The Return of the Pancreas: Evaluating impact of CFTR modulators on pancreatic function
Molly Bozic, MD
Primary Investigator
Brief description of study
What is the purpose of this study?
This study aims to see how often children with cystic fibrosis (CF) develop normal pancreatic function after starting treatment with CFTR modulators and to identify what factors might influence this change. We will also look at whether it is safe for these patients to stop taking pancreatic enzyme supplements and monitor for complications such as malbsorption and acute pancreatitis. Participants will complete surveys and provide lab samples, and we will review their medical records.
Detailed description of study
What will happen during the study?
The participants will submit the following lab samples after enrollment. When the stool sample for fecal elastse
is obtained, this will be known as day 0 of the study. Fecal elastase results will determine which arm of the
study the participant will be.
1. Group A: fecal elastase result is < 200
2. Group B: fecal elastase result is ≥ 200
Participants in Group A and Group B must submit the following blood tests on day 0 (± 28 days):
• Hepatic function panel
• Trikafta/Kalydeco trough levels (to be obtained 22 to 26 hours from the most recent CFTRm dose)
Participants in Group A and Group B will complete the following survey questions on day 0 (± 28 days). The
parent or guardian of participants age <7 years will complete the survey for the participant. For participants ≥ 7
years, the participant will complete the survey with assistance from their parent or guardian. Included within this
survey is the validated PAGI-SYM © Acute scoring system with consent for use obtained from the Mapi
Research Trust valid through June 2026. The second validated scoring system is The Hunger Vital
Sign™. The rest of the survey questions are original to this study. The survey questions can be found within
the Protocol Attachments.
After the submission of the day 0 fecal elastase, blood work, and online survey, participants in Group A
completed the study. After the submission of the day 0 fecal elastase, blood work, and online survey, Group B
will discontinue PERT.
At 3 months (± 28 days), participants in Group B must complete the online survey.
At 6 months (± 28 days), participants must submit a fecal elastase, blood work, and the online survey. After the
submission of the 6-month fecal elastase, blood work, and online survey, Group B completed the study. After
the completion of the study, the participants who discontinued enzymes may continue off of enzymes with the
plan to continue routine every 3-month follow-up with the cystic fibrosis team which includes pediatric
pulmonology and dietician. Pediatric gastroenterology will be available for as needed follow-up for participants
who have discontinued enzymes. The blood work required at this visit includes:
• Vitamin A (Retinol)
• Vitamin D (25-hydroxyvitamin D)
• Vitamin E (alpha tocopherol)
• INR
• Hepatic function panel
• Trikafta/Kalydeco trough levels (to be obtained 22 to 26 hours from the most recent CFTRm dose)
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Cystic Fibrosis, CF, Pancreatic insufficiency, Riley
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Age: 18 years or below
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Gender: All
Inclusion:
• People with cystic fibrosis and pancreatic insufficiency as defined by fecal pancreatic elastase <200 at
baseline
• Age ≤ 18
• Current use of Elexacaftor/tezacaftor/ivacaftor or ivakaftor*
Exclusion:
• CF-related diabetes requiring current insulin use
• Advanced CF Liver disease as defined by nodular liver, advanced fibrosis (F4), multi-lobular cirrhosis
with or without portal hypertension, non-cirrhotic portal hypertension
• Short Gut syndrome as defined by need for parenteral nutrition for > 60 days or bowel length less than
25%.
• History of surgical bowel resection
• Moderate to severe malnutrition as defined by either weight for length or BMI z score -2 or greater.
