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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)

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2016 by Institut Curie
Sponsor:
Information provided by (Responsible Party):
Institut Curie
ClinicalTrials.gov Identifier:
NCT01868269
First received: May 27, 2013
Last updated: September 1, 2016
Last verified: August 2016
  Purpose

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 Pédiatric) 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.


Condition Intervention Phase
Opsoclonus Myoclonus Syndrome
Neuroblastoma
Drug: Dexamethasone acetate
Drug: dexamethasone and cyclophosphamide
Drug: dexamethasone and rituximab
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Multinational European Trial for Children With the Opsoclonus Myoclonus Syndrome / Dancing Eye Syndrome

Resource links provided by NLM:


Further study details as provided by Institut Curie:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 100
Study Start Date: February 2013
Estimated Primary Completion Date: February 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Dexametasone Cyclophosphamide Rituximab Drug: Dexamethasone acetate

First step:

immunosuppressive treatment with dexamethasone

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

  Eligibility

Ages Eligible for Study:   6 Months to 8 Years   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01868269

Contacts
Contact: Patricia TRESCA, MD +33156245765 emmanuelle.bouffier@curie.fr

  Show 37 Study Locations
Sponsors and Collaborators
Institut Curie
Investigators
Principal Investigator: Gudrun Schleiermacher, MD Institut Curie
  More Information

Responsible Party: Institut Curie
ClinicalTrials.gov Identifier: NCT01868269     History of Changes
Other Study ID Numbers: IC 2011-02 
Study First Received: May 27, 2013
Last Updated: September 1, 2016
Health Authority: France: Agence Nationale de Sécurité du Médicament et des produits de santé

Additional relevant MeSH terms:
Syndrome
Opsoclonus-Myoclonus Syndrome
Paraneoplastic Syndromes, Nervous System
Paraneoplastic Syndromes
Neuroblastoma
Myoclonus
Ocular Motility Disorders
Disease
Pathologic Processes
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Nervous System Neoplasms
Neoplasms by Site
Central Nervous System Diseases
Cranial Nerve Diseases
Neurodegenerative Diseases
Eye Diseases
Dexamethasone acetate
Dexamethasone

ClinicalTrials.gov processed this record on September 23, 2016