PurTox (Botulinum Toxin Type A) for the Treatment of Adult Onset Spasmodic Torticollis/Cervical Dystonia

Study Overview
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Brief Summary
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The safety and efficacy of Mentor Purified Toxin, Botulinum Toxin Type A, Purified Neurotoxin, injected intramuscularly into the neck, is being evaluated as a treatment for painful and non-painful spasmodic torticollis/cervical dystonia. It is hypothesized that treatment will decrease symptom severity and will not have any significant side effects.

Official Title
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A Phase I, Single-dose, Double-blind, Placebo Controlled, Dose Escalation Study to Evaluate the Safety of PurTox for the Treatment of Adult Onset Spasmodic Torticollis/Cervical Dystonia and to Explore Dose-associated Efficacy

Conditions
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Spasmodic Torticollis

Intervention / Treatment
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* Drug: Mentor Purified Toxin, Botulinum Toxn Type A, Purified Neurotoxin
* Other: Physiologic saline


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of torticollis with noticeable symptoms for at least 6 months Total TWSTRS score greater or equal to 20, TWSTRS severity score greater than or equal to 10 and TWSTRS Disability score greater than or equal to 3 (must meet all 3 criteria) Exclusion Criteria: Diagnosis of isolated anterocollis Concurrent myopathic disease, myotonic dystrophy (or other form of muscular dystrophy), myasthenia gravis Currently receiving aminoglycoside antibiotic therapy, curare-like drugs, quinidine, succinylcholine, polymixins, anticholinesterases, magnesium sulfate, or lincosamides Any illness that is considered by the Investigator to make the subject an inappropriate candidate Cervical spine injury within 18 months or head and neck surgery within 6 months prior to screening Pre-existing dysphagia History of active autoimmune disease Exposure to botulinum toxin based pharmaceutical within 3 months prior to screening History of primary non-response or secondary resistance with prior exposure to botulinum based pharmaceuticals History of chemotherapy/radiation for malignant disease within 24 months Any investigational drug/device during the 30 days prior to screening

Ages Eligible for Study
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Minimum Age: 30 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: 1<br> | Drug: Mentor Purified Toxin, Botulinum Toxn Type A, Purified Neurotoxin<br>* 100U vials, doses ranged from 50U-200U, single injection.<br>|
| Placebo Comparator: 2<br> | Other: Physiologic saline<br>* Single injection.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Absence of significant drug related adverse effects |  | Post-injection | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Decrease in symptom severity (TWSTRS) |  | 30 days post-injection | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Torticollis, Cervical Dystonia