Cervical Dystonia
Conditions
Brief summary
Open label study to evaluate the safety and immunogenicity of MYOBLOC (Botulinum Toxin Type B) in adult patients with a clinical diagnosis of Cervical Dystonia (CD).
Detailed description
This is an open-label study aimed to assess long-term safety and immunogenicity of MYOBLOC (Botulinum Toxin Type B) in adult patients with a clinical diagnosis of Cervical Dystonia (CD). Up to 500 adult patients with a confirmed clinical diagnosis of CD are planned for enrollment into this open label study. These patients will be Type B toxin naive patients with CD. During this study, patients will receive repeat injections of MYOBLOC every 12 weeks when deemed appropriate by the Investigator. Injections should not occur more frequently than every 12 weeks. Total duration of exposure to MYOBLOC will be targeted for at least two years, with potential exposure for up to 7 years in patients with earliest enrollment.
Interventions
BIOLOGICALBotulinum Toxin Type B
Eligible patients could receive injections of MYOBLOC once every 12 weeks. Total Dose could range from 5,000 Units up to 25,000 Units.
Sponsors
Solstice Neurosciences, LLC, a subsidiary of US WorldMeds, LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 18 years of age or older * body weight of at least 46 kilograms * History of Cervical Dystonia of at least one year's duration who in the opinion of the Investigator requires treatment
Exclusion criteria
* Inability to give informed consent * Patient who has been previously treated with botulinum Toxin Type B * Patient who has received a Botulinum toxin Type A injection in the last 12 weeks. * History of phenol injections involving the neck or shoulder region in the last 12 months. * Patients with neck contractures or cervical spine disease
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment Assessment Scale (TAS), 4 Wks Post-injection Compared to Baseline (Time 0), Rating of Cervical Dystonia Symptoms | Session 1 - Time 0, 4 weeks post-injection compared to baseline | 7 point rating scale (-3 = significantly worse, 0 = no change, +3 = significantly better. Comparison at Wk 4 to baseline. Rating of Cervical Dystonia Symptoms |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Treatment Assessment Scale (TAS), Approx Wk 12 + 4 Wks Post-Injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | Session 2 (12 wks) - 4 weeks post-injection compared to baseline | 7 point rating scale (-3=significantly worse, 0=no change, +3=significantly better), comparison of Wk 12 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms |
| Treatment Assessment Scale (TAS), Approx Wk 24 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | Session 3 (24 Wks) - 4 weeks post-injection compared to baseline | 7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison of Wk 24 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms |
| Treatment Assessment Scale (TAS), Approx Wk 36 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | Session 4 (36 Wks) - 4 weeks post-injection compared to baseline | 7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison of Wk 36 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms |
| Treatment Assessment Scale (TAS), Approx Wk 48 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | Session 5 (48 Wks) - 4 weeks post-injection compared to baseline | 7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison of Wk 48 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms |
| Treatment Assessment Scale (TAS), Approx Wk 60 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | Session 6 (60 Wks) - 4 weeks post-injection compared to baseline | 7 point scale (-3=significantly worse, 0=no change, +3=significantly better). comparison Wk 60 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms |
| Treatment Assessment Scale (TAS), Approx Wk 72 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | Session 7 (72 Wks) - 4 weeks post-injection compared to baseline | 7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison Wk 72 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms |
| Treatment Assessment Scale (TAS), Approx Wk 84 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | Session 8 (84 Wks) - 4 weeks post-injection compared to baseline | 7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison Wk 84 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms |
Contacts
STUDY_DIRECTORJonathan T Rubin, MD
Supernus Pharmaceuticals, Inc.
Participant flow
Recruitment details
Approximately 500 subjects were recruited from 36 centers in North America.
Pre-assignment details
Subjects had to be Botulinum Toxin Type B naive and could not have received a Botulinum Toxin Type A treatment within 12 weeks of enrollment.
Participants by arm
| Arm | Count |
|---|---|
| MYOBLOC Open Label Treatment All patients meeting enrollment criteria received open-label MYOBLOC. Patients were eligible for repeat injections approximately every 12 weeks. | 502 |
| Total | 502 |
Baseline characteristics
| Characteristic | MYOBLOC Open Label Treatment |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 83 Participants |
| Age, Categorical Between 18 and 65 years | 419 Participants |
| Age, Continuous | 54 years STANDARD_DEVIATION 11 |
| Region of Enrollment North America | 502 participants |
| Sex: Female, Male Female | 341 Participants |
| Sex: Female, Male Male | 161 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| other Total, other adverse events | 494 / — |
| serious Total, serious adverse events | 133 / — |
Outcome results
Primary
Treatment Assessment Scale (TAS), 4 Wks Post-injection Compared to Baseline (Time 0), Rating of Cervical Dystonia Symptoms
7 point rating scale (-3 = significantly worse, 0 = no change, +3 = significantly better. Comparison at Wk 4 to baseline. Rating of Cervical Dystonia Symptoms
Time frame: Session 1 - Time 0, 4 weeks post-injection compared to baseline
Population: Safety Population/Intent to Treat
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| MYOBLOC Open Label Treatment | Treatment Assessment Scale (TAS), 4 Wks Post-injection Compared to Baseline (Time 0), Rating of Cervical Dystonia Symptoms | 0.9 points on a scale | Standard Error 0.1 |
Secondary
Treatment Assessment Scale (TAS), Approx Wk 12 + 4 Wks Post-Injection Compared to Baseline. Rating of Cervical Dystonia Symptoms
7 point rating scale (-3=significantly worse, 0=no change, +3=significantly better), comparison of Wk 12 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms
Time frame: Session 2 (12 wks) - 4 weeks post-injection compared to baseline
Population: safety population/Intent to Treat
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| MYOBLOC Open Label Treatment | Treatment Assessment Scale (TAS), Approx Wk 12 + 4 Wks Post-Injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | 1.3 points on a scale | Standard Deviation 0.1 |
Secondary
Treatment Assessment Scale (TAS), Approx Wk 24 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms
7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison of Wk 24 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms
Time frame: Session 3 (24 Wks) - 4 weeks post-injection compared to baseline
Population: safety population/Intent to Treat
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| MYOBLOC Open Label Treatment | Treatment Assessment Scale (TAS), Approx Wk 24 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | 1.5 points on a scale | Standard Deviation 0.1 |
Secondary
Treatment Assessment Scale (TAS), Approx Wk 36 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms
7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison of Wk 36 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms
Time frame: Session 4 (36 Wks) - 4 weeks post-injection compared to baseline
Population: safety population/Intent to Treat
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| MYOBLOC Open Label Treatment | Treatment Assessment Scale (TAS), Approx Wk 36 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | 1.4 points on a scale | Standard Deviation 0.1 |
Secondary
Treatment Assessment Scale (TAS), Approx Wk 48 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms
7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison of Wk 48 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms
Time frame: Session 5 (48 Wks) - 4 weeks post-injection compared to baseline
Population: Safety Population/Intent to Treat
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| MYOBLOC Open Label Treatment | Treatment Assessment Scale (TAS), Approx Wk 48 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | 1.5 points on a scale | Standard Deviation 0.1 |
Secondary
Treatment Assessment Scale (TAS), Approx Wk 60 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms
7 point scale (-3=significantly worse, 0=no change, +3=significantly better). comparison Wk 60 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms
Time frame: Session 6 (60 Wks) - 4 weeks post-injection compared to baseline
Population: Safety Population/Intent to Treat
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| MYOBLOC Open Label Treatment | Treatment Assessment Scale (TAS), Approx Wk 60 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | 1.4 points on a scale | Standard Deviation 0.1 |
Secondary
Treatment Assessment Scale (TAS), Approx Wk 72 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms
7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison Wk 72 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms
Time frame: Session 7 (72 Wks) - 4 weeks post-injection compared to baseline
Population: Safety Population/Intent to Treat
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| MYOBLOC Open Label Treatment | Treatment Assessment Scale (TAS), Approx Wk 72 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | 1.5 points on a scale | Standard Deviation 0.1 |
Secondary
Treatment Assessment Scale (TAS), Approx Wk 84 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms
7 point scale (-3=significantly worse, 0=no change, +3=significantly better). Comparison Wk 84 + 4, compared to baseline. Rating of Cervical Dystonia Symptoms
Time frame: Session 8 (84 Wks) - 4 weeks post-injection compared to baseline
Population: Safety Population/Intent to Treat
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| MYOBLOC Open Label Treatment | Treatment Assessment Scale (TAS), Approx Wk 84 + 4 Wks Post-injection Compared to Baseline. Rating of Cervical Dystonia Symptoms | 1.4 points on a scale | Standard Deviation 0.1 |