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

Sponsor

State University of New York at Buffalo (Other)

Overall Status

Completed

CT.gov ID

NCT00304291

Collaborator

EMD Serono (Industry)

Enrollment

Location

Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Neuromyelitis Optica Myelitis, Transverse Demyelinating Autoimmune Diseases, CNS Autoimmune Diseases of the Nervous System	Drug: Mitoxantrone	Phase 4

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 Design

Study Type:

Interventional

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Aug 1, 2001

Study Completion Date :

May 1, 2004

Outcome Measures

Primary Outcome Measures

Relapse rate []

Secondary Outcome Measures

Safety []

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 55 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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