A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele
Status:
Recruiting
Trial end date:
2022-06-15
Target enrollment:
Participant gender:
Summary
This is a Phase 2 open label 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.
Up to 16 patients will be enrolled in the trial; up 4 patients will be homozygotes to G542X,
and the remaining patients will be compound heterozygotes with G542X and 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 upon the patients
observed safety and tolerability, PK at previous doses and the results of laboratory
tests