Opsoclonus Myoclonus SyndromePhase 3NCT01868269
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)
A Phase 3 study testing Dexamethasone acetate, dexamethasone and cyclophosphamide and dexamethasone and rituximab in people with Opsoclonus Myoclonus Syndrome and Neuroblastoma, run by Institut Curie at 36 sites in 7 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 months to 8 years
- Conditions the study is for
- Opsoclonus Myoclonus Syndrome, Neuroblastoma
- What is being tested?
- Dexamethasone acetate, dexamethasone and cyclophosphamide and dexamethasone and rituximab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 102 participants
- Where
- 36 sitesin 7 countries
- Study dates
- 18 Apr 2013to 17 Jan 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 8 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
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- 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.
Who cannot join
- •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)
Trial locations and contacts
36 sites in 7 countries.
36 sites in 7 countries
France29 sites
Chu Amiens
Amiens, 80054
Chu Angers
Angers, 49933
Hopital Jean Minjoz
Besançon, 25030
Chr Pellegrin
Bordeaux, 33076
CHU CAEN
Caen, 14033
Chu D'Estaing
Clermont-Ferrand, 63003
Chu Dijon
Dijon, 21079
Chu de Grenoble
Grenoble, 38045
Chu de Bicetre
Le Kremlin-Bicêtre, LE Kremlin Bicetre, 94275
Centre Oscar Lambret
Lille, Lille Cedex, 59020
Chu de Limoges
Limoges, 87042
Centre Leon Berard
Lyon, LYON Cedex 08, 69373
Hopita D'Enfants de La Timone
Marseille, Marseille Cedex 5, 13385
Hopital Arnaud de Villeneuve
Montpellier, Montpellier Cedex 4, 34295
Chr de Nantes
Nantes, Nantes Cedex01, 44093
Chu de Nice Archet 2
Nice, NICE Cedex 03, 06202
Ch Trousseau
Paris, Paris Cedex 12, 75571
Institut Curie
Paris, 75005
Chu de Poitiers
Poitiers, 86021
Chu de Reims
Reims, 51092
Chu Hopital Sud
Rennes, Rennes Cedex 02, 35056
Chu de Rouen
Rouen, Rouen Cedex, 76031
CHU LA REUNION Site Félix Guyon
Saint-Denis, 97400
Chu Saint Etienne
Saint-Etienne, 42055
CHU DE STRASBOURG HOPITAL Hautepierre
Strasbourg, Strasbourg Cedex, 67098
Chu Toulouse Hopital Des Enfants
Toulouse, Toulouse Cedex 9, 31059
Chu Tours Hopital Clocheville
Tours, 37044
Hopital Nancy Brabois
Vandœuvre-lès-Nancy, 54500
Institut de Cancerologie Gustave Roussy
Villejuif, 94805
Switzerland2 sites
Universitätskinderklinik
Bern, CH 3010
Kinderspital Zurich
Zurich, 8032
Austria1 site
St. Anna Kinderkrebsforschung e.V. CHILDREN'S CANCER RESEARCH INSTITUTE
Vienna, 1090
Spain1 site
The Fundación para la Formación e Investigación Sanitarias de la Región de Murcia
El Palmar, 30120
About this study
Official title: Multinational European Trial for Children With the Opsoclonus Myoclonus Syndrome / Dancing Eye Syndrome
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.
- Conditions
- Opsoclonus Myoclonus Syndrome, Neuroblastoma
- Being tested
- Dexamethasone acetate, dexamethasone and cyclophosphamide, dexamethasone and rituximab
- Medical subject headings
- Opsoclonus-Myoclonus Syndrome, Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dexamethasone acetateDrug
First step:
immunosuppressive treatment with dexamethasone
dexamethasone and cyclophosphamideDrug
second step (in case of insufficient response): immunosuppressive treatment with dexamethasone and cyclophosphamide
dexamethasone and rituximabDrug
third step (in case of insufficient response): immunosuppressive treatment with dexamethasone and rituximab
| Study group | Role | What they receive |
|---|---|---|
| Dexamethasone Cyclophosphamide Rituximab | Other | Not described Receives: Drug: Dexamethasone acetate; Drug: dexamethasone and cyclophosphamide; Drug: dexamethasone and rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
The response to treatment schedule as defined by the percentage of patients with disappearance of all symptoms.
Measured: at 48 weeks after treatment start
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2013 (estimated)
- Study start18 Apr 2013
- Primary completion9 May 2021
- Study completion17 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Opsoclonus Myoclonus SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 102 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut Curie
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gudrun Schleiermacher, MD, Principal investigator, Institut Curie
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01868269
- Sponsor's protocol ID
- IC 2011-02
- Status last verified
- Jan 2025
- First submitted
- 27 May 2013
- Last updated
- 3 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Sponsor will share de-identified data sets. Documents generated under the project will be disseminated in accordance with Institut Curie policies.
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multinational European Trial for Children With the Opsoclonus Myoclonus Syndrome / Dancing Eye Syndrome
- ClinicalTrials.gov ID
- NCT01868269
- Acronym / sponsor ID
- IC 2011-02
- Conditions
- Opsoclonus Myoclonus Syndrome, Neuroblastoma
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 102 participants (actual)
- Lead sponsor
- Institut CurieUniversity, hospital or other
- Study start
- 18 Apr 2013 (actual)
- Primary completion
- 9 May 2021 (actual)
- Study completion
- 17 Jan 2025 (actual)
- First posted
- 4 Jun 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dexamethasone Cyclophosphamide Rituximab | Other | Drug: Dexamethasone acetate, Drug: dexamethasone and cyclophosphamide, Drug: dexamethasone and rituximab |
Who may qualify
- Ages
- 6 months to 8 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Children with newly diagnosed OMS/DES either NB-pos or NB-neg.
- 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.
Key exclusion criteria (5 listed)
- •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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dexamethasone acetateFirst step: immunosuppressive treatment with dexamethasone | Drug | Dexamethasone Cyclophosphamide Rituximab |
| dexamethasone and cyclophosphamidesecond step (in case of insufficient response): immunosuppressive treatment with dexamethasone and cyclophosphamide | Drug | Dexamethasone Cyclophosphamide Rituximab |
| dexamethasone and rituximabthird step (in case of insufficient response): immunosuppressive treatment with dexamethasone and rituximab | Drug | Dexamethasone Cyclophosphamide Rituximab |
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2025.
- Individual participant dataSharing planned: Yes. Sponsor will share de-identified data sets. Documents generated under the project will be disseminated in accordance with Institut Curie policies.
Related trials for Opsoclonus Myoclonus Syndrome
Active, not recruitingPhase 2
Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases
Autoimmune Disease, Neurologic Autoimmune Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gudrun Schleiermacher, MD principal investigator
Institut Curie
36 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dexamethasone acetate, dexamethasone and cyclophosphamide and dexamethasone and rituximab in people with Opsoclonus Myoclonus Syndrome and Neuroblastoma, run by Institut Curie at 36 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 sites in France, Switzerland, Austria, Italy, Spain and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2025.