NCT04069260

A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis

A Phase 2, Single Center, Open-Label, Multiple Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Daily Subcutaneously Administered ELX-02 in Patients With Nephropathic Cystinosis Bearing One or More CTNS Gene (Cystinosin) Nonsense Mutations

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Eloxx Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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)

Conditions

• Genetic Disease
• Nonsense Mutation
• Cystinosis

Interventions

Type	Name	Description
DRUG	ELX-02	ELX-02 is a small molecule, new chemical entity being developed for the treatment of genetic diseases caused by nonsense mutations. ELX-02 is a eukaryotic ribosomal selective glycoside (ERSG).

Timeline

• Start date: 2019-08-02
• Primary completion: 2019-12-17
• Completion: 2019-12-17
• First posted: 2019-08-28
• Last updated: 2020-05-27

Locations

1 site across 1 country: Canada

Regulatory

• FDA-regulated drug study