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Clinical Trial 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)


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT04069260
Study type Interventional
Source Eloxx Pharmaceuticals, Inc.
Contact
Status Terminated
Phase Phase 2
Start date August 2, 2019
Completion date December 17, 2019

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