Trial Outcomes & Findings for A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (NCT NCT05267600)
NCT ID: NCT05267600
Last Updated: 2025-10-23
Results Overview
CRoff = complete remission while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
98 participants
Primary outcome timeframe
at week 36
Results posted on
2025-10-23
Participant Flow
Parts A and B were identical in schedule, structure, eligibility requirements, assessments, and conduct therefore the results are shown with the pooled data from part A and part B.
Participant milestones
| Measure |
Efgartigimod PH20 SC
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Overall Study
STARTED
|
50
|
48
|
|
Overall Study
COMPLETED
|
35
|
35
|
|
Overall Study
NOT COMPLETED
|
15
|
13
|
Reasons for withdrawal
| Measure |
Efgartigimod PH20 SC
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
4
|
|
Overall Study
Death
|
1
|
2
|
|
Overall Study
Lack of Efficacy
|
1
|
2
|
|
Overall Study
Lost to Follow-up
|
3
|
0
|
|
Overall Study
Physician Decision
|
4
|
0
|
|
Overall Study
Treatment Unblinded
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
5
|
4
|
Baseline Characteristics
A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
Baseline characteristics by cohort
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Total
n=98 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
71.0 years
STANDARD_DEVIATION 12.02 • n=5 Participants
|
70.9 years
STANDARD_DEVIATION 9.37 • n=7 Participants
|
70.9 years
STANDARD_DEVIATION 10.75 • n=5 Participants
|
|
Age, Customized
18 - <65 years
|
12 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Age, Customized
65 - <75 years
|
18 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
35 Participants
n=5 Participants
|
|
Age, Customized
75 - <85 years
|
15 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
32 Participants
n=5 Participants
|
|
Age, Customized
>=85 years
|
5 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
37 Participants
n=5 Participants
|
35 Participants
n=7 Participants
|
72 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
AMERICAN INDIAN OR ALASKA NATIVE
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
ASIAN
|
8 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
MULTIPLE
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
WHITE
|
40 Participants
n=5 Participants
|
39 Participants
n=7 Participants
|
79 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
HISPANIC OR LATINO
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
NOT HISPANIC OR LATINO
|
49 Participants
n=5 Participants
|
48 Participants
n=7 Participants
|
97 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: at week 36CRoff = complete remission while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Number of Participants With CRoff at Week 36
|
8 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: up to week 36OCS = oral corticosteroid
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Cumulative OCS Dose
|
0.242 mg/kg/day
Standard Deviation 0.138
|
0.289 mg/kg/day
Standard Deviation 0.173
|
SECONDARY outcome
Timeframe: at week 36IGA-BP = Investigator Global Assessment of Bullous Pemphigoid. IGA-BP scores range from 0-4, with a higher score representing severe BP. OCS = oral corticosteroid
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Number of Participants Who Achieve an IGA-BP Score of 0 While Off OCS Therapy for ≥8 Weeks at Week 36
|
9 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: up to week 36Control of disease activity (CDA) is defined as the point at which new lesions ceased to form, established lesions began to heal, and pruritic symptoms started to abate. Relapse is defined as the appearance of 3 or more new lesions a month or at least 1 large lesion that did not heal within 1 week, or extension of established lesions or daily pruritus in a participant who had achieved CDA.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Number of Participants With CDA Who Remained Free of Relapse Through Week 36
|
7 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: at week 36CRmin = complete remission receiving minimal oral corticosteroids for at least 8 weeks. Minimal oral corticosteroid (OCS) therapy is defined as ≤0.1 mg/kg/day of prednisone (or equivalent).
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Number of Participants With CRmin at Week 36
|
8 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: up to week 36Population: Only participants with an assessment at baseline and at week 36 are reported.
Itch NRS = Itch Numerical Rating Scale. The Itch NRS scores range from 0-10 with 10 representing the worst imaginable itch.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=19 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=19 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Change From Baseline to Week 36 in the 24-Hour Average Itch NRS Score
|
-4.4 score on a scale
Standard Deviation 3.35
|
-2.4 score on a scale
Standard Deviation 4.26
|
SECONDARY outcome
Timeframe: up to week 36Population: Only participants with an assessment at baseline and week 36 are reported here.
BPDAI = Bullous Pemphigoid Disease Area Index (BPDAI) Total Activity Score is a tool to objectively measure disease activity. BPDAI scores range from 0 to 360 with a higher score indicating more severe disease (max 240 for total skin activity and 120 for mucosal activity).
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=22 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=19 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Changes From Baseline in the BPDAI Total Activity Score
|
-43.13 score on a scale
Standard Deviation 19.633
|
-43.56 score on a scale
Standard Deviation 32.584
|
SECONDARY outcome
Timeframe: up to week 36CDA = control of disease activity, the point at which new lesions ceased to form, established lesions began to heal, and pruritic symptoms started to abate.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=48 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=47 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Time to CDA
|
16.0 days
Interval 10.0 to 17.0
|
15.0 days
Interval 13.0 to 22.0
|
SECONDARY outcome
Timeframe: up to week 36CR = complete remission, the absence of new lesions, complete healing of existing lesions, and absence of pruritus.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Time to CR
|
134.0 days
Interval 85.0 to
The 95% CI could not be reported because of insufficient number of participants with events
|
99.0 days
Interval 57.0 to 225.0
|
SECONDARY outcome
Timeframe: up to week 36CRmin = complete remission, the absence of new lesions, complete healing of existing lesions, and absence of pruritus while being on minimal dose of OCS for ≥8 Weeks. OCS = oral corticosteroid. Minimal OCS therapy is defined as an oral prednisone dosage of ≤0.10 mg/kg/day (or equivalent).
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Time to CRmin
|
NA days
Interval 247.0 to
The median and 95% CI could not be reported because of insufficient number of participants with event
|
NA days
The median and 95% CI could not be reported because of insufficient number of participants with event
|
SECONDARY outcome
Timeframe: up to week 36CRoff= complete remission (the absence of new lesions, complete healing of existing lesions, and absence of pruritus) while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. PRoff = partial remission (the presence of only new transient lesions) while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Time to CRoff/PRoff
|
NA days
The median and 95% CI could not be reported because of insufficient number of participants with events
|
NA days
The median and 95% CI could not be reported because of insufficient number of participants with events
|
SECONDARY outcome
Timeframe: up to week 36CRoff is defined as the absence of new lesions, complete healing of existing lesions, and absence of pruritus while being off OCS therapy for ≥8 weeks.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Time to CRoff
|
NA days
The median and 95% CI could not be reported because of insufficient number of participants with events
|
NA days
The median and 95% CI could not be reported because of insufficient number of participants with events
|
SECONDARY outcome
Timeframe: up to week 36Population: Only participants achieving CDA are included in this section.
CDA is defined as the point at which new lesions ceased to form, established lesions began to heal, and pruritic symptoms started to abate. Relapse is defined as the appearance of 3 or more new lesions a month or at least 1 large lesion that did not heal within 1 week, or extension of established lesions or daily pruritus in a participant who had achieved CDA.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=48 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=42 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Time From CDA to Achieve Relapse
|
114.0 days
Interval 78.0 to 160.0
|
105.0 days
Interval 71.0 to 163.0
|
SECONDARY outcome
Timeframe: at week 36Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Number of Participants Who Receive Rescue Therapy Before Week 36
|
19 Participants
|
18 Participants
|
SECONDARY outcome
Timeframe: up to week 36Population: Only participants with a GTI assessment at baseline and at week 36 are reported here.
The Aggregate Improvement Score (AIS) from the Glucocorticoid Toxicity Index (GTI) can range from -346 to 439 with a higher score representing greater corticosteroid toxicity. If a study drug is effective at lowering greater corticosteroid toxicity over time, the score will be lower.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=21 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=19 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
The AIS From the GTI
|
5.7 score on a scale
Standard Deviation 56.98
|
10.9 score on a scale
Standard Deviation 45.42
|
SECONDARY outcome
Timeframe: up to week 36Population: Only participants with a GTI assessment at baseline and at week 36 are reported here.
The Cumulative Worsening Score (CWS) from the Glucocorticoid Toxicity Index (GTI) can range from 0 to 439 with a higher score representing greater corticosteroid toxicity.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=21 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=19 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
The CWS From the GTI
|
56.1 score on a scale
Standard Deviation 40.71
|
47.5 score on a scale
Standard Deviation 46.68
|
SECONDARY outcome
Timeframe: up to week 36Population: Only participants with an assessment at baseline and at week 36 are reported here.
The EuroQol 5-Dimension 5-Level Visual Analog Scale (EQ-5D-5L VAS) scores range from 0-100 with 0 representing the worst health.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=19 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=19 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
EQ-5D-5L VAS Scores Over Time
|
12.7 score on a scale
Standard Deviation 15.94
|
4.5 score on a scale
Standard Deviation 24.44
|
SECONDARY outcome
Timeframe: up to week 36Population: Only participants with an assessment at baseline and at week 36 are reported here.
Dermatology Life Quality Index (DLQI) assess the participant's perception of the impact of skin diseases on different aspects of their health-related QoL. Scores range from 0 to 30 with a higher score representing a worse quality of life.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=19 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=19 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
DLQI Scores Over Time
|
-7.3 score on a scale
Standard Deviation 6.25
|
-6.7 score on a scale
Standard Deviation 7.14
|
SECONDARY outcome
Timeframe: up to week 36Population: Only participants with an assessment at baseline and at week 36 are analyzed.
Autoimmune Bullous Disease Quality of Life Index (ABQoL) questionnaire assesses the impact of Autoimmune Bullous Disease and their therapies on the daily life of patients. Scores range from 0 to 51 with a higher score representing a worse quality of life.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=19 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=19 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
ABQoL Scores Over Time
|
-12.3 score on a scale
Standard Deviation 6.44
|
-12.0 score on a scale
Standard Deviation 10.92
|
SECONDARY outcome
Timeframe: up to week 36Population: The number analyzed differs from overall number analyzed because the following samples were excluded from analysis: Pre-dose samples that were taken after dosing on that day, samples taken outside the visit window, and when the study drug administration prior to the sample is missed.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Efgartigimod Serum Concentrations
Week 1
|
35.7 ug/mL
Standard Deviation 13.6
|
—
|
|
Efgartigimod Serum Concentrations
Day 10
|
96.4 ug/mL
Standard Deviation 23.9
|
—
|
|
Efgartigimod Serum Concentrations
Week 2
|
37.3 ug/mL
Standard Deviation 12.5
|
—
|
|
Efgartigimod Serum Concentrations
Week 4
|
26.0 ug/mL
Standard Deviation 11.0
|
—
|
|
Efgartigimod Serum Concentrations
Week 8
|
25.8 ug/mL
Standard Deviation 10.5
|
—
|
|
Efgartigimod Serum Concentrations
Week 12
|
24.4 ug/mL
Standard Deviation 8.92
|
—
|
|
Efgartigimod Serum Concentrations
Week 16
|
24.3 ug/mL
Standard Deviation 8.51
|
—
|
|
Efgartigimod Serum Concentrations
Week 20
|
23.9 ug/mL
Standard Deviation 9.12
|
—
|
|
Efgartigimod Serum Concentrations
Week 24
|
24.3 ug/mL
Standard Deviation 9.98
|
—
|
|
Efgartigimod Serum Concentrations
Week 26
|
24.6 ug/mL
Standard Deviation 10.2
|
—
|
|
Efgartigimod Serum Concentrations
Week 28
|
23.3 ug/mL
Standard Deviation 10.3
|
—
|
|
Efgartigimod Serum Concentrations
Week 32
|
26.1 ug/mL
Standard Deviation 11.3
|
—
|
|
Efgartigimod Serum Concentrations
Week 36
|
22.2 ug/mL
Standard Deviation 8.59
|
—
|
SECONDARY outcome
Timeframe: up to 36 weeksPopulation: Only participants with an assessment at baseline and at week 36 are reported here.
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=36 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=34 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Percent Change of Total IgG Serum Levels From Baseline Over Time
|
-46.91 percent change
Standard Deviation 42.244
|
-7.65 percent change
Standard Deviation 25.374
|
SECONDARY outcome
Timeframe: up to 36 weeksPopulation: Only participants with an assessment at baseline and at week 36 are reported here (31+34 with values for anti-BP180 and 11+12 with values for anti-BP230).
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=31 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=34 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time
Percent change of Anti-BP180 antibodies from baseline over time
|
-58.58 Percent change in RU/mL
Standard Deviation 56.983
|
-57.98 Percent change in RU/mL
Standard Deviation 33.470
|
|
Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time
Percent change of Anti-BP230 antibodies from baseline over time
|
-46.72 Percent change in RU/mL
Standard Deviation 35.950
|
-50.95 Percent change in RU/mL
Standard Deviation 28.617
|
SECONDARY outcome
Timeframe: up to 46 weeksOutcome measures
| Measure |
Efgartigimod PH20 SC
n=50 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20
ADA against efgartigimod
|
2 Participants
|
2 Participants
|
|
Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20
Antibodies against rHuPH20
|
9 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: up to week 32Population: The number analyzed for participants and caregivers is the total number who received training and therefore were eligible to be assessed for this outcome (5+4 participants received training and 2+3 caregivers received training).
Outcome measures
| Measure |
Efgartigimod PH20 SC
n=8 Participants
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=18 Participants
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC
Participants
|
5 Participants
|
4 Participants
|
|
Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC
Caregivers
|
2 Participants
|
3 Participants
|
Adverse Events
Efgartigimod PH20 SC
Serious events: 12 serious events
Other events: 47 other events
Deaths: 1 deaths
Placebo PH20 SC
Serious events: 12 serious events
Other events: 46 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Efgartigimod PH20 SC
n=50 participants at risk
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 participants at risk
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Blood and lymphatic system disorders
ANAEMIA
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Cardiac disorders
ACUTE MYOCARDIAL INFARCTION
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Cardiac disorders
ATRIAL FIBRILLATION
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Cardiac disorders
CARDIAC ARREST
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Cardiac disorders
CARDIAC FAILURE
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Cardiac disorders
CARDIAC FAILURE ACUTE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Cardiac disorders
CARDIAC FAILURE CONGESTIVE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Cardiac disorders
COR PULMONALE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Cardiac disorders
CORONARY ARTERY STENOSIS
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Cardiac disorders
SINUS NODE DYSFUNCTION
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Endocrine disorders
CUSHING'S SYNDROME
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Eye disorders
LENS DISLOCATION
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Gastrointestinal disorders
INGUINAL HERNIA
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Gastrointestinal disorders
INTESTINAL MASS
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
General disorders
ASTHENIA
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
General disorders
CONDITION AGGRAVATED
|
4.0%
2/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
General disorders
NON-CARDIAC CHEST PAIN
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Infections and infestations
CELLULITIS GANGRENOUS
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Infections and infestations
PNEUMONIA
|
4.0%
2/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Infections and infestations
PNEUMONIA ASPIRATION
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Infections and infestations
SEPTIC SHOCK
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Injury, poisoning and procedural complications
ANKLE FRACTURE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Injury, poisoning and procedural complications
FALL
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Injury, poisoning and procedural complications
FEMUR FRACTURE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Injury, poisoning and procedural complications
MULTIPLE INJURIES
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Injury, poisoning and procedural complications
SKIN LACERATION
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Injury, poisoning and procedural complications
STRESS FRACTURE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Injury, poisoning and procedural complications
THORACIC VERTEBRAL FRACTURE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Injury, poisoning and procedural complications
VASCULAR GRAFT OCCLUSION
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Metabolism and nutrition disorders
DEHYDRATION
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Metabolism and nutrition disorders
DIABETIC KETOACIDOSIS
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
OVARIAN EPITHELIAL CANCER
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Nervous system disorders
CAROTID ARTERY OCCLUSION
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Nervous system disorders
CEREBROVASCULAR DISORDER
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Nervous system disorders
ENCEPHALOPATHY
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Nervous system disorders
HYPOTONIA
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Nervous system disorders
ISCHAEMIC STROKE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Respiratory, thoracic and mediastinal disorders
ACUTE RESPIRATORY FAILURE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Respiratory, thoracic and mediastinal disorders
CHRONIC OBSTRUCTIVE PULMONARY DISEASE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY EMBOLISM
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Respiratory, thoracic and mediastinal disorders
RESPIRATORY FAILURE
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Skin and subcutaneous tissue disorders
SKIN ULCER
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Vascular disorders
DEEP VEIN THROMBOSIS
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
4.2%
2/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
Other adverse events
| Measure |
Efgartigimod PH20 SC
n=50 participants at risk
All participants from Part A and Part B receiving efgartigimod PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
Placebo PH20 SC
n=48 participants at risk
All participants from Part A and Part B receiving placebo PH20 SC. Participants received concurrent oral prednisone (or equivalent) at a starting dose of 0.5 mg/kg/day. The dose of prednisone was adjusted according to recommendations as per protocol.
|
|---|---|---|
|
Blood and lymphatic system disorders
INCREASED TENDENCY TO BRUISE
|
10.0%
5/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
14.6%
7/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Blood and lymphatic system disorders
LEUKOCYTOSIS
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
8.3%
4/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Blood and lymphatic system disorders
LYMPHOPENIA
|
10.0%
5/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Endocrine disorders
CUSHINGOID
|
8.0%
4/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Gastrointestinal disorders
CONSTIPATION
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Gastrointestinal disorders
DIARRHOEA
|
10.0%
5/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Gastrointestinal disorders
NAUSEA
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Gastrointestinal disorders
VOMITING
|
4.0%
2/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
8.3%
4/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
General disorders
CONDITION AGGRAVATED
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
General disorders
FATIGUE
|
8.0%
4/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
General disorders
INJECTION SITE ERYTHEMA
|
8.0%
4/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
4.2%
2/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
General disorders
INJECTION SITE PARAESTHESIA
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
4.2%
2/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
General disorders
OEDEMA
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
General disorders
OEDEMA PERIPHERAL
|
10.0%
5/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
10.4%
5/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Infections and infestations
COVID-19
|
4.0%
2/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
8.3%
4/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Infections and infestations
NASOPHARYNGITIS
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Infections and infestations
PNEUMONIA
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Infections and infestations
URINARY TRACT INFECTION
|
10.0%
5/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
12.5%
6/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Injury, poisoning and procedural complications
FALL
|
4.0%
2/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
8.3%
4/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Investigations
ALANINE AMINOTRANSFERASE INCREASED
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Investigations
GLYCOSYLATED HAEMOGLOBIN INCREASED
|
8.0%
4/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Metabolism and nutrition disorders
DIABETES MELLITUS
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
4.2%
2/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Metabolism and nutrition disorders
HYPOKALAEMIA
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
8.0%
4/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
10.0%
5/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
10.4%
5/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Musculoskeletal and connective tissue disorders
MUSCLE SPASMS
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Musculoskeletal and connective tissue disorders
MUSCULAR WEAKNESS
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Musculoskeletal and connective tissue disorders
MYOPATHY
|
6.0%
3/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
8.3%
4/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Nervous system disorders
DIZZINESS
|
8.0%
4/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
4.2%
2/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Nervous system disorders
HEADACHE
|
14.0%
7/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
4.2%
2/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Psychiatric disorders
DEPRESSION
|
10.0%
5/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
2.1%
1/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Psychiatric disorders
INSOMNIA
|
14.0%
7/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
16.7%
8/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Psychiatric disorders
MANIA
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Renal and urinary disorders
CHRONIC KIDNEY DISEASE
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Renal and urinary disorders
GLYCOSURIA
|
0.00%
0/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
12.0%
6/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Skin and subcutaneous tissue disorders
DRY SKIN
|
4.0%
2/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Skin and subcutaneous tissue disorders
HIRSUTISM
|
2.0%
1/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
6.2%
3/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Skin and subcutaneous tissue disorders
INTERTRIGO
|
8.0%
4/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
0.00%
0/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Skin and subcutaneous tissue disorders
PRURITUS
|
8.0%
4/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
10.4%
5/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
|
Vascular disorders
HYPERTENSION
|
12.0%
6/50 • up to 46 weeks
Adverse events were assessed during participant visits.
|
4.2%
2/48 • up to 46 weeks
Adverse events were assessed during participant visits.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place