Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 2:22 PM
Ignite Modification Date:
2025-12-24 @ 2:22 PM
NCT ID:
NCT04135495
Brief Summary:
This is a Phase 2 open-label, dose-escalation study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele.
In total, up to 16 patients will be enrolled in the trial; up to 4 patients will be homozygotes for G542X, and the remaining patients will be compound heterozygotes with one G542X or phenotypically similar nonsense allele and any Class 1 or Class 2 mutation.
Each patient will receive up to 5 escalating doses as follows:
* ELX-02 0.3 mg/kg per day SC
* ELX-02 0.75 mg/kg per day SC
* ELX-02 1.5 mg/kg per day SC
* An individualized dose of ELX-02, as high as 3.0 mg/kg per day SC, based on the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests.
* ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid
Study:
NCT04135495
Study Brief:
Protocol Section:
NCT04135495