NCT04069260 - A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

August 2, 2019

Primary Completion Date

December 17, 2019

Study Completion Date

December 17, 2019

Conditions

Genetic DiseaseNonsense MutationCystinosis

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).

Trial Locations (1)

H3H 2R9

McGill University Health Center, Montreal