The Phase 1 study evaluated CLZ-2002, now being developed by Liferna as LFR-1501. The study was conducted in South Korea and sponsored by Cellatoz Therapeutics.
No treatment-related adverse reactions, serious adverse events or dose-limiting toxicities were reported.
Improvements from baseline were observed in CMTNSv2 and ONLS-leg at Week 24, providing an encouraging exploratory efficacy signal.
The exploratory efficacy assessments suggested possible clinical benefit and support further evaluation in a larger, controlled study.
Interpretation: A single intramuscular dose of LFR-1501 was safe and well tolerated in participants with CMT1. Exploratory efficacy assessments suggested possible clinical benefit and support continued evaluation in larger, controlled clinical studies.
Study resources
View on ClinicalTrials.gov
NCT05947578
Watch the study-results presentation
Video presentation of key findings
Read the peer-reviewed publication
Full study report
LFR-1501 is investigational and has not been approved by the U.S. Food and Drug Administration or other regulatory authorities.
