Trial Outcomes & Findings for Comparison of Efficacy and Safety of Two Different Types of Botulinum Toxin Type A in Moderate to Severe Cervical Dystonia (NCT NCT00528541)

NCT ID: NCT00528541

Last Updated: 2011-12-16

Results Overview

Dysphagia Incidence (difficulty swallowing) was defined as the number of patients reporting at least 1 treatment-emergent dysphagia event at any point in the study. Occurrences of dysphagia were captured as spontaneous events or were assessed during study visits using the Structured Symptom Interview (SSI) and the Dystonia Study Group Dysphagia Interview (DSGDI) for symptoms of difficulty swallowing; coughing while eating and drinking; choking while eating or drinking; or difficulty swallowing solids or liquids.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 145 participants
• Primary outcome timeframe: 10 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	75	70
Overall Study COMPLETED	73	69
Overall Study NOT COMPLETED	2	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Comparison of Efficacy and Safety of Two Different Types of Botulinum Toxin Type A in Moderate to Severe Cervical Dystonia

Baseline characteristics by cohort

Measure	BOTOX® n=75 Participants botulinum toxin type A (BOTOX®)	Dysport® n=70 Participants botulinum toxin type A (Dysport®)	Total n=145 Participants Total of all reporting groups
Age Continuous	49.4 years STANDARD_DEVIATION 12.2 • n=5 Participants	48.6 years STANDARD_DEVIATION 11.5 • n=7 Participants	49.0 years STANDARD_DEVIATION 11.9 • n=5 Participants
Sex: Female, Male Female	46 Participants n=5 Participants	39 Participants n=7 Participants	85 Participants n=5 Participants
Sex: Female, Male Male	29 Participants n=5 Participants	31 Participants n=7 Participants	60 Participants n=5 Participants

PRIMARY outcome

Timeframe: 10 weeks

Population: Modified intent to treat: all patients who were randomized to treatment and received 1 dose at Visit 1.

Dysphagia Incidence (difficulty swallowing) was defined as the number of patients reporting at least 1 treatment-emergent dysphagia event at any point in the study. Occurrences of dysphagia were captured as spontaneous events or were assessed during study visits using the Structured Symptom Interview (SSI) and the Dystonia Study Group Dysphagia Interview (DSGDI) for symptoms of difficulty swallowing; coughing while eating and drinking; choking while eating or drinking; or difficulty swallowing solids or liquids.

Outcome measures

Measure	BOTOX® n=75 Participants botulinum toxin type A (BOTOX®)	Dysport® n=70 Participants botulinum toxin type A (Dysport®)
Dysphagia Incidence Over 10 Weeks	10 Number of patients	12 Number of patients

SECONDARY outcome

Timeframe: Baseline, Week 4

Population: Modified intent to treat: all patients who were randomized to treatment and received 1 dose at Visit 1.

The TWSTRS assessments were conducted at each study visit. The TWSTRS is an assessment scale used to measure the impact of cervical dystonia on patients. It 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=75 Participants botulinum toxin type A (BOTOX®)	Dysport® n=70 Participants botulinum toxin type A (Dysport®)
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4 Baseline	40.0 Scores on a Scale Interval 19.0 to 70.0	39.5 Scores on a Scale Interval 25.0 to 68.0
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4 Week 4	22.0 Scores on a Scale Interval 3.0 to 55.0	25.0 Scores on a Scale Interval 11.0 to 49.0

SECONDARY outcome

Timeframe: Baseline, Week 4

Population: Modified intent to treat: all patients who were randomized to treatment and received 1 dose at Visit 1.

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=75 Participants botulinum toxin type A (BOTOX®)	Dysport® n=70 Participants botulinum toxin type A (Dysport®)
Physician Assessment of Cervical Dystonia Severity at Week 4 Baseline	6.0 Units on a Scale Interval 2.0 to 9.0	7.0 Units on a Scale Interval 2.0 to 9.0
Physician Assessment of Cervical Dystonia Severity at Week 4 Week 4	2.0 Units on a Scale Interval 1.0 to 7.0	3.0 Units on a Scale Interval 1.0 to 7.0

SECONDARY outcome

Timeframe: Week 4

Population: Modified intent to treat: all patients who were randomized to treatment and received 1 dose at Visit 1.

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=75 Participants botulinum toxin type A (BOTOX®)	Dysport® n=70 Participants botulinum toxin type A (Dysport®)
Global Assessment of Benefit by Physician at Week 4	3.0 Units on a Scale Interval -1.0 to 3.0	3.0 Units on a Scale Interval -1.0 to 3.0

SECONDARY outcome

Timeframe: Week 4

Population: Modified intent to treat: all patients who were randomized to treatment and received 1 dose at Visit 1.

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=75 Participants botulinum toxin type A (BOTOX®)	Dysport® n=70 Participants botulinum toxin type A (Dysport®)
Global Assessment of Benefit by Patient at Week 4	3.0 Units on a Scale Interval -2.0 to 3.0	3.0 Units on a Scale Interval -1.0 to 3.0

SECONDARY outcome

Timeframe: Baseline, Week 4

Population: Modified intent to treat: all patients who were randomized to treatment and received 1 dose at Visit 1. 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=73 Participants botulinum toxin type A (BOTOX®)	Dysport® n=69 Participants botulinum toxin type A (Dysport®)
Patient Assessment of Need for Retreatment at Week 4 Absolutely requires injection at Baseline	54 Number of Responses	54 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Very much requires injection at Baseline	19 Number of Responses	15 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Somewhat requires injection at Baseline	1 Number of Responses	1 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	17 Number of Responses	15 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Very much requires injection at Week 4	10 Number of Responses	15 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Somewhat requires injection at Week 4	18 Number of Responses	18 Number of Responses
Patient Assessment of Need for Retreatment at Week 4 Does not require injection at Week 4	2 Number of Responses	21 Number of Responses

SECONDARY outcome

Timeframe: Baseline, Week 4

Population: Modified intent to treat: all patients who were randomized to treatment and received 1 dose at Visit 1.

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=75 Participants botulinum toxin type A (BOTOX®)	Dysport® n=70 Participants botulinum toxin type A (Dysport®)
Patient Visual Analog Assessment of Pain at Week 4 Baseline	30.5 Units on a Scale Interval 0.0 to 100.0	39.5 Units on a Scale Interval 0.0 to 92.0
Patient Visual Analog Assessment of Pain at Week 4 Week 4	8.0 Units on a Scale Interval 0.0 to 72.0	13.0 Units on a Scale Interval 0.0 to 75.0

SECONDARY outcome

Timeframe: Week 10

Population: Modified intent to treat: all patients who were randomized to treatment and received 1 dose at Visit 1. 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=73 Participants botulinum toxin type A (BOTOX®)	Dysport® n=69 Participants botulinum toxin type A (Dysport®)
Physician Comparison of Benefit to Previous Injections at Week 10 Much Worse	0 Number of Responses	0 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 10 Worse	0 Number of Responses	1 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 10 Somewhat Worse	7 Number of Responses	7 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 10 Same as Previous	30 Number of Responses	31 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 10 Somewhat Better	25 Number of Responses	15 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 10 Better	7 Number of Responses	12 Number of Responses
Physician Comparison of Benefit to Previous Injections at Week 10 Much Better	4 Number of Responses	3 Number of Responses

SECONDARY outcome

Timeframe: Week 10

Population: Modified intent to treat: all patients who were randomized to treatment and received 1 dose at Visit 1. Only completed assessments at this timepoint 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=72 Participants botulinum toxin type A (BOTOX®)	Dysport® n=68 Participants botulinum toxin type A (Dysport®)
Patient Comparison of Benefit to Previous Injections at Week 10 Much Worse	0 Number of Responses	0 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 10 Worse	1 Number of Responses	2 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 10 Somewhat Worse	8 Number of Responses	10 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 10 Same as Previous	30 Number of Responses	20 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 10 Somewhat Better	24 Number of Responses	17 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 10 Better	8 Number of Responses	12 Number of Responses
Patient Comparison of Benefit to Previous Injections at Week 10 Much Better	1 Number of Responses	7 Number of Responses

Adverse Events

BOTOX®

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Dysport®

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	BOTOX® n=75 participants at risk botulinum toxin type A (BOTOX®)	Dysport® n=70 participants at risk botulinum toxin type A (Dysport®)
Gastrointestinal disorders Dysphagia	6.7% 5/75	5.7% 4/70

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