Neuromyelitis OpticaPhase 4NCT00304291
A Pilot Study of Mitoxantrone for the Treatment of Recurrent Neuromyelitis Optica (Devic's Disease)
A Phase 4 study testing Mitoxantrone in people with Neuromyelitis Optica and Myelitis, Transverse, run by State University of New York at Buffalo at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Neuromyelitis Optica, Myelitis, Transverse, Demyelinating Autoimmune Diseases, CNS
- What is being tested?
- Mitoxantrone
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2001to May 2004
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Baird Multiple Sclerosis Center
Buffalo, New York, 14203
About this study
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.
Read the 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.
- Conditions
- Neuromyelitis Optica, Myelitis, Transverse, Demyelinating Autoimmune Diseases, CNS, Autoimmune Diseases of the Nervous System
- Being tested
- Mitoxantrone
- Keywords
- Novantrone
- Medical subject headings
- Neuromyelitis Optica, Myelitis, Transverse, Demyelinating Autoimmune Diseases, CNS, Autoimmune Diseases of the Nervous System
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MitoxantroneDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Relapse rate
Secondary outcomes (1)
Safety
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2006 (estimated)
- Study startAug 2001
- Study completionMay 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Neuromyelitis OpticaStudies of the same condition, open and under way.
- More trials run by State University of New York at BuffaloThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants
Sponsor and investigators
- Lead sponsor
- State University of New York at Buffalo
- Sponsor type
- University, hospital or other
- Collaborators
- EMD Serono
- Responsible party
- Principal investigator, Bianca Weinstock-Guttman, State University of New York at Buffalo
- Investigators
- Bianca Weinstock-Guttman, M.D., Study director, Baird Multiple Sclerosis Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00304291
- Sponsor's protocol ID
- JNI-NMO-101
- Status last verified
- Dec 2020
- First submitted
- 15 Mar 2006
- Last updated
- 28 Dec 2020
- Results posted
- No
Publications
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.
PubMed 16831964
Study at a glance
From the registry record, last updated by the sponsor on 28 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Mitoxantrone for the Treatment of Recurrent Neuromyelitis Optica (Devic's Disease)
- ClinicalTrials.gov ID
- NCT00304291
- Acronym / sponsor ID
- JNI-NMO-101
- Conditions
- Neuromyelitis Optica, Myelitis, Transverse, Demyelinating Autoimmune Diseases, CNS, Autoimmune Diseases of the Nervous System
- Keywords
- Novantrone
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants
- Lead sponsor
- State University of New York at BuffaloUniversity, hospital or other
- Collaborators
- EMD Serono
- Study start
- Aug 2001
- Study completion
- May 2004
- First posted
- 17 Mar 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mitoxantrone | Drug | – |
Primary outcome
Relapse rate
1 secondary outcome
Safety
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Dec 2020.
- Results publication: PubMed 16831964Weinstock-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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bianca Weinstock-Guttman, M.D. study director
Baird Multiple Sclerosis Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Mitoxantrone in people with Neuromyelitis Optica and Myelitis, Transverse, run by State University of New York at Buffalo at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Relapse rate. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00304291, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Dec 2020.