Personalized Theratyping Trial

• Status: Recruiting
• Trial end date: 2023-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Alabama at Birmingham
• Treatments: Ivacaftor

Criteria

Inclusion Criteria:

• Diagnosis of CF

• Age ≥6 y.o.

• CFTR mutation that may respond to approved correctors/potentiators in the opinion of the study investigators

• Informed Consent/Assent

• Stable CF pulmonary regimen

Exclusion Criteria:

• Exacerbation requiring antibiotic or steroids for >28 days before trial entry

• Ongoing participation in a CFTR modulator study

• Active smoking in the past 6 months

• History of solid organ transplant

• Any condition which precludes the use of CFTR modulators: e.g. advanced cirrhosis, End-stage Renal Disease (ESRD)

• Any condition that precludes the patient from participation in the opinion of the investigator

• Any meds that have significant drug-drug interactions or any other off label use of CFTR modulators