A new multicenter, randomized, double-blind, placebo-controlled phase 2 clinical trial aims to assess the safety and effectiveness of mitoxantrone hydrochloride liposome injection in patients with neuromyelitis optica spectrum disorder (NMOSD).
The study will include 45 patients aged between 18 and 60 years with an NMOSD diagnosis made as determined by the 2015 International Consensus Diagnostic Criteria for NMOSD. At the moment of inclusion, patients must have experienced a minimum of 2 attacks in the last 12 months or 3 in the last 12. All patients must have an Expanded Disability Status Scale below 7.5 points.
Exclusion criteria include: active infections, pregnancy, lactation, surgical procedures with 4 weeks of screening, and previous clinical trial participation in the last 3 months. Drug or alcohol abuse, evidence of an infectious disease such as tuberculosis, viral hepatitis, HIV infection, and syphilis are also a part of the trial’s exclusion criteria.
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No patients with a history of severe heart disease, autoimmune diseases requiring high glucocorticoid doses, severe drug allergies, or a history of malignancy can participate in the study.
Patients with NMOSD, who also have a recent history of bone marrow transplantation, B-cell depleting agents within 6 months of screening, or monoclonal antibody therapy will not participate in the study.
Participants will be divided into 2 experimental arms and 1 control arm. Patients in the experimental arms will receive either a 8 mg/m2 or 12 mg/m2 dose of mitoxantrone hydrochloride liposome injection once every 12 weeks, while patients in the control arm will receive a placebo injection once every 12 weeks.
The primary outcome measure of the study will be the time between randomization and the occurrence of relapse. Relapse will be defined as any sign or symptom of neurological deterioration attributable to NMOSD. The timeframe of the study will be 48 weeks.
The study is estimated to start on November 15, 2022, and the estimated primary completion date is October 15, 2024.
Efficacy and safety of mitoxantrone hydrochloride liposome injection in the treatment of neuromyelitis optica spectrum disorder (NMOSD). ClinicalTrials.gov. September 22, 2022. Accessed September 28, 2022.