Moderate to Severe Glabellar Frown Lines
Conditions
Brief summary
The purpose of the study is to investigate the safety and duration of effect following different doses of Botulinum Toxin A (NT 201) in the treatment of glabellar frown lines (GFL).
Detailed description
This prospective, randomized, double-blind, multi-Center clinical study consists of a two stage experimental main period comparing different dose groups, followed by an optional open-label extension period (20 Units follow-up treatment).
Interventions
DRUGNT 201
Clostridium Botulinum neurotoxin Type A free from complexing proteins. Solution for injection prepared by reconstitution of powder with 0.9% unpreserved Sodium Chloride (NaCl).
Sponsors
Merz Pharmaceuticals GmbH
Merz Aesthetics GmbH
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female participant 18 years or over. * Moderate (score=2) to severe (score=3) GFL at maximum frown as assessed by investigator on the 4-point facial wrinkle scale (FWS). * Moderate (score=2) to severe (score=3) GFL at maximum frown as assessed by participant on the 4-point FWS.
Exclusion criteria
* Previous treatment with Botulinum neurotoxin (BoNT) of any serotype in the facial area within the last 12 months before injection. * Previous treatment with any facial cosmetic procedure (example, chemical peeling, photo rejuvenation, mesotherapy, photodynamic therapy, laser treatment, tattooing of eyebrows) in the glabellar area within the last 12 months before injection. * Previous treatment with any biodegradable filler in the glabellar area within the last 12 months before injection. * Inability to substantially reduce GFL by physically spreading them apart as assessed by the investigator. * Excessively thick sebaceous skin or hypertrophic muscles in the upper third part of the face. * Any surgery or scars in the glabellar area. * Marked facial asymmetry. * Eyelid ptosis. * Marked brow ptosis and/or dermatochalasis. * Ongoing severe or unstable medical conditions, example, systemic infection, or pulmonary disease, at the discretion of the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With at Least One Treatment Related TESAE | From the time of first treatment up to Day 390 | — |
| Duration of Effect Defined as Time Between Treatment and Relapse to Baseline Status Assessed by the Investigator on the FWS | From the time of first treatment up to Day 360 | Duration of effect: time between treatment and first occurrence of relapse to baseline status. If no effect was observed, duration of effect was set to 0. Effect: any improvement (at least 1 point) at maximum frown as assessed by investigator on FWS. Investigator's FWS assessed status of GFL according to 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). Duration of effect was analyzed using Kaplan-meier analysis, and 95 percent (%) confidence interval (CI) were calculated using log-log transformation. |
| Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE) | From the time of first treatment up to Day 390 | — |
| Number of Participants With at Least One Treatment-emergent Serious Adverse Event (TESAE) | From the time of first treatment up to Day 390 | — |
| Number of Participants With at Least One Treatment-emergent Adverse Event of Special Interest (TEAESI) | From the time of first treatment up to Day 390 | — |
| Number of Participants With at Least One Treatment Related TEAE | From the time of first treatment up to Day 390 | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Duration of Effect Whereby Effect Was Defined as Score of None (0) or Mild (1) at Maximum Frown as Assessed by the Investigator According to FWS | From the time of first treatment up to Day 360 | Duration of effect was defined as time between treatment and first point in time when score was moderate or severe again. If no effect was observed, duration of effect was set to 0. Effect was defined by a score of none (0) or mild (1) at maximum frown as assessed by investigator according to FWS. Investigator's FWS assessed status of GFL according to 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). Duration of effect was analyzed using Kaplan-meier analysis, and 95% CI were calculated using log-log transformation. |
| Duration of Effect Whereby Effect Was Defined by 2-point Improvement From Baseline at Maximum Frown as Assessed by the Investigator According to FWS | From the time of first treatment up to Day 360 | Duration of effect: time between treatment and first point in time when improvement was less than 2 points again. If no effect was observed, duration of effect was set to 0. Effect: at least a 2-point improvement from baseline at maximum frown as assessed by investigator on the FWS. Investigator's FWS assessed status of GFL according to 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). Duration of effect was analyzed using Kaplan-meier analysis, and 95% CI were calculated using log-log transformation. |
| Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Investigator's Rating on FWS | At Day 180 | Investigator's FWS assessed status of GFL according to the 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). 95% CIs for percentage of participants were based on Pearson-clopper method. |
| Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Participant's Rating on FWS | At Day 180 | Participant's FWS assessed status of GFL according to the 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for participant's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). 95% CI for percentage of participants were based on Pearson-clopper method. |
| Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Investigator's Rating on FWS | At Day 180 | Investigator's FWS assessed status of GFL according to the 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). 95% CI for percentage of participants were based on Pearson-clopper method. |
| Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Participant's Rating on FWS | At Day 180 | Participant's FWS assessed status of GFL according to the 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for participant's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). 95% CI for percentage of participants were based on Pearson-clopper method. |
Countries
Germany, United States
Participant flow
Recruitment details
Study was conducted at 9 sites in Germany and the United States from 23 January 2019 to 8 October 2020. Out of 256 participants, 15 participants were screen failures and 241 participants were enrolled and randomized in the study. Study had 2 periods: Main Period (MP) and an optional Open-label Extension (OLEX) Period. As planned, combined safety data for MP and OLEX Period was reported.
Pre-assignment details
In MP, Stage 1, participants received NT 201: 20 unit (U), 50 U or 75 U. Based on safety data of Stage 1, Stage 2 was started, and participants received NT 201 20 U or 100 U. After completion of MP, participants had the opportunity to receive an optional follow-up treatment with NT 201 20 U in OLEX period. As planned, 20 U group data from Stages 1 and 2 were pooled.
Participants by arm
| Arm | Count |
|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U Participants from Stages 1 and 2 received NT 201 20 U, powder for solution for injection, intramuscularly with the total amount of 0.25 mL injected into the glabellar area in equal aliquots administered to 5 injection sites (0.05 mL per injection site) on Day 1 of MP. This arm consisted of pooled data of all participants who received NT 201 20 U in Stages 1 and 2 of MP. Participants had the opportunity to receive an optional follow-up treatment with the dose of NT 201 20 U with the total amount of 0.5 mL injected into the glabellar area in equal aliquots administered to 5 injection sites (0.1 mL per injection site) in the OLEX period. | 61 |
| NT 201 50 U Participants from Stage 1 received NT 201 50 U, powder for solution for injection, intramuscularly with the total amount of 0.25 mL injected into the glabellar area in equal aliquots administered to 5 injection sites (0.05 mL per injection site) on Day 1 of MP. Participants had the opportunity to receive an optional follow-up treatment with the dose of NT 201 20 U with the total amount of 0.5 mL injected into the glabellar area in equal aliquots administered to 5 injection sites (0.1 mL per injection site) in the OLEX period. | 60 |
| NT 201 75 U Participants from Stage 1 received NT 201 75 U, powder for solution for injection, intramuscularly with the total amount of 0.25 mL injected into the glabellar area in equal aliquots administered to 5 injection sites (0.05 mL per injection site) on Day 1 of MP. Participants had the opportunity to receive an optional follow-up treatment with the dose of NT 201 20 U with the total amount of 0.5 mL injected into the glabellar area in equal aliquots administered to 5 injection sites (0.1 mL per injection site) in the OLEX period. | 61 |
| NT 201 100 U Participants from Stage 2 received NT 201 100 U, powder for solution for injection, intramuscularly with the total amount of 0.25 mL injected into the glabellar area in equal aliquots administered to 5 injection sites (0.05 mL per injection site) on Day 1 of MP. Participants had the opportunity to receive an optional follow-up treatment with the dose of NT 201 20 U with the total amount of 0.5 mL injected into the glabellar area in equal aliquots administered to 5 injection sites (0.1 mL per injection site) in the OLEX period. | 59 |
| Total | 241 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Main Period | Lost to Follow-up | 2 | 2 | 0 | 0 |
| Main Period | Physician Decision | 0 | 0 | 0 | 1 |
| Main Period | Pregnancy | 0 | 0 | 1 | 0 |
| Main Period | Withdrawal by Subject | 2 | 1 | 1 | 2 |
| OLEX Period | Due to coronavirus disease (COVID) 19 pandemic | 0 | 0 | 0 | 1 |
Baseline characteristics
| Characteristic | Stage 1 and 2 Pooled: NT 201 20 U | NT 201 50 U | NT 201 75 U | NT 201 100 U | Total |
|---|---|---|---|---|---|
| Age, Continuous | 52.0 years STANDARD_DEVIATION 11.43 | 46.9 years STANDARD_DEVIATION 10.27 | 49.2 years STANDARD_DEVIATION 13.75 | 49.4 years STANDARD_DEVIATION 11.19 | 49.4 years STANDARD_DEVIATION 11.81 |
| Facial Wrinkle Scale (FWS) severity at maximum frown as assessed by the investigator Moderate | 8 Participants | 9 Participants | 9 Participants | 8 Participants | 34 Participants |
| Facial Wrinkle Scale (FWS) severity at maximum frown as assessed by the investigator Severe | 53 Participants | 51 Participants | 52 Participants | 51 Participants | 207 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 3 Participants | 9 Participants | 3 Participants | 13 Participants | 28 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 58 Participants | 51 Participants | 58 Participants | 46 Participants | 213 Participants |
| Race/Ethnicity, Customized Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Female | 53 Participants | 51 Participants | 54 Participants | 50 Participants | 208 Participants |
| Sex: Female, Male Male | 8 Participants | 9 Participants | 7 Participants | 9 Participants | 33 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 62 | 0 / 60 | 0 / 61 | 0 / 58 |
| other Total, other adverse events | 13 / 62 | 11 / 60 | 12 / 61 | 12 / 58 |
| serious Total, serious adverse events | 0 / 62 | 0 / 60 | 0 / 61 | 1 / 58 |
Outcome results
Primary
Duration of Effect Defined as Time Between Treatment and Relapse to Baseline Status Assessed by the Investigator on the FWS
Duration of effect: time between treatment and first occurrence of relapse to baseline status. If no effect was observed, duration of effect was set to 0. Effect: any improvement (at least 1 point) at maximum frown as assessed by investigator on FWS. Investigator's FWS assessed status of GFL according to 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). Duration of effect was analyzed using Kaplan-meier analysis, and 95 percent (%) confidence interval (CI) were calculated using log-log transformation.
Time frame: From the time of first treatment up to Day 360
Population: The FAS was subset of participants exposed to study medication for whom any efficacy variable was available.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Duration of Effect Defined as Time Between Treatment and Relapse to Baseline Status Assessed by the Investigator on the FWS | 175 days |
| NT 201 50 U | Duration of Effect Defined as Time Between Treatment and Relapse to Baseline Status Assessed by the Investigator on the FWS | 185 days |
| NT 201 75 U | Duration of Effect Defined as Time Between Treatment and Relapse to Baseline Status Assessed by the Investigator on the FWS | 210 days |
| NT 201 100 U | Duration of Effect Defined as Time Between Treatment and Relapse to Baseline Status Assessed by the Investigator on the FWS | 215 days |
p-value: =0.88195% CI: [0.7, 1.51]Wald Chi-square test
p-value: =0.08995% CI: [0.49, 1.05]Wald Chi-square test
p-value: 0.003595% CI: [0.38, 0.83]Wald Chi-square test
Primary
Number of Participants With at Least One Treatment-emergent Adverse Event of Special Interest (TEAESI)
Time frame: From the time of first treatment up to Day 390
Population: The SES was the subset of all participants who were exposed to study medication.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Number of Participants With at Least One Treatment-emergent Adverse Event of Special Interest (TEAESI) | 1 Participants |
| NT 201 50 U | Number of Participants With at Least One Treatment-emergent Adverse Event of Special Interest (TEAESI) | 0 Participants |
| NT 201 75 U | Number of Participants With at Least One Treatment-emergent Adverse Event of Special Interest (TEAESI) | 2 Participants |
| NT 201 100 U | Number of Participants With at Least One Treatment-emergent Adverse Event of Special Interest (TEAESI) | 2 Participants |
Primary
Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE)
Time frame: From the time of first treatment up to Day 390
Population: The SES was the subset of all participants who were exposed to study medication.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE) | 25 Participants |
| NT 201 50 U | Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE) | 23 Participants |
| NT 201 75 U | Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE) | 26 Participants |
| NT 201 100 U | Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE) | 22 Participants |
Primary
Number of Participants With at Least One Treatment-emergent Serious Adverse Event (TESAE)
Time frame: From the time of first treatment up to Day 390
Population: The SES was the subset of all participants who were exposed to study medication.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Number of Participants With at Least One Treatment-emergent Serious Adverse Event (TESAE) | 0 Participants |
| NT 201 50 U | Number of Participants With at Least One Treatment-emergent Serious Adverse Event (TESAE) | 0 Participants |
| NT 201 75 U | Number of Participants With at Least One Treatment-emergent Serious Adverse Event (TESAE) | 0 Participants |
| NT 201 100 U | Number of Participants With at Least One Treatment-emergent Serious Adverse Event (TESAE) | 1 Participants |
Primary
Number of Participants With at Least One Treatment Related TEAE
Time frame: From the time of first treatment up to Day 390
Population: The SES was the subset of all participants who were exposed to study medication.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Number of Participants With at Least One Treatment Related TEAE | 7 Participants |
| NT 201 50 U | Number of Participants With at Least One Treatment Related TEAE | 6 Participants |
| NT 201 75 U | Number of Participants With at Least One Treatment Related TEAE | 8 Participants |
| NT 201 100 U | Number of Participants With at Least One Treatment Related TEAE | 7 Participants |
Primary
Number of Participants With at Least One Treatment Related TESAE
Time frame: From the time of first treatment up to Day 390
Population: The SES was the subset of all participants who were exposed to study medication.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Number of Participants With at Least One Treatment Related TESAE | 0 Participants |
| NT 201 50 U | Number of Participants With at Least One Treatment Related TESAE | 0 Participants |
| NT 201 75 U | Number of Participants With at Least One Treatment Related TESAE | 0 Participants |
| NT 201 100 U | Number of Participants With at Least One Treatment Related TESAE | 0 Participants |
Secondary
Duration of Effect Whereby Effect Was Defined as Score of None (0) or Mild (1) at Maximum Frown as Assessed by the Investigator According to FWS
Duration of effect was defined as time between treatment and first point in time when score was moderate or severe again. If no effect was observed, duration of effect was set to 0. Effect was defined by a score of none (0) or mild (1) at maximum frown as assessed by investigator according to FWS. Investigator's FWS assessed status of GFL according to 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). Duration of effect was analyzed using Kaplan-meier analysis, and 95% CI were calculated using log-log transformation.
Time frame: From the time of first treatment up to Day 360
Population: The FAS was subset of participants exposed to study medication for whom any efficacy variable was available.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Duration of Effect Whereby Effect Was Defined as Score of None (0) or Mild (1) at Maximum Frown as Assessed by the Investigator According to FWS | 113 days |
| NT 201 50 U | Duration of Effect Whereby Effect Was Defined as Score of None (0) or Mild (1) at Maximum Frown as Assessed by the Investigator According to FWS | 121 days |
| NT 201 75 U | Duration of Effect Whereby Effect Was Defined as Score of None (0) or Mild (1) at Maximum Frown as Assessed by the Investigator According to FWS | 129 days |
| NT 201 100 U | Duration of Effect Whereby Effect Was Defined as Score of None (0) or Mild (1) at Maximum Frown as Assessed by the Investigator According to FWS | 148 days |
Secondary
Duration of Effect Whereby Effect Was Defined by 2-point Improvement From Baseline at Maximum Frown as Assessed by the Investigator According to FWS
Duration of effect: time between treatment and first point in time when improvement was less than 2 points again. If no effect was observed, duration of effect was set to 0. Effect: at least a 2-point improvement from baseline at maximum frown as assessed by investigator on the FWS. Investigator's FWS assessed status of GFL according to 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). Duration of effect was analyzed using Kaplan-meier analysis, and 95% CI were calculated using log-log transformation.
Time frame: From the time of first treatment up to Day 360
Population: The FAS was subset of participants exposed to study medication for whom any efficacy variable was available.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Duration of Effect Whereby Effect Was Defined by 2-point Improvement From Baseline at Maximum Frown as Assessed by the Investigator According to FWS | 96 days |
| NT 201 50 U | Duration of Effect Whereby Effect Was Defined by 2-point Improvement From Baseline at Maximum Frown as Assessed by the Investigator According to FWS | 118 days |
| NT 201 75 U | Duration of Effect Whereby Effect Was Defined by 2-point Improvement From Baseline at Maximum Frown as Assessed by the Investigator According to FWS | 122 days |
| NT 201 100 U | Duration of Effect Whereby Effect Was Defined by 2-point Improvement From Baseline at Maximum Frown as Assessed by the Investigator According to FWS | 145 days |
Secondary
Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Investigator's Rating on FWS
Investigator's FWS assessed status of GFL according to the 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). 95% CI for percentage of participants were based on Pearson-clopper method.
Time frame: At Day 180
Population: The FAS was subset of participants exposed to study medication for whom any efficacy variable was available.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Investigator's Rating on FWS | 32.8 percentage of participants |
| NT 201 50 U | Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Investigator's Rating on FWS | 43.3 percentage of participants |
| NT 201 75 U | Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Investigator's Rating on FWS | 52.5 percentage of participants |
| NT 201 100 U | Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Investigator's Rating on FWS | 52.5 percentage of participants |
Secondary
Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Participant's Rating on FWS
Participant's FWS assessed status of GFL according to the 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for participant's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). 95% CI for percentage of participants were based on Pearson-clopper method.
Time frame: At Day 180
Population: The FAS was subset of participants exposed to study medication for whom any efficacy variable was available.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Participant's Rating on FWS | 37.7 percentage of participants |
| NT 201 50 U | Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Participant's Rating on FWS | 36.7 percentage of participants |
| NT 201 75 U | Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Participant's Rating on FWS | 52.5 percentage of participants |
| NT 201 100 U | Percentage of Participants Rated as at Least 1-point Improvement Compared to Baseline at Maximum Frown by Participant's Rating on FWS | 45.8 percentage of participants |
Secondary
Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Investigator's Rating on FWS
Investigator's FWS assessed status of GFL according to the 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for investigator's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). 95% CIs for percentage of participants were based on Pearson-clopper method.
Time frame: At Day 180
Population: The FAS was subset of participants exposed to study medication for whom any efficacy variable was available.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Investigator's Rating on FWS | 8.2 percentage of participants |
| NT 201 50 U | Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Investigator's Rating on FWS | 8.3 percentage of participants |
| NT 201 75 U | Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Investigator's Rating on FWS | 16.4 percentage of participants |
| NT 201 100 U | Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Investigator's Rating on FWS | 18.6 percentage of participants |
Secondary
Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Participant's Rating on FWS
Participant's FWS assessed status of GFL according to the 4-point severity grades at maximum frown as: 0 (none), 1 (mild), 2 (moderate), 3 (severe). Descriptors of each severity grade for participant's FWS assessment at maximum frown were as: 0 (no muscle action at all), 1 (some even slight muscle action possible \[that is, visible furrows\]), 2 (moderately strong muscle action possible \[that is, visible muscle bulges\]), 3 (strong muscle action possible which may cause local pallor). 95% CI for percentage of participants were based on Pearson-clopper method.
Time frame: At Day 180
Population: The FAS was subset of participants exposed to study medication for whom any efficacy variable was available.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Stage 1 and 2 Pooled: NT 201 20 U | Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Participant's Rating on FWS | 6.6 percentage of participants |
| NT 201 50 U | Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Participant's Rating on FWS | 10.0 percentage of participants |
| NT 201 75 U | Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Participant's Rating on FWS | 19.7 percentage of participants |
| NT 201 100 U | Percentage of Participants Rated as None (0) or Mild (1) at Maximum Frown by Participant's Rating on FWS | 16.9 percentage of participants |