Oromandibular DystoniaPhase 2NCT02199509
Efficacy of Levetiracetam in Oromandibular and Cranial Dystonia
A Phase 2 study testing Levetiracetam in people with Oromandibular Dystonia and Cranial Dystonia, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial summary
Background: People with dystonia cannot control their muscle contractions. This disorder can affect different body areas. When it affects the face, tongue, and jaw, it is called oromandibular dystonia (OMD) or cranial dystonia (CD). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Oromandibular Dystonia, Cranial Dystonia
- What is being tested?
- Levetiracetam
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2014to Mar 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Be at least 18 years of age and less than or equal to 80 years of age.
- Must be able to provide consent.
- Primary OMD or CD diagnosed by movement disorders specialist.
- No history of receiving LVT.
- If the subject is using other medications for their dystonia such as anticholinergics, baclofen, benzodiazepines or tetrabenazine, the dosage must stay the same starting 4 weeks before participation and throughout the duration of the study. Subjects will also be prohibited from starting new medications for their dystonia.
- If the subject was injected with BoNT, the latest dose must be injected at least 12 weeks before participation in this study.
- Subject is willing to not receive any BoNT injections during the entire study.
Who cannot join
- Psychiatric co-morbidities such as depression, psychosis or phobic disorders.
- Has had a history of brain tumor, stroke, documented history of peripheral trauma to the mouth, jaw or face within a year from the onset of dystonia, epilepsy or seizures.
- Secondary OMD or CD.
- Postural instability, frequent falling, severe vertigo or dizziness and severe ataxia.
- Inability to take medication via oral route due to severe degree of OMD.
- An estimated creatinine clearance (eCrCL) less than 50 mL/min.
- Pregnant, lactating or planning to become pregnant in 6 months. Women who are able to get pregnant must be willing to use an effective method of contraception from the time of enrollment until 3 months after the last dose of the study medication.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Efficacy of Levetiracetam in Oromandibular and Cranial Dystonia: A Randomized, Double-Blind Placebo-Controlled Cross-Over Study
Background:
- People with dystonia cannot control their muscle contractions. This disorder can affect different body areas. When it affects the face, tongue, and jaw, it is called oromandibular dystonia (OMD) or cranial dystonia (CD). Researchers want to find out if a drug that treats seizures may help people with this kind of dystonia.
Read more
Objective:
- To see if levetiracetam can improve symptoms of jaw or face dystonia.
Eligibility:
- Adults ages 18 to 80 years with OMD or CD.
Design:
- Participants will be screened with a medical history and physical exam. Researchers will test how severe their dystonia is.
- Participants will have blood drawn through a needle in the arm.
- Participants will be assigned to take either levetiracetam or placebo.
- Phase 1:
- Participants will start with one 500-mg tablet twice daily. The dose will be increased by 500 mg every 3 days. The maximum dose will be 4000 mg a day over 3 weeks. Participants who cannot tolerate that will take the highest dose they can.
- Participants will return for study visits at weeks 3 and 6. They will be asked about their health, side effects, and symptoms of depression. They will have a neurological examination and test of their dystonia.
- After the week 6 visit, participants will taper and stop the study drug over about 1 week.
- Phase 2 begins one week later. Participants will repeat phase 1, but with the other drug.
- After phase 2, participants will return to their usual clinics. They will be told how to stop taking the drug. They will have a follow-up phone call 2 weeks later.
Read the detailed description
Objectives:
--To determine the efficacy of levetiracetam (LVT) for reducing the symptoms of subjects with oromandibular dystonia (OMD) or cranial dystonia (CD) as assessed by dystonia rating scales and to report all adverse events in this study.
Sample Size and Population:
--We plan to recruit 10 subjects with either primary OMD or CD from the Movement Disorders and Botulinum Toxin (BoNT) Clinics of HMCS.
Design:
--This is a randomized, double-blind placebo controlled cross-over study. All subjects will receive a baseline evaluation and have their dystonia assessed by the eyes, mouth, speech and swallowing subscores of the Burke-Fahn-Marsden (BFM) dystonia scale, and the eyes and upper and lower face, jaw, and tongue subscores of the Global Dystonia Severity (GDS) rating scale. They will then be randomized into two groups. Either LVT or placebo with a titration schedule up to a total daily dose of 4000 mg will be prescribed for six weeks. After week 6, all subjects will undergo tapering and a wash-out period (one week for tapering off and one week for the washout). The two groups will then be switched to the opposite intervention (LVT or placebo), following the same titration and tapering pattern. We will evaluate both groups at weeks 3, 6, 11 and 14 using the same scales. This study requires six outpatient visits to the NIH CC; the total duration of this study will be 15 weeks.
Outcome measurement:
Primary outcome: The percent change of sum of the eyes, mouth, speech and swallowing subscores of BFM dystonia scale at weeks 6 and 14 comparing to the baseline.
Secondary outcome: The percent change of the sum of the eyes, mouth, speech and swallowing subscores of BFM dystonia scale at weeks 3 and 11 and sum of eyes and upper face, lower face and jaw and tongue subscores of the GDS rating scale at weeks 3, 6, 11 and 14 comparing to baseline and all adverse events.
- Conditions
- Oromandibular Dystonia, Cranial Dystonia
- Being tested
- Levetiracetam
- Keywords
- Levetiracetam, Cranial Dystonia, Oromandibular Dystonia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LevetiracetamDrug
Anticonvulsant
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change of the Sum of the Eyes, Mouth, Speech and Swallowing Subscores of the Burke-Fahn-Marsden (BFM) Dystonia Scale.
The Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) is a measure of dystonia severity. The scale consists of evaluation of nine body parts (eyes, mouth, speech, swallowing, neck, trunk, right arm, right leg, left arm and left leg). The severity and provoking factors for each part are rated using a 5-point scale. These range from 0 (indicating no dystonia) to 4 (indicating the presence of dystonia at rest). The primary outcome measure includes the sum of the eyes, mouth, speech and swallowing subscores and represents the percent change from baseline to either 6 weeks or 14 weeks (representing the end of the 3 week period at the maximum tolerated dose for Levetiracetam or Placebo). The total range for these combined sub scores is 0-16, with higher scores indicating more severe dystonia and 0 indicating absence of dystonia.
Measured: 6 and 14 weeks compared to baseline
Secondary outcomes (2)
Percent Change of the Sum of the Eyes, Mouth, Speech and Swallowing Subscores of Burke-Fahn-Marsten Dystonia Rating Scale (BFM)
The Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) is a measure of dystonia severity. The scale consists of evaluation of ten body parts (eyes, mouth, speech, swallowing, neck, trunk, right arm, right leg, left arm and left leg). The severity and provoking factors for each part are rated using a 5-point scale. These range from 0 (indicating no dystonia) to 4 (indicating the presence of dystonia at rest). The secondary outcome measure includes the sum of the eyes, mouth, speech and swallowing subscores and represents the percent change from baseline to either 3 weeks or 11 weeks (representing the end of the three week titration period up to the maximum tolerated dose for Levetiracetam or Placebo). The total range for these combined sub scores is 0-16, with higher scores indicating more severe dystonia and 0 indicating absence of dystonia.
Measured: 3 and 11 weeks compared to baseline
Percent Change of the Sum of Eyes and Upper Face, Lower Face and Jaw and Tongue Subscores of the GDS Rating Scale
The Global Dystonia Severity Scale provides a severity rating for ten body regions, i.e.,1) eyes and upper face, 2) lower face, 3) jaw and tongue, 4) larynx, 5) neck, 6) shoulder and proximal arm, 7) distal arm and hand including elbow, 8) pelvis and upper leg, 9) distal leg and foot, and 10) trunk. Each body area is rated from 0 to 10, with 0 representing no dystonia present in that body area and 10 representing severe dystonia. The secondary outcome measure includes the sum of the eyes and upper face, lower face and jaw and tongue subscores of the GDS rating scale and represents the percent change from baseline to either 3 and 6 weeks or 11 and 14 weeks (representing the end of the three week titration period (3 weeks and 11 weeks) and the post-3 week period (6 weeks and 14 weeks) at the maximum tolerated dose for Levetiracetam or Placebo). The total range of these combined sub scores is 0-30, with higher scores indicating more severe dystonia and 0 indicating absence of
Measured: 3, 6, 11 and 14 compared to baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2014 (estimated)
- Study startJul 2014
- Primary completionMar 2016
- Study completionMar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Oromandibular DystoniaStudies of the same condition, open and under way.
- More trials run by National Institute of Neurological Disorders and Stroke (NINDS)The 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 2
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Mark Hallett, M.D., Principal investigator, National Institute of Neurological Disorders and Stroke (NINDS)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02199509
- Sponsor's protocol ID
- 140152
- Other IDs
- 14-N-0152
- Status last verified
- Jun 2017
- First submitted
- 23 Jul 2014
- Last updated
- 11 Jul 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Levetiracetam in Oromandibular and Cranial Dystonia: A Randomized, Double-Blind Placebo-Controlled Cross-Over Study
- ClinicalTrials.gov ID
- NCT02199509
- Acronym / sponsor ID
- 140152
- Conditions
- Oromandibular Dystonia, Cranial Dystonia
- Keywords
- Levetiracetam, Cranial Dystonia, Oromandibular Dystonia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- Jul 2014
- Primary completion
- Mar 2016 (actual)
- Study completion
- Mar 2016 (actual)
- First posted
- 24 Jul 2014 (estimated)
- Results
- Posted 11 Jul 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
- People with dystonia cannot control their muscle contractions. This disorder can affect different body areas. When it affects the face, tongue, and jaw, it is called oromandibular dystonia (OMD) or cranial dystonia (CD). Researchers want to find out if a drug that treats seizures may help people with this kind of dystonia.
Objective:
- To see if levetiracetam can improve symptoms of jaw or face dystonia.
Eligibility:
- Adults ages 18 to 80 years with OMD or CD.
Design:
- Participants will be screened with a medical history and physical exam. Researchers will test how severe their dystonia is.
- Participants will have blood drawn through a needle in the arm.
- Participants will be assigned to take either levetiracetam or placebo.
- Phase 1:
- Participants will start with one 500-mg tablet twice daily. The dose will be increased by 500 mg every 3 days. The maximum dose will be 4000 mg a day over 3 weeks. Participants who cannot tolerate that will take the highest dose they can.
- Participants will return for study visits at weeks 3 and 6. They will be asked about their health, side effects, and symptoms of depression. They will have a neurological examination and test of their dystonia.
- After the week 6 visit, participants will taper and stop the study drug over about 1 week.
- Phase 2 begins one week later. Participants will repeat phase 1, but with the other drug.
- After phase 2, participants will return to their usual clinics. They will be told how to stop taking the drug. They will have a follow-up phone call 2 weeks later.
Detailed description
Objectives:
--To determine the efficacy of levetiracetam (LVT) for reducing the symptoms of subjects with oromandibular dystonia (OMD) or cranial dystonia (CD) as assessed by dystonia rating scales and to report all adverse events in this study.
Sample Size and Population:
--We plan to recruit 10 subjects with either primary OMD or CD from the Movement Disorders and Botulinum Toxin (BoNT) Clinics of HMCS.
Design:
--This is a randomized, double-blind placebo controlled cross-over study. All subjects will receive a baseline evaluation and have their dystonia assessed by the eyes, mouth, speech and swallowing subscores of the Burke-Fahn-Marsden (BFM) dystonia scale, and the eyes and upper and lower face, jaw, and tongue subscores of the Global Dystonia Severity (GDS) rating scale. They will then be randomized into two groups. Either LVT or placebo with a titration schedule up to a total daily dose of 4000 mg will be prescribed for six weeks. After week 6, all subjects will undergo tapering and a wash-out period (one week for tapering off and one week for the washout). The two groups will then be switched to the opposite intervention (LVT or placebo), following the same titration and tapering pattern. We will evaluate both groups at weeks 3, 6, 11 and 14 using the same scales. This study requires six outpatient visits to the NIH CC; the total duration of this study will be 15 weeks.
Outcome measurement:
Primary outcome: The percent change of sum of the eyes, mouth, speech and swallowing subscores of BFM dystonia scale at weeks 6 and 14 comparing to the baseline.
Secondary outcome: The percent change of the sum of the eyes, mouth, speech and swallowing subscores of BFM dystonia scale at weeks 3 and 11 and sum of eyes and upper face, lower face and jaw and tongue subscores of the GDS rating scale at weeks 3, 6, 11 and 14 comparing to baseline and all adverse events.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
Key exclusion criteria (7 listed)
- Psychiatric co-morbidities such as depression, psychosis or phobic disorders.
- Has had a history of brain tumor, stroke, documented history of peripheral trauma to the mouth, jaw or face within a year from the onset of dystonia, epilepsy or seizures.
- Secondary OMD or CD.
- Postural instability, frequent falling, severe vertigo or dizziness and severe ataxia.
- Inability to take medication via oral route due to severe degree of OMD.
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 |
|---|---|---|
| LevetiracetamAnticonvulsant | Drug | – |
Primary outcome
Percent Change of the Sum of the Eyes, Mouth, Speech and Swallowing Subscores of the Burke-Fahn-Marsden (BFM) Dystonia Scale.
Time frame: 6 and 14 weeks compared to baseline
The Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) is a measure of dystonia severity. The scale consists of evaluation of nine body parts (eyes, mouth, speech, swallowing, neck, trunk, right arm, right leg, left arm and left leg). The severity and provoking factors for each part are rated using a 5-point scale. These range from 0 (indicating no dystonia) to 4 (indicating the presence of dystonia at rest). The primary outcome measure includes the sum of the eyes, mouth, speech and swallowing subscores and represents the percent change from baseline to either 6 weeks or 14 weeks (representing the end of the 3 week period at the maximum tolerated dose for Levetiracetam or Placebo). The total range for these combined sub scores is 0-16, with higher scores indicating more severe dystonia and 0 indicating absence of dystonia.
2 secondary outcomes
Percent Change of the Sum of the Eyes, Mouth, Speech and Swallowing Subscores of Burke-Fahn-Marsten Dystonia Rating Scale (BFM)
Time frame: 3 and 11 weeks compared to baseline
Percent Change of the Sum of Eyes and Upper Face, Lower Face and Jaw and Tongue Subscores of the GDS Rating Scale
Time frame: 3, 6, 11 and 14 compared to baseline
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Oromandibular Dystonia
RecruitingObservational study
Brain Networks in Dystonia
Spasmodic Dysphonia, Singer's Dystonia
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.
Mark Hallett, M.D. principal investigator
National Institute of Neurological Disorders and Stroke (NINDS)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Levetiracetam in people with Oromandibular Dystonia and Cranial Dystonia, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
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 7 inclusion and 7 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 11 Jul 2017. 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 United States. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Jul 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percent Change of the Sum of the Eyes, Mouth, Speech and Swallowing Subscores of the Burke-Fahn-Marsden (BFM) Dystonia Scale. (time frame: 6 and 14 weeks compared to baseline). 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 11 Jul 2017.