Personalized Theratyping Trial
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.
Condition
- Cystic Fibrosis
Eligibility
- Eligible Ages
- Over 6 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Early Phase 1
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Symdeko |
Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, depending on the in vitro response pattern |
|
|
Experimental Ivacaftor |
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. |
|
|
Experimental Orkambi |
Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Orkambi, depending on the in vitro response pattern |
|
Recruiting Locations
Birmingham, Alabama 35233
More Details
- Status
- Recruiting
- Sponsor
- University of Alabama at Birmingham
Detailed Description
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.