Myoclonus DystoniaPhase 3EpsilonZêtaNCT01806805
Efficacy Trial of Zonisamide for Myoclonus Dystonia
A Phase 3 study testing zonegran in people with Myoclonus Dystonia, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Myoclonus Dystonia is a disease in which myoclonus distort the precision of movements and so cause a handicap in the movements of the everyday life. Response to oral medications may be incomplete and surgery may cause operating risk. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Myoclonus Dystonia
- What is being tested?
- zonegran
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteFrance
- Study dates
- Feb 2012to Jul 2014
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age >18 and < 60
- Diagnosis of myoclonus dystonia including the isolated myoclonus caused by epsilon-sarcoglycans mutation or deletion.
- Myoclonus present in both hands
- Myoclonus decrease quality of life
- Insufficient efficiency of the benzodiazepine's tolerated maximal dose during one year
- Agreement to use a medically acceptable method of contraception throughout the study for female of childbearing potential
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- Normal physical and neurological examination, except myoclonus dystonia
- No hepatic disease
- No renal disease
- Able to comply with study visits and procedures
- Has voluntarily signed consent form
- Taking no medications or stable doses medication for 4 weeks prior to the Baseline visit
Who cannot join
- Patients who are not enrolled at social security
- Individual who have MMS ≤ 24/30 or patients legally protected or inability to provide an informed consent
- Pregnancy, breast feeding women and women who are of childbearing age and not practicing adequate birth control
- Weight < 40 kg
- history of serious psychiatric illness
- history of renal stones
- history of allergy to sulfonamides
- taking medications : topiramate, rifampicin, ketoconazole, cimetidine
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Comparative Study of the Efficiency of Zonisamide in Myoclonus Dystonia: A Monocentric , Randomized in Cross Over and Double Blind Study Versus Placebo Study
Myoclonus Dystonia is a disease in which myoclonus distort the precision of movements and so cause a handicap in the movements of the everyday life. Response to oral medications may be incomplete and surgery may cause operating risk.
Zonisamide is an antiepileptic drug which could bring a therapeutic profit in Myoclonus Dystonia on the severity of the myoclonus.
Read the detailed description
In "dystonia", the involuntary abnormal movements cause a driving handicap and a change of the quality of life. A particular shape of dystonia, the Myoclonus Dystonia, is characterized by the ascendancy of myoclonias (abrupt and brief movements) associated with the abnormal dystonia. Myoclonus is an additional source of handicap in the movements of the everyday life, because they distort the precision of movements. Response to oral medications may be incomplete and the tolerance poor, such that deep brain stimulation (DBS) surgery is useful for the major forms but it is also an invasive therapeutics which the operating risk is not totally estimated in the absence of controlled study. Therefore, it is necessary to investigate other pharmacological therapeutic tracks which present a good ratio profit / risk.
Zonisamide is usually used in France in the epilepsy's treatment. It showed its efficiency in the progressive myoclonus epilepsy, not only on the seizure but also on the myoclonia. Therefore, it showed its efficiency on post-anoxic and propriospinal myoclonus. So, we make the hypothesis that this medicine could bring a therapeutic profit in the Myoclonus Dystonia.
The aim of this study is to demonstrate the efficiency of the zonisamide on the severity of myoclonus (UMRS) at those patients. The others outcomes are to estimate the impact of the treatment on the myoclonus's neurophysiological characteristics, the dystonia's severity (BFM score), the quality of life (SF-36 and CGI scores), but also to investigate the tolerance of the treatment.
We conducted a randomized, placebo-controlled, double-blind, two-period cross-over design to evaluate the effect on severity of myoclonus in response to placebo or zonisamide (until 300 mg) in 32 patients.
The study includes an evaluation at the beginning and at the end of every period (4 evaluations at all). Each period includes a phase of titration (six weeks) followed by a phase of fixed dose (three weeks). Those two periods are separated by a period of wash-out (3 weeks) preceded by a phase of progressive decrease of doses (two weeks).
- Conditions
- Myoclonus Dystonia
- Being tested
- zonegran, placebo
- Keywords
- Movement disorder, Myoclonus dystonia, DYT 11, Antiepileptic drugs, Zonisamide, Clinical Trial, Double-mind method
- Medical subject headings
- Myoclonic dystonia, Movement Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
zonegranDrug
placeboDrug
| Study group | Role | What they receive |
|---|---|---|
| Zonegran | Experimental | Zonegran / Placebo Zonisamide (Zonegran ®) and its placebo appear under the shape of virgin capsules of size 1. Each drug will be dispensed successively in a box containing blister packs of 14 capsules. For every period (A and B), box will contain 26 blister packs. A phase of progressive increase of doses by stages of 50 mg / week is planned before reaching the fixed dose of 300 in the daytime during 4 weeks. Then, a progressive diminution over two weeks is planned before the stop. Receives: Drug: zonegran; Drug: placebo |
| placebo | Placebo comparator | Placebo /Experimental Zonisamide (Zonegran ®) and its placebo appear under the shape of virgin capsules of size 1. Each drug will be dispensed successively in a box containing blister packs of 14 capsules. For every period (A and B), box will contain 26 blister packs. A phase of progressive increase of doses by stages of 50 mg / week is planned before reaching the fixed dose of 300 in the daytime during 4 weeks. Then, a progressive diminution over two weeks is planned before the stop. Receives: Drug: zonegran; Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measure of the evolution of the severity of myoclonus by a specific scale (UMRS)
Measured: from day 0 to week 23
Secondary outcomes (2)
Measure of the evolution of the severity of dystonia by a specific scale (BFM)
Measured: from day 0 to week 23
measure of the evolution of the severity of myoclonus by electromyographic recording
Measured: from day 0 to week 23
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2013 (estimated)
- Study startFeb 2012
- Primary completionJul 2014
- Study completionJul 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myoclonus DystoniaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- 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
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Emmanuel Roze, Principal investigator, APHP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01806805
- Sponsor's protocol ID
- P091104
- Acronym
- EpsilonZêta
- Status last verified
- Apr 2015
- First submitted
- 6 Mar 2013
- Last updated
- 24 Apr 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparative Study of the Efficiency of Zonisamide in Myoclonus Dystonia: A Monocentric , Randomized in Cross Over and Double Blind Study Versus Placebo Study
- ClinicalTrials.gov ID
- NCT01806805
- Acronym / sponsor ID
- EpsilonZêta / P091104
- Conditions
- Myoclonus Dystonia
- Keywords
- Movement disorder, Myoclonus dystonia, DYT 11, Antiepileptic drugs, Zonisamide, Clinical Trial, Double-mind method
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Feb 2012
- Primary completion
- Jul 2014 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 7 Mar 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.
Myoclonus Dystonia is a disease in which myoclonus distort the precision of movements and so cause a handicap in the movements of the everyday life. Response to oral medications may be incomplete and surgery may cause operating risk.
Zonisamide is an antiepileptic drug which could bring a therapeutic profit in Myoclonus Dystonia on the severity of the myoclonus.
Detailed description
In "dystonia", the involuntary abnormal movements cause a driving handicap and a change of the quality of life. A particular shape of dystonia, the Myoclonus Dystonia, is characterized by the ascendancy of myoclonias (abrupt and brief movements) associated with the abnormal dystonia. Myoclonus is an additional source of handicap in the movements of the everyday life, because they distort the precision of movements. Response to oral medications may be incomplete and the tolerance poor, such that deep brain stimulation (DBS) surgery is useful for the major forms but it is also an invasive therapeutics which the operating risk is not totally estimated in the absence of controlled study. Therefore, it is necessary to investigate other pharmacological therapeutic tracks which present a good ratio profit / risk.
Zonisamide is usually used in France in the epilepsy's treatment. It showed its efficiency in the progressive myoclonus epilepsy, not only on the seizure but also on the myoclonia. Therefore, it showed its efficiency on post-anoxic and propriospinal myoclonus. So, we make the hypothesis that this medicine could bring a therapeutic profit in the Myoclonus Dystonia.
The aim of this study is to demonstrate the efficiency of the zonisamide on the severity of myoclonus (UMRS) at those patients. The others outcomes are to estimate the impact of the treatment on the myoclonus's neurophysiological characteristics, the dystonia's severity (BFM score), the quality of life (SF-36 and CGI scores), but also to investigate the tolerance of the treatment.
We conducted a randomized, placebo-controlled, double-blind, two-period cross-over design to evaluate the effect on severity of myoclonus in response to placebo or zonisamide (until 300 mg) in 32 patients.
The study includes an evaluation at the beginning and at the end of every period (4 evaluations at all). Each period includes a phase of titration (six weeks) followed by a phase of fixed dose (three weeks). Those two periods are separated by a period of wash-out (3 weeks) preceded by a phase of progressive decrease of doses (two weeks).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Zonegran | Experimental | Drug: zonegran, Drug: placeboZonegran / Placebo Zonisamide (Zonegran ®) and its placebo appear under the shape of virgin capsules of size 1. Each drug will be dispensed successively in a box containing blister packs of 14 capsules. For every period (A and B), box will contain 26 blister packs. A phase of progressive increase of doses by stages of 50 mg / week is planned before reaching the fixed dose of 300 in the daytime during 4 weeks. Then, a progressive diminution over two weeks is planned before the stop. |
| placebo | Placebo comparator | Drug: zonegran, Drug: placeboPlacebo /Experimental Zonisamide (Zonegran ®) and its placebo appear under the shape of virgin capsules of size 1. Each drug will be dispensed successively in a box containing blister packs of 14 capsules. For every period (A and B), box will contain 26 blister packs. A phase of progressive increase of doses by stages of 50 mg / week is planned before reaching the fixed dose of 300 in the daytime during 4 weeks. Then, a progressive diminution over two weeks is planned before the stop. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (12 listed)
- Age >18 and < 60
- Diagnosis of myoclonus dystonia including the isolated myoclonus caused by epsilon-sarcoglycans mutation or deletion.
- Myoclonus present in both hands
- Myoclonus decrease quality of life
- Insufficient efficiency of the benzodiazepine's tolerated maximal dose during one year
Key exclusion criteria (8 listed)
- Patients who are not enrolled at social security
- Individual who have MMS ≤ 24/30 or patients legally protected or inability to provide an informed consent
- Pregnancy, breast feeding women and women who are of childbearing age and not practicing adequate birth control
- Weight < 40 kg
- history of serious psychiatric illness
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 |
|---|---|---|
| zonegran | Drug | Zonegran, placebo |
| placebo | Drug | Zonegran, placebo |
Primary outcome
Measure of the evolution of the severity of myoclonus by a specific scale (UMRS)
Time frame: from day 0 to week 23
2 secondary outcomes
Measure of the evolution of the severity of dystonia by a specific scale (BFM)
Time frame: from day 0 to week 23
measure of the evolution of the severity of myoclonus by electromyographic recording
Time frame: from day 0 to week 23
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 24 Apr 2015.
Related trials for Myoclonus Dystonia
RecruitingObservational study
Dystonia Genotype-Phenotype Correlation
Dystonia, Dystonia; Idiopathic
RecruitingObservational study
A Retrospective Survey-based Multicenter Study to Delineate the Molecular and Phenotypic Spectrum of Epilepsy-dyskinesia Syndromes
Epilepsy in Children, Dyskinesias
Recruiting
Clinical Validation of DystoniaNet Deep Learning Platform for Diagnosis of Isolated Dystonia
Dystonia, Drug Induced Dystonia
RecruitingObservational study
Registry and Natural History of Epilepsy-Dyskinesia Syndromes
Epilepsy-Dyskinesia, Epilepsy
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.
Emmanuel Roze principal investigator
APHP
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing zonegran in people with Myoclonus Dystonia, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 12 inclusion and 8 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 24 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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. Some participants in this study may receive a placebo. 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?
Measure of the evolution of the severity of myoclonus by a specific scale (UMRS) (time frame: from day 0 to week 23). 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 24 Apr 2015.