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RecruitingNCT03428009Updated Sep 23, 2026

Dystonia Genotype-Phenotype Correlation

An observational study in Dystonia, Dystonia; Idiopathic and Dystonia, Primary, sponsored by University of Texas Southwestern Medical Center. Recruiting at 1 site in United States. Open to participants aged 11 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-09-23.

Sponsored by University of Texas Southwestern Medical Center · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
200
Ages
11 Years and older
Sex
All
01

Study summary

The purpose of this study is to (1) investigate the effect of known dystonia-causing mutations on brain structure and function, to (2) identify structural brain changes that differ between clinical phenotypes of dystonia, and to (3) collect DNA, detailed family history, and clinical phenotypes from patients with idiopathic dystonia with the goal of identifying new dystonia-related genes. Investigators will be recruiting both healthy control subjects and subjects with any form of dystonia. For this study there will be a maximum of two study visit involving a clinical assessment, collection of medical and family history, task training session, an MRI using the learned tasks, and finally a blood draw for genetic analysis. In total, these visits will take 3-5 hours. If the dystonia subjects receive botulinum toxin injections for treatment, the participants and their matched controls will be asked to come for a second visit.

Read the detailed description
  1. Identify a cohort of individuals with known dystonia-related gene mutations, and individuals with idiopathic but presumed-genetic dystonia. Some of these individuals may receive botulinum toxin injections to treat their dystonia per standard of care; in these patients, investigators will image before and after injections to assess for imaging correlates of treatment response.
  2. Analyze DNA samples from both the dystonia and healthy individual cohorts to detect the presence of mutations and/or polymorphisms in genes associated with dystonia
  3. Collect systematic clinical information, including Tsui Torticollis, Burke-Fahn-Marsden, Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS), Voice Disability Index, Unified Myoclonus Rating Scale, Beck Depression Inventory, Beck Anxiety Inventory and Spielberg Trait Anxiety scales. Scales will be tailored to the type of dystonia, as determined by the clinician referring into the study (i.e., torticollis scales will only be performed on patients with cervical dystonia).
  4. Use functional MRI (fMRI), diffusion tensor imaging (DTI), and structural MRI to a) analyze brain activity and structure pre- and post-botulinum toxin injections, b) determine how different stages of movement (execution, preparation, sequencing) influence dystonia and the underlying neural mechanisms, c) identify structural abnormalities shared between clinical sub-types of dystonia. As new MR imaging methods are introduced that may improve the investigators ability to identify or distinguish these abnormalities, the investigator will incorporate these novel sequences into the imaging protocol.
  5. Correlate brain activity and structural data with ratings of dystonia severity, location of dystonia, genetic status, and response to treatment (medications and/or botulinum toxin injections).
  6. Correlate polymorphism data with dystonia severity, response to botulinum toxin, depression/anxiety severity, and brain activity/structure.
02

Conditions studied

  • Dystonia
  • Dystonia; Idiopathic
  • Dystonia, Primary
  • Dystonia, Secondary
  • Dystonia, Familial
  • Dystonia Disorder
  • Dystonias, Sporadic
  • Dystonia; Orofacial
  • Dystonia Lenticularis
  • Dystonia, Paroxysmal
  • Dystonia 6
  • Dystonia 5
  • Dystonia 8
  • Dystonia 9
  • Dystonia 19
  • Dystonia 10
  • Dystonia 11
  • Dystonia 20
  • Dystonia 12
  • Dystonia, Focal
  • Dystonia of Head
  • Dystonia, Diurnal

Keywords

  • Dystonia
  • Control
  • Magnetic Resonance Imagine
  • Genotype
  • Phenotype
03

Who can participate

Ages eligible
11 Years and older
Sexes eligible
All
Accepts healthy volunteers
Yes
Sampling method
Non-probability sample

Study population

Investigators will recruit both healthy participants and individuals diagnosed with dystonia of any form for this study. Each Dystonia subject will be matched 1:1 with an unrelated control subject based on: 1) age, +/- 3 years (adults), +/- 1 year (16-18y), +/- 6 months (11-15y); 2) handedness, as determined by the Edinburgh Handedness Inventory, 3) gender, and 4) self-identified racial or ethnic background.

General Exclusion (both Dystonia and Control groups):

  • Metal in any part of the body (including metal injury to the eye) OR carrying a medical device incompatible with MRI (e.g., metal implants such as surgical clips or pacemakers) OR positive screening per UTSW MRI screening form
  • Claustrophobia
  • Non-fluent English
  • Weight incompatible with MRI safety
  • History of head trauma with neurological sequelae, including multiple concussions and/or history of stroke
  • Pregnancy
  • Serious medical illness or history of serious medical illness, including cancer that was treated with radiation or chemotherapy, heart attack, or a known history of HIV-1 + status
  • Subjects with Hepatitis C (by Hepatitis C+ titer)
  • Subjects with insulin dependent diabetes mellitus (IDDM)
  • Severe respiratory compromise
  • In the opinion of the investigator, not able to safely participate in this study

Inclusion criteria

Inclusion Criteria:

  • Dystonia group

Previous diagnosis of dystonia which include but is not limited to:

  • cervical dystonia (50 subjects)
  • blepharospasm (25 subjects)
  • limb dystonia (50 subjects)
  • spasmodic dysphonia (25 subjects)
  • segmental dystonia
  • multi-focal dystonia
  • Any childhood-onset dystonia (25 subjects) Age > 11 years

    • Control group:

No prior dystonia diagnosis (175 subjects) Age > 11 years

Exclusion criteria

Exclusion Criteria:

  • Dystonia group Prior history of or concurrent neurological or psychiatric diagnosis - depression and/or anxiety accepted Current use of non-dystonia neuroactive medications - SSRI/medication for depression and/or anxiety accepted Current use of cervical brace designed for dystonia treatment Prior structural brain injury

Control group:

History of or current neurological or psychiatric diagnosis - depression and/or anxiety accepted, but must not be in active phase Current use of any neuroactive medication, SSRI/medication for depression and/or anxiety accepted

04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
200 participants (estimated)
Patient registry
No
Biospecimen retention
Samples with dna

Groups and cohorts

  • Dystonia group

    Both groups will have blood drawn, undergo clinical assessments, the collection of medical and family history, and an Magnetic Resonance Imaging. This is an observational study and there is no intervention.

    Other: Magnetic Resonance Imaging

  • Control Group

    Both groups will have blood drawn, undergo clinical assessments, the collection of medical and family history, and an Magnetic Resonance Imaging. This is an observational study and there is no intervention.

    Other: Magnetic Resonance Imaging

Interventions

  • OtherMagnetic Resonance Imaging

    Study interventions are minimal risk.

    Also known as: Blood Draw for Genetic testing, Clinical Assessments

05

What researchers measure

Primary outcomes

  1. Structural or functional imaging of dystonia and control groups

    Identify structural or functional imaging measures that distinguish (a) dystonia patients from matched controls, (b) between clinically-defined forms of dystonia

    Time frame: 3-5 hours at each study visit

  2. Genetic Analysis of dystonia and control groups

    Identify polymorphisms in genes known to cause dystonia that affect the structural or functional imaging measures in dystonia patients and to identify new genes associated with dystonia.

    Time frame: 30 min

06

Study locations

1 of 1 sites recruiting
07

References and documents

Individual participant data

Plan to share: Yes — Data and/or sample collection was specifically for research and subjects indicated that their data could be shared with other scientists. Investigators will only send coded data and/or samples with no identifying information. In addition, all whole blood for DNA samples collected for this protocol will be sent for DNA sequencing and genetic analysis at Massachusetts General Hospital in Dr. Nutan Sharma's lab. In addition at Massachusetts General Tissue Culture Core in the Dystonia Partners Research Biobank investigators will contribute the subject's deidentified DNA sample from this protocol for use in future genetic research regarding dystonia.

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT03428009
Lead sponsor
University of Texas Southwestern Medical Center
Collaborators
Massachusetts General Hospital
Responsible party
Jeff Waugh, MD, PhD (Associate Professor, University of Texas Southwestern Medical Center) — Principal investigator
First posted
Feb 9, 2018
Start date
Mar 1, 2018
Primary completion
Sep 2027 (estimated)
Completion
Sep 2027 (estimated)
Last update
Sep 23, 2026

Study contacts

Alyssa Boudreau
Contact
alyssa.boudreau@utsouthwestern.edu
214-456-2106
Jeff Waugh, MD, PhD
Contact
Jeff.Waugh@UTSouthwestern.edu
214-867-6906

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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