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A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele

NCT04135495 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 17

Last updated 2022-11-22

No results posted yet for this study

Summary

This is a Phase 2 open-label, dose-escalation study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele.

In total, up to 16 patients will be enrolled in the trial; up to 4 patients will be homozygotes for G542X, and the remaining patients will be compound heterozygotes with one G542X or phenotypically similar nonsense allele and any Class 1 or Class 2 mutation.

Each patient will receive up to 5 escalating doses as follows:

* ELX-02 0.3 mg/kg per day SC
* ELX-02 0.75 mg/kg per day SC
* ELX-02 1.5 mg/kg per day SC
* An individualized dose of ELX-02, as high as 3.0 mg/kg per day SC, based on the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests.
* ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid

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 eukaroyotic ribosomal selective glycoside (ERSG)

DRUG

Ivacaftor

CFTR potentiator

Sponsors & Collaborators

  • Eloxx Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-11-25
Primary Completion
2022-10-03
Completion
2022-10-03
FDA Drug
Yes

Countries

  • United States
  • 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 NCT04135495 on ClinicalTrials.gov