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Recruiting EARLY_Phase 1 NCT03587961

Personalized Theratyping Trial

Trial Parameters

Condition Cystic Fibrosis
Sponsor George Solomon
Study Type INTERVENTIONAL
Phase EARLY_Phase 1
Enrollment 20
Sex ALL
Min Age 6 Years
Max Age N/A
Start Date 2019-08-01
Completion 2027-01-01
Interventions
Symdeko

Brief 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 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

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