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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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01868269
Recruitment Status : Recruiting
First Posted : June 4, 2013
Last Update Posted : December 12, 2018
Sponsor:
Information provided by (Responsible Party):
Institut Curie

Tracking Information
First Submitted Date  ICMJE May 27, 2013
First Posted Date  ICMJE June 4, 2013
Last Update Posted Date December 12, 2018
Actual Study Start Date  ICMJE April 18, 2013
Estimated Primary Completion Date February 2027   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 30, 2013)
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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01868269 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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  ICMJE Multinational European Trial for Children With the Opsoclonus Myoclonus Syndrome / Dancing Eye Syndrome
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Opsoclonus Myoclonus Syndrome
  • Neuroblastoma
Intervention  ICMJE
  • 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
Study Arms  ICMJE Dexamethasone Cyclophosphamide Rituximab
Interventions:
  • Drug: Dexamethasone acetate
  • Drug: dexamethasone and cyclophosphamide
  • Drug: dexamethasone and rituximab
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 30, 2013)
100
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 2031
Estimated Primary Completion Date February 2027   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months to 8 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Patricia TRESCA, MD +33156245765 emmanuelle.bouffier@curie.fr
Listed Location Countries  ICMJE Austria,   France,   Italy,   Spain,   Sweden,   Switzerland,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01868269
Other Study ID Numbers  ICMJE IC 2011-02
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Institut Curie
Study Sponsor  ICMJE Institut Curie
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Gudrun Schleiermacher, MD Institut Curie
PRS Account Institut Curie
Verification Date December 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP