Clinical trial NCT00304291: A Pilot Study of Mitoxantrone for the Treatment of Recurrent Neuromyelitis Optica (Devic's Disease)
Official title: A Pilot Study of Mitoxantrone for the Treatment of Recurrent Neuromyelitis Optica (Devic's Disease)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Neuromyelitis Optica
Myelitis, Transverse
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Keywords: Novantrone
Lead sponsor: State University of New York at Buffalo
Sponsor class: OTHER
Collaborators: name: EMD Serono
First posted: 2006-03-17
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Relapse rate
Secondary outcomes:
measure: Safety
Other outcomes:
Locations:
city: Buffalo
country: United States
facility: Baird Multiple Sclerosis Center
state: New York
Study officials:
affiliation: Baird Multiple Sclerosis Center
name: Bianca Weinstock-Guttman, M.D.
role: STUDY_DIRECTOR
References:
citation: Weinstock-Guttman B, Ramanathan M, Lincoff N, Napoli SQ, Sharma J, Feichter J, Bakshi R. Study of mitoxantrone for the treatment of recurrent neuromyelitis optica (Devic disease). Arch Neurol. 2006 Jul;63(7):957-63. doi: 10.1001/archneur.63.7.957.