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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00304291
Recruitment Status : Completed
First Posted : March 17, 2006
Last Update Posted : November 28, 2006
EMD Serono
Information provided by:
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.

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 Type : Interventional  (Clinical Trial)
Enrollment : 5 participants
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 : August 2001
Study Completion Date : May 2004

Primary Outcome Measures :
  1. Relapse rate

Secondary Outcome Measures :
  1. Safety

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00304291

United States, New York
Baird Multiple Sclerosis Center
Buffalo, New York, United States, 14203
Sponsors and Collaborators
State University of New York at Buffalo
EMD Serono
Study Director: Bianca Weinstock-Guttman, M.D. Baird Multiple Sclerosis Center

Publications of Results: Identifier: NCT00304291     History of Changes
Other Study ID Numbers: JNI-NMO-101
First Posted: March 17, 2006    Key Record Dates
Last Update Posted: November 28, 2006
Last Verified: March 2006

Keywords provided by State University of New York at Buffalo:

Additional relevant MeSH terms:
Autoimmune Diseases
Neuromyelitis Optica
Nervous System Diseases
Autoimmune Diseases of the Nervous System
Demyelinating Autoimmune Diseases, CNS
Myelitis, Transverse
Immune System Diseases
Optic Neuritis
Optic Nerve Diseases
Cranial Nerve Diseases
Demyelinating Diseases
Eye Diseases
Central Nervous System Infections
Central Nervous System Diseases
Spinal Cord Diseases
Brain Diseases
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Paraneoplastic Syndromes
Neurodegenerative Diseases
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Antineoplastic Agents