Personalized Theratyping Trial
Who is this study for? Patients with Cystic Fibrosis
What treatments are being studied? Symdeko Cystic Fibrosis Transmembrane Conductance Regulator Modulator
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Early Phase 1
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
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• 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
Locations
United States
Alabama
University of Alabama at Birmingham
RECRUITING
Birmingham
Contact Information
Primary
Heather Hathorne, PhD
hhathorne@peds.uab.edu
205-638-9568
Time Frame
Start Date: 2019-08-01
Estimated Completion Date: 2027-09-01
Participants
Target number of participants: 20
Treatments
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
Related Therapeutic Areas
Sponsors
Leads: George Solomon