Spasmodic DysphoniaNCT03042962
Brain Networks in Dystonia
An observational study of Spasmodic Dysphonia and Singer's Dystonia, run by Massachusetts Eye and Ear Infirmary at 1 site in United States.
Trial summary
To date, there is only limited knowledge about the distinct neural abnormalities that lead to the development of different forms of focal dystonia. The goal of this study is to dissect the pathophysiological mechanisms underlying this clinical phenomenon using multi-level brain network analysis in patients with focal… Read more about this study
- Who is it for?
- Ages 21 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Spasmodic Dysphonia, Singer's Dystonia, Writer's Cramp
- What is being tested?
- MRI and Blood draw
- Type of treatment
- Surgery or procedure
- How many people
- 141 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2015to 31 Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 21 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Patients with focal dystonia, thier unaffected blood relatives, and healthy volunteers
Who may be able to join
- Patients will have clinically documented focal dystonia
- Unaffected relatives of patients with focal dystonia
- Healthy controls will be healthy volunteers with a negative history of neurological, laryngeal or psychiatric problems
- Age from 21 to 80 years.
- Native English speakers.
- Right-handedness (based on Edinburgh Handedness Inventory).
Who cannot join
- Subjects who are incapable of giving an informed consent.
- Pregnant or breastfeeding women until a time when they are no longer pregnant or breastfeeding. All women of childbearing potential will have a urine pregnancy test performed, which must be negative for participation in the imaging studies.
- Subjects with past or present medical history of (a) neurological problems, such as stroke, movement disorders (other than dystonia in the patient groups), brain tumors, traumatic brain injury with loss of consciousness, ataxias, myopathies, myasthenia gravis, demyelinating diseases, alcoholism, drug depend-ence; (b) psychiatric problems, such as schizophrenia, major and/or bipolar depression, obsessive-compulsive disorder; (c) laryngeal problems, such as vocal fold paralysis, paresis, vocal fold nodules and polyps, carcinoma, chronic laryngitis.
- Patients who are not symptomatic due to treatment with botulinum toxin injections into the laryngeal muscles. The duration of positive effects of botulinum toxin vary from patient to patient but lasts on average for 3-4 months. All patients will be evaluated to ensure that they are fully symptomatic prior to entering the study.
- Patients with other forms of dystonia.
- Patients who have dystonia symptoms at rest in order to avoid the potential confound of dystonic spasms occurring during the scanning.
Show all 9Show fewer
- To avoid the possibility of confounding effects of drugs acting upon the central nervous system, all study participants will be questioned about any prescribed or over-the-counter medications as part of their initial intake screening. Those patients who receive medication(s) affecting the central nervous system will be excluded from the study.
- The patients will be asked whether they have undergone any head, neck, or hand surgeries, which resulted in changes in regional anatomy or innervation. Because brain, hand and laryngeal surgery may potentially lead to the brain structure and function re-organization, all patients with history of brain, hand and/or laryngeal surgery will be excluded from the study.
- Subjects who have tattoos, ferromagnetic objects in their bodies (e.g., implanted stimulators, surgical clips, prosthesis, artificial heart valve, etc.) that cannot be removed for the purpose of study participation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Massachusetts Eye and Ear
Boston, Massachusetts, 02114
Kristina Simonyan, MD, PhD, 617-573-6016, simonyan_lab@meei.harvard.edu
Kristina Simonyan, MD, PhD
About this study
To date, there is only limited knowledge about the distinct neural abnormalities that lead to the development of different forms of focal dystonia. The goal of this study is to dissect the pathophysiological mechanisms underlying this clinical phenomenon using multi-level brain network analysis in patients with focal dystonia.
Read the detailed description
Among the many causes of craniofacial disease are focal dystonias such as blepharospasm (BSP) and oromandibular dystonia (OMD), affecting the eyes and jaw, respectively, as well as Meige Syndrome, which combines features of both. Craniofacial dystonias are poorly understood and have limited treatment options. The investigators hypothesize that craniofacial dystonia (CFD) may be caused by both rare and common genetic variants. To identify neural correlates of different genetic causes of CFD, the investigators will perform structural and functional whole-brain imaging and examine the organization of the neural network in these patients compared to healthy individuals and their unaffected blood relatives.
- Conditions
- Spasmodic Dysphonia, Singer's Dystonia, Writer's Cramp, Musician's Focal Hand Dystonia, Craniofacial Dystonia, Blepharospasm Oromandibular Dystonia
- Being tested
- MRI, Blood draw
- Keywords
- dystonia, imaging, genetics
- Medical subject headings
- Dysphonia, Dystonic Disorders, Meige Syndrome, Dystonia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRIOther
Functional and structural MRI of the brain will be conducted to identify disorder specific neural markers
Blood drawProcedure or surgery
Blood samples will be collected, the DNA will be extracted and banked for genetic studies
| Study group | Role | What they receive |
|---|---|---|
| Patients with dystonia and their unaffected relatives | Patients with dystonia and their unaffected blood relatives will undergo MRI of the brain and blood draw. Receives: Other: MRI; Procedure: Blood draw | |
| Healthy volunteers | Healthy subjects without any neurological, psychiatric or otolaryngological problems will undergo MRI of the brain and blood draw. Receives: Other: MRI; Procedure: Blood draw |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of brain structural and functional changes
Identify changes in brain activity and gray and white matter in patients with dystonia vs. unaffected relatives vs. healthy controls
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2017 (estimated)
- Study start1 Aug 2015
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (26)Papers the sponsor has linked to this study.
- Other trials for Spasmodic DysphoniaStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 141 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts Eye and Ear Infirmary
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Dental and Craniofacial Research (NIDCR)
- Responsible party
- Principal investigator, Kristina Simonyan, Massachusetts Eye and Ear Infirmary
- Investigators
- Kristina Simonyan, MD, PhD, Principal investigator, Massachusetts Eye and Ear Infirmary
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03042962
- Sponsor's protocol ID
- 2019P001183
- Other IDs
- R01DE030464
- Status last verified
- Dec 2025
- First submitted
- 31 Jan 2017
- Last updated
- 8 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Battistella G, Termsarasab P, Ramdhani RA, Fuertinger S, Simonyan K. Isolated Focal Dystonia as a Disorder of Large-Scale Functional Networks. Cereb Cortex. 2017 Feb 1;27(2):1203-1215. doi: 10.1093/cercor/bhv313.
PubMed 26679193Fuertinger S, Simonyan K. Stability of Network Communities as a Function of Task Complexity. J Cogn Neurosci. 2016 Dec;28(12):2030-2043. doi: 10.1162/jocn_a_01026. Epub 2016 Aug 30.
PubMed 27575646Rittiner JE, Caffall ZF, Hernandez-Martinez R, Sanderson SM, Pearson JL, Tsukayama KK, Liu AY, Xiao C, Tracy S, Shipman MK, Hickey P, Johnson J, Scott B, Stacy M, Saunders-Pullman R, Bressman S, Simonyan K, Sharma N, Ozelius LJ, Cirulli ET, Calakos N. Functional Genomic Analyses of Mendelian and Sporadic Disease Identify Impaired eIF2alpha Signaling as a Generalizable Mechanism for Dystonia. Neuron. 2016 Dec 21;92(6):1238-1251. doi: 10.1016/j.neuron.2016.11.012. Epub 2016 Dec 8.
PubMed 27939583Fuertinger S, Simonyan K. Connectome-Wide Phenotypical and Genotypical Associations in Focal Dystonia. J Neurosci. 2017 Aug 2;37(31):7438-7449. doi: 10.1523/JNEUROSCI.0384-17.2017. Epub 2017 Jul 3.
PubMed 28674168Simonyan K, Barkmeier-Kraemer J, Blitzer A, Hallett M, Houde JF, Jacobson Kimberley T, Ozelius LJ, Pitman MJ, Richardson RM, Sharma N, Tanner K; The NIH/NIDCD Workshop on Research Priorities in Spasmodic Dysphonia/Laryngeal Dystonia. Laryngeal Dystonia: Multidisciplinary Update on Terminology, Pathophysiology, and Research Priorities. Neurology. 2021 May 25;96(21):989-1001. doi: 10.1212/WNL.0000000000011922. Epub 2021 Apr 15.
PubMed 33858994Worthley A, Simonyan K. Suicidal Ideations and Attempts in Patients With Isolated Dystonia. Neurology. 2021 Mar 16;96(11):e1551-e1560. doi: 10.1212/WNL.0000000000011596. Epub 2021 Jan 27.
PubMed 33504639Valeriani D, Simonyan K. A microstructural neural network biomarker for dystonia diagnosis identified by a DystoniaNet deep learning platform. Proc Natl Acad Sci U S A. 2020 Oct 20;117(42):26398-26405. doi: 10.1073/pnas.2009165117. Epub 2020 Oct 1.
PubMed 33004625Hanekamp S, Simonyan K. The large-scale structural connectome of task-specific focal dystonia. Hum Brain Mapp. 2020 Aug 15;41(12):3253-3265. doi: 10.1002/hbm.25012. Epub 2020 Apr 20.
PubMed 32311207Maguire F, Reilly RB, Simonyan K. Normal Temporal Discrimination in Musician's Dystonia Is Linked to Aberrant Sensorimotor Processing. Mov Disord. 2020 May;35(5):800-807. doi: 10.1002/mds.27984. Epub 2020 Jan 13.
PubMed 31930574Lungu C, Ozelius L, Standaert D, Hallett M, Sieber BA, Swanson-Fisher C, Berman BD, Calakos N, Moore JC, Perlmutter JS, Pirio Richardson SE, Saunders-Pullman R, Scheinfeldt L, Sharma N, Sillitoe R, Simonyan K, Starr PA, Taylor A, Vitek J; participants and organizers of the NINDS Workshop on Research Priorities in Dystonia. Defining research priorities in dystonia. Neurology. 2020 Mar 24;94(12):526-537. doi: 10.1212/WNL.0000000000009140. Epub 2020 Feb 25.
PubMed 32098856Guiry S, Worthley A, Simonyan K. A separation of innate and learned vocal behaviors defines the symptomatology of spasmodic dysphonia. Laryngoscope. 2019 Jul;129(7):1627-1633. doi: 10.1002/lary.27617. Epub 2018 Dec 24.
PubMed 30582159Bianchi S, Fuertinger S, Huddleston H, Frucht SJ, Simonyan K. Functional and structural neural bases of task specificity in isolated focal dystonia. Mov Disord. 2019 Apr;34(4):555-563. doi: 10.1002/mds.27649. Epub 2019 Mar 6.
PubMed 30840778Simonyan K. Recent advances in understanding the role of the basal ganglia. F1000Res. 2019 Jan 30;8:F1000 Faculty Rev-122. doi: 10.12688/f1000research.16524.1. eCollection 2019.
PubMed 30755797Fuertinger S, Simonyan K. Task-specificity in focal dystonia is shaped by aberrant diversity of a functional network kernel. Mov Disord. 2018 Dec;33(12):1918-1927. doi: 10.1002/mds.97. Epub 2018 Sep 27.
PubMed 30264427Simonyan K. Neuroimaging Applications in Dystonia. Int Rev Neurobiol. 2018;143:1-30. doi: 10.1016/bs.irn.2018.09.007. Epub 2018 Oct 23.
PubMed 30473192
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Brain Networks in Dystonia
- ClinicalTrials.gov ID
- NCT03042962
- Acronym / sponsor ID
- 2019P001183
- Conditions
- Spasmodic Dysphonia, Singer's Dystonia, Writer's Cramp, Musician's Focal Hand Dystonia, Craniofacial Dystonia, Blepharospasm Oromandibular Dystonia
- Keywords
- dystonia, imaging, genetics
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 141 participants (estimated)
- Lead sponsor
- Massachusetts Eye and Ear InfirmaryUniversity, hospital or other
- Collaborators
- National Institute of Dental and Craniofacial Research (NIDCR)
- Study start
- 1 Aug 2015 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 3 Feb 2017 (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.
To date, there is only limited knowledge about the distinct neural abnormalities that lead to the development of different forms of focal dystonia. The goal of this study is to dissect the pathophysiological mechanisms underlying this clinical phenomenon using multi-level brain network analysis in patients with focal dystonia.
Detailed description
Among the many causes of craniofacial disease are focal dystonias such as blepharospasm (BSP) and oromandibular dystonia (OMD), affecting the eyes and jaw, respectively, as well as Meige Syndrome, which combines features of both. Craniofacial dystonias are poorly understood and have limited treatment options. The investigators hypothesize that craniofacial dystonia (CFD) may be caused by both rare and common genetic variants. To identify neural correlates of different genetic causes of CFD, the investigators will perform structural and functional whole-brain imaging and examine the organization of the neural network in these patients compared to healthy individuals and their unaffected blood relatives.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with dystonia and their unaffected relatives | – | Other: MRI, Procedure: Blood drawPatients with dystonia and their unaffected blood relatives will undergo MRI of the brain and blood draw. |
| Healthy volunteers | – | Other: MRI, Procedure: Blood drawHealthy subjects without any neurological, psychiatric or otolaryngological problems will undergo MRI of the brain and blood draw. |
Who may qualify
- Ages
- 21 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with focal dystonia, thier unaffected blood relatives, and healthy volunteers
Key inclusion criteria (6 listed)
- Patients will have clinically documented focal dystonia
- Unaffected relatives of patients with focal dystonia
- Healthy controls will be healthy volunteers with a negative history of neurological, laryngeal or psychiatric problems
- Age from 21 to 80 years.
- Native English speakers.
Key exclusion criteria (9 listed)
- Subjects who are incapable of giving an informed consent.
- Pregnant or breastfeeding women until a time when they are no longer pregnant or breastfeeding. All women of childbearing potential will have a urine pregnancy test performed, which must be negative for participation in the imaging studies.
- Subjects with past or present medical history of (a) neurological problems, such as stroke, movement disorders (other than dystonia in the patient groups), brain tumors, traumatic brain injury with loss of consciousness, ataxias, myopathies, myasthenia gravis, demyelinating diseases, alcoholism, drug depend-ence; (b) psychiatric problems, such as schizophrenia, major and/or bipolar depression, obsessive-compulsive disorder; (c) laryngeal problems, such as vocal fold paralysis, paresis, vocal fold nodules and polyps, carcinoma, chronic laryngitis.
- Patients who are not symptomatic due to treatment with botulinum toxin injections into the laryngeal muscles. The duration of positive effects of botulinum toxin vary from patient to patient but lasts on average for 3-4 months. All patients will be evaluated to ensure that they are fully symptomatic prior to entering the study.
- Patients with other forms of dystonia.
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 |
|---|---|---|
| MRIFunctional and structural MRI of the brain will be conducted to identify disorder specific neural markers | Other | Healthy volunteers, Patients with dystonia and their unaffected relatives |
| Blood drawBlood samples will be collected, the DNA will be extracted and banked for genetic studies | Procedure or surgery | Healthy volunteers, Patients with dystonia and their unaffected relatives |
Primary outcome
Incidence of brain structural and functional changes
Time frame: 5 years
Identify changes in brain activity and gray and white matter in patients with dystonia vs. unaffected relatives vs. healthy controls
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2025.
- Results publication: PubMed 26679193Battistella G, Termsarasab P, Ramdhani RA, Fuertinger S, Simonyan K. Isolated Focal Dystonia as a Disorder of Large-Scale Functional Networks. Cereb Cortex. 2017 Feb 1;27(2):1203-1215. doi: 10.1093/cercor/bhv313.
- Results publication: PubMed 27575646Fuertinger S, Simonyan K. Stability of Network Communities as a Function of Task Complexity. J Cogn Neurosci. 2016 Dec;28(12):2030-2043. doi: 10.1162/jocn_a_01026. Epub 2016 Aug 30.
- Results publication: PubMed 27939583Rittiner JE, Caffall ZF, Hernandez-Martinez R, Sanderson SM, Pearson JL, Tsukayama KK, Liu AY, Xiao C, Tracy S, Shipman MK, Hickey P, Johnson J, Scott B, Stacy M, Saunders-Pullman R, Bressman S, Simonyan K, Sharma N, Ozelius LJ, Cirulli ET, Calakos N. Functional Genomic Analyses of Mendelian and Sporadic Disease Identify Impaired eIF2alpha Signaling as a Generalizable Mechanism for Dystonia. Neuron. 2016 Dec 21;92(6):1238-1251. doi: 10.1016/j.neuron.2016.11.012. Epub 2016 Dec 8.
- Results publication: PubMed 28674168Fuertinger S, Simonyan K. Connectome-Wide Phenotypical and Genotypical Associations in Focal Dystonia. J Neurosci. 2017 Aug 2;37(31):7438-7449. doi: 10.1523/JNEUROSCI.0384-17.2017. Epub 2017 Jul 3.
- Results publication: PubMed 33858994Simonyan K, Barkmeier-Kraemer J, Blitzer A, Hallett M, Houde JF, Jacobson Kimberley T, Ozelius LJ, Pitman MJ, Richardson RM, Sharma N, Tanner K; The NIH/NIDCD Workshop on Research Priorities in Spasmodic Dysphonia/Laryngeal Dystonia. Laryngeal Dystonia: Multidisciplinary Update on Terminology, Pathophysiology, and Research Priorities. Neurology. 2021 May 25;96(21):989-1001. doi: 10.1212/WNL.0000000000011922. Epub 2021 Apr 15.
- Results publication: PubMed 33504639Worthley A, Simonyan K. Suicidal Ideations and Attempts in Patients With Isolated Dystonia. Neurology. 2021 Mar 16;96(11):e1551-e1560. doi: 10.1212/WNL.0000000000011596. Epub 2021 Jan 27.
- Results publication: PubMed 33004625Valeriani D, Simonyan K. A microstructural neural network biomarker for dystonia diagnosis identified by a DystoniaNet deep learning platform. Proc Natl Acad Sci U S A. 2020 Oct 20;117(42):26398-26405. doi: 10.1073/pnas.2009165117. Epub 2020 Oct 1.
- Results publication: PubMed 32311207Hanekamp S, Simonyan K. The large-scale structural connectome of task-specific focal dystonia. Hum Brain Mapp. 2020 Aug 15;41(12):3253-3265. doi: 10.1002/hbm.25012. Epub 2020 Apr 20.
- Results publication: PubMed 31930574Maguire F, Reilly RB, Simonyan K. Normal Temporal Discrimination in Musician's Dystonia Is Linked to Aberrant Sensorimotor Processing. Mov Disord. 2020 May;35(5):800-807. doi: 10.1002/mds.27984. Epub 2020 Jan 13.
- Results publication: PubMed 32098856Lungu C, Ozelius L, Standaert D, Hallett M, Sieber BA, Swanson-Fisher C, Berman BD, Calakos N, Moore JC, Perlmutter JS, Pirio Richardson SE, Saunders-Pullman R, Scheinfeldt L, Sharma N, Sillitoe R, Simonyan K, Starr PA, Taylor A, Vitek J; participants and organizers of the NINDS Workshop on Research Priorities in Dystonia. Defining research priorities in dystonia. Neurology. 2020 Mar 24;94(12):526-537. doi: 10.1212/WNL.0000000000009140. Epub 2020 Feb 25.
- Results publication: PubMed 30582159Guiry S, Worthley A, Simonyan K. A separation of innate and learned vocal behaviors defines the symptomatology of spasmodic dysphonia. Laryngoscope. 2019 Jul;129(7):1627-1633. doi: 10.1002/lary.27617. Epub 2018 Dec 24.
- Results publication: PubMed 30840778Bianchi S, Fuertinger S, Huddleston H, Frucht SJ, Simonyan K. Functional and structural neural bases of task specificity in isolated focal dystonia. Mov Disord. 2019 Apr;34(4):555-563. doi: 10.1002/mds.27649. Epub 2019 Mar 6.
- NIH grant descriptionLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Kristina Simonyan, MD, PhD principal investigator
Massachusetts Eye and Ear Infirmary
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Spasmodic Dysphonia and Singer's Dystonia, run by Massachusetts Eye and Ear Infirmary 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 6 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 8 Dec 2025. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kristina Simonyan, MD, PhD (617-573-6016, Simonyan_Lab@MEEI.HARVARD.EDU), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of brain structural and functional changes (time frame: 5 years). 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 NCT03042962, which also lists 26 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 8 Dec 2025.