A Study of ELX-02 in Patients With Alport Syndrome
Status:
Not yet recruiting
Trial end date:
2023-05-30
Target enrollment:
Participant gender:
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.