Birmingham, Alabama 35233

  • Cystic Fibrosis

Purpose:

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.


Study 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. Symdeko (Tezacaftor/Ivacaftor), Orkambi (Ivacaftor and Lumacaftor), correctors of CFTR misfolding and Kalydeco (Ivacaftor), a potentiator of abnormal CFTR gating, will be explored as a treatment for patients with other CF mutations than those currently approved. Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, or Orkambi, depending on the in vitro response pattern. Patients who have mutation response to a potentiator of CFTR function will be given Ivacaftor monotherapy. Patients with a mutation equivalent to wild type will be given Ivacaftor. If the patient is 6-12 years old, we will only study Orkambi or ivacaftor as symdeko is not yet FDA approved in this patient population.


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


NCT ID:

NCT03587961


Primary Contact:

Heather Hathorne, PhD
Phone: 205-638-9568
Email: hhathorne@peds.uab.edu


Backup Contact:

N/A


Location Contact:

Birmingham, Alabama 35233
United States

Heather Hathorne, PhD
Phone: 205-638-9568
Email: hhathorne@peds.uab.edu

Site Status: Recruiting


Data Source: ClinicalTrials.gov

Date Processed: June 18, 2021

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