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)
- Conditions
- NeuroblastomaOpsoclonus Myoclonus Syndrome
- Interventions
- Registration Number
- NCT01868269
- Lead Sponsor
- Institut Curie
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 102
- 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.
•Patients with opsoclonus, myoclonus or ataxia caused by other identified disease (e.g. current active CNS infection, neurometabolic disorder or demyelination).
An identified viral precursor is not an exclusion criterion.
- 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)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Dexamethasone Cyclophosphamide Rituximab dexamethasone and cyclophosphamide - Dexamethasone Cyclophosphamide Rituximab dexamethasone and rituximab - Dexamethasone Cyclophosphamide Rituximab Dexamethasone acetate -
- Primary Outcome Measures
Name Time Method The response to treatment schedule as defined by the percentage of patients with disappearance of all symptoms. at 48 weeks after treatment start
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (36)
Chu Amiens
🇫🇷Amiens, France
St. Anna Kinderkrebsforschung e.V. CHILDREN'S CANCER RESEARCH INSTITUTE
🇦🇹Wien, Austria
Chu de Bicetre
🇫🇷Le Kremlin-Bicêtre, LE Kremlin Bicetre, France
Centre Oscar Lambret
🇫🇷Lille, Lille Cedex, France
Hopita D'Enfants de La Timone
🇫🇷Marseille, Marseille Cedex 5, France
Centre Leon Berard
🇫🇷Lyon, LYON Cedex 08, France
Chr de Nantes
🇫🇷Nantes, Nantes Cedex01, France
Hopital Arnaud de Villeneuve
🇫🇷Montpellier, Montpellier Cedex 4, France
Chu de Nice Archet 2
🇫🇷Nice, NICE Cedex 03, France
Ch Trousseau
🇫🇷Paris, Paris Cedex 12, France
Chu de Rouen
🇫🇷Rouen, Rouen Cedex, France
Chu Toulouse Hopital Des Enfants
🇫🇷Toulouse, Toulouse Cedex 9, France
CHU DE STRASBOURG HOPITAL Hautepierre
🇫🇷Strasbourg, Strasbourg Cedex, France
Chu Angers
🇫🇷Angers, France
Hopital Jean Minjoz
🇫🇷Besancon, France
Chr Pellegrin
🇫🇷Bordeaux, France
CHU CAEN
🇫🇷Caen, France
Chu D'Estaing
🇫🇷Clermont Ferrand, France
Chu de Limoges
🇫🇷Limoges, France
Chu Dijon
🇫🇷Dijon, France
Chu de Grenoble
🇫🇷Grenoble, France
Institut Curie
🇫🇷Paris, France
CHU LA REUNION Site Félix Guyon
🇫🇷Saint-denis, France
Chu Saint Etienne
🇫🇷Saint-Étienne, France
Chu Tours Hopital Clocheville
🇫🇷Tours, France
Hopital Nancy Brabois
🇫🇷Vandoeuvre Les Nancy, France
G. Gaslini Institut
🇮🇹Genova, Italy
Institut de Cancerologie Gustave Roussy
🇫🇷Villejuif, France
The Fundación para la Formación e Investigación Sanitarias de la Región de Murcia
🇪🇸El Palmar, Spain
Universitätskinderklinik
🇨🇭Bern, Switzerland
University Hospital Lund
🇸🇪Lund, Sweden
Kinderspital Zurich
🇨🇭Zurich, Switzerland
John Radcliffe Hospital
🇬🇧Oxford, United Kingdom
Chu de Reims
🇫🇷Reims, France
Chu Hopital Sud
🇫🇷Rennes, Rennes Cedex 02, France
Chu de Poitiers
🇫🇷Poitiers, France