What is the Purpose of this Study?
This study focuses on people who have been diagnosed with a form of isolated (or primary) dystonia that involves one (focal) or more (segmental) areas of the body, including neck, face or limbs. Dystonia is a movement disorder that causes involuntary abnormal movements, sustained postures, or both; the condition may be painful and can affect the ability to complete daily tasks. The study will collect information to help researchers understand how dystonia impacts the patient’s life and how the condition has changed over time. The study may also help scientists to better understand clinical scales used as standard of care. Clinical scales are tools that examine how the patient is doing in various areas of life, such as the ability to control their movements, wellbeing, pain, relationships, social interactions, and progress over time.
Participants will be asked to complete 2 study visits (Visit 1 and Visit 2). During Visit 1, they will be asked about their background, medical and dystonia history, and current health/medications. They will be asked to complete interview-based rating scales: the Burke-Fahn-Marsden (BFM) rating scale and Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS-2). The Clinical Global Impression-Severity (CGI-S) Rating Scale will be completed by their doctor. During Visit 2, participants will be asked about any changes to their health and dystonia symptoms and any medicines they are taking. They will also be asked to complete the same scales (BFM, TWSTRS-2); the CGI-S will be completed by their doctor.
Eligibility
- * The participant is willing to sign an informed consent form indicating that he/she understands the purpose of the study; understands and can perform, complete, and comply with all the assessments that are required during the study; and is willing to participate in the study.
- * The participant is a male or female between 12 and 65 years of age (inclusive).
- * The participant has a clinical diagnosis of isolated (or primary) dystonia for at least one year prior to Visit 1 and meets the study definition of segmental/multifocal or generalized dystonia at Visit 1.
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- * The participant is willing to sign an informed consent form indicating that he/she understands the purpose of the study; understands and can perform, complete, and comply with all the assessments that are required during the study; and is willing to participate in the study.
- * The participant is a male or female between 12 and 65 years of age (inclusive).
- * The participant has a clinical diagnosis of isolated (or primary) dystonia for at least one year prior to Visit 1 and meets the study definition of segmental/multifocal or generalized dystonia at Visit 1.
- * The participant has a diagnosis of focal dystonia at Visit 1.
- * The participant has a diagnosis of dopa-responsive dystonia, secondary dystonia, or psychogenic/functional dystonia.
- * The participant has any significant ongoing disease, disorder, laboratory abnormalities, alcohol or illicit drug abuse or dependence, environmental factor, or any ongoing or history of any medical or surgical condition that in the judgment of the investigator, in consultation with the medical monitor and/or sponsor's designee, might jeopardize the participant's safety, impact the scientific objectives of the clinical study, or interfere with participation in the clinical study.
- * The participant has a history of any psychiatric or cognitive impairment disorder that, in the opinion of the investigator, may interfere with study conduct.
- * The participant has had myotomy or denervation surgery in any affected muscles (e.g., peripheral denervation and/or spinal cord stimulation).
- * The participant has marked limitation on passive range of motion that suggests contractures or other structural abnormality (e.g., cervical contractures or cervical spine syndrome).
- * The participant has undergone deep brain stimulation (DBS) surgery (even if implant deactivated).
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Where can I participate?
Beverly
More about this Clinical Trial
What is the full name of this clinical trial?
An Observational Study of Individuals with Isolated Dystonia