Safety and target engagement of an oral
small-molecule sequestrant in adolescents
with autism spectrum disorder: an open-
label phase 1b/2a trial
In the format provided by the
authors and unedited
Supplementary information
https://doi.org/10.1038/s41591-022-01683-9
Supplementary Information
(SI):
Deidentified subject demographics and assessment
scores for participants in clinical trial
.
SI Table 1. Demographics (completer group).
SI Table 2. ABC Subscales
SI Table 3. PARS
SI Table 4. VABS
SI Table 5. SRS
Supplementary Information Table 1. Demographics (completers)
Subject ID
Age
Wt (kg)
Wt (lb)
Ht (cm)
Ht (in)
BMI
ADOS Severi ty
001-001
12.1
64.8
143
152
60
28.05
8
001-003
14.6
49.5
109
175.5
70
16.07
9
001-005
15.5
62.4
137
174
69
20.61
7
001-007
12.1
30.1
66
147
58
13.93
8
002-001
13
70.3
155
169
67
24.61
10
002-002
17.3
65.1
143
177.6
70
20.64
Auti sm
002-004
12.9
58.2
128
152.2
60
25.12
6
002-005
13.5
84
185
164.5
65
31.04
10
002-006
13.1
42.5
94
171.7
68
14.42
6
002-010
13.4
57.9
127
169.2
67
20.22
3
002-011
13.3
44.3
97
159
63
17.52
8
002-012
14.2
56.2
124
160
63
21.95
9
002-014
13.8
70.2
154
163
65
26.42
9
002-015
12.8
39.2
86
145.7
58
18.47
9
003-001
13.7
92.25
203
171.1
68
31.51
10
003-002
16.1
56.25
124
172.4
68
18.93
8
003-005
13.5
49
108
170.1
68
16.94
6
003-006
16.8
70.35
155
170.2
68
24.29
Auti sm
003-007
13.4
42.65
94
158.6
63
16.96
9
003-010
15
67.1
148
176.02
70
21.66
7
003-012
14.5
58.35
128
178
71
18.42
7
003-013
15.9
55.15
121
177.2
70
17.56
5
003-014
16.3
67.75
149
176.9
70
21.65
Auti sm
003-015
15.2
49.6
109
175.9
70
16.03
6
003-016
13.5
44
97
170.1
68
15.21
6
003-017
16.2
53.15
117
174.7
69
17.41
Auti sm Spectrum
Note: ADOS severity of either Autism or Autism Spectrum is reflective of the fact that the subject was
tested using Module 4 of the ADOS and as such a numerical score isn't available
Subject
Number
Visit
Subscale I
(Irritability)
Subscale II (Social
Wi thdr awal )
Subscale III
(Stereotypic
Behavi our )
Subscale IV
(Hyperactivity/Noncompliance)
Subscale V (Inappropriate
Speech)
001-001
V1 (Screening)
22
12
.
.
.
001-001
V5 (EOT/ET)
13
3
6
6
0
001-001
V6
12
0
5
6
1
001-003
V1 (Screening)
16
44
18
13
5
001-003
V5 (EOT/ET)
6
26
14
7
4
001-003
V6
2
13
4
4
1
001-005
V1 (Screening)
3
9
3
4
6
001-005
V5 (EOT/ET)
5
9
1
4
4
001-005
V6
2
6
1
5
6
001-007
V1 (Screening)
26
15
5
24
1
001-007
V5 (EOT/ET)
8
8
0
11
0
001-007
V6
12
7
0
18
2
002-001
V1 (Screening)
0
0
0
0
0
002-001
V5 (EOT/ET)
0
4
1
9
2
002-001
V6
0
9
0
10
5
002-002
V1 (Screening)
6
11
1
20
2
002-002
V5 (EOT/ET)
8
11
1
15
3
002-002
V6
2
8
3
15
2
002-004
V1 (Screening)
24
22
5
22
4
002-004
V5 (EOT/ET)
15
4
4
14
1
002-004
V6
16
7
2
17
4
002-005
V1 (Screening)
29
27
6
19
6
002-005
V5 (EOT/ET)
28
33
1
22
8
002-005
V6
26
30
4
24
7
002-006
V1 (Screening)
2
21
0
4
1
002-006
V5 (EOT/ET)
1
16
1
5
0
002-006
V6
2
15
2
4
0
002-010
V1 (Screening)
20
13
5
23
7
002-010
V5 (EOT/ET)
15
12
7
17
1
002-010
V6
22
21
5
22
0
002-011
V1 (Screening)
1
0
0
2
0
002-011
V5 (EOT/ET)
1
0
0
2
0
002-011
V6
1
0
0
0
0
002-012
V1 (Screening)
15
14
2
17
2
002-012
V5 (EOT/ET)
3
8
0
4
1
002-012
V6
15
16
2
17
4
002-014
V1 (Screening)
8
10
4
14
1
002-014
V5 (EOT/ET)
2
10
0
9
2
002-014
V6
7
5
1
10
2
002-015
V1 (Screening)
15
8
4
16
1
002-015
V5 (EOT/ET)
11
5
1
15
1
002-015
V6
15
9
0
15
1
003-001
V1 (Screening)
40
27
6
36
7
003-001
V5 (EOT/ET)
17
8
0
12
3
003-001
V6
37
19
3
27
3
003-002
V1 (Screening)
2
20
3
8
2
003-002
V5 (EOT/ET)
0
9
0
4
1
003-002
V6
0
6
2
5
2
003-005
V1 (Screening)
5
8
4
8
1
003-005
V5 (EOT/ET)
3
10
0
11
2
003-005
V6
9
12
3
13
3
003-006
V1 (Screening)
0
8
0
10
0
003-006
V5 (EOT/ET)
0
6
0
6
1
003-006
V6
0
9
0
8
1
003-007
V1 (Screening)
24
20
10
26
8
003-007
V5 (EOT/ET)
24
21
8
25
7
003-007
V6
25
19
10
34
7
003-010
V1 (Screening)
23
16
10
28
5
003-010
V5 (EOT/ET)
14
10
6
19
1
003-010
V6
24
10
7
27
5
003-012
V1 (Screening)
4
1
1
0
0
003-012
V5 (EOT/ET)
3
1
0
2
1
003-012
V6
0
1
0
0
0
003-013
V1 (Screening)
5
1
0
7
0
003-013
V5 (EOT/ET)
7
2
1
11
0
003-013
V6
14
10
2
15
0
003-014
V1 (Screening)
2
14
6
2
2
003-014
V5 (EOT/ET)
4
13
4
9
3
003-014
V6
1
12
3
5
2
003-015
V1 (Screening)
10
18
5
15
5
003-015
V5 (EOT/ET)
8
15
0
12
1
003-015
V6
3
4
2
6
1
003-016
V1 (Screening)
14
4
6
22
4
003-016
V5 (EOT/ET)
14
14
5
20
4
003-017
V1 (Screening)
1
19
10
1
1
003-017
V5 (EOT/ET)
3
27
11
4
4
S
upplementary Information Table 2. ABC Subscales
Supplementary Information Table 3. PARS
Subject
V1 (Baseline)
V5 (EOT)
V6 (Recovery)
001-001
4
10
6
001-003
15
4
1
001-005
8
11
6
001-007
11
6
6
002-001
9
0
2
002-002
13
7
0
002-004
8
15
5
002-005
11
9
16
002-006
11
10
6
002-010
7
12
20
002-011
7
0
5
002-012
12
0
0
002-014
3
1
7
002-015
16
10
15
003-001
19
6
13
003-002
10
0
2
003-005
10
15
7
003-006
11
8
8
003-007
18
15
18
003-010
18
16
18
003-012
12
11
0
003-013
2
10
10
003-014
4
15
1
003-015
14
11
11
003-016
11
003-017
10
Subjects were not able to
complete V5 or V6
Supplementary Information Table 4. VABS
age
Subject ID
Total ABC
Scores
Standardiz
ed
Composi te
Scores
Communi cati on
Standard Score
Dai l y
Li vi ng
Standard
Score
Socializati
on
Standard
Score
12.1
001-001
V1 (Screening)
81
28
20
31
30
12.1
001-001
V5 (EOT)
88
30
20
38
30
12.1
001-001
V6
97
33
20
41
36
14.6
001-003
V1 (Screening)
247
79
81
100
66
14.6
001-003
V5 (EOT)
226
74
81
70
75
14.6
001-003
V6
214
71
84
77
53
15.5
001-005
V1 (Screening)
234
76
66
92
76
15.5
001-005
V5 (EOT)
238
77
70
89
79
15.5
001-005
V6
265
85
84
92
89
12.1
001-007
V1 (Screening)
202
68
76
72
54
12.1
001-007
V5 (EOT)
237
77
100
93
44
12.1
001-007
V6
287
93
102
104
81
13.1
002-001
V1 (Screening)
248
79
85
100
63
13.1
002-001
V5 (EOT)
314
105
108
133
73
13.1
002-001
V6
334
114
108
132
94
17.3
002-002
V1 (Screening)
260
83
64
100
96
17.3
002-002
V5 (EOT)
234
76
77
89
68
17.3
002-002
V6
271
87
79
96
96
12.9
002-004
V1 (Screening)
361
126
106
133
122
12.9
002-004
V5 (EOT)
264
84
100
118
46
12.9
002-004
V6
257
82
83
116
58
13.5
002-005
V1 (Screening)
189
63
70
89
30
13.5
002-005
V5 (EOT)
217
72
68
89
60
13.5
002-005
V6
222
73
68
93
61
13.1
002-006
V1 (Screening)
246
79
81
95
70
13.1
002-006
V5 (EOT)
268
85
85
100
83
13.1
002-006
V6
251
80
78
98
75
13.4
002-010
V1 (Screening)
207
69
77
76
54
13.4
002-010
V5 (EOT)
212
70
70
79
63
13.4
002-010
V6
221
73
92
79
50
13.8
002-011
V1 (Screening)
337
116
104
133
100
13.8
002-011
V5 (EOT)
356
124
110
132
114
13.8
002-011
V6
347
121
113
122
112
14.2
002-012
V1 (Screening)
148
51
30
65
53
14.2
002-012
V5 (EOT)
302
101
77
140
85
14.2
002-012
V6
185
62
46
85
54
13.8
002-014
V1 (Screening)
295
98
85
116
94
13.8
002-014
V5 (EOT)
184
62
48
73
63
13.8
002-014
V6
221
73
68
76
77
12.8
002-015
V1 (Screening)
208
69
68
82
58
12.8
002-015
V5 (EOT)
229
75
83
85
61
12.8
002-015
V6
232
75
83
93
56
13.7
003-001
V1 (Screening)
185
62
58
66
61
13.7
003-001
V5 (EOT)
217
72
70
79
68
13.7
003-001
V6
189
63
52
77
60
16.1
003-002
V1 (Screening)
238
77
82
83
73
16.1
003-002
V5 (EOT)
241
78
88
85
68
16.1
003-002
V6
246
79
82
88
76
13.5
003-005
V1 (Screening)
259
83
81
102
76
13.5
003-005
V5 (EOT)
309
102
83
126
100
13.5
003-005
V6
283
91
81
104
98
16.8
003-006
V1 (Screening)
273
87
94
96
83
16.8
003-006
V5 (EOT)
282
91
96
92
94
16.8
003-006
V6
291
95
103
94
94
13.4
003-007
V1 (Screening)
182
62
60
80
42
13.4
003-007
V5 (EOT)
224
73
78
85
61
13.4
003-007
V6
217
72
75
82
60
15
003-010
V1 (Screening)
202
68
75
79
48
15
003-010
V5 (EOT)
206
69
81
81
44
15
003-010
V6
204
68
77
83
44
14.5
003-012
V1 (Screening)
93
32
32
23
38
14.5
003-012
V5 (EOT)
168
57
38
59
71
14.5
003-012
V6
168
57
38
59
71
15.9
003-013
V1 (Screening)
227
74
75
92
60
15.9
003-013
V5 (EOT)
231
75
79
88
64
15.9
003-013
V6
231
75
79
88
64
16.3
003-014
V1 (Screening)
255
82
88
109
58
16.3
003-014
V5 (EOT)
257
82
91
96
70
16.3
003-014
V6
292
96
85
111
96
15.2
003-015
V1 (Screening)
220
72
79
70
71
15.2
003-015
V5 (EOT)
263
84
96
92
75
15.2
003-015
V6
242
78
82
77
83
003-016
003-017
Analysis not completed due to parental illness
Note:Red subject numbers not included because their assessments were not valid based on assessment
criteria
Subject
interval_name
SRS
Composite
Social
Awareness
Social
Cognition
Social
Communication
Social
Moti vi ati on
Repetitive
Behavior
001-001
V1 (Screening)
80
89
81
69
62
89
001-001
V5 (EOT/ET)
74
73
70
67
62
87
001-001
V6
68
73
70
63
58
68
001-003
V1 (Screening)
98
76
87
100
93
98
001-003
V5 (EOT/ET)
85
73
76
86
77
89
001-003
V6
85
76
74
90
79
78
001-005
V1 (Screening)
70
51
72
74
62
68
001-005
V5 (EOT/ET)
68
45
66
68
67
75
001-005
V6
69
54
74
67
62
71
001-007
V1 (Screening)
92
79
91
89
87
87
001-007
V5 (EOT/ET)
81
81
81
83
71
68
001-007
V6
71
67
66
71
75
62
002-001
V1 (Screening)
58
60
52
56
62
61
002-001
V5 (EOT/ET)
71
67
65
69
71
71
002-001
V6
70
67
61
75
58
71
002-002
V1 (Screening)
75
54
72
74
77
76
002-002
V5 (EOT/ET)
76
73
74
75
67
73
002-002
V6
69
54
63
72
73
66
002-004
V1 (Screening)
64
67
59
58
62
71
002-004
V5 (EOT/ET)
75
86
72
77
67
64
002-004
V6
70
76
70
70
56
68
002-005
V1 (Screening)
96
95
96
91
95
82
002-005
V5 (EOT/ET)
86
86
91
78
79
82
002-005
V6
91
89
87
90
79
87
002-006
V1 (Screening)
75
79
66
74
73
71
002-006
V5 (EOT/ET)
72
54
66
69
77
76
002-006
V6
73
64
61
73
75
76
002-010
V1 (Screening)
87
82
81
81
81
89
002-010
V5 (EOT/ET)
80
73
76
74
69
89
002-010
V6
88
86
81
85
79
89
002-011
V1 (Screening)
62
51
65
62
54
68
002-011
V5 (EOT/ET)
58
57
63
55
46
66
002-011
V6
54
57
57
55
42
57
002-012
V1 (Screening)
83
79
79
81
87
73
002-012
V5 (EOT/ET)
74
73
79
74
69
61
002-012
V6
88
82
87
90
83
73
002-014
V1 (Screening)
73
64
79
73
62
69
002-014
V5 (EOT/ET)
85
73
83
93
69
76
002-014
V6
74
76
78
70
62
73
002-015
V1 (Screening)
76
76
78
70
69
76
002-015
V5 (EOT/ET)
73
73
76
70
67
68
002-015
V6
70
67
72
67
67
68
003-001
V1 (Screening)
79
70
79
79
67
80
003-001
V5 (EOT/ET)
83
76
89
84
67
75
003-001
V6
74
60
78
78
67
66
003-002
V1 (Screening)
75
79
63
84
64
66
003-002
V5 (EOT/ET)
70
76
57
78
71
53
003-002
V6
71
79
57
78
69
59
003-005
V1 (Screening)
77
73
72
79
75
69
003-005
V5 (EOT/ET)
76
73
72
81
75
59
003-005
V6
78
76
76
81
73
64
003-006
V1 (Screening)
52
57
48
44
54
62
003-006
V5 (EOT/ET)
59
51
52
58
60
68
003-006
V6
61
64
50
61
58
68
003-007
V1 (Screening)
87
79
85
85
79
82
003-007
V5 (EOT/ET)
86
82
83
91
71
78
003-007
V6
85
73
83
85
77
80
003-010
V1 (Screening)
82
82
79
79
71
80
003-010
V5 (EOT/ET)
80
86
78
80
64
76
003-010
V6
72
79
74
72
58
66
003-012
V1 (Screening)
69
64
59
67
71
73
003-012
V5 (EOT/ET)
67
60
65
70
67
61
003-012
V6
58
57
59
52
60
59
003-013
V1 (Screening)
60
60
55
66
54
57
003-013
V5 (EOT/ET)
59
76
57
65
46
48
003-013
V6
58
64
57
68
42
52
003-014
V1 (Screening)
83
86
74
81
83
76
003-014
V5 (EOT/ET)
68
79
59
67
69
61
003-014
V6
68
79
61
68
69
61
003-015
V1 (Screening)
81
79
78
82
67
78
003-015
V5 (EOT/ET)
76
70
76
77
71
68
003-015
V6
77
70
78
75
71
75
Supplementary Information Table 5. SRS