A Pilot Study of Mitoxantrone for the Treatment of Recurrent Neuromyelitis Optica (Devic's Disease)

Phase 4

Completed

• Conditions: Demyelinating Autoimmune Diseases, CNS; Neuromyelitis Optica; Autoimmune Diseases of the Nervous System; Myelitis, Transverse

• Registration Number: NCT00304291
• Lead Sponsor: State University of New York at Buffalo

Brief Summary

Neuromyelitis optica (NMO) is a severe demyelinating disease that selectively involves the optic nerves and the spinal cord but usually spares the brain. NMO is considered to have a B cell induced pathogenesis. Mitoxantrone (MITO, Novantrone®), a synthetic anthracenedione approved for worsening relapsing-remitting multiple sclerosis (MS) and secondary progressive MS, has been shown to primarily suppress the humoral response. We conducted a prospective 2-year study to evaluate the benefit of MITO in five relapsing NMO patients.

Detailed Description

The treatment protocol consisted of monthly 12 mg/m2 MITO intravenous infusions for 6 months followed by 3 additional treatments every 3 months. Neurological assessment including the determination of the Expanded Disability Status Scale (EDSS) score was performed every 3 months and during relapses. Orbital, brain and spinal cord MRI was performed at baseline, 3, 6, 12, 18, and 24 months. Visual evoked potentials and ophthalmologic evaluations were performed at baseline and annually.

Study Timeline

• Study Start: 2001-08
• Study Completion: 2004-05
• Study First Submitted: 2006-03-15
• Study First Submitted QC: 2006-03-15
• Study First Posted: 2006-03-17
• Status Verified: 2020-12
• Last Update Submitted: 2020-12-23
• Last Update Posted: 2020-12-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• Recurrent longitudinal myelitis (>3 segments of spinal cord involvement by MRI) with or without recurrent ON (unilateral or bilateral) but with normal brain MRI.
• Patients with recurrent longitudinally extensive myelitis without optic neuritis have an underlying pathology and serology similar to NMO and it is appropriate to consider this a form of NMO10.
• Cerebrospinal fluid required no intrathecal IgG synthesis or oligoclonal bands.
• Age was required to be 18-55 years
• Extended Disability Status Score ≤ 7.

Exclusion Criteria

• Cardiac risk factors (e.g history of congestive heart failure and left ventricular ejection fraction (LVEF) < 50%
• Systemic diseases such as lupus, Sjogren's syndrome, anti-phospholipid antibody syndrome, sarcoidosis, rheumatoid arthritis, or vitamin B12 deficiency
• Previous treatment with mitoxantrone or anthracyclines

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Relapse rate

Secondary Outcome Measures

Name	Time	Method
Safety

Trial Locations

Locations (1)

Baird Multiple Sclerosis Center; 🇺🇸 Buffalo, New York, United States