Evaluating Safety and Effectiveness of NouvSoma001 Given by Spinal Injection for Neuromyelitis Optica Spectrum Disorders

What this Trial Is About

Researchers are evaluating the safety, tolerability, and effectiveness of NouvSoma001, a treatment made from human-induced neural stem cell-derived extracellular vesicles, for patients with neuromyelitis optica spectrum disorders NMOSD. This trial is a single-center, randomized, open-label, placebo-controlled, dose-escalation study designed to explore different dosing levels and extend treatment based on initial findings. The study aims to better understand how this treatment may help people with NMOSD, a condition diagnosed by specific criteria and associated with severe symptoms affecting movement, pain, and bladder or bowel function. The trial consists of two parts Part 1 involves escalating doses of NouvSoma001 given by intrathecal injection, starting with 5 109 particles and increasing to 3 1010 particles if no significant toxicities occur. Participants are grouped in cohorts of three to nine, with dose adjustments based on safety findings within 2 weeks after the first dose. Part 2 involves 60 participants randomized in a 21 ratio to receive either the selected dose of NouvSoma001 or a placebo by the same intrathecal injection method. Both groups receive their assigned treatment once in a single day. Participants will undergo various evaluations including monitoring for adverse events up to six months after treatment, imaging scans of the brain and spinal cord, and assessments of pain, disability, visual function, mental health, quality of life, and several biological markers in blood and cerebrospinal fluid. These measures will be compared to baseline and placebo groups to assess changes. Safety and tolerability will be closely followed, with additional assessments continuing up to 18 months. Overall, participation involves medical evaluations, imaging, laboratory tests, and questionnaires over a period extending beyond six months.

The Safety and Efficacy of NouvSoma001 in Neuromyelitis Optica Spectrum Disorders