U.S., Nov. 20 -- ClinicalTrials.gov registry received information related to the study (NCT07236606) titled 'RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)' on Nov. 05.
Brief Summary: RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety, efficacy, and pharmacodynamic dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II (Hunter Syndrome)
Study Start Date: Nov. 19
Study Type: INTERVENTIONAL
Condition:
MPS II
Hunter Syndrome (MPS II)
Intervention:
GENETIC: RGX-121-3102
Recombinant adeno-associated virus serotype 9 [AAV9] capsid containing huma...