Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations
Status:
Recruiting
Trial end date:
2022-11-01
Target enrollment:
Participant gender:
Summary
Based on previous clinical findings, the investigator hypothesize that ivacaftor will have
synergistic effects with drugs that facilitate truncated but partially active W1282X CFTR
protein processing (tezacaftor) in patients with W1282X CFTR. In the current study, the
investigators propose to directly test the efficacy of tezacaftor/ivacaftor (TEZ/IVA) and
Trikafta for W1282X CFTR therapy in the clinic in comparison to ivacaftor alone.