A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis
Status:
Terminated
Trial end date:
2019-12-17
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 cystinosis with nonsense
mutation in at least one allele.
Six patients will be enrolled in the trial.
The study will comprise of the following periods for each patient:
- A screening period of up to 6 weeks
- A total treatment period of 4 weeks
- A safety follow-up period of 4 weeks after the last treatment
Each patient will receive three escalating doses as follows:
- Treatment period 1: ELX-02 0.5 mg/kg SC daily for 7 days (total dose not to exceed 3.5
mg/kg for this week; the daily dose will be individualized to achieve the target weekly
exposure of about 47.5 µg*h/mL)
- Treatment period 2: ELX-02 1.0 mg/kg SC daily for 7 days (total dose not to exceed 7.0
mg/kg for this week; the daily dose will be individualized to achieve the target weekly
exposure of about 95 µg*h/mL)
- Treatment period 3: ELX-02 2.0 mg/kg SC daily for 14 days (total dose not to exceed 14
mg/kg for these two weeks; the daily dose will be individualized to achieve the target
weekly exposure of about 190 µg*h/mL)