UK Multicentre Study of Children with Opsoclonus Myoclonus Syndrome (UMSCOM)

UK Multicentre Study of Children with Opsoclonus Myoclonus Syndrome (UMSCOM) This is the UK arm of EU study "Multinational Européan Trial for Children with the Opsoclonus Myoclonus Syndrome/Dancing Eye Syndrome". - UK Multicentre Study of Children with Opsoclonus Myoclonus Syndrome v1

Status

Not yet recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-000990-29-GB

Enrollment

100

Registered

2013-08-13

Start date

2013-08-13

Completion date

Unknown

Last updated

2013-09-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Opsoclonus myoclonus Syndrome/Dancing Eye Syndrome (OMS/DES) in children with and without neuroblastoma (NBpos and NBneg) MedDRA version: 16.0 Level: PT Classification code 10053854 Term: Opsoclonus myoclonus System Organ Class: 10015919 - Eye disorders

Interventions

Product Name: CYCLOPHOSPHAMIDE Pharmaceutical Form: Solution for injection Product Name: DEXAMETHASONE Pharmaceutical Form: Oral solution Product Name: RITUXIMAB Pharmaceutical Form: Solution for in

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: For inclusion in the study all three numbered criteria below must be satisfied: 1) A diagnosis of OMS: Three out of the following four components must be evident: • Opsoclonus or ocular flutter (but not nystagmus) • Ataxia and/or myoclonus • Behavioural change and/or sleep disturbance • Neuroblastoma 2) Age at diagnosis between 6 months and 7 years (up to 8th birthday). 3) Signed informed consent has been given Are the trial subjects under 18? yes Number of subjects for this age range: 15 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: 1. Opsoclonus, myoclonus or ataxia caused by another identified disease. 2. Prior or parallel use of chemotherapy (other than required for treatment of NB) 3. Steroid treatment lasting 14 days or more immediately before start of the study.

Design outcomes

Primary

Measure	Time frame
Main Objective: The principal question is: • How effective are certain drugs and drug combinations for treating OMS? This question will be addressed by evaluating clinical remission, i.e. the disappearance of symptoms, as well as longer term outcomes, namely cognition, behaviour and quality of life, in relation to early symptoms and programme of drug treatment.;Secondary Objective: Secondary research questions are: • For OMS patients, in what percentage is NB detected using a uniform and systematic investigation protocol? • Does the presence and stage of NB influence OMS disease progression and drug responses? • What is the relationship of both remission and outcomes to age at diagnosis of OMS, severity of symptoms at onset, time between onset of symptoms and start of treatment? • What percentage of patients experience side effects at each stage of the study? • How do the results of the study compare with published series (very limited) and to results from the ongoing US COG trial (steroids and CP with or without intra-venous immunoglobulin in NB-associated OMS)? • What can we learn about event free survival of OMS patients (how long patients continue to be free of symptoms) and overall survival (number remaining alive) for OMS patients with NB? • Which laboratory tests (NB histology, blood and CSF markers such as B cell numbers) predict disease progres;Primary end point(s): Remission of OMS symptoms;Timepoint(s) of evaluation of this end point: 48 weeks after treatment start.	—

Measure

Time frame

Main Objective: The principal question is: • How effective are certain drugs and drug combinations for treating OMS? This question will be addressed by evaluating clinical remission, i.e. the disappearance of symptoms, as well as longer term outcomes, namely cognition, behaviour and quality of life, in relation to early symptoms and programme of drug treatment.;Secondary Objective: Secondary research questions are: • For OMS patients, in what percentage is NB detected using a uniform and systematic investigation protocol? • Does the presence and stage of NB influence OMS disease progression and drug responses? • What is the relationship of both remission and outcomes to age at diagnosis of OMS, severity of symptoms at onset, time between onset of symptoms and start of treatment? • What percentage of patients experience side effects at each stage of the study? • How do the results of the study compare with published series (very limited) and to results from the ongoing US COG trial (steroids and CP with or without intra-venous immunoglobulin in NB-associated OMS)? • What can we learn about event free survival of OMS patients (how long patients continue to be free of symptoms) and overall survival (number remaining alive) for OMS patients with NB? • Which laboratory tests (NB histology, blood and CSF markers such as B cell numbers) predict disease progres;Primary end point(s): Remission of OMS symptoms;Timepoint(s) of evaluation of this end point: 48 weeks after treatment start.

—

Secondary

Measure	Time frame
Secondary end point(s): Response (CRF scores) at 12, 24 and 48 weeks after treatment start Number of OMS/DES relapses Percentage of OMS-NBpos Evaluation of treatment burden (neurological and oncological treatment) Comparison of OMS-NBpos and OMS-NBneg in terms of presentation, severity and treatment response. Neuropsychological longterm outcome Long term quality of life Evaluation of factors influencing long term outcome and quality of life Comparison of long term outcome to other OMS cohorts Event free survival and overall survival in OMS-NBpos.;Timepoint(s) of evaluation of this end point: Depending on endpoint: 12, 24 and 48 weeks after treatment start (CRF scores), 48 weeks (relapses, NB positivity, treatment burden)and at 2 years after entry to study and at the 5th birthday (for cognitive, behavioural and quality of life assessments)	—

Measure

Time frame

Secondary end point(s): Response (CRF scores) at 12, 24 and 48 weeks after treatment start Number of OMS/DES relapses Percentage of OMS-NBpos Evaluation of treatment burden (neurological and oncological treatment) Comparison of OMS-NBpos and OMS-NBneg in terms of presentation, severity and treatment response. Neuropsychological longterm outcome Long term quality of life Evaluation of factors influencing long term outcome and quality of life Comparison of long term outcome to other OMS cohorts Event free survival and overall survival in OMS-NBpos.;Timepoint(s) of evaluation of this end point: Depending on endpoint: 12, 24 and 48 weeks after treatment start (CRF scores), 48 weeks (relapses, NB positivity, treatment burden)and at 2 years after entry to study and at the 5th birthday (for cognitive, behavioural and quality of life assessments)

—

Countries

Sweden, Switzerland, United Kingdom

Contacts

Public ContactHeather House

Oxford University Hospitals NHS Trust

heather.house@orh.nhs.uk01865 572245

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026