U.S., Dec. 9 -- ClinicalTrials.gov registry received information related to the study (NCT07269665) titled 'First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration' on Sept. 16.
Brief Summary: The goal of this first in human study is to evaluate the preliminary safety and tolerability of AXV-101 in participants with BBS1. The main questions it aims to answer are:
* Is AXV-101 safe and tolerable to use in participants with BBS1?
* To determine the therapeutic dose of AXV-101 in participants with BBS1
* To investigate the concentration of AXV-101 in blood, urine and tears (both eyes)
Participants will undergo comprehensive ophthalmic assessments to evaluate functional and structural changes from baseline to ...