Multinational européan trial for children with the opsoclonus myoclonus syndrome/dancing eye syndrome.

Phase 1

• Conditions: Opsoclonus myoclonus Syndrome/Dancing Eye Syndrome (OMS/DES) in childrenwith and without neuroblastoma (NBpos and NBneg); MedDRA version: 15.0Level: PTClassification code 10053854Term: Opsoclonus myoclonusSystem Organ Class: 10015919 - Eye disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2011-000990-29-FR
• Lead Sponsor: INSTITUT CURIE

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-09-04
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

•children with newly diagnosed OMS/DES
(with or without neuroblastoma) using the diagnostic criteria of this trial
• no prior use of immunosuppressive medication
•staging for neuroblastoma according the guidelines of the trial
•age > 6 months and < 8 years
•informed consent by parents / legal representatives

Are the trial subjects under 18? yes
Number of subjects for this age range: 100
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

exclusion criteria:
•opsoclonus, myoclonus or ataxia caused by other diseases

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Primary endpoint:<br>•response to treatment at 48 weeks after treatment start<br>;Secondary Objective: Secondary endpoints:<br>•response at 3 months, 6 months, 12 months, and 2 years after treatment start<br>•number of OMS/DES relapses<br>•percentage of OMS-NBpos<br>•need for chemotherapy in OMS-NBpos<br>•evaluation of treatment burden <br>(neurological and oncological treatment)<br>•comparison of OMS-NBpos and OMS-NBneg in terms of presentation, severity and treatment response<br>•neuropsychological longterm outcome<br>•long term quality of life<br>•evaluation of factors influencing long term outcome and quality of life<br>•comparison of long term outcome to other OMS cohorts<br>•Event free Survival and Overall survival in OMS-NBpos<br>;Primary end point(s): Response to treatme nt at 48 weeks after treatment start.;Timepoint(s) of evaluation of this end point: 48 weeks after treatment start.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Response at 3 months, 6 months, 12 months, and 2 years after <br>treatment start<br>Number of OMS/DES relapses <br>percentage of OMS-NBpos.<br>Evaluation of treatment burden (neurological and oncological treatment)<br>Comparison of OMS-NBpos and OMS-NBneg in terms of presentation, severity and treatment response.<br>Neuropsychological longterm outcome<br>Long term quality of life<br>Evaluation of factors influencing long term outcome and quality of life<br>Comparison of long term outcome to other OMS cohorts<br>Event free survival and overall survival in OMS-NBpos.