Overview

This trial is active, not recruiting.

Conditions oral dystonia, tardive dystonia
Treatment abobotulinumtoxina
Phase phase 1/phase 2
Sponsor Emory University
Collaborator Ipsen
Start date August 2013
End date December 2016
Trial size 20 participants
Trial identifier NCT01921270, IRB00064292

Summary

The purpose of this study is to study the efficacy, safety, and proper dosing of AbobotulinumtoxinA (Dysport) for use in Oromandibular Dystonia (OMD). The study hypothesis is that one of the dosing levels used will have adequate safety and efficacy.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking double blind (subject, outcomes assessor)
Primary purpose treatment
Arm
(Active Comparator)
This arm refers to Aim 1 of the study in which different dosages will be administered. For a particular muscle that is appropriate to inject, the established "high" dose will be given. It will be injected once at Week 0. Dosage of AbobotulinumtoxinA (Dysport) per muscle: Medial Pterygoid: 100 units Masseter: 75 units Lateral Pterygoid: 100 units Anterior digastrics: 30 units Genioglossus: 35 units
abobotulinumtoxina Dysport
Medication will be reconstituted at 1.5 cc of normal saline for every 300 units of abobotulinumtoxinA, then injected.
(Active Comparator)
This arm refers to Aim 1 of the study in which different dosages will be administered. For a particular muscle that is appropriate to inject, the established "medium" dose will be given. It will be injected once at Week 0. Dosage of AbobotulinumtoxinA (Dysport) per muscle: Medial Pterygoid: 75 units Masseter: 50 units Lateral Pterygoid: 75 units Anterior digastrics: 20 units Genioglossus: 25 units
abobotulinumtoxina Dysport
Medication will be reconstituted at 1.5 cc of normal saline for every 300 units of abobotulinumtoxinA, then injected.
(Active Comparator)
This arm refers to Aim 1 of the study in which different dosages will be administered. For a particular muscle that is appropriate to inject, the established "low" dose will be given. It will be injected once at Week 0. Dosage of AbobotulinumtoxinA (Dysport) per muscle: Medial Pterygoid: 50 units Masseter: 25 units Lateral Pterygoid: 50 units Anterior digastrics: 10 units Genioglossus: 15 units
abobotulinumtoxina Dysport
Medication will be reconstituted at 1.5 cc of normal saline for every 300 units of abobotulinumtoxinA, then injected.

Primary Outcomes

Measure
Global Dystonia Rating Scale
time frame: 0,6 weeks

Secondary Outcomes

Measure
Global Dystonia Rating Scale
time frame: 0,12 weeks
Global Dystonia Rating Scale
time frame: 0,6,12 weeks
Analogue Pain Scale
time frame: 0, 6, 12 weeks
SCS-PD (Sialorrhea Clinical Scale for PD)
time frame: 0,6,12 weeks
Estimated amount of tongue bites per day
time frame: 0,6,12 weeks
Swallowing Disturbance Questionnaire(SDQ)
time frame: 0,6,12 weeks
Fahn-Marsden Part B "Speech" Question
time frame: 0,6, 12 weeks
Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25)
time frame: 0,6,12 weeks
Global Clinical Impression Scale (CGI) with Efficacy Index
time frame: 0,6,12 weeks
Unified Dystonia Rating Scale (UDRS)
time frame: 0,6,12 weeks

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - a diagnosis of primary or tardive OMD - moderate or severe severity, defined as GDS score ≥4 in either "lower face" or "jaw and tongue" section - age>18 years - capability of attending the scheduled visits - only those who have been previously injected with onabotulinumtoxinA and responded to that treatment, and are at least 12 weeks post last injection - Women of childbearing age need to use contraception in order to be included. Exclusion Criteria: - Existence of a systemic disease that could confound the evaluation - previous placement of Deep Brain Stimulation electrodes to treat dystonia - concomitant oral medications that could interfere with the action of botulinum toxin Type A (e.g., aminoglycosides) - on an unstable dosage of any medication prescribed to treat dystonia (e.g., benzodiazepines, baclofen or anticholinergics) - any known hypersensitivity to any botulinum toxin preparation and allergy to cow's milk protein - immunoresistance to other forms of botulinum toxin type A - existence of a concomitant neuromuscular disorder (e.g., Myasthenia Gravis or Lambert-Eaton syndrome, etc) - infection at the proposed injection sites - pregnant women - women of childbearing age NOT on contraception - breastfeeding women - inability to comply with scheduled visits - patients who had been previously injected with botulinum toxin type A but who did not respond

Additional Information

Official title A Pilot Dose Ranging Study of Dysport® (AbobotulinumtoxinA) in the Treatment of Oromandibular Dystonia
Principal investigator Stewart A Factor, DO
Description Study Design: Interventional , single site, prospective study with 2 parts: 1. Dose raging (aim 1): 3 oromandibular Dystonia (OMD) groups undergoing 3 different Dysport® dosages with a step-wise approach based on safety response. 6 weeks duration for the 3 groups. 2. Efficacy & safety assessment of Dysport® selected dose from study part 1 (aim 2): 1 single arm with OMD subjects. 12 weeks duration. Study Specific Aims: Aim 1: To determine the best dose range for Dysport® for the treatment of OMD In order to address Aim 1, patients with OMD who have been previously treated with Botox® (onabotulinumtoxinA) and responded will be recruited into this study. The muscles to be injected will be tailored to the individual's presentation and up to the injector's discretion, but if a muscle is chosen it will be given a set amount of Dysport® (abobotulinumtoxinA )(see table 2). The subject will be injected with Dysport® only, with an unblinded open-label disclosure. There will be three dose levels and three subjects will be injected in each (OMD type for each group: 2 jaw opening + 1 jaw closing). This optimal dosage will be the safest dosage that provides adequate efficacy. This will be based on the Clinical Global Impression Scale's Efficacy Index. Subjects from Aim 1 will be offered enrollment into Aim 2. Aim 2: To determine the efficacy and safety of Dysport® for the treatment of oromandibular dystonia 20 subjects will be included in the second part of the study and will be injected with the determined optimal dosage found at the end of the first study part. They would be injected for Aim 2 after at least 12 weeks have passed, given that the effect from the prior injection had either completely abated or at least declined significantly. The 9 subjects from Aim 1 will be offered to enter study part 2 unless they have severe adverse event (AE), ongoing AE or AE considered inappropriate for study continuation. Their previous injections with Dysport® should not confound the results of Aim 2 since both Botox® and Dysport® should both have minimal clinical effect at 12 weeks out from injection. Since the local population suffering from OMD is small, the inclusion of the Aim 1 subjects into Aim 2 would lead to a much more rapid completion of recruitment. In both Aim 1 and Aim 2, the safety and efficacy will be recorded for all subjects that undergo injection. All subjects will be examined and videotaped at the injection visit, then at 6 and 12 weeks after injection with a standardized protocol. The primary outcome will be blinded examination scores of the videos performed after the study is complete. The evaluators will be two different movement disorders experts, not otherwise involved in the study, who will review the videotaped examinations, presented in a random order, using the Global Dystonia Rating scale (GDS). They will rate the dystonia at baseline (injection visit) and 6 weeks after injection. The change in GDS score from baseline to 6 weeks after injection is the best objective measure of efficacy and will serve as the study's primary endpoint. The investigators chose the GDS over other dystonia scales because of its ease of use, sensitivity to clinical changes because of its continuous rating system, internal consistency, and inter-rater reliability.
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Emory University.