Clinical trial NCT01868269: 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)
Official title: Multinational European Trial for Children With the Opsoclonus Myoclonus Syndrome / Dancing Eye Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Opsoclonus Myoclonus Syndrome
Neuroblastoma
Lead sponsor: Institut Curie
Sponsor class: OTHER
First posted: 2013-06-04
Enrollment: 102
Sex: ALL
Minimum age (years): 6
Maximum age (years): 8
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

•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)

Primary outcomes:
measure: The response to treatment schedule as defined by the percentage of patients with disappearance of all symptoms.
timeFrame: at 48 weeks after treatment start
Secondary outcomes:
Other outcomes:
Locations:
city: Wien
country: Austria
facility: St. Anna Kinderkrebsforschung e.V. CHILDREN'S CANCER RESEARCH INSTITUTE
city: Le Kremlin-Bicêtre
country: France
facility: Chu de Bicetre
state: LE Kremlin Bicetre
city: Lille
country: France
facility: Centre Oscar Lambret
state: Lille Cedex
city: Lyon
country: France
facility: Centre Leon Berard
state: LYON Cedex 08
city: Marseille
country: France
facility: Hopita D'Enfants de La Timone
state: Marseille Cedex 5
city: Montpellier
country: France
facility: Hopital Arnaud de Villeneuve
state: Montpellier Cedex 4
city: Nantes
country: France
facility: Chr de Nantes
state: Nantes Cedex01
city: Nice
country: France
facility: Chu de Nice Archet 2
state: NICE Cedex 03
city: Paris
country: France
facility: Ch Trousseau
state: Paris Cedex 12
city: Rennes
country: France
facility: Chu Hopital Sud
state: Rennes Cedex 02
city: Rouen
country: France
facility: Chu de Rouen
state: Rouen Cedex
city: Strasbourg
country: France
facility: CHU DE STRASBOURG HOPITAL Hautepierre
state: Strasbourg Cedex
city: Toulouse
country: France
facility: Chu Toulouse Hopital Des Enfants
state: Toulouse Cedex 9
city: Amiens
country: France
facility: Chu Amiens
city: Angers
country: France
facility: Chu Angers
city: Besancon
country: France
facility: Hopital Jean Minjoz
city: Bordeaux
country: France
facility: Chr Pellegrin
city: Caen
country: France
facility: CHU CAEN
city: Clermont Ferrand
country: France
facility: Chu D'Estaing
city: Dijon
country: France
facility: Chu Dijon
city: Grenoble
country: France
facility: Chu de Grenoble
city: Limoges
country: France
facility: Chu de Limoges
city: Paris
country: France
facility: Institut Curie
city: Poitiers
country: France
facility: Chu de Poitiers
city: Reims
country: France
facility: Chu de Reims
city: Saint-denis
country: France
facility: CHU LA REUNION Site Félix Guyon
city: Saint-Étienne
country: France
facility: Chu Saint Etienne
city: Tours
country: France
facility: Chu Tours Hopital Clocheville
city: Vandoeuvre Les Nancy
country: France
facility: Hopital Nancy Brabois
city: Villejuif
country: France
facility: Institut de Cancerologie Gustave Roussy
city: Genova
country: Italy
facility: G. Gaslini Institut
city: El Palmar
country: Spain
facility: The Fundación para la Formación e Investigación Sanitarias de la Región de Murcia
city: Lund
country: Sweden
facility: University Hospital Lund
city: Bern
country: Switzerland
facility: Universitätskinderklinik
city: Zurich
country: Switzerland
facility: Kinderspital Zurich
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
Study officials:
affiliation: Institut Curie
name: Gudrun Schleiermacher, MD
role: PRINCIPAL_INVESTIGATOR
References: