Multinational european trial for children with the opsoclonus myoclonus syndrome/dancing eye syndrome.
Phase 1
- Conditions
- Opsoclonus myoclonus Syndrome/Dancing Eye Syndrome (OMS/DES) in children with and without neuroblastoma (NBpos and NBneg)MedDRA version: 20.0Level: PTClassification code 10053854Term: Opsoclonus myoclonusSystem Organ Class: 10015919 - Eye disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2011-000990-29-BE
- Lead Sponsor
- Institut Curie
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- 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
•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: To evaluate prospectively the response to the treatment schedule as defined by the percentage of patients with disappearance of all symptoms;Secondary Objective: - the response to the treatment schedule as defined by the percentage of patients with disappearance of all symptoms <br>- the response to the treatment schedule as given by absolute and relative scoring scales and by the parents’ estimation<br>- the response as estimated by the parents <br>- the response with respect to each treatment step<br>- to assess the number of OMS/DES relapses by treatment step given <br>- to determine the percentage of neuroblastoma(NB)-associated OMS/DES (OMS-NBpos) and neuroblastoma(NB)-negative OMS/DES (OMS-NBneg) in a patient cohort investigated at diagnosis according to the uniform guidelines given in this trial;Primary end point(s): Response to treatment 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.;Timepoint(s) of evaluation of this end point: 3 months, 6 months, 12 months and 2 years after treatment start