Personalized Theratyping Trial

Recruiting Early Phase 1 Interventional Study
Cystic Fibrosis
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
6 and older
Sex
Any
Trial phase
Early Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
20 (estimated)
Sponsor
George Solomon · Other
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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.

Trial Locations
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Eligibility 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 d…
Contacts

Heather Hathorne, PhD

205-638-9568

hhathorne@peds.uab.edu

CONTACT