A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis
- Registration Number
- NCT04069260
- Lead Sponsor
- Eloxx Pharmaceuticals, Inc.
- Brief 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)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 3
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ELX-02 ELX-02 Eukaryotic ribosomal selective glycoside (ERSG)
- Primary Outcome Measures
Name Time Method Maximum observed plasma concentration (Cmax) Day 1 of treatment periods 1, 2, and 3 Full PK profile 12 blood samples over 24 hours
AEs associated with different dose levels of ELX-02 From the time of first dosing through the follow-up visit, an average of approximately 10 weeks Observed plasma concentration at 1 hour post dose (C1h) Days 1, 2, 5, and 7 of treatment periods 1-2; Days 1, 2, 5, 7, 10, and 14 of treatment period 3 Sparse sampling, blood sampling only, pre-dose and 1 hour post dose
Amount of ELX-02 excreted in urine from 0 to 24 hours (Ae24h) Day 1 of treatment periods 1, 2, and 3 6 urine collections over 24 hours
Renal clearance on Day 1 (Ae24h/plasma AUC0-24h) Day 1 of treatment periods 1, 2, and 3 6 urine collections over 24 hours
Area under the plasma concentration curve from time zero to 24 hours (AUC0-24h) Day 1 of treatment periods 1, 2, and 3 Full PK profile 12 blood samples over 24 hours
- Secondary Outcome Measures
Name Time Method Changes from baseline in WBC cystine levels Screening; Days 1, 2, 5, and 7 of treatment periods 1-2; Days 1, 2, 5, 7, 10, and 14 of treatment period 3, and 4-week safety follow-up
Trial Locations
- Locations (1)
McGill University Health Center
🇨🇦Montréal, Quebec, Canada