Clinical trial NCT07022327: 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.
Official title: IMPACT Study: Intervention Monitoring for Pancreatitis in an Adolescent Undergoing Clinical Treatment
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Chronic Pancreatitis
Lead sponsor: Mission: Cure
Sponsor class: OTHER
First posted: 2025-06-15
Enrollment: 1
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population consists of a single adolescent under the age of 16 with a clinical diagnosis of chronic pancreatitis, who has experienced recurrent acute-on-chronic pancreatitis episodes despite standard treatments. The participant does not carry a cystic fibrosis-associated CFTR mutation associated with Cystic Fibrosis and is under the ongoing care of a multidisciplinary team. This individual was selected for observation based on persistent disease burden, lack of response to conventional therapies, and medical decision-making that included initiating off-label Ivacaftor use as part of the participant's clinical care.

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).


Eligibility criteria: 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.

Primary outcomes:
measure: Time Between Acute Pancreatitis Episodes While on Ivacaftor
timeFrame: 12 months
Secondary outcomes:
Other outcomes:
Locations:
city: Seattle
country: United States
facility: Mission: Cure
state: Washington
Study officials:
affiliation: Net.bio
name: Nicholas Schork, PhD
role: PRINCIPAL_INVESTIGATOR
References: