- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04613128
The PROMISE Pediatric Study 6 to 11 Years Old
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (the PROMISE Pediatric 6 to 11 Years Old Study)
Study Overview
Status
Conditions
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 Type
Enrollment (Estimated)
Contacts and Locations
Study Locations
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Alabama
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Birmingham, Alabama, United States, 35294
- University of Alabama at Birmingham
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California
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Palo Alto, California, United States, 94304
- Stanford University Medical Center
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
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Illinois
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Chicago, Illinois, United States, 60611
- Ann & Robert H. Lurie Children's Hospital of Chicago
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Indiana
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Indianapolis, Indiana, United States, 46202
- Riley Hospital for Children
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Iowa
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Iowa City, Iowa, United States, 52242
- University of Iowa
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Maryland
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Baltimore, Maryland, United States, 21287
- John Hopkins University
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Boston Children's Hospital, Brigham & Women's Hospital
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Minnesota
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Minneapolis, Minnesota, United States, 55455
- The Minnesota Cystic Fibrosis Center
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Missouri
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Kansas City, Missouri, United States, 64108
- Children's Mercy Kansas City
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Saint Louis, Missouri, United States, 63110
- Washington University School of Medicine
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North Carolina
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Chapel Hill, North Carolina, United States, 27599
- University of North Carolina at Chapel Hill
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital Medical Center
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Cleveland, Ohio, United States, 44146
- Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
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Oregon
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Portland, Oregon, United States, 97239
- Oregon Health Sciences University
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Texas
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Houston, Texas, United States, 77030
- Baylor College of Medicine
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Virginia
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Richmond, Virginia, United States, 23219
- Virginia Commonwealth University
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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Wisconsin
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Madison, Wisconsin, United States, 53792
- University of Wisconsin
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Milwaukee, Wisconsin, United States, 53226
- Children's Hospital of Wisconsin
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
Inclusion Criteria:
- 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.
- 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).
Exclusion Criteria:
- 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.
Study Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
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Pediatric
Cystic Fibrosis pediatric patients (6-11 years old) prescribed ETI CFTR modulator Therapy.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Sweat Chloride at 6 months
Time Frame: 6 months
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Change in sweat chloride from Baseline to 6 months.
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6 months
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Sweat Chloride at 24 months
Time Frame: 24 months
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Change sweat chloride from Baseline to 24 months.
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24 months
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Forced expiratory volume at one second (FEV1) at 6 months
Time Frame: 6 months
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Change in FEV1 from Baseline to 6 months.
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6 months
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Forced expiratory volume at one second (FEV1) at 24 months
Time Frame: 24 months
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Change in FEV1 from Baseline to 24 months.
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24 months
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Lung Clearance Index (LCI) at 6 months
Time Frame: 6 months
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Change Lung Clearance Index (LCI) from baseline to 6 months.
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6 months
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Lung Clearance Index (LCI) at 24 months
Time Frame: 24 months
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Change Lung Clearance Index (LCI) from baseline to 24 months.
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24 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Weight at 6 Months
Time Frame: 6 months
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Change in weight from Baseline to 6 months.
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6 months
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Weight at 24 Months
Time Frame: 24 months
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Change in weight from Baseline to 24 months.
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24 months
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BMI at 6 Months
Time Frame: 6 months
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Change in BMI from Baseline to 6 months.
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6 months
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BMI at 24 Months
Time Frame: 24 months
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Change in BMI from Baseline to 24 months.
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24 months
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Cystic Fibrosis Questionnaire Revised (CFQ-R) at 6 Months
Time Frame: 6 months
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Change in CFQ-R (respiratory domain) from Baseline to 6 months.
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6 months
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Cystic Fibrosis Questionnaire Revised (CFQ-R) at 24 Months
Time Frame: 24 months
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Change in CFQ-R (respiratory domain) from Baseline to 24 months.
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24 months
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Steven Rowe, MD, University of Alabama at Birmingham
- Principal Investigator: David Nichols, MD, University of Washington
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- PROMISE-OB-18 Pediatric Study
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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