NCT04135495

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

A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients With Cystic Fibrosis With at Least One G542X Allele

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Eloxx Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

• Cystic Fibrosis

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 eukaroyotic ribosomal selective glycoside (ERSG)
DRUG	Ivacaftor	CFTR potentiator

Timeline

• Start date: 2019-11-25
• Primary completion: 2022-10-03
• Completion: 2022-10-03
• First posted: 2019-10-22
• Last updated: 2022-11-22

Locations

10 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study