No Early Effect of Intrathecal Rituximab in Progressive Multiple Sclerosis (EFFRITE Clinical Trial)

Area of Science:

• Neurology
• Immunology
• Clinical Trials

Background:

• Progressive multiple sclerosis (MS) involves intrathecal inflammation resistant to standard treatments.
• Targeting central nervous system inflammation via direct injection is a potential therapeutic strategy.

Purpose of the Study:

• To evaluate the safety and efficacy of intrathecal (IT) rituximab in progressive MS.
• To assess changes in cerebrospinal fluid (CSF) biomarkers and clinical/MRI outcomes.

Main Methods:

• Phase II, single-center, randomized controlled trial (EFFRITE) of IT rituximab in progressive MS.
• Patients randomized to control, IT rituximab, or intravenous+IT rituximab arms.
• Primary outcome: change in CSF osteopontin; Secondary outcomes: CSF neurofilament light chain, clinical, and MRI changes.

Main Results:

• Ten patients included; no adverse events reported.
• CSF osteopontin levels remained stable; CSF neurofilament light chain decreased slightly (-8.7%).
• Clinical parameters and MRI findings showed no significant changes.

Conclusions:

• Intrathecal rituximab did not significantly alter inflammation biomarkers or clinical outcomes in progressive MS.
• Limited drug efficiency within the CSF may explain the observed results.
• Future study considerations for drug delivery are discussed.