Chronic Pancreatitis
Conditions
Brief summary
The goal of this observational study is to learn about the long-term effects of Ivacaftor in an adolescent without a cystic fibrosis-associated CFTR mutation who is taking Ivacaftor to treat chronic pancreatitis. The main question it aims to answer is: Does Ivacaftor reduce the frequency or severity of acute pancreatitis episodes in this adolescent compared to their historical medical data? The participant, who is already taking Ivacaftor as part of their medical care, will be monitored over the course of 12 months. Flare frequency, hospitalizations, and wellness data will be tracked through caregiver reports, lab data, and physiological data collected from a wearable health monitoring device (Apple Watch).
Interventions
DRUGIvacaftor 150 MG (milligrams)
Ivacaftor is a CFTR potentiator approved for the treatment of cystic fibrosis in patients with specific gating mutations. In this observational, single-patient study, Ivacaftor is being used off-label in an adolescent without a cystic fibrosis-associated CFTR mutation to evaluate its potential impact on chronic pancreatitis. The intervention is not assigned by the study team but is part of the participant's ongoing medical care. The study monitors clinical outcomes and wearable health data over a 12-month period.
Sponsors
Mission: Cure
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
The subject has been diagnosed with recurrent acute pancreatitis or chronic pancreatitis and a clinical decision has been made to treat the subject with one or more FDA-approved therapies, which may be being used off-label) to treat or prevent pancreatitis symptoms.
Exclusion criteria
Participant has a known cystic fibrosis-associated CFTR mutation that would qualify them for standard Ivacaftor use. Participant is unable to tolerate Ivacaftor due to prior adverse reactions. Participant has severe liver dysfunction or baseline liver enzyme elevations that contraindicate Ivacaftor use, as determined by the treating physician. Participant or parent/guardian is unwilling to comply with study procedures, including regular follow-up and data sharing. Any other medical or psychological condition, in the judgment of the treating physician or counselor, that would make participation unsafe or not in the best interest of the participant.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time Between Acute Pancreatitis Episodes While on Ivacaftor | 12 months | The number of days between clinically confirmed acute pancreatitis attacks during the 12-month Ivacaftor treatment period, compared to historical pre-treatment intervals. |
Countries
United States
Outcome results
None listed