Ivacaftor Impact in an Adolescent Without a Cystic Fibrosis-Associated CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Mutation for Chronic Pancreatitis: Single-Patient Study Tracking Flare Frequency and Health Data Compared to Historical Records.

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).

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Chronic Pancreatitis Chronic Disease Digestive System Diseases Disease Attributes Pancreatic Diseases Pancreatitis Pancreatitis, Chronic Pathologic Processes Pathological Conditions, Signs and Symptoms

Sex eligibility

All sexes

Study type

Observational

Primary location

Mission: Cure

Seattle, Washington, 98199, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Ivacaftor 150 MG (milligrams)

Drug

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.

Primary outcomes

Time Between Acute Pancreatitis Episodes While on Ivacaftor

Time frame: 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.

Sponsors and collaborators

Lead sponsor

Mission: Cure

Other

Registry information

Official study title

IMPACT Study: Intervention Monitoring for Pancreatitis in an Adolescent Undergoing Clinical Treatment

Acronym: IMPACT

Important dates

Study start: 2025
Primary completion: 2026
Study completion: 2026
First posted: Jun 15, 2025
Registry last updated: Jun 15, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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