Study of Cytokines in Children With Opsoclonus-Myoclonus Syndrome

NCT00806182 | Completed

• 1 other identifier: Thrasher Award 02826-2
• Study Type: observational
• Target: 400
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Opsoclonus-myoclonus Syndrome

Keywords

paraneoplastic; neuroblastoma; Kinsbourne syndrome; autoimmune disease; immunotherapy; ataxia

Outcome Measures

Primary Outcomes (1)

• Reduction in inflammatory cytokines

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

  6 and 12 months

Secondary Outcomes (1)

• Correlation of cytokine concentration and clinical severity score.

  6 and 12 months

Study Arms (2)

Pediatric case-controls

These are children who underwent lumbar puncture and blood drawing for diagnostic testing for non-inflammatory neurological or non-neurological disorders, and whose samples were retrieved from the clinical lab under a linked Institutional Review Board (IRB) protocol.

Pediatric OMS

These are patients treated by the P.I. based on clinical decision making, not a clinical trial (this is an observational study). The types of treatments are varied, and, on the initial evaluation, the patients may be untreated or already tried on various immunotherapies. They range from monotherapy with steroids, ACTH, or IVIg, to disease modifying agents, such as rituximab, cyclophosphamide, and other chemotherapy, typically adjunctively or as combination therapy.

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Referrals to National Pediatric Myoclonus Center Website, www.omsusa.org

You may qualify if:

• Clinical diagnosis of OMS

You may not qualify if:

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

Sponsors & Collaborators

• National Pediatric Neuroinflammation Organization, Inc.: lead

Study Sites (1)

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

Springfield, Illinois, 62702, United States

Location

Related Publications (11)

• Pranzatelli MR, Tate ED, Hoefgen ER, Swan JA, Colliver JA. Therapeutic down-regulation of central and peripheral B-cell-activating factor (BAFF) production in pediatric opsoclonus-myoclonus syndrome. Cytokine. 2008 Oct;44(1):26-32. doi: 10.1016/j.cyto.2008.06.001. Epub 2008 Aug 9.

  PMID: 18675552 BACKGROUND

• Pranzatelli MR, Travelstead AL, Tate ED, Allison TJ, Moticka EJ, Franz DN, Nigro MA, Parke JT, Stumpf DA, Verhulst SJ. B- and T-cell markers in opsoclonus-myoclonus syndrome: immunophenotyping of CSF lymphocytes. Neurology. 2004 May 11;62(9):1526-32. doi: 10.1212/wnl.62.9.1526.

  PMID: 15136676 BACKGROUND

• Pranzatelli MR, Tate ED, McGee NR, Colliver JA. Cytokines, cytokine antagonists, and soluble adhesion molecules in pediatric OMS and other neuroinflammatory disorders. J Neurol Sci. 2013 Mar 15;326(1-2):53-8. doi: 10.1016/j.jns.2013.01.011. Epub 2013 Feb 4.

  PMID: 23380454 RESULT

• Pranzatelli MR, Tate ED, McGee NR, Travelstead AL, Verhulst SJ, Ransohoff RM. Expression of CXCR3 and its ligands CXCL9, -10 and -11 in paediatric opsoclonus-myoclonus syndrome. Clin Exp Immunol. 2013 Jun;172(3):427-36. doi: 10.1111/cei.12065.

  PMID: 23600831 RESULT

• Pranzatelli MR, Tate ED, McGee NR, Colliver JA. Pediatric reference ranges for proinflammatory and anti-inflammatory cytokines in cerebrospinal fluid and serum by multiplexed immunoassay. J Interferon Cytokine Res. 2013 Sep;33(9):523-8. doi: 10.1089/jir.2012.0132. Epub 2013 May 9.

  PMID: 23659672 RESULT

• Pranzatelli MR, Tate ED, McGee NR, Ransohoff RM. CCR7 signaling in pediatric opsoclonus-myoclonus: upregulated serum CCL21 expression is steroid-responsive. Cytokine. 2013 Oct;64(1):331-6. doi: 10.1016/j.cyto.2013.05.020. Epub 2013 Jun 10.

  PMID: 23764550 RESULT

• Pranzatelli MR, Tate ED, McGee NR, Verhulst SJ. CSF neurofilament light chain is elevated in OMS (decreasing with immunotherapy) and other pediatric neuroinflammatory disorders. J Neuroimmunol. 2014 Jan 15;266(1-2):75-81. doi: 10.1016/j.jneuroim.2013.11.004. Epub 2013 Nov 16.

  PMID: 24342231 RESULT

• Pranzatelli MR, Tate ED, Travelstead AL, Verhulst SJ. Chemokine/cytokine profiling after rituximab: reciprocal expression of BCA-1/CXCL13 and BAFF in childhood OMS. Cytokine. 2011 Mar;53(3):384-9. doi: 10.1016/j.cyto.2010.12.004. Epub 2011 Jan 5.

  PMID: 21211990 RESULT

• Pranzatelli MR, Tate ED, McGee NR, Colliver JA, Ransohoff RM. CCR4 agonists CCL22 and CCL17 are elevated in pediatric OMS sera: rapid and selective down-regulation of CCL22 by ACTH or corticosteroids. J Clin Immunol. 2013 May;33(4):817-25. doi: 10.1007/s10875-013-9867-4. Epub 2013 Jan 23.

  PMID: 23340773 RESULT

• Pranzatelli MR, Tate ED, McGee NR, Travelstead AL, Colliver JA, Ness JM, Ransohoff RM. BAFF/APRIL system in pediatric OMS: relation to severity, neuroinflammation, and immunotherapy. J Neuroinflammation. 2013 Jan 16;10:10. doi: 10.1186/1742-2094-10-10.

  PMID: 23324534 RESULT

• Pranzatelli MR, Tate ED, McGee NR, Travelstead AL, Ransohoff RM, Ness JM, Colliver JA. Key role of CXCL13/CXCR5 axis for cerebrospinal fluid B cell recruitment in pediatric OMS. J Neuroimmunol. 2012 Feb 29;243(1-2):81-8. doi: 10.1016/j.jneuroim.2011.12.014. Epub 2012 Jan 20.

  PMID: 22264765 RESULT

Biospecimen

Retention: SAMPLES WITHOUT DNA

serum, plasma, cerebrospinal fluid

MeSH Terms

Conditions

Opsoclonus-Myoclonus Syndrome; Neuroblastoma; Autoimmune Diseases; Ataxia

Condition Hierarchy (Ancestors)

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

Study Officials

• Michael R Pranzatelli, MD

  National Pediatric Neuroinflammation Organization, Inc.

  PRINCIPAL INVESTIGATOR

• Elizabeth D Tate, FNP, MN

  National Pediatric Neuroinflammation Organization, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director

Study Record Dates

• First Submitted: December 8, 2008
• First Posted: December 10, 2008
• Study Start: January 1, 2008
• Primary Completion: December 1, 2016
• Study Completion: December 1, 2016
• Last Updated: May 23, 2017

Record last verified: 2017-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)