MyoclonusPhase 2NCT00506012
Pilot Efficacy Study of T2000 in Myoclonus Dystonia
A Phase 2 study testing T2000 in people with Myoclonus, run by Sun Pharmaceutical Industries, Inc. at 1 site in Canada.
Trial summary
This pilot study will evaluate the safety and efficacy of once daily T2000 when used to treat patients with Myoclonus Dystonia over a 12 week period. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Myoclonus
- What is being tested?
- T2000
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteCanada
- Study dates
- Aug 2007to Oct 2011
- Phase
- Phase 2
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients should meet diagnostic criteria for M-D based on the following criteria:
- myoclonus is the primary feature; focal or segmental dystonia of any severity may also be present
- symptoms began by age 20
- a familial pattern should be present
- neurological history should not be suggestive of a different neurological condition
- investigations such as imaging, EEG and evoked potential tests should be normal
- Patients will be eligible for this study if they are symptomatic on their current treatment, cannot tolerate current therapies, or are treatment naïve patients who have been explained treatment alternatives.
Who cannot join
- Patients adequately controlled without side effects on a current M-D treatment
- Current treatment with a barbiturate such as phenobarbital or primidone
- Pregnant patients or patients who may become pregnant during the study
- Patients who must take medications that alter liver metabolism as well as patients with liver disease or coagulation disorders
- Patients with seizure disorders
- Patients with a history of allergy or hypersensitivity reaction to barbiturates or other related medications, such as phenobarbital or phenytoin
- Patient with significant general medical or clinical laboratory abnormalities
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Efficacy and Safety of Taro Pharmaceuticals' Pro-Drug T2000 (1,3-Dimethoxymethyl-5,5-Diphenyl-Barbituric Acid) In Patients With Myoclonus Dystonia: An Open Label Sequential Dose Escalation Study
This pilot study will evaluate the safety and efficacy of once daily T2000 when used to treat patients with Myoclonus Dystonia over a 12 week period.
Read the detailed description
Myoclonus Dystonia (M-D) is a rare, inherited movement disorder in which patients experience myoclonus - sudden, brief, jerky involuntary motions, often in association with dystonia - involuntary sustained contractions causing twisting or abnormal posture. While most M-D patients respond significantly to alcohol, there are no approved medications for M-D. A variety of medications are currently used to treat M-D, but these treatments work in a small proportion of patients and provide only partial improvement in symptoms; their use is also limited by side-effects in many patients.
T2000 is a medication currently under development for the treatment of movement disorders, including essential tremor (ET). Although T2000 is a new medication, it belongs to a class of medications that has been used for many years for the treatment of a variety of medical conditions. In previous studies, T2000 appeared to be effective in controlling symptoms of ET and some patients with severe ET had major improvements in tremor. As would be expected for medications in this class, T2000 can cause sedation at high blood levels, such as may be seen when large doses are given to older individuals. In younger patients, T2000 caused only minimal side effects even when administered at high doses and for periods of several weeks to several months.
The current study will evaluate the safety and efficacy of T2000 in patients with M-D. Patients will receive doses of T2000 beginning at 200 mg a day and increasing every other week by an additional 200 mg a day up to a maximal dose of 1000 mg a day. The total duration of treatment will be 12 weeks. Patient's symptoms of myoclonus and dystonia, as well as overall neurological examination, will be monitored throughout the study. The response to T2000 will be determined by comparing the severity of myoclonus and dystonia while patients are receiving T2000 compared to the symptoms observed without active medication.
- Conditions
- Myoclonus
- Being tested
- T2000
- Keywords
- Myoclonus Dystonia, Essential Myoclonus, Inherited Myoclonus, Dyskinesia, Movement Disorder
- Medical subject headings
- Myoclonus, Myoclonic dystonia, Dyskinesias, Movement Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
T2000Drug
T2000 at doses of 200 mg a day to 1000 mg a day
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | T2000 Receives: Drug: T2000 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of treatment on the movement disorder will be measured by a myoclonus scale and a dystonia scale as well as by assessment of overall functional status. Response at various dosages will be compared to baseline for all patients.
Measured: Up to 12 weeks
Secondary outcomes (1)
Safety parameters including neurological examination, blood tests and EKG will be monitored throughout the treatment period and during withdrawal of the medication.
Measured: Up to 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2007 (estimated)
- Study startAug 2007
- Primary completionAug 2011
- Study completionOct 2011
Why it stopped: Slow Recruitment of eligible patients
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for MyoclonusStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sun Pharmaceutical Industries, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00506012
- Sponsor's protocol ID
- T2000-0633
- Status last verified
- Nov 2013
- First submitted
- 20 Jul 2007
- Last updated
- 23 Dec 2013
- Results posted
- No
Publications
Melmed C, Moros D, Rutman H. Treatment of essential tremor with the barbiturate T2000 (1,3-dimethoxymethyl-5,5-diphenyl-barbituric acid). Mov Disord. 2007 Apr 15;22(5):723-7. doi: 10.1002/mds.21321.
PubMed 17265458
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Efficacy and Safety of Taro Pharmaceuticals' Pro-Drug T2000 (1,3-Dimethoxymethyl-5,5-Diphenyl-Barbituric Acid) In Patients With Myoclonus Dystonia: An Open Label Sequential Dose Escalation Study
- ClinicalTrials.gov ID
- NCT00506012
- Acronym / sponsor ID
- T2000-0633
- Conditions
- Myoclonus
- Keywords
- Myoclonus Dystonia, Essential Myoclonus, Inherited Myoclonus, Dyskinesia, Movement Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow Recruitment of eligible patients
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Sun Pharmaceutical Industries, Inc.Industry (pharma, biotech, devices)
- Study start
- Aug 2007
- Primary completion
- Aug 2011 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 25 Jul 2007 (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.
This pilot study will evaluate the safety and efficacy of once daily T2000 when used to treat patients with Myoclonus Dystonia over a 12 week period.
Detailed description
Myoclonus Dystonia (M-D) is a rare, inherited movement disorder in which patients experience myoclonus - sudden, brief, jerky involuntary motions, often in association with dystonia - involuntary sustained contractions causing twisting or abnormal posture. While most M-D patients respond significantly to alcohol, there are no approved medications for M-D. A variety of medications are currently used to treat M-D, but these treatments work in a small proportion of patients and provide only partial improvement in symptoms; their use is also limited by side-effects in many patients.
T2000 is a medication currently under development for the treatment of movement disorders, including essential tremor (ET). Although T2000 is a new medication, it belongs to a class of medications that has been used for many years for the treatment of a variety of medical conditions. In previous studies, T2000 appeared to be effective in controlling symptoms of ET and some patients with severe ET had major improvements in tremor. As would be expected for medications in this class, T2000 can cause sedation at high blood levels, such as may be seen when large doses are given to older individuals. In younger patients, T2000 caused only minimal side effects even when administered at high doses and for periods of several weeks to several months.
The current study will evaluate the safety and efficacy of T2000 in patients with M-D. Patients will receive doses of T2000 beginning at 200 mg a day and increasing every other week by an additional 200 mg a day up to a maximal dose of 1000 mg a day. The total duration of treatment will be 12 weeks. Patient's symptoms of myoclonus and dystonia, as well as overall neurological examination, will be monitored throughout the study. The response to T2000 will be determined by comparing the severity of myoclonus and dystonia while patients are receiving T2000 compared to the symptoms observed without active medication.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: T2000T2000 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients should meet diagnostic criteria for M-D based on the following criteria:
- Patients will be eligible for this study if they are symptomatic on their current treatment, cannot tolerate current therapies, or are treatment naïve patients who have been explained treatment alternatives.
Key exclusion criteria (7 listed)
- Patients adequately controlled without side effects on a current M-D treatment
- Current treatment with a barbiturate such as phenobarbital or primidone
- Pregnant patients or patients who may become pregnant during the study
- Patients who must take medications that alter liver metabolism as well as patients with liver disease or coagulation disorders
- Patients with seizure disorders
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 |
|---|---|---|
| T2000T2000 at doses of 200 mg a day to 1000 mg a day | Drug | 1 |
Primary outcome
Effect of treatment on the movement disorder will be measured by a myoclonus scale and a dystonia scale as well as by assessment of overall functional status. Response at various dosages will be compared to baseline for all patients.
Time frame: Up to 12 weeks
1 secondary outcome
Safety parameters including neurological examination, blood tests and EKG will be monitored throughout the treatment period and during withdrawal of the medication.
Time frame: Up to 16 weeks
Results and safety
The study was stopped early (reason given: Slow Recruitment of eligible patients). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2013.
- Background: PubMed 17265458Melmed C, Moros D, Rutman H. Treatment of essential tremor with the barbiturate T2000 (1,3-dimethoxymethyl-5,5-diphenyl-barbituric acid). Mov Disord. 2007 Apr 15;22(5):723-7. doi: 10.1002/mds.21321.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 T2000 in people with Myoclonus, run by Sun Pharmaceutical Industries, Inc. at 1 site in Canada.
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: stopped early, as of the sponsor's last update on 23 Dec 2013. 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 Canada. 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?
The study was stopped early (reason given: Slow Recruitment of eligible patients). Results are not available in the source record.
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?
Effect of treatment on the movement disorder will be measured by a myoclonus scale and a dystonia scale as well as by assessment of overall functional status. Response at various dosages will be compared to baseline for all patients. (time frame: Up to 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00506012, which also lists 1 linked publication.
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 23 Dec 2013.