Trial Outcomes & Findings for A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves) (NCT NCT04281472)
NCT ID: NCT04281472
Last Updated: 2024-08-20
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
322 participants
Primary outcome timeframe
Up to 12 weeks during the open-label stage A
Results posted on
2024-08-20
Participant Flow
ARGX-113-1802 enrolled a broad and global (North America, Asia, Europe, and the rest of the world \[ROW\]) population of treatment-naïve participants and participants who were previously treated for CIDP (corticosteroids, IVIg, or SCIg) with confirmed, active disease and a wide range of disease severity. A total of 322 participants received efgartigimod PH20 SC in Stage A, and 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
Participant milestones
| Measure |
Stage A: Efgartigimod PH20 SC
Participants receiving efgartigimod PH20 SC in Stage A
|
Stage B: Efgartigimod PH20 SC
Participants who completed stage A and received efgartigimod PH20 SC in stage B
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|---|
|
Stage A
STARTED
|
322
|
0
|
0
|
|
Stage A
COMPLETED
|
221
|
0
|
0
|
|
Stage A
NOT COMPLETED
|
101
|
0
|
0
|
|
Stage B
STARTED
|
0
|
111
|
110
|
|
Stage B
COMPLETED
|
0
|
64
|
74
|
|
Stage B
NOT COMPLETED
|
0
|
47
|
36
|
Reasons for withdrawal
| Measure |
Stage A: Efgartigimod PH20 SC
Participants receiving efgartigimod PH20 SC in Stage A
|
Stage B: Efgartigimod PH20 SC
Participants who completed stage A and received efgartigimod PH20 SC in stage B
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|---|
|
Stage A
Lack of Efficacy
|
8
|
0
|
0
|
|
Stage A
All events required for primary analysis achieved (rolled over to ARGX-1113-1902)
|
22
|
0
|
0
|
|
Stage A
Adverse Event
|
20
|
0
|
0
|
|
Stage A
Death
|
1
|
0
|
0
|
|
Stage A
Lack of efficacy before end of Stage A
|
28
|
0
|
0
|
|
Stage A
Lost to Follow-up
|
2
|
0
|
0
|
|
Stage A
Non-compliance with study drug
|
1
|
0
|
0
|
|
Stage A
Physician Decision
|
1
|
0
|
0
|
|
Stage A
Prohibited medications
|
2
|
0
|
0
|
|
Stage A
Withdrawal by Subject
|
11
|
0
|
0
|
|
Stage A
Other
|
5
|
0
|
0
|
|
Stage B
All events required for primary analysis achieved (rolled over to ARGX-1113-1902)
|
0
|
35
|
26
|
|
Stage B
Adverse Event
|
0
|
3
|
0
|
|
Stage B
Death
|
0
|
0
|
1
|
|
Stage B
Lack of Efficacy
|
0
|
0
|
1
|
|
Stage B
Lost to Follow-up
|
0
|
0
|
2
|
|
Stage B
Prohibited medications
|
0
|
2
|
1
|
|
Stage B
Protocol Violation
|
0
|
1
|
1
|
|
Stage B
Sponsor decision
|
0
|
0
|
1
|
|
Stage B
Withdrawal by Subject
|
0
|
3
|
3
|
|
Stage B
Other
|
0
|
3
|
0
|
Baseline Characteristics
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
Baseline characteristics by cohort
| Measure |
Stage A: Efgartigimod PH20 SC
n=322 Participants
Participants receiving efgartigimod PH20 SC in Stage A
|
Stage B: Efgartigimod PH20 SC
n=111 Participants
Participants who completed stage A and received efgartigimod PH20 SC in stage B
|
Stage B: Placebo PH20 SC
n=110 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
Total
n=543 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
Stage A · <=18 years
|
0 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Age, Categorical
Stage A · Between 18 and 65 years
|
247 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
247 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Age, Categorical
Stage A · >=65 years
|
75 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
75 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Age, Categorical
Stage B · <=18 years
|
0 Participants
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Age, Categorical
Stage B · Between 18 and 65 years
|
0 Participants
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
86 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
88 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
174 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Age, Categorical
Stage B · >=65 years
|
0 Participants
A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
25 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
22 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
47 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Age, Continuous
Stage A
|
54.0 years
STANDARD_DEVIATION 13.92 • n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
54.0 years
STANDARD_DEVIATION 13.92 • n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Age, Continuous
Stage B
|
—
|
54.5 years
STANDARD_DEVIATION 13.18 • n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
51.3 years
STANDARD_DEVIATION 14.47 • n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
52.9 years
STANDARD_DEVIATION 13.90 • n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Sex: Female, Male
Stage A · Female
|
114 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
114 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Sex: Female, Male
Stage A · Male
|
208 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
208 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Sex: Female, Male
Stage B · Female
|
—
|
38 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
41 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
79 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Sex: Female, Male
Stage B · Male
|
—
|
73 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
69 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
142 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Ethnicity (NIH/OMB)
Stage A · Hispanic or Latino
|
23 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
23 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Ethnicity (NIH/OMB)
Stage A · Not Hispanic or Latino
|
288 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
288 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Ethnicity (NIH/OMB)
Stage A · Unknown or Not Reported
|
11 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
11 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Ethnicity (NIH/OMB)
Stage B · Hispanic or Latino
|
—
|
9 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
4 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
13 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Ethnicity (NIH/OMB)
Stage B · Not Hispanic or Latino
|
—
|
99 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
102 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
201 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Ethnicity (NIH/OMB)
Stage B · Unknown or Not Reported
|
—
|
3 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
4 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
7 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage A · American Indian or Alaska Native
|
0 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
0 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage A · Asian
|
89 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
89 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage A · Native Hawaiian or Other Pacific Islander
|
1 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
1 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage A · Black or African American
|
4 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
4 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage A · White
|
211 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
211 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage A · More than one race
|
0 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
0 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage A · Unknown or Not Reported
|
17 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
—
|
—
|
17 Participants
n=322 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage B · American Indian or Alaska Native
|
—
|
0 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage B · Asian
|
—
|
33 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
34 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
67 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage B · Native Hawaiian or Other Pacific Islander
|
—
|
0 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage B · Black or African American
|
—
|
1 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
1 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
2 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage B · White
|
—
|
73 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
71 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
144 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage B · More than one race
|
—
|
0 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
0 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
|
Race (NIH/OMB)
Stage B · Unknown or Not Reported
|
—
|
4 Participants
n=111 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
4 Participants
n=110 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
8 Participants
n=221 Participants • A total of 322 participants were enrolled in Stage A. Of those 322, 221 participants were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.
|
PRIMARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=322 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Percentage of Participants With Confirmed Evidence of Clinical Improvement(ECI)
|
66.5 percentage of participants
Interval 61.0 to 71.6
|
—
|
PRIMARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: mITT (modified intent-to-treat) analysis set; Participants who were randomized in Stage B and who received at least 1 dose or part of a dose of IMP in Stage B.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=110 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline
|
NA Days
Insufficient number of participants with events, less than 50% of the participants had clinically deteriorated.
|
140.0 Days
Interval 75.0 to
Insufficient number of participants with events to calculate the upper limit of the confidence interval.
|
SECONDARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=322 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI)
25th percentile
|
22 days
Interval 22.0 to 23.0
|
—
|
|
Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI)
50th percentile (median)
|
43.0 days
Interval 31.0 to 51.0
|
—
|
|
Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI)
75th percentile
|
71.0 days
Interval 70.0 to 78.0
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A
Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scores range from 0-10 with a score of 10 indicating the greatest degree of disability.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=316 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Adjusted INCAT Score
|
-0.9 score on a scale
Standard Deviation 1.71
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A
The Medical Research Council (MRC) Sum scores range from 0 to 60 with a lower score indicating greater muscle weakness.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=318 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Medical Research Council (MRC) Sum Score
|
3.8 score on a scale
Standard Error 0.41
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A
The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=320 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in I-RODS Disability Scores
|
7.7 score on a scale
Standard Deviation 15.48
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A
The Timed Up and Go (TUG) score is calculated as the number of seconds needed to complete a series of actions. The longer time needed to complete this test (expressed in seconds) indicates lower mobility.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=288 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in TUG Score
|
-4.3 seconds
Standard Error 0.83
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A
This is measured with a handheld device called a vigometer
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=317 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Mean Grip Strength
dominant hand
|
12.3 Kilopascal (kPa)
Standard Deviation 18.68
|
—
|
|
Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Mean Grip Strength
nondominant hand
|
11.2 Kilopascal (kPa)
Standard Deviation 21.12
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A
Treatment-emergent (serious) AEs expressed in number of events/100 PYFU (participant years of follow-up)
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=322 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events
Exposure Adjusted Occurrence of Treatment-emergent Adverse Events
|
1343.1 Events/100 PYFU
|
—
|
|
Stage A: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events
Exposure Adjusted Occurrence of Treatment-emergent Serious Adverse Events
|
51.2 Events/100 PYFU
|
—
|
SECONDARY outcome
Timeframe: Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI))Population: The number analyzed in rows decreases due to participants withdrawing from the study and missing participant data. Week 13 was an optional additional week for the confirmation of the ECI. PK-A (Stage A PK analysis set): Participants from the SAF-A for whom at least 1 serum PK concentration during Stage A is available
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=298 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 1
|
14.9 ug/mL
Standard Deviation 6.92
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 2
|
19.6 ug/mL
Standard Deviation 8.55
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 3
|
19.7 ug/mL
Standard Deviation 9.62
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 4
|
18.9 ug/mL
Standard Deviation 9.96
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 5
|
18.4 ug/mL
Standard Deviation 8.38
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 6
|
19.2 ug/mL
Standard Deviation 9.62
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 7
|
17.3 ug/mL
Standard Deviation 8.89
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 8
|
18.8 ug/mL
Standard Deviation 8.93
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 9
|
17.8 ug/mL
Standard Deviation 8.84
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 10
|
17.3 ug/mL
Standard Deviation 7.62
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 11
|
20.1 ug/mL
Standard Deviation 9.64
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 12
|
20.0 ug/mL
Standard Deviation 6.89
|
—
|
|
Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 13
|
14.9 ug/mL
Standard Deviation 7.24
|
—
|
SECONDARY outcome
Timeframe: Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI))Population: The number analyzed in rows decreases due to participants advancing to stage B or withdrawing from the study and missing patient data. Week 13 was an optional additional week for the confirmation of the evidence of clinical improvement (ECI). PD-A (Stage A PD analysis set): Participants from the SAF-A for whom at least 1 serum PD concentration during Stage A is available.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=321 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 1
|
-36.1 percent change
Standard Error 0.58
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 2
|
-54.6 percent change
Standard Error 0.80
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 3
|
-63.5 percent change
Standard Error 0.71
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 4
|
-66.2 percent change
Standard Error 0.89
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 5
|
-67.9 percent change
Standard Error 0.74
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 6
|
-67.7 percent change
Standard Error 1.42
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 7
|
-66.4 percent change
Standard Error 1.96
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 8
|
-64.5 percent change
Standard Error 4.28
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 9
|
-68.8 percent change
Standard Error 1.17
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 10
|
-67.1 percent change
Standard Error 1.90
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 11
|
-70.0 percent change
Standard Error 1.41
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 12
|
-67.3 percent change
Standard Error 4.44
|
—
|
|
Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
Percent change from Baseline to Week 13
|
-51.0 percent change
Standard Error 10.00
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: The number of participants analyzed is different since it is based on the number of evaluable participants for each drug. For Ab and NAb against rHuPH20 only 316 participants were evaluable, whereas for ADA and NAb towards efgartigimod 317 participants were evaluable. IMM-A (Stage A immunogenicity analysis set): Participants from the SAF-A for whom at least 1 ADA sample during Stage A is available
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=317 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
ADA towards Efgartigimod incidence
|
20 Participants
|
—
|
|
Stage A: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
Ab towards rHuPH20 incidence
|
45 Participants
|
—
|
|
Stage A: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
NAb against Efgartigimod incidence
|
1 Participants
|
—
|
|
Stage A: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
NAb against rHuPH20 incidence
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks during the open-label stage APopulation: SAF-A (Stage A safety analysis set): Participants who received at least 1 dose or part of a dose of efgartigimod PH20 SC in Stage A
Scores range from 0-100 with 100 indicating the best health state. Therefore, positive changes indicate higher health-related quality of life reported by the patient.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=275 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage A: Changes From Stage A Baseline to Last Assessment in Stage A, in EQ-5D-5L Visual Analog Scale (VAS) Over Time
|
10.7 score on a scale
Standard Error 1.34
|
—
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: mITT (modified intent-to-treat) analysis set; Participants who were randomized in Stage B and who received at least 1 dose or part of a dose of IMP in Stage B.
Time to chronic inflammatory demyelinating polyneuropathy (CIDP) disease progression is defined by the time from first dose of double-blind IMP to the first I-RODS score decrease ≥4 points compared to Stage B baseline using the centile metric.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=110 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Time to CIDP Disease Progression
|
NA days
Interval 247.0 to
The median (95% CI) time to CIDP Disease Progression could not be calculated because less than 50% of the participants showed CIDP Disease Progression in the efgartigimod PH20 SC group
|
85 days
Interval 50.0 to 253.0
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: mITT (modified intent-to-treat) analysis set; Participants who were randomized in Stage B and who received at least 1 dose or part of a dose of IMP in Stage B.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=110 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Number of Participants With Improved Functional Level Compared to Stage B Baseline
|
50 Participants
|
40 Participants
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: mITT (modified intent-to-treat) analysis set; Participants who were randomized in Stage B and who received at least 1 dose or part of a dose of IMP in Stage B.
Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scores range from 0-10 with a score of 10 indicating the greatest degree of disability.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=109 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in Adjusted INCAT Score
|
0.1 score on a scale
Standard Deviation 1.08
|
0.9 score on a scale
Standard Deviation 1.98
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: mITT (modified intent-to-treat) analysis set; Participants who were randomized in Stage B and who received at least 1 dose or part of a dose of IMP in Stage B.
The Medical Research Council (MRC) Sum scores range from 0 to 60 with a lower score indicating greater muscle weakness.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=109 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in MRC Sum Score
|
-0.3 score on a scale
Standard Error 0.43
|
-3 score on a scale
Standard Error 0.86
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: mITT (modified intent-to-treat) analysis set; Participants who were randomized in Stage B and who received at least 1 dose or part of a dose of IMP in Stage B.
The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=108 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in 24-item I-RODS Disability Score
|
0.8 score on a scale
Standard Deviation 12.33
|
-7.0 score on a scale
Standard Deviation 19.10
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: mITT (modified intent-to-treat) analysis set; Participants who were randomized in Stage B and who received at least 1 dose or part of a dose of IMP in Stage B.
The Timed Up and Go (TUG) score is calculated as the number of seconds needed to complete a series of actions. The longer time needed to complete this test (expressed in seconds) indicates lower mobility.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=108 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=102 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in TUG Score
|
0.8 seconds
Standard Error 0.36
|
1.9 seconds
Standard Error 0.60
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BThis is measured with a handheld device called a vigometer
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=109 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in Mean Grip Strength
dominant hand
|
2.1 Kilopascal (kPa)
Standard Deviation 13.29
|
-8.2 Kilopascal (kPa)
Standard Deviation 20.69
|
|
Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in Mean Grip Strength
nondominant hand
|
2.0 Kilopascal (kPa)
Standard Deviation 17.33
|
-6.9 Kilopascal (kPa)
Standard Deviation 21.30
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: mITT (modified intent-to-treat) analysis set; Participants who were randomized in Stage B and who received at least 1 dose or part of a dose of IMP in Stage B.
The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=110 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Time to 10% Decrease in the 24-item I-RODS
|
NA days
Interval 260.0 to
The median (95% CI) time to 10% decrease in I-RODS could not be calculated because less than 50% of the participants showed 10% decrease in I-RODS in the efgartigimod PH2O SC group
|
111 days
Interval 84.0 to
Could not be calculated
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: SAF-B (Stage B safety analysis set): Participants from the SCR who received at least 1 dose or part of a dose of IMP in Stage B
Treatment-emergent (serious) AEs expressed in number of events/100 PYFU (participant years of follow-up)
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=110 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events
treatment-emergent adverse events
|
347.6 Events/100 PYFU
|
510.9 Events/100 PYFU
|
|
Stage B: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events
serious adverse events
|
14.1 Events/100 PYFU
|
19.0 Events/100 PYFU
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: The number analyzed in rows decreases due to participants completing the study, withdrawing from the study, and missing participant data. PK-B (Stage B PK analysis set): Participants from the SAF-B for whom at least 1 serum PK concentration during Stage B is available
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=110 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 24
|
16.0 ug/mL
Standard Deviation 7.89
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 28
|
18.5 ug/mL
Standard Deviation 10.2
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 32
|
18.0 ug/mL
Standard Deviation 9.50
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 4
|
18.4 ug/mL
Standard Deviation 10.3
|
0.267 ug/mL
Standard Deviation 0.365
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 8
|
17.2 ug/mL
Standard Deviation 9.11
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 12
|
18.1 ug/mL
Standard Deviation 9.48
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 16
|
16.9 ug/mL
Standard Deviation 9.03
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 20
|
16.8 ug/mL
Standard Deviation 8.36
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 36
|
17.9 ug/mL
Standard Deviation 10.7
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 40
|
16.2 ug/mL
Standard Deviation 8.20
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 44
|
18.1 ug/mL
Standard Deviation 10.2
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
|
Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time
Week 48
|
16.3 ug/mL
Standard Deviation 8.18
|
NA ug/mL
Standard Deviation NA
Data was below the limit of quantification (\< 0.200 ug/mL)
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: The number analyzed in rows decreases due to participants completing the study, withdrawing from the study, and missing participant data. PD-B (Stage B PD analysis set): Participants from the SAF-B for whom at least 1 serum PD concentration during Stage B is available
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=110 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 4
|
-68.2 percent change
Standard Error 1.14
|
-36.7 percent change
Standard Error 1.91
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 8
|
-68.2 percent change
Standard Error 1.24
|
-10.9 percent change
Standard Error 2.41
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 12
|
-69.8 percent change
Standard Error 1.09
|
-7.1 percent change
Standard Error 2.54
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 16
|
-67.4 percent change
Standard Error 1.49
|
-2.1 percent change
Standard Error 3.09
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 20
|
-67.8 percent change
Standard Error 1.30
|
2.0 percent change
Standard Error 3.61
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 24
|
-67.2 percent change
Standard Error 1.52
|
1.7 percent change
Standard Error 4.22
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 28
|
-67.9 percent change
Standard Error 1.42
|
-2.3 percent change
Standard Error 4.31
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 32
|
-68.5 percent change
Standard Error 1.63
|
0.6 percent change
Standard Error 5.18
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 36
|
-64.7 percent change
Standard Error 2.82
|
-3.6 percent change
Standard Error 4.12
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 40
|
-68.0 percent change
Standard Error 1.91
|
-4.2 percent change
Standard Error 4.98
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 44
|
-67.8 percent change
Standard Error 1.92
|
-3.6 percent change
Standard Error 6.50
|
|
Stage B: Percent Changes of Serum IgG Levels Over Time
Percent change from Baseline to Week 48
|
-66.6 percent change
Standard Error 1.85
|
-0.9 percent change
Standard Error 7.36
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: IMM-B (Stage B immunogenicity analysis set): Participants from the SAF-B for whom at least 1 ADA sample during Stage B is available
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=111 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=109 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
Ab towards rHuPH20 incidence
|
52 participants
|
32 participants
|
|
Stage B: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
NAb against efgartigimod incidence
|
0 participants
|
13 participants
|
|
Stage B: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
NAb against rHuPH20 incidence
|
5 participants
|
2 participants
|
|
Stage B: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
ADA towards Efgartigimod incidence
|
2 participants
|
64 participants
|
SECONDARY outcome
Timeframe: Up to 48 weeks during the randomized placebo-controlled stage BPopulation: SAF-B (Stage B safety analysis set): Participants from the SCR who received at least 1 dose or part of a dose of IMP in Stage B
Scores range from 0-100 with 100 indicating the best health state. Therefore, positive changes indicate higher health-related quality of life reported by the patient.
Outcome measures
| Measure |
Stage A: Efgartigimod PH20 SC
n=97 Participants
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Placebo PH20 SC
n=90 Participants
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|
|
Stage B: Changes From Stage B Baseline to Last Assessment in Stage B, in EQ-5D-5L Visual Analog Scale (VAS) Over Time
|
0.5 score on a scale
Standard Error 1.77
|
-10.2 score on a scale
Standard Error 2.47
|
Adverse Events
Stage A: Efgartigimod PH20 SC
Serious events: 21 serious events
Other events: 60 other events
Deaths: 2 deaths
Stage B: Efgartigimod PH20 SC
Serious events: 6 serious events
Other events: 31 other events
Deaths: 0 deaths
Stage B: Placebo PH20 SC
Serious events: 6 serious events
Other events: 15 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Stage A: Efgartigimod PH20 SC
n=322 participants at risk
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Efgartigimod PH20 SC
n=111 participants at risk
Participants who completed stage A and received efgartigimod PH20 SC in stage B
|
Stage B: Placebo PH20 SC
n=110 participants at risk
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|---|
|
Cardiac disorders
CARDIAC ARREST
|
0.31%
1/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Infections and infestations
CLOSTRIDIUM DIFFICILE COLITIS
|
0.31%
1/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Infections and infestations
COVID-19
|
0.31%
1/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Infections and infestations
COVID-19 PNEUMONIA
|
0.31%
1/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.90%
1/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Infections and infestations
SUSPECTED COVID-19
|
0.31%
1/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
SQUAMOUS CELL CARCINOMA OF SKIN
|
0.31%
1/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Nervous system disorders
CHRONIC INFLAMMATORY DEMYELINATING POLYRADICULONEUROPATHY
|
4.3%
14/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.91%
1/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Nervous system disorders
QUADRIPARESIS
|
0.31%
1/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Renal and urinary disorders
CALCULUS URINARY
|
0.31%
1/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Ear and labyrinth disorders
DEAFNESS UNILATERAL
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.91%
1/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Hepatobiliary disorders
CHOLELITHIASIS
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.90%
1/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Infections and infestations
APPENDICITIS
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.91%
1/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Infections and infestations
PNEUMONIA
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
1.8%
2/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Injury, poisoning and procedural complications
CONCUSSION
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.90%
1/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Injury, poisoning and procedural complications
FOOT FRACTURE
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.90%
1/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Metabolism and nutrition disorders
DEHYDRATION
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.91%
1/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
LIPOMA
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.91%
1/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
PROSTATE CANCER
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.90%
1/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
TRANSITIONAL CELL CARCINOMA
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.90%
1/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Renal and urinary disorders
URETHRAL STENOSIS
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.90%
1/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Renal and urinary disorders
URINARY BLADDER POLYP
|
0.00%
0/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.90%
1/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
Other adverse events
| Measure |
Stage A: Efgartigimod PH20 SC
n=322 participants at risk
Participants receiving efgartigimod PH20 SC in stage A
|
Stage B: Efgartigimod PH20 SC
n=111 participants at risk
Participants who completed stage A and received efgartigimod PH20 SC in stage B
|
Stage B: Placebo PH20 SC
n=110 participants at risk
Participants who completed stage A and received placebo PH20 SC in stage B
|
|---|---|---|---|
|
General disorders
INJECTION SITE ERYTHEMA
|
10.2%
33/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
5.4%
6/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.00%
0/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Nervous system disorders
HEADACHE
|
5.0%
16/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
3.6%
4/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
1.8%
2/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
General disorders
INJECTION SITE BRUISING
|
1.2%
4/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
5.4%
6/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
0.91%
1/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Infections and infestations
COVID-19
|
1.9%
6/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
17.1%
19/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
12.7%
14/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
3.4%
11/322 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
1.8%
2/111 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
10.0%
11/110 • 60 weeks (12 weeks during Stage A and 48 weeks during Stage B)
Adverse events will be assessed at each participant visit. Treatment-emergent adverse events are reported in these tables.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place