A Study to Evaluate the Efficacy, Safety, and Tolerability of VIM0423 in Individuals With Isolated Dystonia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: VIM0423, VIM0423 Placebo.
- Who it may be relevant to
- Registry conditions: Dystonia. Basic parameters: 18 years — 65 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of VIM0423 in Individuals With Isolated Dystonia (Stride Dystonia)
Overview
Stride Dystonia is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of VIM0423 in individuals with isolated dystonia. The main objectives of this clinical trial are to determine the following: * Does VIM0423 therapy improve dystonia symptoms compared to placebo? * Is VIM0423 well tolerated in individuals with isolated dystonia? and * Do the therapeutic effects of VIM0423 confer improvements on daily function and quality of life?
Detailed description
Isolated dystonia, previously referred to as primary dystonia, is a rare movement disorder subclassified according to parts of the body affected. Approximately 160,000 individuals in the United States are affected. Dystonia is characterized by sustained muscle contractions which cause abnormal movements and/or postures. These can be patterned, twisting, or tremulous.
Botulinum toxins (BoNT) are the only approved drug products in the US for the treatment of dystonia; however, these injections are only approved for individuals with a diagnosis of cervical (neck) dystonia or blepharospasm (eye lid) and act locally to weaken affected muscles. There are no approved treatment options for approximately 50% of individuals with other subtypes of isolated dystonia that affect more than one body part, and there are no approved oral treatments for dystonia.
VIM0423 is being developed as a once-daily oral medication that will address the root cause of dystonia, which is known to be a chemical imbalance in specific brain regions. As such, VIM0423 is intended to treat most people with isolated dystonia, regardless of the number of body parts affected.
Stride Dystonia (Study VIM0423-201) is a Phase 2, multicenter, randomized, double-blind, placebo-controlled study designed to assess the efficacy, safety, and tolerability of VIM0423 in adults with isolated dystonia that affects two or more body regions. Up to 120 individuals will be enrolled in the trial.
Participants will be randomized (1:1) to receive either VIM0423 or matching placebo (up to 6 pills a day, taken before bed each evening) for 16 weeks. The Investigator and Participant will not know what patients are receiving VIM0423 or placebo, but that information will be available if needed.
The total time of participation in the trial is up to 32 weeks and requires 6 in person visits. The study schedule includes Screening and Baseline (up to 14 weeks, 2 visits), Dose Titration, Dose Maintenance, Dose Taper (16 weeks, 3 Visits), and Safety follow-up (2 weeks, 1 Visit).
Assessments of changes in dystonia including video recordings will be made by study personnel. Clinical labs, EKGs, and Adverse events will be monitored throughout the study. Participants will be asked to perform self-assessments of their dystonia and its impact on their activities of daily living.
Interventions
- Drug VIM0423
VIM0423 is a combination drug product containing two active ingredients (VMA-1001 and VMA-1002) - Drug VIM0423 Placebo
Matching VIM0423 placebo product containing no active ingredient
Primary outcome measures
- Change from baseline in Burke-Fahn-Marsden (BFM) Dystonia Rating Scale [Time frame: From baseline to Week 14]
Secondary outcome measures (4)
- Change from baseline in Clinical Global Impression of Severity (CGI-S) as assessed by the clinician [Time frame: From baseline to Week 14]
- Change from baseline in the Toronto Western Spasmodic Torticollis Rating Scale including head tremor (TWSTRS; as applicable) [Time frame: From baseline to Week 14]
- Clinical Global Impression of Change (CGI-C) [Time frame: From baseline to Week 14]
- Patient Global Impression of Change (PGI-C) [Time frame: From baseline to Week 14]
Eligibility criteria
Inclusion criteria
- Participant must be male or nonpregnant female between 18 and 65 years of age (inclusive) at Visit 1 (Screening).
- The participant has a clinical diagnosis of isolated dystonia for at least one year prior to Visit 1 (Screening); has two or more body regions affected by dystonia at Visit 1 (Screening); and has been confirmed by Investigator and Enrollment Authorization Committee.
- The participant may be untreated with BoNT; OR if the participant is being treated with BoNT as part of their standard of care for dystonia, they must meet protocol-specified criteria.
- The participants must meet protocol-specified requirements for baseline scores on the BFM and CGI-S.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 37 centers
- Vima Site #033 — Birmingham
- Vima Site #039 — Scottsdale
- Vima Site #004 — Scottsdale
- Vima Site #028 — Sun City
- Vima Site #042 — Irvine
- Vima Site #030 — Los Angeles
- Vima Site #019 — Denver
- Vima Site #009 — Gainesville
- … and 29 more centers
Identifiers
NCT: NCT07304089 · VIM0423-201