RecruitingEarly Phase 1NCT03587961

Personalized Theratyping Trial

Sponsor

George Solomon

Enrollment

20 participants

Start Date

Aug 1, 2019

Study Type

INTERVENTIONAL

Conditions

Cystic Fibrosis

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.

Eligibility

Min Age: 6 Years

Inclusion Criteria5

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 Criteria7

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

Interventions

DRUGSymdeko

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.