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. (IMPACT)

Mission: Cure

Status: Active, not recruiting

Conditions: Chronic Pancreatitis

Treatments: Drug: Ivacaftor 150 MG (milligrams)

Study type: Observational

Funder types: Other

Identifiers

NCT07022327

20251496 (Other Identifier)

N1-IVAC-01

Details and patient eligibility

About

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).

Enrollment: 1 patient

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design