A Study to Evaluate the Efficacy, Safety, and Tolerability of VIM0423 in Individuals With Isolated Dystonia
Purpose
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?
Condition
- Dystonia
Eligibility
- Eligible Ages
- Between 18 Years and 65 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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.
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Intervention Model Description
- Parallel cohorts of participants receiving active or placebo drug products
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Masking Description
- The study sponsor, participants, the CRO, Investigator, and all site personnel (except pharmacists and pharmacy staff) will be blinded to treatment assignments until the database has been locked and restricted from further edits.
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Active Comparator Segmental/multifocal Active |
Segmental/Multifocal isolated dystonia (enrolling participants with at least two body parts affected receiving active n~40 |
|
|
Placebo Comparator Segmental/multifocal Placebo |
Segmental/Multifocal isolated dystonia (enrolling participants with at least two body parts affected) receiving placebo n~40 |
|
|
Active Comparator Generalized Active |
Generalized isolated dystonia (enrolling participants with more than two body parts affected) receiving active n~10 |
|
|
Placebo Comparator Generalized Placebo |
Generalized isolated dystonia (enrolling participants with more than two body parts affected) receiving placebo n~10 |
|
Recruiting Locations
Scottsdale, Arizona 85258
Denver, Colorado 80113
Orlando, Florida 32825
Farmington Hills, Michigan 48334
Albuquerque, New Mexico 87106
New York, New York 10021
Philadelphia, Pennsylvania 19107
Memphis, Tennessee 38157
Georgetown, Texas 78628
Richmond, Virginia 23233
Kirkland, Washington 98034
Spokane, Washington 99202
More Details
- Status
- Recruiting
- Sponsor
- Vima Therapeutics
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.