Personalized Theratyping Trial

Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age6+
SponsorGeorge Solomon

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.

Eligibility criteria

Qualifiers

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

Disqualifiers

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

Trial design

Treatments tested in this trial

  • Symdeko

Treatment groups

20 Participants
are divided into 3 treatment groups

Sponsors and collaborators

George Solomon

Lead sponsor

University of Alabama at Birmingham

Sponsor institution