At a glance
ClinicalIndex Comparison RecordStandardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 2 Open Label Pilot Study to Evaluate the Safety and Efficacy of Subcutaneously Administered ELX-02 in Patients With Alport Syndrome With Col4A5 and Col4A3/4 Nonsense Mutation
In Brief
A Phase 2 clinical trial evaluating ELX-02 for Alport Syndrome. Completed, enrolled 3 participants across 4 sites in 2 countries.
Detailed Summary
This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation. In total, up to 8 participants, with a minimum of 3 adults, will be enrolled in the trial. The study will be comprised of the following periods for each participant: * a Screening period of up to 6 weeks (42 days) * a total Treatment Period of 8 weeks (60 days) * a safety/efficacy Follow-up Period of 12 weeks (90 days) after the last treatment The Treatment Period will be a treatment of ELX-02 0.75 mg/kg SC QD for 8 weeks.
Study Details
Timeline
Interventions
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).