Study of Cytokines in Children With Opsoclonus-Myoclonus Syndrome

The purpose of this study is to determine if cytokines, inflammatory mediators, are increased in spinal fluid and blood, correlate with disease activity, and could serve as biomarkers or therapeutic targets in children with opsoclonus-myoclonus syndrome (OMS), an autoimmune complication of the tumor neuroblastoma.

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Key information

Conditions

Opsoclonus-myoclonus Syndrome Ataxia Autoimmune Diseases Central Nervous System Diseases Cranial Nerve Diseases Dyskinesias Eye Diseases Immune System Diseases Myoclonus Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Nervous System Diseases Nervous System Neoplasms Neuroblastoma Neurodegenerative Diseases Neuroectodermal Tumors Neuroectodermal Tumors, Primitive Neuroectodermal Tumors, Primitive, Peripheral Neurologic Manifestations Ocular Motility Disorders Paraneoplastic Syndromes Paraneoplastic Syndromes, Nervous System Pathological Conditions, Signs and Symptoms Signs and Symptoms

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

National Pediatric Myoclonus Center, Formerly at Dept. of Neurology, Southern Illinois University School of Medicine

Springfield, Illinois, 62702, United States

About this study

In this translational research, immunological mechanisms that underlie the assault of the immune system on the brain in paraneoplastic opsoclonus-myoclonus syndrome (OMS) are under evaluation. To test our principal hypothesis that there is an imbalance of pro-inflammatory (Th1) and anti-inflammatory (Th2) cytokines in OMS, a comprehensive cytokine panel will be measured by enzyme-linked immunosorbent assay (ELISA) and multiplexed fluorescent bead-based immunoassay detection (LUMINEX 100 Lab MAP system)in blood and cerebrospinal fluid (CSF) of 400 children. To test the second hypothesis that cytokines could serve as biomarkers of disease activity in OMS, cytokine concentrations will be correlated with clinical variables, such as disease severity, OMS duration, prior relapses, and remissions, as well as immunological variables, such as lymphocyte subset analysis. The cytokine 'biomarker profile' could aid decision making for early intervention by identifying children at high risk for relapse and poor outcome and allow targeting of the most implicated inflammatory cytokines by cytokine therapies. To test our third hypothesis that lack of response to immunotherapy is due in part to failure to increase the expression of anti-inflammatory Th2 cytokines, we will make paired pre/post comparisons of the impact of immunotherapies given in the course of clinical care [steroids, adrenocorticotropin (ACTH), intravenous immunoglobulins (IVIg), rituximab, chemotherapy, other drugs, combinations] on the cytokine and clinical profile. This research could lead to the application of commercially-available cytokines and cytokine blockers or to the development of new ones for OMS.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Clinical diagnosis of OMS

Exclusion criteria

Equivocal diagnosis
Contraindications to lumbar puncture
Treatment with agents outside the scope of the study

Treatment and study plan

Primary outcomes

Reduction in inflammatory cytokines

Time frame: 6 and 12 months

Reduction in the concentration of inflammatory chemokines/cytokines between clinical time points

Secondary outcomes

Correlation of cytokine concentration and clinical severity score.

Time frame: 6 and 12 months

Statistical correlation of chemokine/cytokine concentration with OMS motor severity as measured using the OMS video evaluation scale

Sponsors and collaborators

Lead sponsor

National Pediatric Neuroinflammation Organization, Inc.

Other

Registry information

Official study title

Cytokines as Biomarkers and Therapeutic Targets in Paraneoplastic Opsoclonus-Myoclonus Syndrome (OMS)

Acronym: OMS

Important dates

Study start: 2008
Primary completion: 2016
Study completion: 2016
First posted: Dec 10, 2008
Registry last updated: May 23, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed