A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (PROMISE)
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| ClinicalTrials.gov Identifier: NCT04038047 |
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Recruitment Status :
Active, not recruiting
First Posted : July 30, 2019
Last Update Posted : September 9, 2022
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| Condition or disease |
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| Cystic Fibrosis |
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 |
| Actual Enrollment : | 490 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Official Title: | A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (The PROMISE Study) |
| Actual Study Start Date : | October 22, 2019 |
| Estimated Primary Completion Date : | October 27, 2024 |
| Estimated Study Completion Date : | October 27, 2024 |
| Group/Cohort |
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Core
Cystic Fibrosis patients prescribed elexacaftor, tezacaftor and ivacaftor CFTR modulator therapy (TCT).
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- 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.
- Weight at 6 Months [ Time Frame: 6 months ]Change in weight from Baseline to 6 months.
- Weight at 30 Months [ Time Frame: 30 months ]Change in weight from Baseline to 30 months.
- BMI at 6 Months [ Time Frame: 6 months ]Change in BMI from Baseline to 6 months.
- BMI at 30 months [ Time Frame: 30 months ]Change in BMI from Baseline to 30 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 30 months [ Time Frame: 30 months ]Change in CFQ-R (respiratory domain) from Baseline to 30 months.
Biospecimen Retention: Samples With DNA
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| Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
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
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| Principal Investigator: | Steven Rowe, MD | University of Alabama at Birmingham | |
| Principal Investigator: | David Nichols, MD | University of Washington |
| Responsible Party: | Nicole Hamblett, Professor of Pediatrics, Division of Pulmonary and Sleep Medicine, University of Washington School of Medicine Adjunct Professor, Biostatistics, University of Washington School of Medicine Co-Executive Director, Cystic Fibrosis Therapeutics Development, Seattle Children's Hospital |
| ClinicalTrials.gov Identifier: | NCT04038047 |
| Other Study ID Numbers: |
PROMISE-OB-18 |
| First Posted: | July 30, 2019 Key Record Dates |
| Last Update Posted: | September 9, 2022 |
| Last Verified: | September 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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CF CFTR modulator triple combination therapy |
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Cystic Fibrosis Pancreatic Diseases Digestive System Diseases Lung Diseases |
Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |