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.

NCT ID: NCT07022327

Last Updated: 2025-06-15

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 1 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2025-05-06
• Study Completion Date: 2026-08-31

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).

Conditions

Chronic Pancreatitis

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: PROSPECTIVE

Interventions

Ivacaftor 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.

Intervention Type: DRUG

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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Mission: Cure

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Nicholas Schork, PhD

Role: PRINCIPAL_INVESTIGATOR

Net.bio

Locations

Mission: Cure

Seattle, Washington, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

20251496

Identifier Type: OTHER

Identifier Source: secondary_id

N1-IVAC-01

Identifier Type: -

Identifier Source: org_study_id