An Observational Study of Individuals With Isolated Dystonia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age12-65
SponsorVima Therapeutics
About this trial
This is an observational study in individuals with isolated (or primary) dystonia that involves more than one body region. The purpose of the study is to collect detailed information to help your physician and other researchers understand how dystonia impacts your and other patients' lives and how the disease changes over time. The study may also help researchers better understand the clinical scales that are commonly used to evaluate dystonia symptoms and how they affect various areas of life such as well-being, pain, relationships and social interactions, and progress over time. Because this is an observational study, you will not receive any interventional treatment in the study.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial population
Isolated (Primary) Dystonia
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
200 Participants
are grouped into 1 trial groupGroup A: Isolated (or Primary) Dystonia Cohort
Trial outcomes
Primary outcomes
1
Burke-Fahn-Marsden (BFM) Dystonia Rating Scale
The BFM was developed to quantitatively assess primary dystonias (Burke 1985). It is composed of 2 subscales: a movement subscale, based on the clinician's evaluation of the patient in nine body areas, and a disability subscale, based on the participant's report of disability in activities of daily living.
Time frame
From start of treatment through 4 monthsSecondary outcomes
1
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS-2)
The TWSTRS-2 scale is used to assess cervical dystonia. It includes 3 subscales that evaluate symptom severity, disability, and pain. The severity scale is clinician-rated and the disability scale is patient-rated based on 6 items related to work, activities of daily living, driving, reading, watching TV, and outside activities.
Time frame
From start of treatment through 4 months2
Clinical Global Impression of Severity (CGI-S)
The CGI-S score is used by the Investigator to determine, in a global sense, how severe the patients' blepharospasm is in the context of other blepharospasm patients. Increased scores indicate more severe symptoms.
Time frame
From start of treatment through 4 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov