Back to resultsOpsoclonus 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)
Primary Purpose
Opsoclonus Myoclonus Syndrome, Neuroblastoma
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Dexamethasone acetate
dexamethasone and cyclophosphamide
dexamethasone and rituximab
Sponsored by
About this trial
This is an interventional treatment trial for Opsoclonus Myoclonus Syndrome
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)
Sites / Locations
- St. Anna Kinderkrebsforschung e.V. CHILDREN'S CANCER RESEARCH INSTITUTE
- Chu de Bicetre
- Centre Oscar Lambret
- Centre Leon Berard
- Hopita D'Enfants de La Timone
- Hopital Arnaud de Villeneuve
- Chr de Nantes
- Chu de Nice Archet 2
- Ch Trousseau
- Chu Hopital Sud
- Chu de Rouen
- CHU DE STRASBOURG HOPITAL Hautepierre
- Chu Toulouse Hopital Des Enfants
- Chu Amiens
- Chu Angers
- Hopital Jean Minjoz
- Chr Pellegrin
- CHU CAEN
- Chu D'Estaing
- Chu Dijon
- Chu de Grenoble
- Chu de Limoges
- Institut Curie
- Chu de Poitiers
- Chu de Reims
- CHU LA REUNION Site Félix Guyon
- Chu Saint Etienne
- Chu Tours Hopital Clocheville
- Hopital Nancy Brabois
- Institut de Cancerologie Gustave Roussy
- G. Gaslini Institut
- The Fundación para la Formación e Investigación Sanitarias de la Región de Murcia
- University Hospital Lund
- Universitätskinderklinik
- Kinderspital Zurich
- John Radcliffe Hospital
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Dexamethasone Cyclophosphamide Rituximab
Arm Description
Outcomes
Primary Outcome Measures
The response to treatment schedule as defined by the percentage of patients with disappearance of all symptoms.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01868269
Brief Title
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
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
April 18, 2013 (Actual)
Primary Completion Date
February 2027 (Anticipated)
Study Completion Date
April 2031 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Institut Curie
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Opsoclonus Myoclonus Syndrome, Neuroblastoma
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
102 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Dexamethasone Cyclophosphamide Rituximab
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
Dexamethasone acetate
Intervention Description
First step:
immunosuppressive treatment with dexamethasone
Intervention Type
Drug
Intervention Name(s)
dexamethasone and cyclophosphamide
Intervention Description
second step (in case of insufficient response): immunosuppressive treatment with dexamethasone and cyclophosphamide
Intervention Type
Drug
Intervention Name(s)
dexamethasone and rituximab
Intervention Description
third step (in case of insufficient response): immunosuppressive treatment with dexamethasone and rituximab
Primary Outcome Measure Information:
Title
The response to treatment schedule as defined by the percentage of patients with disappearance of all symptoms.
Time Frame
at 48 weeks after treatment start
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
No
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gudrun Schleiermacher, MD
Organizational Affiliation
Institut Curie
Official's Role
Principal Investigator
Facility Information:
Facility Name
St. Anna Kinderkrebsforschung e.V. CHILDREN'S CANCER RESEARCH INSTITUTE
City
Wien
ZIP/Postal Code
1090
Country
Austria
Facility Name
Chu de Bicetre
City
Le Kremlin-Bicêtre
State/Province
LE Kremlin Bicetre
ZIP/Postal Code
94275
Country
France
Facility Name
Centre Oscar Lambret
City
Lille
State/Province
Lille Cedex
ZIP/Postal Code
59020
Country
France
Facility Name
Centre Leon Berard
City
Lyon
State/Province
LYON Cedex 08
ZIP/Postal Code
69373
Country
France
Facility Name
Hopita D'Enfants de La Timone
City
Marseille
State/Province
Marseille Cedex 5
ZIP/Postal Code
13385
Country
France
Facility Name
Hopital Arnaud de Villeneuve
City
Montpellier
State/Province
Montpellier Cedex 4
ZIP/Postal Code
34295
Country
France
Facility Name
Chr de Nantes
City
Nantes
State/Province
Nantes Cedex01
ZIP/Postal Code
44093
Country
France
Facility Name
Chu de Nice Archet 2
City
Nice
State/Province
NICE Cedex 03
ZIP/Postal Code
06202
Country
France
Facility Name
Ch Trousseau
City
Paris
State/Province
Paris Cedex 12
ZIP/Postal Code
75571
Country
France
Facility Name
Chu Hopital Sud
City
Rennes
State/Province
Rennes Cedex 02
ZIP/Postal Code
35056
Country
France
Facility Name
Chu de Rouen
City
Rouen
State/Province
Rouen Cedex
ZIP/Postal Code
76031
Country
France
Facility Name
CHU DE STRASBOURG HOPITAL Hautepierre
City
Strasbourg
State/Province
Strasbourg Cedex
ZIP/Postal Code
67098
Country
France
Facility Name
Chu Toulouse Hopital Des Enfants
City
Toulouse
State/Province
Toulouse Cedex 9
ZIP/Postal Code
31059
Country
France
Facility Name
Chu Amiens
City
Amiens
ZIP/Postal Code
80054
Country
France
Facility Name
Chu Angers
City
Angers
ZIP/Postal Code
49933
Country
France
Facility Name
Hopital Jean Minjoz
City
Besancon
ZIP/Postal Code
25030
Country
France
Facility Name
Chr Pellegrin
City
Bordeaux
ZIP/Postal Code
33076
Country
France
Facility Name
CHU CAEN
City
Caen
ZIP/Postal Code
14033
Country
France
Facility Name
Chu D'Estaing
City
Clermont Ferrand
ZIP/Postal Code
63003
Country
France
Facility Name
Chu Dijon
City
Dijon
ZIP/Postal Code
21079
Country
France
Facility Name
Chu de Grenoble
City
Grenoble
ZIP/Postal Code
38045
Country
France
Facility Name
Chu de Limoges
City
Limoges
ZIP/Postal Code
87042
Country
France
Facility Name
Institut Curie
City
Paris
ZIP/Postal Code
75005
Country
France
Facility Name
Chu de Poitiers
City
Poitiers
ZIP/Postal Code
86021
Country
France
Facility Name
Chu de Reims
City
Reims
ZIP/Postal Code
51092
Country
France
Facility Name
CHU LA REUNION Site Félix Guyon
City
Saint-denis
ZIP/Postal Code
97400
Country
France
Facility Name
Chu Saint Etienne
City
Saint-Étienne
ZIP/Postal Code
42055
Country
France
Facility Name
Chu Tours Hopital Clocheville
City
Tours
ZIP/Postal Code
37044
Country
France
Facility Name
Hopital Nancy Brabois
City
Vandoeuvre Les Nancy
ZIP/Postal Code
54500
Country
France
Facility Name
Institut de Cancerologie Gustave Roussy
City
Villejuif
ZIP/Postal Code
94805
Country
France
Facility Name
G. Gaslini Institut
City
Genova
ZIP/Postal Code
16148
Country
Italy
Facility Name
The Fundación para la Formación e Investigación Sanitarias de la Región de Murcia
City
El Palmar
ZIP/Postal Code
30120
Country
Spain
Facility Name
University Hospital Lund
City
Lund
ZIP/Postal Code
22185
Country
Sweden
Facility Name
Universitätskinderklinik
City
Bern
ZIP/Postal Code
CH 3010
Country
Switzerland
Facility Name
Kinderspital Zurich
City
Zurich
ZIP/Postal Code
8032
Country
Switzerland
Facility Name
John Radcliffe Hospital
City
Oxford
ZIP/Postal Code
0X3 9DU
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
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)
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