Cystic FibrosisNCT04613128
The PROMISE Pediatric Study 6 to 11 Years Old
An observational study of Cystic Fibrosis and Cystic Fibrosis in Children, run by Nicole Hamblett at 20 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 children (6-11 years of old) with one or more copies of the F508del mutation, study the effects of ETI across a number of CF… Read more about this study
- Who is it for?
- Ages 6 to 11 years
- Conditions the study is for
- Cystic Fibrosis, Cystic Fibrosis in Children
- How many people
- 125 participants
- Where
- 20 sitesUnited States
- Study dates
- 11 Jun 2021to 8 Dec 2025
- 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 6 to 11 years
- All sexes
- No healthy volunteers
- Child
Study population: Cystic Fibrosis, 6-11 years old prescribed ETI.
Who may be able to join
- Written parental informed consent and assent obtained from subject and the subject's legal guardian.
- Be willing and able to adhere to the study visit schedule and other protocol requirements.
- All genders 6-11 years old on Day 1.
- Diagnosis of CF.
- CFTR mutations consistent with the FDA labeled indication for the ETI.
- Physician intent to prescribe the ETI.
Show all 9Show fewer
- Able to attempt 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 (and inclusive of Visit 1).
Who cannot join
- Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.
- Use of any ETI within the 180 days prior to Visit 1.
- Any acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids within the 14 days prior to Visit 1 (inclusive of 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 (inclusive of 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
20 sites in 1 country.
20 sites in 1 country
United States20 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
John Hopkins University
Baltimore, Maryland, 21287
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Boston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts, 02115
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44146
Baylor College of Medicine
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
University of Iowa
Iowa City, Iowa, 52242
Children's Mercy Kansas City
Kansas City, Missouri, 64108
University of Wisconsin
Madison, Wisconsin, 53792
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, 55455
Stanford University Medical Center
Palo Alto, California, 94304
Oregon Health Sciences University
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23219
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (the PROMISE Pediatric 6 to 11 Years Old 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 children (6-11 years of old) 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 ETI" visit within 30 days before initiation of the therapy and five "after ETI" visits over a 24-month follow-up period. Participants who have participated in the original PROMISE Pediatric Sub-Study have the option of participating in a long-term extension with annual visits performed at the 36- and 48-month timepoints. The durability of the clinical and biological changes in the PROMISE Pediatric Sub-Study 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. The duration of participation for each subject is 24 months (with an additional 24 months if participants agree to the optional long-term extension). NOTE: FDA has granted approval for elexacaftor, tezacaftor and ivacaftor in the 6-11 age group.
Read the detailed description
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 (elexacaftor and tezacaftor) plus the potentiator ivacaftor have been developed and approved as a triple combination therapy for CF patients (12 years old and above) with one or two copies of the F508del mutation. We predict that over 90% of pediatric CF patients (age 6-11 y/o) will be eligible for highly effective CFTR modulator therapy in the U.S.
The PROMISE Pediatric Study is designed to measure the direct and indirect CFTR dependent anion secretion by collecting and analyzing clinical research outcomes and biomarkers on pediatric patients both before and after they begin treatment with ETI. This study will investigate the impact of ETI 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 Pediatric Study will provide a coordinated collection of clinical research outcomes data that can be linked with these specimens.
- Conditions
- Cystic Fibrosis, Cystic Fibrosis in Children
- Keywords
- CF, Cystic Fibrosis, CFTR Modulator, Trikafta, Triple Combination Therapy, Pediatric, ETI, elexacaftor/texacaftor/ivacaftor
- 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 |
|---|---|---|
| Pediatric | Cystic Fibrosis pediatric patients (6-11 years old) prescribed ETI CFTR modulator Therapy. |
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 24 months
Change sweat chloride from Baseline to 24 months.
Measured: 24 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 24 months
Change in FEV1 from Baseline to 24 months.
Measured: 24 months
Lung Clearance Index (LCI) at 6 months
Change Lung Clearance Index (LCI) from baseline to 6 months.
Measured: 6 months
Lung Clearance Index (LCI) at 24 months
Change Lung Clearance Index (LCI) from baseline to 24 months.
Measured: 24 months
Secondary outcomes (6)
Weight at 6 Months
Change in weight from Baseline to 6 months.
Measured: 6 months
Weight at 24 Months
Change in weight from Baseline to 24 months.
Measured: 24 months
BMI at 6 Months
Change in BMI from Baseline to 6 months.
Measured: 6 months
BMI at 24 Months
Change in BMI from Baseline to 24 months.
Measured: 24 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 24 Months
Change in CFQ-R (respiratory domain) from Baseline to 24 months.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2020
- Study start11 Jun 2021
- Primary completion8 Dec 2025
- Study completion8 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 125 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
- NCT04613128
- Sponsor's protocol ID
- PROMISE-OB-18 Pediatric Study
- Status last verified
- Apr 2026
- First submitted
- 27 Oct 2020
- Last updated
- 27 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2026. 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 Pediatric 6 to 11 Years Old Study)
- ClinicalTrials.gov ID
- NCT04613128
- Acronym / sponsor ID
- PROMISE-OB-18 Pediatric Study
- Conditions
- Cystic Fibrosis, Cystic Fibrosis in Children
- Keywords
- CF, Cystic Fibrosis, CFTR Modulator, Trikafta, Triple Combination Therapy, Pediatric, ETI, elexacaftor/texacaftor/ivacaftor
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 125 participants (actual)
- Lead sponsor
- Nicole HamblettUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- 11 Jun 2021 (actual)
- Primary completion
- 8 Dec 2025 (actual)
- Study completion
- 8 Dec 2025 (actual)
- First posted
- 3 Nov 2020 (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 children (6-11 years of old) 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 ETI" visit within 30 days before initiation of the therapy and five "after ETI" visits over a 24-month follow-up period. Participants who have participated in the original PROMISE Pediatric Sub-Study have the option of participating in a long-term extension with annual visits performed at the 36- and 48-month timepoints. The durability of the clinical and biological changes in the PROMISE Pediatric Sub-Study 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. The duration of participation for each subject is 24 months (with an additional 24 months if participants agree to the optional long-term extension). NOTE: FDA has granted approval for elexacaftor, tezacaftor and ivacaftor in the 6-11 age group.
Detailed description
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 (elexacaftor and tezacaftor) plus the potentiator ivacaftor have been developed and approved as a triple combination therapy for CF patients (12 years old and above) with one or two copies of the F508del mutation. We predict that over 90% of pediatric CF patients (age 6-11 y/o) will be eligible for highly effective CFTR modulator therapy in the U.S.
The PROMISE Pediatric Study is designed to measure the direct and indirect CFTR dependent anion secretion by collecting and analyzing clinical research outcomes and biomarkers on pediatric patients both before and after they begin treatment with ETI. This study will investigate the impact of ETI 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 Pediatric 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 |
|---|---|---|
| Pediatric | – | –Cystic Fibrosis pediatric patients (6-11 years old) prescribed ETI CFTR modulator Therapy. |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Cystic Fibrosis, 6-11 years old prescribed ETI.
Key inclusion criteria (9 listed)
- Written parental informed consent and assent obtained from subject and the subject's legal guardian.
- Be willing and able to adhere to the study visit schedule and other protocol requirements.
- All genders 6-11 years old on Day 1.
- Diagnosis of CF.
- CFTR mutations consistent with the FDA labeled indication for the ETI.
Key exclusion criteria (8 listed)
- Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.
- Use of any ETI within the 180 days prior to Visit 1.
- Any acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids within the 14 days prior to Visit 1 (inclusive of 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 (inclusive of Visit 1).
- Use of an investigational agent 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 24 months
Time frame: 24 months
Change sweat chloride from Baseline to 24 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 24 months
Time frame: 24 months
Change in FEV1 from Baseline to 24 months.
Lung Clearance Index (LCI) at 6 months
Time frame: 6 months
Change Lung Clearance Index (LCI) from baseline to 6 months.
Lung Clearance Index (LCI) at 24 months
Time frame: 24 months
Change Lung Clearance Index (LCI) from baseline to 24 months.
6 secondary outcomes
Weight at 6 Months
Time frame: 6 months
Weight at 24 Months
Time frame: 24 months
BMI at 6 Months
Time frame: 6 months
BMI at 24 Months
Time frame: 24 months
Cystic Fibrosis Questionnaire Revised (CFQ-R) at 6 Months
Time frame: 6 months
Cystic Fibrosis Questionnaire Revised (CFQ-R) at 24 Months
Time frame: 24 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 27 Apr 2026.
- 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
20 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 and Cystic Fibrosis in Children, run by Nicole Hamblett at 20 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 9 inclusion and 8 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 27 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 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 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Apr 2026.