Trial Outcomes & Findings for An Observational Study of BOTOX® as Headache Prophylaxis for Chronic Migraine (NCT NCT01686581)
NCT ID: NCT01686581
Last Updated: 2018-11-09
Results Overview
The Baseline value included participants who had been admitted to the hospital for headache in the last 3 months prior to the baseline visit (first administration of BOTOX®); the Last Follow-up value included participants who been admitted to the hospital for headache since the previous visit.
COMPLETED
641 participants
Baseline (previous 3 months prior to first dose of BOTOX®) and Last Follow-up Visit (FU last) [median 21.20 months]
2018-11-09
Participant Flow
Participant milestones
| Measure |
BOTOX®
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Overall Study
STARTED
|
641
|
|
Overall Study
Safety Analysis Set; Received Study Drug
|
633
|
|
Overall Study
Completed 24 Months
|
128
|
|
Overall Study
COMPLETED
|
464
|
|
Overall Study
NOT COMPLETED
|
177
|
Reasons for withdrawal
| Measure |
BOTOX®
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Overall Study
Side-effects or Health Problems
|
15
|
|
Overall Study
Lack of Efficacy
|
90
|
|
Overall Study
Participant Concerned about Risks
|
6
|
|
Overall Study
Thinks Injections Too Painful
|
8
|
|
Overall Study
Thinks Injections Take Too Much Time
|
2
|
|
Overall Study
Trying to get Pregnant or Nursing
|
2
|
|
Overall Study
Thinks it is Inconvenient
|
17
|
|
Overall Study
Other Miscellaneous Reasons
|
36
|
|
Overall Study
Reason Missing
|
1
|
Baseline Characteristics
An Observational Study of BOTOX® as Headache Prophylaxis for Chronic Migraine
Baseline characteristics by cohort
| Measure |
BOTOX®
n=633 Participants
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Age, Continuous
|
45.4 years
STANDARD_DEVIATION 11.7 • n=5 Participants
|
|
Sex: Female, Male
Female
|
540 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
93 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline (previous 3 months prior to first dose of BOTOX®) and Last Follow-up Visit (FU last) [median 21.20 months]Population: Participants from the Safety Analysis Set, all participants who received at least one dose of BOTOX®, with data available for analysis at the given time-point.
The Baseline value included participants who had been admitted to the hospital for headache in the last 3 months prior to the baseline visit (first administration of BOTOX®); the Last Follow-up value included participants who been admitted to the hospital for headache since the previous visit.
Outcome measures
| Measure |
BOTOX®
n=633 Participants
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Percentage of Participants Admitted to the Hospital for Headache
Baseline
|
6.0 percentage of participants
|
|
Percentage of Participants Admitted to the Hospital for Headache
FU last
|
2.1 percentage of participants
|
PRIMARY outcome
Timeframe: Baseline (previous 3 months prior to first dose of BOTOX@) to FU last [median 21.20 months]Population: Participants from the Safety Analysis Set, all participants who received at least one dose of BOTOX®, who were hospitalized.
The number of admission days is presented as the mean, normalized to a period of 90 days. The Baseline value included admissions during the last 3 months prior to the baseline visit (first administration of BOTOX®); the Last Follow-up value included admissions since the second-to-last visit.
Outcome measures
| Measure |
BOTOX®
n=38 Participants
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Mean Number of Days of Headache-related Hospital Admissions
Baseline
|
8.9 days
Standard Deviation 20.14
|
|
Mean Number of Days of Headache-related Hospital Admissions
FU last
|
4.8 days
Standard Deviation 10.98
|
PRIMARY outcome
Timeframe: Baseline (previous 3 months prior to first dose of BOTOX®) to FU last [median 21.20 months]Population: Participants from the Safety Analysis Set, all participants who received at least one dose of BOTOX®, with data available for analysis at the given time-point.
The Baseline value included participants who had visited an HCP in the last 3 months prior to the baseline visit (first administration of Botox); the value for FU last included participants who visited an HCP since the second-to-last visit.
Outcome measures
| Measure |
BOTOX®
n=633 Participants
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Accident and Emergency Visit: Baseline
|
6.3 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Other: FU last
|
0.5 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Any HCP Visit: Baseline
|
45.8 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Any HCP Visit: FU last
|
17.1 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Primary Care Consultant Visit: Baseline
|
30.6 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Primary Care Consultant Visit: FU last
|
12.7 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Outpatient Consultation: Baseline
|
33.3 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Outpatient Consultation: FU last
|
6.8 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Accident and Emergency Visit: FU last
|
2.6 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Alternative Practitioner: Baseline
|
6.3 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Alternative Practitioner: FU last
|
2.0 percentage of participants
|
|
Percentage of Participants Who Visited Any Healthcare Professional (HCP)
Other: Baseline
|
4.3 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline to Last Administration of BOTOX® (ADM last) [median 20.30 months]Population: Safety Analysis Set, all participants who received at least one dose of BOTOX®, with data available for analysis at the given time-point.
The MSQ is a 14-item questionnaire to measure health-related quality-of-life attributed to migraine in the past 4 weeks. Each item is scored on a 6-point scale where: 1=none of the time to 6=all of the time. There are 3 dimensions: Role-function Restrictive (questions 1 to 7; score range 7 to 42), Role-function Preventive (questions 8 to 11; score range 4 to 24) and Emotional-function (questions 12 to 14; score range 3 to 18). The individual dimension scores were converted to a score of 0 to 100; the total score ranged from 0 to 300 with higher numbers representing a better quality of life. A positive change from baseline in the dimension scores and the total score indicates that quality of life has improved.
Outcome measures
| Measure |
BOTOX®
n=633 Participants
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Change From Baseline in Migraine-Specific Quality of Life Questionnaire (MSQ) Score
Role Function-Restrictive: Baseline (BL)
|
37.14 score on a scale
Interval 0.0 to 100.0
|
|
Change From Baseline in Migraine-Specific Quality of Life Questionnaire (MSQ) Score
Emotional Function: Baseline
|
40.00 score on a scale
Interval 0.0 to 100.0
|
|
Change From Baseline in Migraine-Specific Quality of Life Questionnaire (MSQ) Score
Emotional Function: Change from BL to ADM last
|
26.67 score on a scale
Interval -60.0 to 100.0
|
|
Change From Baseline in Migraine-Specific Quality of Life Questionnaire (MSQ) Score
Total Score: Baseline
|
132.86 score on a scale
Interval 0.0 to 300.0
|
|
Change From Baseline in Migraine-Specific Quality of Life Questionnaire (MSQ) Score
Role Function-Restrictive: Change BL to ADM last
|
25.71 score on a scale
Interval -28.6 to 97.1
|
|
Change From Baseline in Migraine-Specific Quality of Life Questionnaire (MSQ) Score
Role Function-Preventive: Baseline
|
50.0 score on a scale
Interval 0.0 to 100.0
|
|
Change From Baseline in Migraine-Specific Quality of Life Questionnaire (MSQ) Score
Role Function-Preventive: Change BL to ADM last
|
20.00 score on a scale
Interval -55.0 to 100.0
|
|
Change From Baseline in Migraine-Specific Quality of Life Questionnaire (MSQ) Score
Total Score: Change from BL to ADM last
|
70.95 score on a scale
Interval -96.2 to 297.1
|
SECONDARY outcome
Timeframe: Baseline (prior to first dose of BOTOX®) to ADM last [median 20.30 months]Population: Participant from the Safety Analysis Set, all participants who received at least one dose of BOTOX®, with data available at the given time-point.
The EQ-5D consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) assessed by the participant using a 3-point scale: 1=no problems, 2=some problems and 3=extreme problems. The combination of levels from the 5 dimensions results in a health state code. The total score is calculated by converting the health state code into a score: start with score 1.000=\[11111\] (perfect health state), subtract 0.081 (constant) for any other state, subtract nothing for level 1 on any dimension, subtract appropriate level 2 or level 3 value for each dimension from a table of constants \[Level 2: Mobility 0.069, Self-care 0.104, Usual activity 0.036, Pain/discomfort 0.123, Anxiety/depression 0.071\] \[Level 3: Mobility 0.314, Self-care 0.214, Usual activity 0.094, Pain/discomfort 0.386, Anxiety/depression 0.236\], subtract 0.269 if any dimension has a level 3 problem. Higher numbers indicate better health. A positive change from Baseline indicates improvement.
Outcome measures
| Measure |
BOTOX®
n=633 Participants
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Change From Baseline in the EQ-5D Questionnaire Total Score
Change from Baseline to ADM last
|
0.107 score on a scale
Interval -0.889 to 1.39
|
|
Change From Baseline in the EQ-5D Questionnaire Total Score
Baseline
|
0.69 score on a scale
Interval -0.59 to 1.0
|
SECONDARY outcome
Timeframe: Baseline (prior to first dose of BOTOX®) to ADM last [median 20.30 months]Population: Participants from the Safety Analysis Set, all participants who received at least one dose of BOTOX®, with data available at the given time-point.
The EQ-5D health state scale is a visual analog scale that ranges from 0 (worst imaginable health state) to 100 (best imaginable health state), with higher scores indicating a better state of health. Participants were asked to rate their health state on a drawn line that started at 0 and ended at 100. A positive change from baseline in the health state score indicates that the participant's health state has improved.
Outcome measures
| Measure |
BOTOX®
n=633 Participants
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Change From Baseline in the Health State Score of the EQ-5D Questionnaire
Baseline
|
50.0 score on a scale
Interval 0.0 to 100.0
|
|
Change From Baseline in the Health State Score of the EQ-5D Questionnaire
Change from Baseline to ADM last
|
10.0 score on a scale
Interval -86.0 to 91.0
|
SECONDARY outcome
Timeframe: Baseline (prior to first dose of BOTOX®) to ADM last [median 20.30 months]Population: Participants from the Safety Analysis Set, all participants who received at least one dose of BOTOX®, with data available at the given time-point.
At each visit, participants were asked to provide the number of headache days he/she experienced in the last month. A headache day was defined as 4 or more hours of continuous headache. A negative change from Baseline (less headache days) indicates improvement.
Outcome measures
| Measure |
BOTOX®
n=633 Participants
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Change From Baseline in the Number of Headache Days
Baseline
|
20.0 days
Interval 8.0 to 31.0
|
|
Change From Baseline in the Number of Headache Days
Change from Baseline to ADM last
|
-11.0 days
Interval -30.0 to 15.0
|
SECONDARY outcome
Timeframe: ADM last [median 20.30 months]Population: Participants from the Safety Analysis Set, all participants who received at least one dose of BOTOX®, with data available.
At each visit, participants and physicians were asked to indicate the level of satisfaction that he/she had with the treatment. Physicians indicated the level of satisfaction with the patient's treatment. The 4- point scale consisted of the following responses: insufficient, moderate, good and very good. The combined percentage of "good" and "very good" responses by participants and physicians are reported.
Outcome measures
| Measure |
BOTOX®
n=571 Participants
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Percentage of Participants With Good or Very Good Responses on the 4-Point Treatment Satisfaction Scale
Physician Assessment
|
78.1 percentage of participants
|
|
Percentage of Participants With Good or Very Good Responses on the 4-Point Treatment Satisfaction Scale
Patient Assessment
|
76.0 percentage of participants
|
Adverse Events
BOTOX®
Serious adverse events
| Measure |
BOTOX®
n=633 participants at risk
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Spinal disorder
|
0.16%
1/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
|
Psychiatric disorders
Mental disorder
|
0.16%
1/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
|
Psychiatric disorders
Depression
|
0.16%
1/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
|
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous
|
0.16%
1/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
|
Psychiatric disorders
Psychosomatic disease
|
0.16%
1/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
|
Nervous system disorders
Migraine
|
0.16%
1/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.16%
1/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
|
Nervous system disorders
Headache
|
0.16%
1/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
|
Gastrointestinal disorders
Vomiting
|
0.16%
1/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
Other adverse events
| Measure |
BOTOX®
n=633 participants at risk
BOTOX® (botulinum toxin Type A) administered according to physician prescription for the treatment of chronic migraine; all treatment decisions lie with the physician.
|
|---|---|
|
Eye disorders
Eyelid ptosis
|
5.4%
34/633 • First dose of study drug to FU last [median 21.20 months]
As per the protocol, Adverse Drug Reactions and Serious Adverse Drug Reactions were collected.
|
Additional Information
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 and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER