Opsoclonus Myoclonus Syndrome/Dancing Eye Syndrome (OMS/DES) in Children With and Without Neuroblastoma (NBpos and NBneg)Opsoclonus Myoclonus Syndrome/Dancing Eye Syndrome (OMS/DES) in Children With and Without Neuroblastoma (NBpos and NBneg)

NCT01868269 | Completed Phase 3 DMC

• 1 other identifier: IC 2011-02
• Study Type: interventional
• Target: 102
• Locations: 7 countries
• Sites: 36

Brief Summary

The OMS/DES study is a multinational European Trial for Children with the Opsoclonus Myoclonus Syndrome / Dancing Eye Syndrome. This trial brought on the way by specialists of the EPNS (European Paediatric Neurology Society), the GPOH (Gesellschaft für Pädiatrische Hematologic und Oncologie) and the SIOPEN (SIOP (International Society Oncology Pediatric) Europe Neuroblastoma). This protocol will investigate an escalating treatment schedule starting with a corticosteroid standard treatment with dexamethasone pulses (first step), which is followed, if response has been inadequate after 3 months of treatment, by the addition of CP (second step) and, if still no sufficient improvement, by the replacement of CP by Rituximab (third step). Treatment intensification is decided on the basis of standardized scoring of OMS/DES severity.

Conditions

Opsoclonus Myoclonus Syndrome; Neuroblastoma

Outcome Measures

Primary Outcomes (1)

• The response to treatment schedule as defined by the percentage of patients with disappearance of all symptoms.

  at 48 weeks after treatment start

Study Arms (1)

Dexamethasone Cyclophosphamide Rituximab

OTHER

Drug: Dexamethasone acetate; Drug: dexamethasone and cyclophosphamide; Drug: dexamethasone and rituximab

Interventions

Dexamethasone acetate DRUG

First step: immunosuppressive treatment with dexamethasone

Dexamethasone Cyclophosphamide Rituximab

dexamethasone and cyclophosphamide DRUG

second step (in case of insufficient response): immunosuppressive treatment with dexamethasone and cyclophosphamide

Dexamethasone Cyclophosphamide Rituximab

dexamethasone and rituximab DRUG

third step (in case of insufficient response): immunosuppressive treatment with dexamethasone and rituximab

Dexamethasone Cyclophosphamide Rituximab

Eligibility Criteria

• Age: 6 Months - 8 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children with newly diagnosed OMS/DES either NB-pos or NB-neg.
• Three out of the following four components are necessary for the diagnosis of OMS/DES:
• Opsoclonus or ocular flutter (but not nystagmus)
• Ataxia and/or myoclonus
• Behavioural change and/or sleep disturbance
• Neuroblastoma The diagnosis of OMS/DES may be difficult in some patients. Opsoclonus, in particular, may be intermittent or late in onset. A video example will be available at www.dancingeyes.org.uk. If uncertain, please contact the national coordinator for support in interpreting clinical features.
• Age 6 months or over up to less than 8 years (\< 8th birthday) The date of diagnosis of OMS/DES is the date on which a doctor confirms the condition to be OMS/DES. The date of symptom onset needs also to be documented.
• Treatment start with the standard corticosteroid treatment with dexamethasone pulses as proposed by the guidelines given in this trial protocol (see 11.10, page 71).
• In patients with presumed NB-neg OMS/DES, neuroblastoma must be excluded according the guidelines of this trial (see chapter 4.4.1.4, page 30, and appendix 11.9, page 70)
• Documented informed consent for treatment and enrolment in the trial by parents / legal representatives.

You may not qualify if:

• Patients with opsoclonus, myoclonus or ataxia caused by other identified disease (e.g. current active CNS infection, neurometabolic disorder or demyelination).
• prior or parallel use of chemotherapy (other than required for treatment of the neuroblastoma)
• Corticoid steroid for OMS/DES or other reasons lasting 14 days or more immediately before treatment start according the standard treatment proposed (treatment with corticosteroids for less than 14 days will be allowed)
• contre-indication of use of one of the experimental study drug (cf Summary of Product Characteristics used in this study)

Sponsors & Collaborators

• Institut Curie: lead

Study Sites (36)

St. Anna Kinderkrebsforschung e.V. CHILDREN'S CANCER RESEARCH INSTITUTE

Vienna, 1090, Austria

Location

Chu de Bicetre

Le Kremlin-Bicêtre, LE Kremlin Bicetre, 94275, France

Location

Centre Oscar Lambret

Lille, Lille Cedex, 59020, France

Location

Centre Leon Berard

Lyon, LYON Cedex 08, 69373, France

Location

Hopita D'Enfants de La Timone

Marseille, Marseille Cedex 5, 13385, France

Location

Hopital Arnaud de Villeneuve

Montpellier, Montpellier Cedex 4, 34295, France

Location

Chr de Nantes

Nantes, Nantes Cedex01, 44093, France

Location

Chu de Nice Archet 2

Nice, NICE Cedex 03, 06202, France

Location

Ch Trousseau

Paris, Paris Cedex 12, 75571, France

Location

Chu Hopital Sud

Rennes, Rennes Cedex 02, 35056, France

Location

Chu de Rouen

Rouen, Rouen Cedex, 76031, France

Location

CHU DE STRASBOURG HOPITAL Hautepierre

Strasbourg, Strasbourg Cedex, 67098, France

Location

Chu Toulouse Hopital Des Enfants

Toulouse, Toulouse Cedex 9, 31059, France

Location

Chu Amiens

Amiens, 80054, France

Location

Chu Angers

Angers, 49933, France

Location

Hopital Jean Minjoz

Besançon, 25030, France

Location

Chr Pellegrin

Bordeaux, 33076, France

Location

CHU CAEN

Caen, 14033, France

Location

Chu D'Estaing

Clermont-Ferrand, 63003, France

Location

Chu Dijon

Dijon, 21079, France

Location

Chu de Grenoble

Grenoble, 38045, France

Location

Chu de Limoges

Limoges, 87042, France

Location

Institut Curie

Paris, 75005, France

Location

Chu de Poitiers

Poitiers, 86021, France

Location

Chu de Reims

Reims, 51092, France

Location

CHU LA REUNION Site Félix Guyon

Saint-Denis, 97400, France

Location

Chu Saint Etienne

Saint-Etienne, 42055, France

Location

Chu Tours Hopital Clocheville

Tours, 37044, France

Location

Hopital Nancy Brabois

Vandœuvre-lès-Nancy, 54500, France

Location

Institut de Cancerologie Gustave Roussy

Villejuif, 94805, France

Location

G. Gaslini Institut

Genova, 16148, Italy

Location

The Fundación para la Formación e Investigación Sanitarias de la Región de Murcia

El Palmar, 30120, Spain

Location

University Hospital Lund

Lund, 22185, Sweden

Location

Universitätskinderklinik

Bern, CH 3010, Switzerland

Location

Kinderspital Zurich

Zurich, 8032, Switzerland

Location

John Radcliffe Hospital

Oxford, 0X3 9DU, United Kingdom

Location

MeSH Terms

Conditions

Opsoclonus-Myoclonus Syndrome; Neuroblastoma

Interventions

dexamethasone acetate; Dexamethasone; Cyclophosphamide; Rituximab

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

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Gudrun Schleiermacher, MD

  Institut Curie

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 27, 2013
• First Posted: June 4, 2013
• Study Start: April 18, 2013
• Primary Completion: May 9, 2021
• Study Completion: January 17, 2025
• Last Updated: February 3, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: SAP
• Time Frame: Data requests can be submitted starting 9 months after last article publication and will be made accessible for up to 12 months.
• Access Criteria: Access to trial individual participant data can be requested by qualified researchers engaging in independent scientific research, and will be provided following review and approval of a research proposal and Statistical Analysis Plan (SAP) and execution of a data sharing agreement (DSA).

Locations

GB (1); FR (29); AU (1); IT (1); CH (2); ES (1); SE (1)