Study of the Oral Treatment MTR-601 in Cervical Dystonia

Phase 2

Recruiting

• Conditions: Cervical Dystonia
• Interventions: Drug: Placebo; Drug: MTR-601

• Registration Number: NCT06830642
• Lead Sponsor: Motric Bio

Brief Summary

Study MTR-601-201 is an 8-week, randomized, placebo-controlled study to examine the safety, tolerability, and efficacy of MTR-601 in participants with cervical dystonia.

Detailed Description

Study MTR-601-201 is an 8-week, randomized, placebo-controlled study to examine the safety, tolerability, and efficacy of MTR-601 in participants with cervical dystonia.

Participants will be randomized (1:1) to receive either MTR-601 or matching placebo every day for 4 weeks, after which all participants will be followed for an additional 2 weeks through study treatment washout. The Investigator and Participant will be blinded to the assigned arm. Treatment will be administered via capsules and matching placebo capsules. The total sample size will be approximately 80 participants.

The study will be divided into 3 periods: Screening, Treatment and Follow up.

An initial screening assessment (V1) will occur between Day -90 and Day -2, where individuals will undergo informed consent and have their preliminary eligibility reviewed. Individuals who are found to be eligible will be instructed to not receive their next scheduled botulinum toxin treatment prior to entry into the study.

A full Screening visit (V2) will occur between Day -14 and -1.

Individuals who are confirmed to be eligible after V2, including having not received botulinum toxin treatment for ≥3 months (≥6 months for daxibotulinum ToxinA), will return to clinic on Day 1 for randomization and initiation of study treatment (V3). At this visit individuals will be randomized into the study and receive either MTR-601 or matching placebo to take for the duration of the study and will be discharged home.

Individuals will take the first dose and subsequent doses of study treatment once daily while at home, with weekly visits during the treatment period to assess safety, tolerability and efficacy.

At Day 36 and thereafter, participants may resume treatment with botulinum toxin or daxibotulinum toxinA.

Individuals will return to the clinic 14 days after completion of treatment (Day 42) for the end of study visit (V8) where final safety assessments will be performed. Individuals will then be discontinued from the study.

Study Timeline

• Study First Submitted: 2025-01-31
• Study First Submitted QC: 2025-02-12
• Study First Posted: 2025-02-17
• Study Start: 2025-02-28
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-12
• Last Update Posted: 2025-08-14
• Primary Completion: 2026-07-08
• Study Completion: 2026-08-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
MTR-601	MTR-601	-

Primary Outcome Measures

Name	Time	Method
To evaluate the safety and tolerability of MTR-601 by the incidents of treatment-related adverse events as assessed by CTCAE v5.0	Baseline to week 6	To evaluate the safety and tolerability of MTR-601 in participants with cervical dystonia by the incidents of treatments emergent adverse events
To evaluate the efficacy of MTR-601 in participants with cervical dystonia	Baseline to week 4	To evaluate the efficacy of MTR-601 in participants with cervical dystonia by Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) overall score change.

Secondary Outcome Measures

Name	Time	Method
To evaluate the plasma concentration of MTR-601 in participants with cervical dystonia overall and by age	Day 14 and Day 28
To evaluate the effect of MTR-601 on Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) severity sub score	Baseline to week 2
To evaluate the effect of MTR-601 on Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) pain sub score	Baseline to week 2
To evaluate the effect of MTR-601 on Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) disability sub score	Baseline to week 2
To evaluate the effect of MTR-601 on Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) overall score	Baseline to week 2

Trial Locations

Locations (14)

Vanderbilt Neurology, The Vanderbilt Clinic; 🇺🇸 Nashville, Tennessee, United States

Arizona Neuroscience Research, LLC; 🇺🇸 Phoenix, Arizona, United States

The Parkinson's and Movement Disorder Institute; 🇺🇸 Fountain Valley, California, United States

Keck Medicine of University of Southern California; 🇺🇸 Los Angeles, California, United States

The Neurology Research Group, LLC Brainstorm Research; 🇺🇸 Miami, Florida, United States

Neurology One; 🇺🇸 Orlando, Florida, United States

University of South Florida; 🇺🇸 Tampa, Florida, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States

Michigan State University, Department of Neurology; 🇺🇸 East Lansing, Michigan, United States

Quest Research Institute; 🇺🇸 Farmington Hills, Michigan, United States

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