Description
Seeking men and women with Adrenomyeloneuropathy for a gene therapy study to try and correct gene defects so that nerve cells can create proteins needed to prevent damage to spinal cord cells and nerves.
Overview
Participants in this study will be randomly assigned to receive either the gene therapy or a sham procedure (similar in everyway to the gene therapy, but no medication will be given). One in four participants (25%) will receive the sham procedure. After 24 months the participants who received the sham procedure will be offered the opportunity to get the investigational gene therapy.
What we're hoping for
We are studying whether the gene therapy is safe and effective in correcting gene defects so that nerve cells can create proteins needed to prevent damage to spinal cord cells and nerves.
Additional Information
ClinicalTrials.gov Identifier: NCT05394064