/o and above) will be eligible for highly effective CFTR modulator therapy in the U.S.
The PROMISE study is designed to measure the direct and indirect CFTR-dependent anion secretion by collecting and analyzing clinical research outcomes and biomarkers on a large number of patients both before and after they begin treatment with elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT). This study will investigate the impact of TCT across a wide range of CF disease manifestations and organ systems. While specific biomarkers of special interest have been selected for detailed analysis in this study, an additional important goal is to collect blood, urine, stool, and airway epithelial cell specimens for long-term storage in a biorepository to enable future research. These samples can be made available for research beyond the current scope of work. The PROMISE study will provide a coordinated collection of clinical research outcomes data that can be linked with these specimens.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Core | – | –Cystic Fibrosis patients prescribed elexacaftor, tezacaftor and ivacaftor CFTR modulator therapy (TCT). |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals with Cystic Fibrosis, prescribed the elexacaftor, tezacaftor and ivacaftor CFTR modulator therapy (TCT).
Key inclusion criteria (8 listed)
- 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).
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Sweat Chloride at 6 months
Time frame: 6 months
Change in sweat chloride from Baseline to 6 months.
Sweat Chloride at 30 months
Time frame: 30 months
Change sweat chloride from Baseline to 30 months.
Forced expiratory volume at one second (FEV1) at 6 months
Time frame: 6 months
Change in FEV1 from Baseline to 6 months.
Forced expiratory volume at one second (FEV1) at 30 months
Time frame: 30 months
Change in FEV1 from Baseline to 30 months.
6 secondary outcomes
Weight at 6 Months
Time frame: 6 months
Weight at 30 Months
Time frame: 30 months
BMI at 6 Months
Time frame: 6 months
BMI at 30 months
Time frame: 30 months
Cystic Fibrosis Questionnaire Revised (CFQ-R) at 6 Months
Time frame: 6 months
Cystic Fibrosis Questionnaire Revised (CFQ-R) at 30 months
Time frame: 30 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jan 2025.
- Derived from this study: PubMed 40961158Sagel SD, Poore TS, Wagner BD, Xie J, Heltshe SL, Cross M, Bratcher PE, Taylor-Cousar JL, Wilson A, McBennett K, Morgan SJ, Singh PK, Nichols DP, Kelly A, Solomon GM; PROMISE Study Group. Long-term reductions in inflammation in people with cystic fibrosis treated with elexacaftor/tezacaftor/ivacaftor. Ann Am Thorac Soc. 2026 Feb 1;23(2):241-251. doi: 10.1513/AnnalsATS.202507-817OC.
- Derived from this study: PubMed 40540670Pittman JE, Morgan S, McCreary M, Vu PT, Jorth P, Heltshe S, Hoffman LR, Kelly A, Sagel SD, Singh PK, Solomon GM, Rosenfeld M, Ratjen F; PROMISE Pediatric substudy Investigators. Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in 6- to 11-Year-Olds with Cystic Fibrosis: An Observational Study. Ann Am Thorac Soc. 2025 Nov;22(11):1696-1708. doi: 10.1513/AnnalsATS.202501-103OC.
- Derived from this study: PubMed 36976651Nichols DP, Morgan SJ, Skalland M, Vo AT, Van Dalfsen JM, Singh SB, Ni W, Hoffman LR, McGeer K, Heltshe SL, Clancy JP, Rowe SM, Jorth P, Singh PK; PROMISE-Micro Study Group. Pharmacologic improvement of CFTR function rapidly decreases sputum pathogen density, but lung infections generally persist. J Clin Invest. 2023 May 15;133(10):e167957. doi: 10.1172/JCI167957.
- Derived from this study: PubMed 36280527Schwarzenberg SJ, Vu PT, Skalland M, Hoffman LR, Pope C, Gelfond D, Narkewicz MR, Nichols DP, Heltshe SL, Donaldson SH, Frederick CA, Kelly A, Pittman JE, Ratjen F, Rosenfeld M, Sagel SD, Solomon GM, Stalvey MS, Clancy JP, Rowe SM, Freedman SD; Promise Study Group. Elexacaftor/tezacaftor/ivacaftor and gastrointestinal outcomes in cystic fibrosis: Report of promise-GI. J Cyst Fibros. 2023 Mar;22(2):282-289. doi: 10.1016/j.jcf.2022.10.003. Epub 2022 Oct 21.
- Derived from this study: PubMed 34784492Nichols DP, Paynter AC, Heltshe SL, Donaldson SH, Frederick CA, Freedman SD, Gelfond D, Hoffman LR, Kelly A, Narkewicz MR, Pittman JE, Ratjen F, Rosenfeld M, Sagel SD, Schwarzenberg SJ, Singh PK, Solomon GM, Stalvey MS, Clancy JP, Kirby S, Van Dalfsen JM, Kloster MH, Rowe SM; PROMISE Study group. Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial. Am J Respir Crit Care Med. 2022 Mar 1;205(5):529-539. doi: 10.1164/rccm.202108-1986OC.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven Rowe, MD principal investigator
University of Alabama at Birmingham
David Nichols, MD principal investigator
University of Washington
56 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Nicole Hamblett at 56 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 56 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Sweat Chloride at 6 months (time frame: 6 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04038047, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jan 2025.