Trial Outcomes & Findings for Randomized, Placebo-Controlled Study of AbobotulinumtoxinA (Dysport®) for the Treatment of Cervical Dystonia (NCT NCT00257660)

NCT ID: NCT00257660

Last Updated: 2022-09-28

Results Overview

TWSTRS is comprised of three different components which are severity, disability \& pain. There is an ordinal scale for each component and the score range for each is the following: for severity from 0 (absence of severity) to 35 (max severity), for disability from 0 (no disability) to 30 (max disability) and for pain from 0 (no pain) to 20 (max pain). TWSTRS total score is the sum of the 3 component scores, with a range from 0 to a maximum of 85. The change in TWSTRS total score is the score at week 4 minus the score at baseline.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

116 participants

Primary outcome timeframe

Baseline and Week 4

Results posted on

2022-09-28

Participant Flow

Patients were recruited at 16 centres in the USA and 4 centres in Russia from October 2005 until September 2006

Participant milestones

Participant milestones
Measure
Dysport 500 Units
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Placebo
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Overall Study
STARTED
55
61
Overall Study
COMPLETED
45
38
Overall Study
NOT COMPLETED
10
23

Reasons for withdrawal

Reasons for withdrawal
Measure
Dysport 500 Units
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Placebo
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Overall Study
Lack of Efficacy
5
23
Overall Study
Withdrawal by Subject
2
0
Overall Study
Lost to Follow-up
1
0
Overall Study
PI & Subject Schedule Conflicts
1
0
Overall Study
No longer wanted to do blood draws
1
0

Baseline Characteristics

Randomized, Placebo-Controlled Study of AbobotulinumtoxinA (Dysport®) for the Treatment of Cervical Dystonia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Dysport 500 Units
n=55 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Placebo
n=61 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Total
n=116 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Age, Categorical
Between 18 and 65 years
46 Participants
n=99 Participants
50 Participants
n=107 Participants
96 Participants
n=206 Participants
Age, Categorical
>=65 years
9 Participants
n=99 Participants
11 Participants
n=107 Participants
20 Participants
n=206 Participants
Age, Continuous
51.9 years
STANDARD_DEVIATION 13.4 • n=99 Participants
53.9 years
STANDARD_DEVIATION 12.5 • n=107 Participants
53.0 years
STANDARD_DEVIATION 12.9 • n=206 Participants
Sex: Female, Male
Female
37 Participants
n=99 Participants
38 Participants
n=107 Participants
75 Participants
n=206 Participants
Sex: Female, Male
Male
18 Participants
n=99 Participants
23 Participants
n=107 Participants
41 Participants
n=206 Participants
Region of Enrollment
United States
41 participants
n=99 Participants
47 participants
n=107 Participants
88 participants
n=206 Participants
Region of Enrollment
Russian Federation
14 participants
n=99 Participants
14 participants
n=107 Participants
28 participants
n=206 Participants

PRIMARY outcome

Timeframe: Baseline and Week 4

Population: The analysis was performed on the intention to treat population which consisted of 55 subjects receiving Dysport and 61 Placebo. There were 4 subjects for Dysport and 3 for placebo who were not assessed on TWSTRS score at Week 4. As there was no imputation of missing TWSTRS score values, these 7 subjects were not taken into account.

TWSTRS is comprised of three different components which are severity, disability \& pain. There is an ordinal scale for each component and the score range for each is the following: for severity from 0 (absence of severity) to 35 (max severity), for disability from 0 (no disability) to 30 (max disability) and for pain from 0 (no pain) to 20 (max pain). TWSTRS total score is the sum of the 3 component scores, with a range from 0 to a maximum of 85. The change in TWSTRS total score is the score at week 4 minus the score at baseline.

Outcome measures

Outcome measures
Measure
Placebo
n=58 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=51 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)
Change in TWSTRS total score
-5.23 points on a scale
Standard Deviation 9.33
-13.99 points on a scale
Standard Deviation 12.33
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)
Baseline TWSTRS total score
45.81 points on a scale
Standard Deviation 8.78
43.83 points on a scale
Standard Deviation 7.97
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)
Week 4 TWSTRS total score
40.22 points on a scale
Standard Deviation 11.75
30.04 points on a scale
Standard Deviation 12.65

SECONDARY outcome

Timeframe: Baseline and Week 8

Population: The analysis was performed on the intention to treat population which consisted of 55 subjects receiving Dysport and 61 Placebo. There were 9 subjects for Dysport and 15 for placebo who were not assessed on TWSTRS score at Week 8. As there was no imputation of missing TWSTRS score values, these 24 subjects were not taken into account.

TWSTRS is comprised of three different components which are severity, disability \& pain. There is an ordinal scale for each component and the score range for each is the following: for severity from 0 (absence of severity) to 35 (max severity), for disability from 0 (no disability) to 30 (max disability) and for pain from 0 (no pain) to 20 (max pain). TWSTRS total score is the sum of the 3 component scores, with a range from 0 to a maximum of 85. The change in TWSTRS total score is the score at week 4 minus the score at baseline.

Outcome measures

Outcome measures
Measure
Placebo
n=46 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=46 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)
Change in TWSTRS total score
-5.59 points on a scale
Standard Deviation 11.37
-13.82 points on a scale
Standard Deviation 11.53
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)
Baseline TWSTRS total score
45.81 points on a scale
Standard Deviation 8.78
43.83 points on a scale
Standard Deviation 7.97
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)
Week 8 TWSTRS total score
39.64 points on a scale
Standard Deviation 13.50
29.31 points on a scale
Standard Deviation 10.99

SECONDARY outcome

Timeframe: Baseline and Week 12

Population: The analysis was performed on the intention to treat population which consisted of 55 subjects receiving Dysport and 61 Placebo. There were 10 subjects for Dysport and 17 for placebo who were not assessed on TWSTRS score at Week 12. As there was no imputation of missing TWSTRS score values, these 27 subjects were not taken into account.

TWSTRS is comprised of three different components which are severity, disability \& pain. There is an ordinal scale for each component and the score range for each is the following: for severity from 0 (absence of severity) to 35 (max severity), for disability from 0 (no disability) to 30 (max disability) and for pain from 0 (no pain) to 20 (max pain). TWSTRS total score is the sum of the 3 component scores, with a range from 0 to a maximum of 85. The change in TWSTRS total score is the score at week 4 minus the score at baseline.

Outcome measures

Outcome measures
Measure
Placebo
n=44 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=45 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score
Change in TWSTRS total score
-4.53 points on a scale
Standard Deviation 7.75
-6.98 points on a scale
Standard Deviation 11.12
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score
Baseline TWSTRS total score
45.81 points on a scale
Standard Deviation 8.78
43.83 points on a scale
Standard Deviation 7.97
Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score
Week 12 TWSTRS total score
40.76 points on a scale
Standard Deviation 11.08
36.04 points on a scale
Standard Deviation 11.76

SECONDARY outcome

Timeframe: Baseline and Week 4

Population: Analysis was performed on intention to treat population which consisted of 55 subjects on Dysport and 61 on Placebo. There were 5 subjects on Dysport and 8 on placebo who were not assessed on the change in subject VAS score for CD symptoms at Week 4. There was no imputation of missing VAS scores, so these 13 subjects were not taken into account.

The assessment was a continuous 100 mm horizonal line with a scale of 0 mm (no symptoms)to 100 mm (worst possible symptoms).

Outcome measures

Outcome measures
Measure
Placebo
n=53 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=50 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Subject Visual Analogue Score (VAS) for Cervical Dystonia (CD) Symptom Assessment
Change in Subject VAS for CD symptoms
-6.7 points on a scale
Standard Deviation 20.8
-24.2 points on a scale
Standard Deviation 29.5
Subject Visual Analogue Score (VAS) for Cervical Dystonia (CD) Symptom Assessment
Baseline Subject VAS for CD symptoms
63.6 points on a scale
Standard Deviation 18.9
67.7 points on a scale
Standard Deviation 19.7
Subject Visual Analogue Score (VAS) for Cervical Dystonia (CD) Symptom Assessment
Week 4 Subject VAS for CD symptoms
55.7 points on a scale
Standard Deviation 20.6
44.3 points on a scale
Standard Deviation 24.4

SECONDARY outcome

Timeframe: Baseline and Week 4

Population: Analysis was performed on intention to treat population which consisted of 55 subjects receiving Dysport and 61 Placebo. There were 3 subjects for Dysport and 5 for placebo who were not assessed on the change in investigator VAS score at Week 4. As there was no imputation of missing VAS scores, these 8 subjects were not taken into account.

The assessment was a continuous 100 mm horizonal line with a scale of 0 mm (no symptoms)to 100 mm (worst possible symptoms).

Outcome measures

Outcome measures
Measure
Placebo
n=56 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=52 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Investigator VAS for CD Symptom Assessment
Change in Investigator VAS for CD symptoms
-9.1 points on a scale
Standard Deviation 20.2
-23.1 points on a scale
Standard Deviation 22.1
Investigator VAS for CD Symptom Assessment
Baseline Investigator VAS for CD symptoms
65.3 points on a scale
Standard Deviation 18.0
62.3 points on a scale
Standard Deviation 15.8
Investigator VAS for CD Symptom Assessment
Week 4 Investigator VAS for CD symptoms
56.8 points on a scale
Standard Deviation 21.8
40.3 points on a scale
Standard Deviation 21.7

SECONDARY outcome

Timeframe: Baseline and Week 8

Population: Analysis was performed on intention to treat population which consisted of 55 subjects on Dysport and 61 on Placebo. Missing assessments at Week 8 were imputed using Last Observation Carried Forward (LOCF) methodology. There was no imputation for 4 patients on placebo with missing baseline and Week 4 values.

The assessment was a continuous 100 mm horizonal line with a scale of 0 mm (no symptoms)to 100mm (worst possible symptoms).

Outcome measures

Outcome measures
Measure
Placebo
n=57 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=55 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Subject VAS for CD Symptom Assessment
Change in subject VAS for CD symptoms
-5.4 points on a scale
Standard Deviation 25.4
-24.6 points on a scale
Standard Deviation 30.0
Subject VAS for CD Symptom Assessment
Baseline subject VAS for CD symptoms
63.6 points on a scale
Standard Deviation 18.9
67.7 points on a scale
Standard Deviation 19.7
Subject VAS for CD Symptom Assessment
Week 8 subject VAS for CD symptoms
57.8 points on a scale
Standard Deviation 27.5
44.7 points on a scale
Standard Deviation 24.9

SECONDARY outcome

Timeframe: Baseline and week 8

Population: Analysis was performed on intention to treat population which consisted of 55 subjects on Dysport and 61 on Placebo. Missing assessments at Week 8 were imputed using Last Observation Carried Forward (LOCF) methodology. There was no imputation for 3 patients with missing baseline and Week 4 values.

The assessment was a continuous 100 mm horizonal line with a scale of 0 mm (no symptoms)to 100 mm (worst possible symptoms).

Outcome measures

Outcome measures
Measure
Placebo
n=59 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=54 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Investigator's VAS for CD Symptom Assessment
Week 8 Investigator VAS for CD symptoms
60.8 points on a scale
Standard Deviation 24.6
40.8 points on a scale
Standard Deviation 22.3
Investigator's VAS for CD Symptom Assessment
Change in Investigator VAS for CD symptoms
-5.0 points on a scale
Standard Deviation 21.3
-20.5 points on a scale
Standard Deviation 22.7
Investigator's VAS for CD Symptom Assessment
Baseline Investigator VAS for CD symptoms
65.3 points on a scale
Standard Deviation 18.0
62.3 points on a scale
Standard Deviation 15.8

SECONDARY outcome

Timeframe: Baseline and week 12

Population: Analysis was performed on intention to treat population which consisted of 55 subjects on Dysport and 61 on Placebo. Missing assessments at Week 12 were imputed using Last Observation Carried Forward (LOCF) methodology. There was no imputation for 4 patients on placebo with missing baseline, Week 4 and Week 8 values.

The assessment was a continuous 100 mm horizonal line with a scale of 0 mm (no symptoms)to 100 mm (worst possible symptoms).

Outcome measures

Outcome measures
Measure
Placebo
n=57 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=55 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Subject VAS for CD Symptom Assessment
Change in subject VAS for CD symptoms
-4.6 points on a scale
Standard Deviation 23.3
-14.4 points on a scale
Standard Deviation 25.2
Subject VAS for CD Symptom Assessment
Baseline subject VAS for CD symptoms
63.6 points on a scale
Standard Deviation 18.9
67.7 points on a scale
Standard Deviation 19.7
Subject VAS for CD Symptom Assessment
Week 12 subject VAS for CD symptoms
58.2 points on a scale
Standard Deviation 25.7
55.4 points on a scale
Standard Deviation 25.5

SECONDARY outcome

Timeframe: Baseline and week 12

Population: Analysis was performed on intention to treat population which consisted of 55 subjects on Dysport and 61 on Placebo. Missing assessments at Week 12 were imputed using Last Observation Carried Forward (LOCF) methodology. There was no imputation for 3 patients with missing baseline, Week 4 and Week 8 values.

The assessment was a continuous 100 mm horizonal line with a scale of 0 mm (no symptoms) to 100 mm (worst possible symptoms).

Outcome measures

Outcome measures
Measure
Placebo
n=59 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=54 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Investigator's VAS for CD Symptom Assessment
Change in Investigator VAS for CD symptoms
-5.8 points on a scale
Standard Deviation 15.8
-8.5 points on a scale
Standard Deviation 18.9
Investigator's VAS for CD Symptom Assessment
Baseline Investigator VAS for CD symptoms
65.3 points on a scale
Standard Deviation 18.0
62.3 points on a scale
Standard Deviation 15.8
Investigator's VAS for CD Symptom Assessment
Week 12 Investigator VAS for CD symptoms
60.0 points on a scale
Standard Deviation 21.5
54.8 points on a scale
Standard Deviation 20.8

SECONDARY outcome

Timeframe: Week 8

Population: Analysis was performed on intention to treat population which consisted of 55 subjects on Dysport and 61 on Placebo. There were 13 subjects on Dysport and 24 on placebo who were not assessed on the change in mental health summary at Week 8. There was no imputation of missing scores, so these 37 subjects were not taken into account.

SF-36 is a Quality of Life scale comprising eight individual domains. The QoL score for each domain is on a scale from 0 (worst health possible) to 100 (best health possible). SF-36 Mental Health Summary Score is derived from four individual domains (vitality, social functioning, role limitations due to emotional problems and mental health).

Outcome measures

Outcome measures
Measure
Placebo
n=37 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=42 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
SF-36 Mental Health Summary Score
Change in SF-36 Mental health summary Score
2.48 points on a scale
Standard Deviation 8.12
4.11 points on a scale
Standard Deviation 9.18
SF-36 Mental Health Summary Score
Baseline SF-36 Mental health summary Score
43.31 points on a scale
Standard Deviation 11.14
44.52 points on a scale
Standard Deviation 10.41
SF-36 Mental Health Summary Score
Week 8 SF-36 Mental health summary Score
43.41 points on a scale
Standard Deviation 12.30
49.00 points on a scale
Standard Deviation 8.69

SECONDARY outcome

Timeframe: Week 8

Population: Analysis was performed on intention to treat population which consisted of 55 subjects on Dysport and 61 on Placebo. There were 13 subjects on Dysport and 24 on placebo who were not assessed on the change in physical health summary at Week 8. There was no imputation of missing scores, so these 37 subjects were not taken into account.

SF-36 is a Quality of Life scale comprising eight individual domains. The QoL score for each domain is on a scale from 0 (worst health possible) to 100 (best health possible). SF-36 Physical Health Summary Score is derived from four individual domains (physical functioning, role physical, bodily pain and general health).

Outcome measures

Outcome measures
Measure
Placebo
n=37 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=42 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
SF-36 Physical Health Summary Score
Change in SF-36 Physical health summary score
-0.64 points on a scale
Standard Deviation 6.41
4.37 points on a scale
Standard Deviation 5.46
SF-36 Physical Health Summary Score
Baseline SF-36 Physical health summary score
43.18 points on a scale
Standard Deviation 7.89
39.42 points on a scale
Standard Deviation 8.84
SF-36 Physical Health Summary Score
Week 8 SF-36 Physical health summary score
42.49 points on a scale
Standard Deviation 8.84
43.70 points on a scale
Standard Deviation 8.76

SECONDARY outcome

Timeframe: Week 12

Population: Analysis was performed on intention to treat population which consisted of 55 participants on Dysport and 61 on Placebo.

The number of participants considered to be overall treatment successes by the investigator at week 12 was assessed.

Outcome measures

Outcome measures
Measure
Placebo
n=61 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Dysport 500 Units
n=55 Participants
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Number of Participants Considered by the Investigator to be Overall Treatment Successes
10 participants
32 participants

Adverse Events

Dysport 500 Units

Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths

Placebo

Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Dysport 500 Units
n=55 participants at risk
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Placebo
n=61 participants at risk
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Psychiatric disorders
Suicide attempt
0.00%
0/55
1.6%
1/61 • Number of events 1

Other adverse events

Other adverse events
Measure
Dysport 500 Units
n=55 participants at risk
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Placebo
n=61 participants at risk
Single intramuscular injection into the clinically indicated neck muscles in a single dosing session wherever possible
Gastrointestinal disorders
Dysphagia
9.1%
5/55 • Number of events 5
0.00%
0/61
General disorders
Injection site pain
5.5%
3/55 • Number of events 3
3.3%
2/61 • Number of events 2
Musculoskeletal and connective tissue disorders
Neck pain
5.5%
3/55 • Number of events 4
4.9%
3/61 • Number of events 4

Additional Information

Senior Director, Medical Development, Neurology

Ipsen

Results disclosure agreements

  • Principal investigator is a sponsor employee A publication of results from all sites in this multicenter study is expected. If the sponsor no longer plans this or 6 months after study completion at all sites, the PI may publish the results. He will supply any proposed publication to the sponsor for review and comment at least 30 days before submission for publication. If the sponsor believes a patent application should be filed, the publication will be delayed for another 60 days or until patent filing; with a maximum delay of 90 days.
  • Publication restrictions are in place

Restriction type: OTHER