U.S., April 14 -- ClinicalTrials.gov registry received information related to the study (NCT07523581) titled 'EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety' on April 05.
Brief Summary: This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations in COL4A3/4/5 genes.
Targeted 24 patients aged 12 and older 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 exaluren 0.75 mg/kg or placebo administered daily subcutaneously for 32 weeks: Part 1: patients ar...