A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (PROMISE)
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|ClinicalTrials.gov Identifier: NCT04038047|
Recruitment Status : Not yet recruiting
First Posted : July 30, 2019
Last Update Posted : August 14, 2019
|Condition or disease|
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 Type :||Observational|
|Estimated Enrollment :||400 participants|
|Official Title:||A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (The PROMISE Study)|
|Estimated Study Start Date :||March 1, 2020|
|Estimated Primary Completion Date :||November 30, 2022|
|Estimated Study Completion Date :||November 30, 2022|
Cystic Fibrosis patients prescribed elexacaftor, tezacaftor and ivacaftor CFTR modulator therapy (TCT).
- 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.
- Weight at 6 Months [ Time Frame: 6 months ]Change in weight from Baseline to 6 months.
- Weight at 24 Months [ Time Frame: 24 months ]Change in weight from Baseline to 24 months.
- BMI at 6 Months [ Time Frame: 6 months ]Change in BMI from Baseline to 6 months.
- BMI at 24 Months [ Time Frame: 24 months ]Change in BMI from Baseline to 24 months.
- Cystic Fibrosis Questionnaire Revised (CFQ-R) at 6 Months [ Time Frame: 6 months ]Change in CFQ-R (respiratory domain) from Baseline to 6 months.
- Cystic Fibrosis Questionnaire Revised (CFQ-R) at 24 Months [ Time Frame: 24 months ]Change in CFQ-R (respiratory domain) from Baseline to 24 months.
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04038047
|Contact: Shannon Kirbyemail@example.com|
Show 55 Study Locations
|Principal Investigator:||Steven Rowe, MD||University of Alabama at Birmingham|
|Principal Investigator:||David Nichols, MD||Seattle Children's Hospital|