Immune System Diseases

All Conditions

Symptoms and General Pathology

Nervous System Diseases

Eye Diseases

Infections

Neoplasms

Rare Diseases

Autoimmune Diseases

Recurrence

Autoimmune Diseases of the Nervous System

Neuromyelitis Optica

Myelitis

Myelitis, Transverse

Demyelinating Autoimmune Diseases, CNS

Neuritis

Optic Neuritis

Optic Nerve Diseases

Cranial Nerve Diseases

Demyelinating Diseases

Communicable Diseases

Central Nervous System Infections

Central Nervous System Diseases

Spinal Cord Diseases

Neuroinflammatory Diseases

Leukoencephalopathies

Brain Diseases

Syndrome

Paraneoplastic Syndromes

Paraneoplastic Syndromes, Nervous System

Nervous System Neoplasms

Neurodegenerative Diseases

Neuromyelitis Optica Spectrum Disorder

Antineoplastic Agents

Analgesics

All Drugs and Chemicals

Mitoxantrone

Enzyme Inhibitors

Bianca Weinstock-Guttman, M.D.

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.

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.

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

EMD Serono

State University of New York at Buffalo

Principal Investigator

Transverse Myelitis

Anti-Inflammatory Agents

Antiemetics

Neuroprotective Agents

Gastrointestinal Agents

Antibodies

Prednisolone

Immunoglobulins

Methylprednisolone

Methylprednisolone Acetate

Methylprednisolone Hemisuccinate

Prednisolone acetate

Prednisolone hemisuccinate

Prednisolone phosphate

Immunoglobulins, Intravenous

gamma-Globulins

Rho(D) Immune Globulin

Glucocorticoids

Hormones

Hormone Antagonists

Protective Agents

Immunologic Factors

Paediatric patients - 30mg/kg or 500mg/m2 up to a maximum daily dose of 1g/day for 5 days.

Adult patients - 1g/day for 5 days.

Paediatric patients \<41.2kg - total dose of 2g/kg in divided doses over 2 days.

All other patients - total dose of 2g/kg in divided doses over 5 days.

PLUS Intravenous Methylprednisolone

Pediatric patients - 30mg/kg or 500mg/m2 up to a maximum daily dose of 1g/day for 5 days.

Adult patients - 1g/day for 5 days.

Intravenous Methylprednisolone

Intravenous Immunoglobulin

Ming Lim, MB, PhD

This multi-center randomized controlled trial evaluates if the addition of intravenous immunoglobulin to standard treatment of corticosteroids improves outcome in children and adults with first episode of Transverse Myelitis of Neuro-myelitis optica. Half of participants will receive corticosteroids alone, whilst the other half will receive corticosteroids plus intravenous immunoglobulin.

Transverse myelitis (TM) is a severe demyelinating condition predominantly affecting young people, which causes significant long-term disability in approximately one third. Current initial treatment is with corticosteroids, although evidence for their use is based on extrapolation from trials in adult multiple sclerosis relapses. In view of the severity of the condition, additional treatments have been trialed.

Intravenous immunoglobulin (IVIG) is often used as second-line treatment in steroid-unresponsive central nervous system demyelination, although evidence for its efficacy is limited to small case series and case reports. Randomized controlled trials have demonstrated that IVIG reduces inflammation and enhances remyelination in a number of neurological conditions, although there have been no randomized controlled trials testing its use in adults and children with TM.

This study will evaluate if additional and early treatment with IVIG is of extra benefit in TM when compared to the current standard therapy of intravenous steroids alone.

A Multicentre randomiSed Controlled TRial of IntraVEnous Immunoglobulin Versus Standard Therapy for Transverse Myelitis

A Multicentre randomiSed Controlled TRial of IntraVEnous Immunoglobulin (IVIg) Versus Standard Therapy for the Treatment of Transverse Myelitis in Adults and Children

King's College London

Barts and the London School of Medicine and Dentistry

Cardiff University

University College, London

King's College Hospital NHS Trust

Great Ormond Street Hospital for Children NHS Foundation Trust

Barts & The London NHS Trust

Alder Hey Children's NHS Foundation Trust

Walton Centre NHS Foundation Trust

Oxford University Hospitals NHS Trust

Birmingham Women's and Children's NHS Foundation Trust

University Hospital Birmingham NHS Foundation Trust

Cardiff and Vale University Health Board

North Bristol NHS Trust

University Hospitals Bristol and Weston NHS Foundation Trust

Manchester University NHS Foundation Trust

Northern Care Alliance NHS Foundation Trust

University Hospital Southampton NHS Foundation Trust

Newcastle-upon-Tyne Hospitals NHS Trust

Nottingham University Hospitals NHS Trust

NHS Lothian

Guy's and St Thomas' NHS Foundation Trust

Paraplegia

Paralysis

Neurologic Manifestations

Acute Graft Versus Host Disease

Observational study with online survey participation highlighting outcomes recovery. The surveys can be completed by the child and parent, or if too young to participate, parent only. The survey asks how the child is doing after hospitalization within 6 months of diagnosis, and every 4 months until study end in 2024.

Benjamin Greenberg, MD

Patients and families are invited to participate in an online registry and data repository specifically for patients with transverse myelitis (TM) or acute flaccid myelitis (AFM). The data generated in this study will come from surveys, interviews, review of medical records.

Data from this study will be utilized to guide future clinical trials for children with an acute case of TM or AFM. Parents and school aged children will complete an online survey 7 banks of questions. Each bundle of survey topics have 7-10 questions. We will have both the parent and child complete a outcomes based survey within 6 months of diagnosis and invite to participate every 4 months until study end in 2024.

Both parent and child will participate in the online questionnaires. The validated questionnaires, PROMIS or Patient-Reported Outcomes Measurement Information System, are blocks of 8-10 questions. 7 blocks of questions for the parent and 7 similar blocks for the child: anxiety, depressive symptoms, fatigue, mobility, pain interference, peer relationships, upper extremity function.

Participation via the Internet: When a child or family enters the Transverse Myelitis Association (TMA) website, they will be directed to information about the CAPTURE study. If the family is interested in participation, the TMA will give them the UT southwestern in Dallas, TX research coordinator information. It is up to the parent/guardian to contact the study staff from University Texas Southwestern. Once initial contact is complete, at the family's request, study staff will send the consent form to the interested family. The family will need to email or mail the consent back. Once the signed consent form is in hand, we will send the research survey to the email the parent provides. One survey for the parent, a separate but similar survey for a school aged child.

We will invite parents of school aged children to complete a school survey. This is a one time only questionnaire. Ideally, the parents will complete this either 6 months or 1 year into their child's recovery of TM/AFM.

We invite families living outside of North America to participate in the online survey. They need to be fluent in English, same as the North American cohort.

Collaborative Assessment of Pediatric Transverse Myelitis: Understand, Reveal, Educate or CAPTURE Study

each parent /guardian and child will fill out a questionnaire examining how you feel about your ability to move and your emotions. This is completed at 3,6,and 12 months after symptoms are diagnosed.

We will measure the motor (movement) and sensory (touch) of the child above and below the spinal lesion. This is completed at 3 time points 3,6,and 12 months post symptom onset. For the secondary outcomes, the family needs to be able to travel to 1 of the 5 centers listed in the study.

Patient-Centered Outcomes Research Institute

University of Texas Southwestern Medical Center

Professor of Medicine

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Pilot Study of Mitoxantrone for the Treatment of Recurrent Neuromyelitis Optica (Devic's Disease)	NCT00304291	Neuromyelitis O... Myelitis, Trans... Demyelinating A... Autoimmune Dise...	Mitoxantrone	18 Years - 55 Years	State University of New York at Buffalo
A Multicentre randomiSed Controlled TRial of IntraVEnous Immunoglobulin Versus Standard Therapy for Transverse Myelitis	NCT02398994	Myelitis, Trans... Neuromyelitis O...	Intravenous Met... Intravenous Imm...	1 Year -	Guy's and St Thomas' NHS Foundation Trust
Collaborative Assessment of Pediatric Transverse Myelitis: Understand, Reveal, Educate or CAPTURE Study	NCT02144935	Myelitis, Trans... Flaccid Paraple... Flaccid Paraple...		- 17 Years	University of Texas Southwestern Medical Center
A Multicentre randomiSed Controlled TRial of IntraVEnous Immunoglobulin Versus Standard Therapy for Transverse Myelitis	NCT02398994	Myelitis, Trans... Neuromyelitis O...	Intravenous Met... Intravenous Imm...	1 Year -	Guy's and St Thomas' NHS Foundation Trust
Collaborative Assessment of Pediatric Transverse Myelitis: Understand, Reveal, Educate or CAPTURE Study	NCT02144935	Myelitis, Trans... Flaccid Paraple... Flaccid Paraple...		- 17 Years	University of Texas Southwestern Medical Center

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