Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations

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.

Timeline

Start

2018-12-01

Primary completion

2019-10-03

Completion

2019-10-03

Outcome

Outcome not reported

Stopped: “summary results revealed ineffective intervention and the trial was terminated prematurely. No formal statistical analysis was performed”

Drugs

Indications