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)

Neuroblastoma Clinical Trial

Official title:

Multinational European Trial for Children With the Opsoclonus Myoclonus Syndrome / Dancing Eye Syndrome

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT01868269
• Other study ID #: IC 2011-02
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: April 18, 2013
• Est. completion date: April 2031

Study information

• Verified date: January 2024
• Source: Institut Curie
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 102
• Est. completion date: April 2031
• Est. primary completion date: February 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 8 Years

Eligibility

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)

Related Conditions & MeSH terms

• Myoclonus
• Neuroblastoma
• Ocular Motility Disorders
• Opsoclonus Myoclonus Syndrome
• Opsoclonus-Myoclonus Syndrome
• Syndrome

Intervention

Drug:
• Dexamethasone acetate
First step: immunosuppressive treatment with dexamethasone
• dexamethasone and cyclophosphamide
second step (in case of insufficient response): immunosuppressive treatment with dexamethasone and cyclophosphamide
• dexamethasone and rituximab
third step (in case of insufficient response): immunosuppressive treatment with dexamethasone and rituximab

Locations

Country	Name	City	State
Austria	St. Anna Kinderkrebsforschung e.V. CHILDREN'S CANCER RESEARCH INSTITUTE	Wien
France	Chu Amiens	Amiens
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 Dijon	Dijon
France	Chu de Grenoble	Grenoble
France	Chu de Bicetre	Le Kremlin-Bicêtre	LE Kremlin Bicetre
France	Centre Oscar Lambret	Lille	Lille Cedex
France	Chu de Limoges	Limoges
France	Centre Leon Berard	Lyon	LYON Cedex 08
France	Hopita D'Enfants de La Timone	Marseille	Marseille Cedex 5
France	Hopital Arnaud de Villeneuve	Montpellier	Montpellier Cedex 4
France	Chr de Nantes	Nantes	Nantes Cedex01
France	Chu de Nice Archet 2	Nice	NICE Cedex 03
France	Ch Trousseau	Paris	Paris Cedex 12
France	Institut Curie	Paris
France	Chu de Poitiers	Poitiers
France	Chu de Reims	Reims
France	Chu Hopital Sud	Rennes	Rennes Cedex 02
France	Chu de Rouen	Rouen	Rouen Cedex
France	CHU LA REUNION Site Félix Guyon	Saint-denis
France	Chu Saint Etienne	Saint-Étienne
France	CHU DE STRASBOURG HOPITAL Hautepierre	Strasbourg	Strasbourg Cedex
France	Chu Toulouse Hopital Des Enfants	Toulouse	Toulouse Cedex 9
France	Chu Tours Hopital Clocheville	Tours
France	Hopital Nancy Brabois	Vandoeuvre Les Nancy
France	Institut de Cancerologie Gustave Roussy	Villejuif
Italy	G. Gaslini Institut	Genova
Spain	The Fundación para la Formación e Investigación Sanitarias de la Región de Murcia	El Palmar
Sweden	University Hospital Lund	Lund
Switzerland	Universitätskinderklinik	Bern
Switzerland	Kinderspital Zurich	Zurich
United Kingdom	John Radcliffe Hospital	Oxford

Sponsors (1)

Lead Sponsor	Collaborator
Institut Curie

Countries where clinical trial is conducted

Austria,  France,  Italy,  Spain,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The response to treatment schedule as defined by the percentage of patients with disappearance of all symptoms.		at 48 weeks after treatment start