A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele
Status:
Recruiting
Trial end date:
2021-06-15
Target enrollment:
Participant gender:
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 in patients with CF with at least
one G542X allele.
In total, up to eight patients will be enrolled in the trial; up to 4 patients will be
homozygotes to G542X, and the remaining patients will be compound heterozygotes with G542X
and with any Class 1 or Class 2 mutation, excluding F508del.
Each patient will receive 4 escalating doses as follows:
- 0.3 mg/kg per day SC
- 0.75 mg/kg per day SC
- 1.5 mg/kg per day SC
- An individualized dose, 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.