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RECRUITINGEarly Phase 1INTERVENTIONAL

Personalized Theratyping Trial

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - Diagnosis of CF - Age ≥6 y.o. - CFTR mutation that may respond to approved correctors/potentiators in the opinion of the study investigators - willing to sign a consent form/Assent - Stable CF pulmonary regimen Who Should NOT Join This Trial: - 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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

DRUG

Symdeko

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.

Locations (1)

University of Alabama at Birmingham
Birmingham, Alabama, United States