- Trial registry record
- Trial registry update
Elpida Therapeutics is testing ELP-02, a gene therapy that delivers a working copy of the FIG4 gene, in CMT4J. It is the first test in people: up to eight children and young adults aged 3 to 20 receive a single dose into the spinal fluid, and everyone gets the treatment.
Stanford in Palo Alto is recruiting; a University of Iowa site is not yet open. The study started in August 2026 and is expected to run to December 2032.
- Source date
- Source updated
- Registry status
- Recruiting
- Results posted to ClinicalTrials.gov
- No
- Genes
- FIG4
- Subtypes
- CMT4J
- Approved