Oral DystoniaPhase 1/2NCT01921270
Dysport for the Treatment of OMD
A Phase 1/2 study testing Low Dose - AbobotulinumtoxinA in people with Oral Dystonia and Tardive Dystonia, run by Emory University at 1 site in United States.
Trial summary
The purpose of this study is to study the efficacy and safety of AbobotulinumtoxinA (Dysport) for use in Oromandibular Dystonia (OMD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Oral Dystonia, Tardive Dystonia
- What is being tested?
- Low Dose - AbobotulinumtoxinA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2013to 8 Feb 2017
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- a diagnosis of primary or tardive OMD
- moderate or severe severity, defined as GDS score ≥4 in either "lower face" or "jaw and tongue" section
- capability of attending the scheduled visits
- only those who have been previously injected with onabotulinumtoxinA and responded to that treatment, and are at least 12 weeks post last injection
- Women of childbearing age need to use contraception in order to be included.
Who cannot join
- Existence of a systemic disease that could confound the evaluation
- previous placement of Deep Brain Stimulation electrodes to treat dystonia
- concomitant oral medications that could interfere with the action of botulinum toxin Type A (e.g., aminoglycosides)
- on an unstable dosage of any medication prescribed to treat dystonia (e.g., benzodiazepines, baclofen or anticholinergics)
- any known hypersensitivity to any botulinum toxin preparation and allergy to cow's milk protein
- immunoresistance to other forms of botulinum toxin type A
Show all 13Show fewer
- existence of a concomitant neuromuscular disorder (e.g., Myasthenia Gravis or Lambert-Eaton syndrome, etc)
- infection at the proposed injection sites
- pregnant women
- women of childbearing age NOT on contraception
- breastfeeding women
- inability to comply with scheduled visits
- patients who had been previously injected with botulinum toxin type A but who did not respond
Trial locations and contacts
1 site in 1 country.
United States1 site
Wesley Woods Health Center; Emory University Hospital
Atlanta, Georgia, 30329
About this study
Official title: A Pilot Dose Ranging Study of Dysport® (AbobotulinumtoxinA) in the Treatment of Oromandibular Dystonia
The purpose of this study is to study the efficacy and safety of AbobotulinumtoxinA (Dysport) for use in Oromandibular Dystonia (OMD).
Read the detailed description
Oromandibular dystonia (OMD) is an uncommon, disabling form of cranial dystonia, involving involuntary movements of the lower facial, masticatory, and lingual muscles. This can cause jaw movements including opening, closure, protrusion, retraction, or deviation. Common additional facial movements involve grimacing or lip pursing. When there is tongue involvement, it usually presents as tongue protrusion or curling. Such patients are impaired in relation to eating, speaking and swallowing
This study aims to evaluate the efficacy and safety of a low dose of Dysport® deemed tolerable during phase 1 in subjects with oromandibular dystonia (OMD).
Participants will be injected with Dysport® only, with an unblinded open-label disclosure. The safety and efficacy pf receiving Dysport® will be recorded for all subjects that undergo injection. All subjects will be examined and videotaped at the injection visit, then at 6 and 12 weeks after injection with a standardized protocol. The primary outcome will be blinded examination scores of the videos performed after the study is complete.The evaluators will be three different movement disorders experts, not otherwise involved in the study, who will review the videotaped examinations, presented in a random order, using the Global Dystonia Rating scale (GDS). Evaluators will rate the dystonia at baseline (injection visit) and 6 weeks after injection.
- Conditions
- Oral Dystonia, Tardive Dystonia
- Being tested
- Low Dose - AbobotulinumtoxinA
- Keywords
- Oromandibular Dystonia, Oral Dystonia, Tardive Dystonia, botulinum toxin, abobotulinumtoxinA
- Medical subject headings
- Tardive Dyskinesia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low Dose - AbobotulinumtoxinADrug
Also called Dysport
Participants will receive low dose AbobotulinumtoxinA injections. Location of injections will be determined by the clinician to treat the individual's dystonic symptom.
Muscles that may be included for in injection for OMD with Jaw Closing:
Medial Pterygoid 50 units, Masseter 25 units
Muscles that may be included in injection for OMD with Jaw Opening:
Lateral Pterygoid 50 units, Anterior digastrics 10 units
Muscle that will be included in injection for OMD with Tongue Protrusion:
Genioglossus 7.5 units
| Study group | Role | What they receive |
|---|---|---|
| Dysport Injections | Experimental | Participants with OMD who have been previously treated with any botulinum toxin Type A will be injected with Dysport®. Receives: Drug: Low Dose - AbobotulinumtoxinA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater
This scale measures the severity of dystonia for the jaw and tongue by a blinded rater. Dystonia is rated from 0 to 10:
0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia
Measured: Baseline, Week 6, Week 12
Secondary outcomes (12)
Change in Analogue Pain Scale Score
Measure of jaw pain by visual analogue scale (0-100) where 0 represents "no pain" and 100 represents the "most severe pain".
Measured: Baseline, Week 12
Mean Sialorrhea Clinical Scale for Parkinson's Disease (SCS-PD) Score
The SCS-PD measures drooling. Individual items are scored on a scale from 0-3 where 0 represents "never" and 3 represents "always". The overall maximum score is 21. A higher score indicates greater drooling severity. A lower score indicates lesser severity.
Measured: Baseline, Week 6, Week 12
Change in Number of Tongue Bites Per Day
The patient will be asked to estimate how many times they tend to accidentally/involuntarily bite their tongue per day.
Measured: Baseline, Week 12
Mean Swallowing Disturbance Questionnaire (SDQ-20) Score
Ease of chewing and swallowing will be assessed by the SDQ-20 (modified to exclude question 5 due to redundancy as it relates to drooling and question 15 which is not relevant to the study as it involves prior aspiration pneumonias). Individual items are scored from 0 (never) to 3 (very frequently). The overall score is the total for all items; a higher score indicating more frequent swallowing disturbance; a lower score indicating no or less frequent disturbance, with a possible maximum score of 39.
Measured: Baseline, Week 6, Week 12
Mean Fahn-Marsden Part B "Speech" Question (BFM-q21) Rating
The Fahn-Marsden Part B "Speech" Question assesses the ease of producing speech. Responses range from 0=Normal, 1=Slightly involved, easily understood, 2=Some difficulty understanding, 3=Marked difficulty understanding.
Measured: Baseline, Week 6, Week 12
Mean Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25) Score
The OMDQ-25 is a subjective quality of life measurement made for patients with Oromandibular Dystonia. The maximum total score is 100 indicating the highest quality of life. A score of 50 indicates a mediocre quality of life. A lower score indicates perceived lower quality of life.
Measured: Baseline, Week 6, Week 12
Mean Global Clinical Impression - Improvement Scale (CGI) Index Score
The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. Responses are scored on a scale from 1 to 7; 1 represents "very much improved" and 7 represents "very much worse".
Measured: Week 6, Week 12
Mean Global Clinical Impression Scale (CGI-S) With Severity Index Score
The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Responses are scored on a scale from 1 to 7; 1 represents "normal, not at all ill" and 7 represents "among the most extremely ill patients".
Measured: Baseline, Week 6, Week 12
Mean Global Clinical Impression- Efficacy Index Score
The Clinical Global Impression - Efficacy Index is a 4×4 rating scale that assesses the therapeutic effect of treatment. Responses range on a scale from 0 to 4 with 4 being the best response.
Measured: Week 6, Week 12
Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Un-blinded Rater
The UDRS measures dystonia severity. The UDRS is being rated by un-blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia.
Measured: Baseline, Week 6, Week 12
Mean Global Dystonia Rating Scale Score as Measured by Un-blinded Rater
This scale measures the severity of dystonia for the jaw and tongue by an un-blinded rater. Dystonia is rated from 0 to 10:
0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia
Measured: Baseline, Week 6, Week 12
Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Blinded Rater
The UDRS measures dystonia severity. The UDRS is being rated by blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia.
Measured: Baseline, Week 6, Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2013 (estimated)
- Study startAug 2013
- Primary completion8 Feb 2017
- Study completion8 Feb 2017
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.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Oral DystoniaStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Ipsen
- Responsible party
- Principal investigator, Stewart Factor, Emory University
- Investigators
- Stewart A Factor, DO, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01921270
- Sponsor's protocol ID
- IRB00064292
- Status last verified
- Oct 2017
- First submitted
- 9 Aug 2013
- Last updated
- 17 Nov 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Tan EK, Jankovic J. Botulinum toxin A in patients with oromandibular dystonia: long-term follow-up. Neurology. 1999 Dec 10;53(9):2102-7. doi: 10.1212/wnl.53.9.2102.
PubMed 10599789Rosales RL, Ng AR, Santos MM, Fernandez HH. The broadening application of chemodenervation in X-linked dystonia-parkinsonism (Part II): an open-label experience with botulinum toxin-A (Dysport(R)) injections for oromandibular, lingual, and truncal-axial dystonias. Int J Neurosci. 2011;121 Suppl 1:44-56. doi: 10.3109/00207454.2011.558260.
PubMed 21348790
Study at a glance
From the registry record, last updated by the sponsor on 17 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Dose Ranging Study of Dysport® (AbobotulinumtoxinA) in the Treatment of Oromandibular Dystonia
- ClinicalTrials.gov ID
- NCT01921270
- Acronym / sponsor ID
- IRB00064292
- Conditions
- Oral Dystonia, Tardive Dystonia
- Keywords
- Oromandibular Dystonia, Oral Dystonia, Tardive Dystonia, botulinum toxin, abobotulinumtoxinA
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Ipsen
- Study start
- Aug 2013
- Primary completion
- 8 Feb 2017 (actual)
- Study completion
- 8 Feb 2017 (actual)
- First posted
- 13 Aug 2013 (estimated)
- Results
- Posted 17 Nov 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to study the efficacy and safety of AbobotulinumtoxinA (Dysport) for use in Oromandibular Dystonia (OMD).
Detailed description
Oromandibular dystonia (OMD) is an uncommon, disabling form of cranial dystonia, involving involuntary movements of the lower facial, masticatory, and lingual muscles. This can cause jaw movements including opening, closure, protrusion, retraction, or deviation. Common additional facial movements involve grimacing or lip pursing. When there is tongue involvement, it usually presents as tongue protrusion or curling. Such patients are impaired in relation to eating, speaking and swallowing
This study aims to evaluate the efficacy and safety of a low dose of Dysport® deemed tolerable during phase 1 in subjects with oromandibular dystonia (OMD).
Participants will be injected with Dysport® only, with an unblinded open-label disclosure. The safety and efficacy pf receiving Dysport® will be recorded for all subjects that undergo injection. All subjects will be examined and videotaped at the injection visit, then at 6 and 12 weeks after injection with a standardized protocol. The primary outcome will be blinded examination scores of the videos performed after the study is complete.The evaluators will be three different movement disorders experts, not otherwise involved in the study, who will review the videotaped examinations, presented in a random order, using the Global Dystonia Rating scale (GDS). Evaluators will rate the dystonia at baseline (injection visit) and 6 weeks after injection.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dysport Injections | Experimental | Drug: Low Dose - AbobotulinumtoxinAParticipants with OMD who have been previously treated with any botulinum toxin Type A will be injected with Dysport®. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- a diagnosis of primary or tardive OMD
- moderate or severe severity, defined as GDS score ≥4 in either "lower face" or "jaw and tongue" section
- capability of attending the scheduled visits
- only those who have been previously injected with onabotulinumtoxinA and responded to that treatment, and are at least 12 weeks post last injection
- Women of childbearing age need to use contraception in order to be included.
Key exclusion criteria (13 listed)
- Existence of a systemic disease that could confound the evaluation
- previous placement of Deep Brain Stimulation electrodes to treat dystonia
- concomitant oral medications that could interfere with the action of botulinum toxin Type A (e.g., aminoglycosides)
- on an unstable dosage of any medication prescribed to treat dystonia (e.g., benzodiazepines, baclofen or anticholinergics)
- any known hypersensitivity to any botulinum toxin preparation and allergy to cow's milk protein
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 |
|---|---|---|
| Low Dose - AbobotulinumtoxinAAlso: DysportParticipants will receive low dose AbobotulinumtoxinA injections. Location of injections will be determined by the clinician to treat the individual's dystonic symptom. Muscles that may be included for in injection for OMD with Jaw Closing: Medial Pterygoid 50 units, Masseter 25 units Muscles that may be included in injection for OMD with Jaw Opening: Lateral Pterygoid 50 units, Anterior digastrics 10 units Muscle that will be included in injection for OMD with Tongue Protrusion: Genioglossus 7.5 units | Drug | Dysport Injections |
Primary outcome
Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater
Time frame: Baseline, Week 6, Week 12
This scale measures the severity of dystonia for the jaw and tongue by a blinded rater. Dystonia is rated from 0 to 10: 0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia
12 secondary outcomes
Change in Analogue Pain Scale Score
Time frame: Baseline, Week 12
Mean Sialorrhea Clinical Scale for Parkinson's Disease (SCS-PD) Score
Time frame: Baseline, Week 6, Week 12
Change in Number of Tongue Bites Per Day
Time frame: Baseline, Week 12
Mean Swallowing Disturbance Questionnaire (SDQ-20) Score
Time frame: Baseline, Week 6, Week 12
Mean Fahn-Marsden Part B "Speech" Question (BFM-q21) Rating
Time frame: Baseline, Week 6, Week 12
Mean Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25) Score
Time frame: Baseline, Week 6, Week 12
Mean Global Clinical Impression - Improvement Scale (CGI) Index Score
Time frame: Week 6, Week 12
Mean Global Clinical Impression Scale (CGI-S) With Severity Index Score
Time frame: Baseline, Week 6, Week 12
Mean Global Clinical Impression- Efficacy Index Score
Time frame: Week 6, Week 12
Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Un-blinded Rater
Time frame: Baseline, Week 6, Week 12
Mean Global Dystonia Rating Scale Score as Measured by Un-blinded Rater
Time frame: Baseline, Week 6, Week 12
Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Blinded Rater
Time frame: Baseline, Week 6, Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 10599789Tan EK, Jankovic J. Botulinum toxin A in patients with oromandibular dystonia: long-term follow-up. Neurology. 1999 Dec 10;53(9):2102-7. doi: 10.1212/wnl.53.9.2102.
- Background: PubMed 21348790Rosales RL, Ng AR, Santos MM, Fernandez HH. The broadening application of chemodenervation in X-linked dystonia-parkinsonism (Part II): an open-label experience with botulinum toxin-A (Dysport(R)) injections for oromandibular, lingual, and truncal-axial dystonias. Int J Neurosci. 2011;121 Suppl 1:44-56. doi: 10.3109/00207454.2011.558260.
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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.
Stewart A Factor, DO principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Low Dose - AbobotulinumtoxinA in people with Oral Dystonia and Tardive Dystonia, run by Emory University 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 5 inclusion and 13 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 17 Nov 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 17 Nov 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?
Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater (time frame: Baseline, Week 6, Week 12). 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 NCT01921270, which also lists 2 linked publications.
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 17 Nov 2017.