Trial Outcomes & Findings for Efficacy and Safety of Two Different Botulinum Toxin Type A Treatments for Moderate to Severe Cervical Dystonia (NCT NCT00432341)

NCT ID: NCT00432341

Last Updated: 2011-12-16

Results Overview

Duration of treatment benefit was measured as the time (days) from Baseline until patients had a loss of therapeutic benefit, as defined by the achievement of their Toronto Western Spasmodic Torticollis Rating Scale Duration Target Score (TDTS) \[loss of 80% of benefit\].

• Recruitment status: TERMINATED
• Study phase: PHASE4
• Target enrollment: 54 participants
• Primary outcome timeframe: 20 Weeks
• Results posted on: 2011-12-16

Participant Flow

Participant milestones

Measure	BOTOX® Botulinum toxin type A (BOTOX®)	Dysport® Botulinum toxin type A (Dysport®)
Overall Study STARTED	24	30
Overall Study COMPLETED	24	30
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Safety of Two Different Botulinum Toxin Type A Treatments for Moderate to Severe Cervical Dystonia

Baseline characteristics by cohort

Measure	BOTOX® n=24 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=30 Participants Botulinum toxin type A (Dysport®)	Total n=54 Participants Total of all reporting groups
Age Continuous	42.0 years STANDARD_DEVIATION 13.1 • n=5 Participants	49.9 years STANDARD_DEVIATION 12.6 • n=7 Participants	46.4 years STANDARD_DEVIATION 13.3 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	12 Participants n=7 Participants	19 Participants n=5 Participants
Sex: Female, Male Male	17 Participants n=5 Participants	18 Participants n=7 Participants	35 Participants n=5 Participants

PRIMARY outcome

Timeframe: 20 Weeks

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided at least 1 post-dose assessment measure.

Duration of treatment benefit was measured as the time (days) from Baseline until patients had a loss of therapeutic benefit, as defined by the achievement of their Toronto Western Spasmodic Torticollis Rating Scale Duration Target Score (TDTS) [loss of 80% of benefit].

Outcome measures

Measure	BOTOX® n=24 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=30 Participants Botulinum toxin type A (Dysport®)
Duration of Treatment Benefit	112.5 Days Interval 102.0 to 126.0	107.0 Days Interval 102.0 to 118.0

SECONDARY outcome

Timeframe: Week 4

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided at least 1 post-dose assessment measure.

The TDTS was the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) score representing a loss of 80% of the treatment benefit at Week 4. The TDTS is calculated from the TWSTRS score and ranges from 0 (least symptoms) to 68 (worst symptoms). The TWSTRS total score which is scored from 0 (least symptoms) to 85 (worst symptoms).

Outcome measures

Measure	BOTOX® n=24 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=30 Participants Botulinum toxin type A (Dysport®)
Toronto Western Spasmodic Torticollis Rating Scale Duration Target Score (TDTS) at Week 4	37.0 Scores on a Scale Interval 16.0 to 56.0	41.0 Scores on a Scale Interval 26.0 to 69.0

SECONDARY outcome

Timeframe: Baseline, Week 4

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided at least 1 post-dose assessment measure.

The TWSTRS is an assessment scale used to measure the impact of cervical dystonia on patients. The score is comprised of 3 subscales: Severity, Disability, and Pain, each of which is scored independently. The total of these 3 comprises the TWSTRS total score which is scored from 0 (least symptoms) to 85 (worst symptoms). Higher scores indicate a greater degree of symptom severity.

Outcome measures

Measure	BOTOX® n=24 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=30 Participants Botulinum toxin type A (Dysport®)
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4 Baseline	42.0 Scores on a Scale Interval 17.0 to 63.0	43.5 Scores on a Scale Interval 30.0 to 73.0
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4 Week 4	26.0 Scores on a Scale Interval 10.0 to 47.0	27.0 Scores on a Scale Interval 11.0 to 52.0

SECONDARY outcome

Timeframe: Week 4

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided at least 1 post-dose assessment measure.

Patient evaluation of benefit from botulinum toxin type A treatment for cervical dystonia. Ratings were on a scale of +4 to -4, with higher scores denoting improvement in cervical dystonia: +4 was 'Complete abolishment of signs and symptoms (about 100% improvement)', 0 represented 'Unchanged', and -4 represented 'Very marked worsening (about 100% worse or greater)'.

Outcome measures

Measure	BOTOX® n=24 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=30 Participants Botulinum toxin type A (Dysport®)
Global Assessment of Benefit by Patient at Week 4	2.0 Units on a Scale Interval 0.0 to 4.0	2.0 Units on a Scale Interval -3.0 to 3.0

SECONDARY outcome

Timeframe: Week 4

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided at least 1 post-dose assessment measure.

Physician evaluation of benefit from botulinum toxin type A treatment for cervical dystonia. Ratings were on a scale of +4 to -4, with higher scores denoting improvement in cervical dystonia: +4 was 'Complete abolishment of signs and symptoms (about 100% improvement)', 0 represented 'Unchanged', and -4 represented 'Very marked worsening (about 100% worse or greater)'.

Outcome measures

Measure	BOTOX® n=24 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=30 Participants Botulinum toxin type A (Dysport®)
Global Assessment of Benefit by Physician at Week 4	2.0 Units on a Scale Interval 1.0 to 4.0	2.0 Units on a Scale Interval 1.0 to 3.0

SECONDARY outcome

Timeframe: Baseline, Week 4

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided as least 1 post-dose assessment measure.

Physician assessment of cervical dystonia severity. The rating was assessed on a scale of 0 to 10, with higher scores denoting greater severity: 0 represented 'No evidence of dystonia' and 10 represented 'Worst cervical dystonia ever'

Outcome measures

Measure	BOTOX® n=24 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=30 Participants Botulinum toxin type A (Dysport®)
Physician Assessment of Cervical Dystonia Severity at Week 4 Baseline	7.0 Units on a Scale Interval 4.0 to 9.0	8.0 Units on a Scale Interval 4.0 to 9.0
Physician Assessment of Cervical Dystonia Severity at Week 4 Week 4	4.0 Units on a Scale Interval 2.0 to 6.0	4.0 Units on a Scale Interval 1.0 to 7.0

SECONDARY outcome

Timeframe: Week 20

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided as least 1 post-dose assessment measure. Only completed assessments at this time point are included.

Physicians assessed the improvement in cervical dystonia after the study treatment compared to previous treatment(s) for each patient. Physicians were required to answer "How would you rate the benefit of the current treatment of cervical dystonia with botulinum toxin type A compared to the previous treatment using the following scale?". The response options were 'much worse', 'worse', 'somewhat worse', 'same as previous', 'somewhat better', 'better', and 'much better'.

Outcome measures

Measure	BOTOX® n=18 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=24 Participants Botulinum toxin type A (Dysport®)
Physician Comparison of Benefit to Previous Injections at Week 20 Much Worse	0 Number of Responses	0 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 20 Worse	0 Number of Responses	1 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 20 Somewhat Worse	0 Number of Responses	3 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 20 Same as Previous	13 Number of Responses	13 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 20 Somewhat Better	4 Number of Responses	5 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 20 Better	0 Number of Responses	1 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 20 Much Better	1 Number of Responses	1 Number of Responses

SECONDARY outcome

Timeframe: Week 20

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided as least 1 post-dose assessment measure. Only completed assessments at this time point are included.

Patients assessed the improvement in cervical dystonia after receiving the study treatment compared to previous treatment(s). Patients were required to answer "How would you rate the benefit of the current treatment of cervical dystonia with botulinum toxin type A compared to the previous treatment using the following scale?". The response options were 'much worse', 'worse', 'somewhat worse', 'same as previous', 'somewhat better', 'better', and 'much better'.

Outcome measures

Measure	BOTOX® n=19 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=21 Participants Botulinum toxin type A (Dysport®)
Patient Comparison of Benefit to Previous Injections at Week 20 Much Worse	0 Number of Responses	0 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 20 Worse	0 Number of Responses	1 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 20 Somewhat Worse	0 Number of Responses	1 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 20 Same as Previous	8 Number of Responses	11 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 20 Somewhat Better	8 Number of Responses	4 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 20 Better	0 Number of Responses	0 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 20 Much Better	3 Number of Responses	4 Number of Responses

SECONDARY outcome

Timeframe: Baseline, Week 4

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided as least 1 post-dose assessment measure.

Patients were required to assess their pain using a Visual Analog Scale in reference to their current perception of pain at that visit. This scale consisted of a line measuring 100 mm, and patients were instructed to put a mark on the line at the point that best described 'How much pain you are having right now'. Higher scores denoted higher pain intensity: 0 indicated 'No pain' and 100 indicated 'Worst possible pain'.

Outcome measures

Measure	BOTOX® n=24 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=30 Participants Botulinum toxin type A (Dysport®)
Patient Visual Analog Assessment of Pain at Week 4 Baseline	49.5 Units on a Scale Interval 0.0 to 81.0	52.0 Units on a Scale Interval 0.0 to 100.0
Patient Visual Analog Assessment of Pain at Week 4 Week 4	20.5 Units on a Scale Interval 0.0 to 58.0	28.0 Units on a Scale Interval 0.0 to 87.0

SECONDARY outcome

Timeframe: Baseline, Week 4

Population: Modified Intent to Treat: included all patients who were randomized to treatment, received treatment at Visit 1, and provided as least 1 post-dose assessment measure. Only completed assessments at the time points are included.

Patients were queried regarding their need for another injection of botulinum toxin type A for cervical dystonia. Patients were required to answer "How would you rate your need for another injection of botulinum toxin type A for cervical dystonia using the following scale?". The response options included 'absolutely requires injection', 'very much requires injection', 'somewhat requires injection', and 'does not require injection'.

Outcome measures

Measure	BOTOX® n=23 Participants Botulinum toxin type A (BOTOX®)	Dysport® n=29 Participants Botulinum toxin type A (Dysport®)
Patient Assessment of Need for Retreatment at Week 4 Very much requires injection at Week 4	7 Number of Responses	5 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Somewhat requires injection at Week 4	4 Number of Responses	3 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Does not require injection at Week 4	11 Number of Responses	17 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Absolutely requires injection at Baseline	19 Number of Responses	24 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Very much requires injection at Baseline	5 Number of Responses	5 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Somewhat requires injection at Baseline	0 Number of Responses	0 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Does not require injection at Baseline	0 Number of Responses	0 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Absolutely requires injection at Week 4	1 Number of Responses	4 Number of Responses

Adverse Events

BOTOX®

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Dysport®

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	BOTOX® n=24 participants at risk Botulinum toxin type A (BOTOX®)	Dysport® n=30 participants at risk Botulinum toxin type A (Dysport®)
Gastrointestinal disorders Dry Mouth	8.3% 2/24	6.7% 2/30
Gastrointestinal disorders Dysphagia	16.7% 4/24	3.3% 1/30
General disorders Fatigue	4.2% 1/24	10.0% 3/30
Musculoskeletal and connective tissue disorders Muscular Weakness	4.2% 1/24	6.7% 2/30
Musculoskeletal and connective tissue disorders Neck Pain	16.7% 4/24	6.7% 2/30
Respiratory, thoracic and mediastinal disorders Choking	0.00% 0/24	6.7% 2/30

Additional Information

Vice President Medical Affairs

Allergan, Inc.

Phone: 714-246-4500

Email: clinicaltrials@allergan.com

Results disclosure agreements

• Principal investigator is a sponsor employee A disclosure restriction on the PI is that the sponsor can review results communications prior to public release for a period that is not less than 60 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication.
• Publication restrictions are in place

Restriction type: OTHER