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

NCT04069260 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2020-05-27

No results posted yet for this study

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

Interventions

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).

Sponsors & Collaborators

  • Eloxx Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Paul Goodyer, MD · McGill University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-08-02
Primary Completion
2019-12-17
Completion
2019-12-17
FDA Drug
Yes

Countries

  • Canada

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04069260 on ClinicalTrials.gov