Hidradenitis Suppurativa
Conditions
Keywords
Bimekizumab, UCB4940, HS, Hidradenitis Suppurativa, Acne inversa
Brief summary
The purpose of the study is to evaluate the efficacy and safety of bimekizumab in study participants with moderate to severe hidradenitis suppurativa (HS)
Interventions
DRUGBimekizumab
Subjects will receive bimekizumab at pre-specified time-points.
OTHERPlacebo
Subjects will receive placebo at pre-specified time-points during the Initial Treatment Period.
Sponsors
UCB Biopharma SRL
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participant must be at least 18 years of age, at the time of signing the informed consent. If a study participant is under the local age of consent and is at least 18 years of age, written informed consent will be obtained from both the study participant and the legal representative * Study participants must have a diagnosis of Hidradenitis Suppurativa (HS) based on clinical history and physical examination for at least 6 months prior to the Baseline visit * Study participant must have HS lesions present in at least 2 distinct anatomic areas (eg, left and right axilla), 1 of which must be at least Hurley Stage II or Hurley Stage III at both the Screening and Baseline visits * Study participant must have moderate to severe HS defined as a total of ≥5 inflammatory lesions (ie, number of abscesses plus number of inflammatory nodules) at both the Screening and Baseline visits * Study participant must have had an inadequate response to a course of a systemic antibiotic for treatment of HS as assessed by the Investigator through study participant interview and review of medical history * A female study participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies: 1. Not a woman of childbearing potential (WOCBP) OR 2. A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 20 weeks after the last dose of investigational medicinal product (IMP)
Exclusion criteria
* Draining tunnel count of \>20 at the Baseline Visit * Any other active skin disease or condition (eg, bacterial cellulitis, candida intertrigo, extensive condyloma) that may, in the opinion of the Investigator, interfere with the assessment of hidradenitis suppurativa (HS) * Study participant has a diagnosis of sarcoidosis, systemic lupus erythematosus, or active inflammatory bowel disease (IBD) * Primary immunosuppressive condition, including taking immunosuppressive therapy following an organ transplant, or has had a splenectomy * Female who is breastfeeding, pregnant, or plans to become pregnant during the study or within 20 weeks following the final dose of investigational medicinal product (IMP) * Active infection or history of certain infection(s) * Active tuberculosis (TB) infection, latent TB infection, high risk of exposure to TB infection, current or history of nontuberculous mycobacterium (NTM) infection * Concurrent malignancy. Study participants with a history of malignancy within the past 5 years prior to the Screening Visit are excluded, EXCEPT if the malignancy was a cutaneous squamous or basal cell carcinoma, or in situ cervical cancer that has been treated and is considered cured * History of a lymphoproliferative disorder including lymphoma or current signs and symptoms suggestive of lymphoproliferative disease * Known hypersensitivity to any components of bimekizumab or comparative drugs as stated in this protocol this protocol * Concomitant and prior medication restrictions * Myocardial infarction or stroke within the 6 months prior to the Screening Visit * Study participant has the presence of active suicidal ideation, or positive suicide behavior using the "Screening" version of the electronic Columbia Suicide Severity Rating Scale (eC-SSRS) * Presence of moderately severe major depression or severe major depression * Subject has a history of chronic alcohol or drug abuse within 6 months prior to Screening
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 50 (HiSCR50) at Week 16 | Week 16 | HiSCR50 was defined as at least a 50 percent (%) reduction from Baseline in the total abscess and inflammatory nodule (AN) count, with no increase from Baseline in abscess or draining tunnel count. Intermittent missing data are imputed using multiple imputation with Markov Chain Monte Carlo (MCMC) method followed by monotone regression for monotone missing data. Lesion counts were imputed and then dichotomized to obtain the response status. Participants who experienced an intercurrent event were treated as nonresponders following the intercurrent event. An intercurrent event was defined as receipt of systemic antibiotic rescue medication or discontinuation of study treatment due to an adverse event (AE) or lack of efficacy. Percentages of participants shown do not account for model effects using the logistic regression model. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 75 (HiSCR75) at Week 16 | Week 16 | HiSCR75 was defined as at least a 75% reduction from Baseline in the total AN count, with no increase from Baseline in abscess or draining tunnel count. Intermittent missing data were imputed using multiple imputation with MCMC method followed by monotone regression for monotone missing data. Lesion counts were imputed and then dichotomized to obtain the response status. Participants who experienced an intercurrent event were treated as nonresponders following the intercurrent event. An intercurrent event was defined as receipt of systemic antibiotic rescue medication or discontinuation of study treatment due to an AE or lack of efficacy. Percentages of participants shown do not account for model effects using the logistic regression model. |
| Percentage of Participants With Flare by Week 16 | From Baseline to Week 16 | Flare was defined as a greater than or equal to (\>=) 25% increase in AN count with an absolute increase in AN count of \>= 2 relative to Baseline. Intermittent missing data were imputed using multiple imputation with MCMC method followed by monotone regression for monotone missing data. Lesion counts were imputed and then dichotomized to obtain the response status. Participants who experienced an intercurrent event prior to experiencing a flare were treated as having experienced a flare at all flare assessments on and after the intercurrent event date. An intercurrent event was defined as receipt of systemic antibiotic rescue medication or discontinuation of study treatment due to an AE or lack of efficacy. Percentages of participants shown do not account for model effects using the logistic regression model. |
| Absolute Change From Baseline in Dermatology Life Quality Index (DLQI) Total Score at Week 16 | Baseline, Week 16 | The DLQI is a patient-reported questionnaire designed for use in adult participants with skin diseases and Hidradenitis Suppurativa (HS). The DLQI is a skin disease-specific questionnaire aimed at the evaluation of how symptoms and treatment affect patients' health related quality of life (HRQoL), with a recall period of 7 days. This instrument asks participants 10 questions about symptoms and feelings, daily activities, leisure, work and school, personal relationships, and treatment. The scoring of each answer for the DLQI is on a scale range of 0 (not at all) to 3 (very much). The DLQI total score was calculated by adding the score of each question. The maximum score is 30, and the minimum score is 0. The higher the score, the more quality of life is impaired. Participants who experienced an intercurrent event were treated as missing following the intercurrent event and imputed using the multiple imputation method for missing data. |
| Absolute Change From Baseline in Worst Skin Pain Score at Week 16 | Baseline, Week 16 | Absolute change from Baseline in worst Skin Pain score at Week 16 was assessed using the worst skin pain item in the Hidradenitis Suppurativa Symptom Daily Diary (HSSDD). Worst skin pain during the past 24 hours was assessed daily using an 11-point numeric rating scale (NRS) which ranges from 0 (no skin pain) to 10 (skin pain as bad as you can imagine). The worst skin pain score was derived from the weekly average of daily scores, defined as the sum of the scored item over the course of the study week divided by the number of days in which the item was completed, relative to each respective visit date. Intermittent missing data are imputed using multiple imputation with MCMC method followed by monotone regression for monotone missing data. Participants who experienced an intercurrent event were treated as missing following the intercurrent event and imputed using the multiple imputation method for missing data. Mean values shown do not account for model effects using the ANCOVA model. |
| Percentage of Participants Achieving Skin Pain Response at Week 16 | Week 16 | Skin pain response at Week 16, as assessed by "worst skin pain" item in HSSDD, was defined as an improvement in weekly worst skin pain score of at least 3 points versus Baseline. Worst skin pain during the past 24 hours was assessed daily using an 11-point numeric rating scale (NRS) which ranges from 0 (no skin pain) to 10 (skin pain as bad as you can imagine). Worst skin pain score was derived from weekly average of daily scores (sum of scored item over study week/number of days in which item completed, relative to each respective visit). Intermittent missing data were imputed using multiple imputation with MCMC method followed by monotone regression for monotone missing data. Weekly pain scores were imputed and then dichotomized to obtain response status. Participants who experienced an intercurrent event were treated as non-responders following the intercurrent event. Percentages of participants shown do not account for model effects using logistic regression model. |
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | From Baseline (Day 1) until Safety Follow-Up (up to Week 71) | An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. Treatment-emergent AEs are defined as those AEs that have a start date on or following the first dose of study treatment through the final dose of study treatment + 140 days (covering the 20-week Safety Follow-Up \[SFU\] period). |
| Percentage of Participants With Serious Treatment-emergent Adverse Events During the Study | From Baseline (Day 1) until Safety Follow-Up (up to Week 71) | A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: Results in death; Is life-threatening, Requires inpatient hospitalization or prolongation of existing hospitalization; Results in persistent disability/incapacity; Is a congenital anomaly/birth defect; Important medical events. Treatment-emergent AEs are defined as those AEs that have a start date on or following the first dose of study treatment through the final dose of study treatment + 140 days (covering the 20-week SFU period). |
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From the Study | From Baseline (Day 1) until Safety Follow-Up (up to Week 71) | An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of IMP, whether or not considered related to the IMP. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. Treatment-emergent AEs are defined as those AEs that have a start date on or following the first dose of study treatment through the final dose of study treatment + 140 days (covering the 20-week SFU period). TEAEs leading to discontinuation of the study are reported. |
Countries
Australia, Bulgaria, Canada, Czechia, France, Germany, Hungary, Ireland, Israel, Japan, Poland, Spain, United Kingdom, United States
Contacts
STUDY_DIRECTORUCB Cares
001 844 599 2273
Participant flow
Recruitment details
The study started to enroll participants in March 2020 and concluded in September 2022.
Pre-assignment details
Participant Flow refers to the Randomized Set (RS) and Maintenance Set (MS).
Participants by arm
| Arm | Count |
|---|---|
| Placebo Participants received placebo during the 16-weeks Initial Treatment Period. | 74 |
| BKZ Dosing Regimen 1 Participants received Bimekizumab (BKZ) dosing regimen 1 subcutaneously (SC) during the 16-weeks Initial Treatment Period. | 144 |
| BKZ Dosing Regimen 2 Participants received BKZ dosing regimen 2 SC during the 16-weeks Initial Treatment Period. | 291 |
| Total | 509 |
Baseline characteristics
| Characteristic | Placebo | BKZ Dosing Regimen 1 | BKZ Dosing Regimen 2 | Total |
|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 7 Participants | 10 Participants | 17 Participants |
| Age, Categorical >=65 years | 4 Participants | 2 Participants | 4 Participants | 10 Participants |
| Age, Categorical Between 18 and 65 years | 70 Participants | 135 Participants | 277 Participants | 482 Participants |
| Age, Continuous | 38.1 Years STANDARD_DEVIATION 13.2 | 35.2 Years STANDARD_DEVIATION 11.9 | 36.9 Years STANDARD_DEVIATION 12.3 | 36.6 Years STANDARD_DEVIATION 12.4 |
| Race/Ethnicity, Customized American Indian or Alaskan Native | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Asian | 5 Participants | 7 Participants | 22 Participants | 34 Participants |
| Race/Ethnicity, Customized Black | 5 Participants | 13 Participants | 22 Participants | 40 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 5 Participants | 10 Participants | 15 Participants | 30 Participants |
| Race/Ethnicity, Customized Missing | 0 Participants | 0 Participants | 2 Participants | 2 Participants |
| Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander | 0 Participants | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 69 Participants | 134 Participants | 274 Participants | 477 Participants |
| Race/Ethnicity, Customized Other/mixed | 0 Participants | 4 Participants | 12 Participants | 16 Participants |
| Race/Ethnicity, Customized White | 64 Participants | 119 Participants | 232 Participants | 415 Participants |
| Sex: Female, Male Female | 31 Participants | 77 Participants | 150 Participants | 258 Participants |
| Sex: Female, Male Male | 43 Participants | 67 Participants | 141 Participants | 251 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk |
|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 74 | 0 / 142 | 0 / 290 | 0 / 69 | 0 / 133 | 0 / 130 | 0 / 131 |
| other Total, other adverse events | 18 / 74 | 40 / 142 | 101 / 290 | 26 / 69 | 49 / 133 | 57 / 130 | 64 / 131 |
| serious Total, serious adverse events | 0 / 74 | 3 / 142 | 9 / 290 | 2 / 69 | 4 / 133 | 3 / 130 | 4 / 131 |
Outcome results
Primary
Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 50 (HiSCR50) at Week 16
HiSCR50 was defined as at least a 50 percent (%) reduction from Baseline in the total abscess and inflammatory nodule (AN) count, with no increase from Baseline in abscess or draining tunnel count. Intermittent missing data are imputed using multiple imputation with Markov Chain Monte Carlo (MCMC) method followed by monotone regression for monotone missing data. Lesion counts were imputed and then dichotomized to obtain the response status. Participants who experienced an intercurrent event were treated as nonresponders following the intercurrent event. An intercurrent event was defined as receipt of systemic antibiotic rescue medication or discontinuation of study treatment due to an adverse event (AE) or lack of efficacy. Percentages of participants shown do not account for model effects using the logistic regression model.
Time frame: Week 16
Population: The RS consisted of all study participants randomized into the study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 50 (HiSCR50) at Week 16 | 32.2 percentage of participants |
| BKZ Dosing Regimen 1 | Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 50 (HiSCR50) at Week 16 | 53.8 percentage of participants |
| BKZ Dosing Regimen 2 | Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 50 (HiSCR50) at Week 16 | 52.0 percentage of participants |
p-value: 0.00497.5% CI: [1.221, 4.804]Regression, Logistic
p-value: 0.00397.5% CI: [1.22, 4.291]Regression, Logistic
Secondary
Absolute Change From Baseline in Dermatology Life Quality Index (DLQI) Total Score at Week 16
The DLQI is a patient-reported questionnaire designed for use in adult participants with skin diseases and Hidradenitis Suppurativa (HS). The DLQI is a skin disease-specific questionnaire aimed at the evaluation of how symptoms and treatment affect patients' health related quality of life (HRQoL), with a recall period of 7 days. This instrument asks participants 10 questions about symptoms and feelings, daily activities, leisure, work and school, personal relationships, and treatment. The scoring of each answer for the DLQI is on a scale range of 0 (not at all) to 3 (very much). The DLQI total score was calculated by adding the score of each question. The maximum score is 30, and the minimum score is 0. The higher the score, the more quality of life is impaired. Participants who experienced an intercurrent event were treated as missing following the intercurrent event and imputed using the multiple imputation method for missing data.
Time frame: Baseline, Week 16
Population: The RS consisted of all study participants randomized into the study. Mean values shown do not account for model effects using the analysis of covariance (ANCOVA) model. Intercurrent event defined as receipt of systemic antibiotic rescue medication or discontinuation of study treatment due to AE or lack of efficacy.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Absolute Change From Baseline in Dermatology Life Quality Index (DLQI) Total Score at Week 16 | -3.2 score on a scale | Standard Error 0.6 |
| BKZ Dosing Regimen 1 | Absolute Change From Baseline in Dermatology Life Quality Index (DLQI) Total Score at Week 16 | -4.7 score on a scale | Standard Error 0.5 |
| BKZ Dosing Regimen 2 | Absolute Change From Baseline in Dermatology Life Quality Index (DLQI) Total Score at Week 16 | -4.6 score on a scale | Standard Error 0.3 |
p-value: <0.00197.5% CI: [-3.92, -0.867]ANCOVA
p-value: <0.00197.5% CI: [-3.705, -0.914]ANCOVA
Secondary
Absolute Change From Baseline in Worst Skin Pain Score at Week 16
Absolute change from Baseline in worst Skin Pain score at Week 16 was assessed using the worst skin pain item in the Hidradenitis Suppurativa Symptom Daily Diary (HSSDD). Worst skin pain during the past 24 hours was assessed daily using an 11-point numeric rating scale (NRS) which ranges from 0 (no skin pain) to 10 (skin pain as bad as you can imagine). The worst skin pain score was derived from the weekly average of daily scores, defined as the sum of the scored item over the course of the study week divided by the number of days in which the item was completed, relative to each respective visit date. Intermittent missing data are imputed using multiple imputation with MCMC method followed by monotone regression for monotone missing data. Participants who experienced an intercurrent event were treated as missing following the intercurrent event and imputed using the multiple imputation method for missing data. Mean values shown do not account for model effects using the ANCOVA model.
Time frame: Baseline, Week 16
Population: The RS consisted of all study participants randomized into the study. Intercurrent event defined as receipt of systemic antibiotic rescue medication or discontinuation of study treatment due to AE or lack of efficacy.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Absolute Change From Baseline in Worst Skin Pain Score at Week 16 | -0.36 score on a scale | Standard Error 0.3 |
| BKZ Dosing Regimen 1 | Absolute Change From Baseline in Worst Skin Pain Score at Week 16 | -1.44 score on a scale | Standard Error 0.24 |
| BKZ Dosing Regimen 2 | Absolute Change From Baseline in Worst Skin Pain Score at Week 16 | -1.83 score on a scale | Standard Error 0.17 |
p-value: 0.0197.5% CI: [-1.684, -0.113]ANCOVA
p-value: <0.00197.5% CI: [-1.978, -0.552]ANCOVA
Secondary
Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 75 (HiSCR75) at Week 16
HiSCR75 was defined as at least a 75% reduction from Baseline in the total AN count, with no increase from Baseline in abscess or draining tunnel count. Intermittent missing data were imputed using multiple imputation with MCMC method followed by monotone regression for monotone missing data. Lesion counts were imputed and then dichotomized to obtain the response status. Participants who experienced an intercurrent event were treated as nonresponders following the intercurrent event. An intercurrent event was defined as receipt of systemic antibiotic rescue medication or discontinuation of study treatment due to an AE or lack of efficacy. Percentages of participants shown do not account for model effects using the logistic regression model.
Time frame: Week 16
Population: The RS consisted of all study participants randomized into the study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 75 (HiSCR75) at Week 16 | 15.6 percentage of participants |
| BKZ Dosing Regimen 1 | Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 75 (HiSCR75) at Week 16 | 33.7 percentage of participants |
| BKZ Dosing Regimen 2 | Percentage of Participants Achieving Clinical Response as Measured by Hidradenitis Suppurativa Clinical Response 75 (HiSCR75) at Week 16 | 35.7 percentage of participants |
p-value: 0.00797.5% CI: [1.182, 6.267]Regression, Logistic
p-value: 0.00297.5% CI: [1.374, 6.581]Regression, Logistic
Secondary
Percentage of Participants Achieving Skin Pain Response at Week 16
Skin pain response at Week 16, as assessed by worst skin pain item in HSSDD, was defined as an improvement in weekly worst skin pain score of at least 3 points versus Baseline. Worst skin pain during the past 24 hours was assessed daily using an 11-point numeric rating scale (NRS) which ranges from 0 (no skin pain) to 10 (skin pain as bad as you can imagine). Worst skin pain score was derived from weekly average of daily scores (sum of scored item over study week/number of days in which item completed, relative to each respective visit). Intermittent missing data were imputed using multiple imputation with MCMC method followed by monotone regression for monotone missing data. Weekly pain scores were imputed and then dichotomized to obtain response status. Participants who experienced an intercurrent event were treated as non-responders following the intercurrent event. Percentages of participants shown do not account for model effects using logistic regression model.
Time frame: Week 16
Population: The RS consisted of all study participants randomized into the study. Here, number of participants analyzed included RS with HSSDD worst skin pain score \>=3 at Baseline. Intercurrent event defined as receipt of systemic antibiotic rescue medication or discontinuation of study treatment due to AE or lack of efficacy.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants Achieving Skin Pain Response at Week 16 | 10.9 percentage of participants |
| BKZ Dosing Regimen 1 | Percentage of Participants Achieving Skin Pain Response at Week 16 | 28.6 percentage of participants |
| BKZ Dosing Regimen 2 | Percentage of Participants Achieving Skin Pain Response at Week 16 | 31.8 percentage of participants |
p-value: 0.02897.5% CI: [0.974, 10.997]Regression, Logistic
p-value: 0.0197.5% CI: [1.189, 11.867]Regression, Logistic
Secondary
Percentage of Participants With Flare by Week 16
Flare was defined as a greater than or equal to (\>=) 25% increase in AN count with an absolute increase in AN count of \>= 2 relative to Baseline. Intermittent missing data were imputed using multiple imputation with MCMC method followed by monotone regression for monotone missing data. Lesion counts were imputed and then dichotomized to obtain the response status. Participants who experienced an intercurrent event prior to experiencing a flare were treated as having experienced a flare at all flare assessments on and after the intercurrent event date. An intercurrent event was defined as receipt of systemic antibiotic rescue medication or discontinuation of study treatment due to an AE or lack of efficacy. Percentages of participants shown do not account for model effects using the logistic regression model.
Time frame: From Baseline to Week 16
Population: The RS consisted of all study participants randomized into the study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants With Flare by Week 16 | 28.0 percentage of participants |
| BKZ Dosing Regimen 1 | Percentage of Participants With Flare by Week 16 | 23.6 percentage of participants |
| BKZ Dosing Regimen 2 | Percentage of Participants With Flare by Week 16 | 28.8 percentage of participants |
p-value: 0.49797.5% CI: [0.378, 1.683]Regression, Logistic
p-value: 0.86897.5% CI: [0.541, 2.041]Regression, Logistic
Secondary
Percentage of Participants With Serious Treatment-emergent Adverse Events During the Study
A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: Results in death; Is life-threatening, Requires inpatient hospitalization or prolongation of existing hospitalization; Results in persistent disability/incapacity; Is a congenital anomaly/birth defect; Important medical events. Treatment-emergent AEs are defined as those AEs that have a start date on or following the first dose of study treatment through the final dose of study treatment + 140 days (covering the 20-week SFU period).
Time frame: From Baseline (Day 1) until Safety Follow-Up (up to Week 71)
Population: The SS consisted of all study participants who received at least 1 dose (full or partial) of IMP. The MS consisted of all study participants who received at least 1 dose (full or partial) of BKZ in the Maintenance Treatment Period.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants With Serious Treatment-emergent Adverse Events During the Study | 0 percentage of participants |
| BKZ Dosing Regimen 1 | Percentage of Participants With Serious Treatment-emergent Adverse Events During the Study | 2.1 percentage of participants |
| BKZ Dosing Regimen 2 | Percentage of Participants With Serious Treatment-emergent Adverse Events During the Study | 3.1 percentage of participants |
| Placebo/BKZ Dosing Regimen 2 | Percentage of Participants With Serious Treatment-emergent Adverse Events During the Study | 2.9 percentage of participants |
| BKZ Dosing Regimen 1/BKZ Dosing Regimen 1 | Percentage of Participants With Serious Treatment-emergent Adverse Events During the Study | 3.0 percentage of participants |
| BKZ Dosing Regimen 2/BKZ Dosing Regimen 1 | Percentage of Participants With Serious Treatment-emergent Adverse Events During the Study | 2.3 percentage of participants |
| BKZ Dosing Regimen 2/BKZ Dosing Regimen 2 | Percentage of Participants With Serious Treatment-emergent Adverse Events During the Study | 3.1 percentage of participants |
Secondary
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. Treatment-emergent AEs are defined as those AEs that have a start date on or following the first dose of study treatment through the final dose of study treatment + 140 days (covering the 20-week Safety Follow-Up \[SFU\] period).
Time frame: From Baseline (Day 1) until Safety Follow-Up (up to Week 71)
Population: The Safety Set (SS) consisted of all study participants who received at least 1 dose (full or partial) of IMP. The MS consisted of all study participants who received at least 1 dose (full or partial) of BKZ in the Maintenance Treatment Period.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 56.8 percentage of participants |
| BKZ Dosing Regimen 1 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 51.4 percentage of participants |
| BKZ Dosing Regimen 2 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 64.5 percentage of participants |
| Placebo/BKZ Dosing Regimen 2 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 71.0 percentage of participants |
| BKZ Dosing Regimen 1/BKZ Dosing Regimen 1 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 72.2 percentage of participants |
| BKZ Dosing Regimen 2/BKZ Dosing Regimen 1 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 77.7 percentage of participants |
| BKZ Dosing Regimen 2/BKZ Dosing Regimen 2 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 77.1 percentage of participants |
Secondary
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From the Study
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of IMP, whether or not considered related to the IMP. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. Treatment-emergent AEs are defined as those AEs that have a start date on or following the first dose of study treatment through the final dose of study treatment + 140 days (covering the 20-week SFU period). TEAEs leading to discontinuation of the study are reported.
Time frame: From Baseline (Day 1) until Safety Follow-Up (up to Week 71)
Population: The SS consisted of all study participants who received at least 1 dose (full or partial) of IMP. The MS consisted of all study participants who received at least 1 dose (full or partial) of BKZ in the Maintenance Treatment Period.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From the Study | 0 percentage of participants |
| BKZ Dosing Regimen 1 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From the Study | 2.1 percentage of participants |
| BKZ Dosing Regimen 2 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From the Study | 4.1 percentage of participants |
| Placebo/BKZ Dosing Regimen 2 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From the Study | 0 percentage of participants |
| BKZ Dosing Regimen 1/BKZ Dosing Regimen 1 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From the Study | 4.5 percentage of participants |
| BKZ Dosing Regimen 2/BKZ Dosing Regimen 1 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From the Study | 3.1 percentage of participants |
| BKZ Dosing Regimen 2/BKZ Dosing Regimen 2 | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From the Study | 1.5 percentage of participants |