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A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03811028
Recruitment Status : Completed
First Posted : January 22, 2019
Results First Posted : February 25, 2022
Last Update Posted : February 25, 2022
Sponsor:
Information provided by (Responsible Party):
Swedish Orphan Biovitrum

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Sanfilippo Syndrome Type A (MPS IIIA)
Intervention Drug: SOBI003
Enrollment 6
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Period Title: Overall Study
Started 3 3
Completed 3 3
Not Completed 0 0
Arm/Group Title Dose Group 1 Dose Group 2 Total
Hide Arm/Group Description

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Total of all reporting groups
Overall Number of Baseline Participants 3 3 6
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 3 participants 3 participants 6 participants
52.0  (23.8) 34.7  (12.7) 43.3  (19.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 6 participants
Female
1
  33.3%
1
  33.3%
2
  33.3%
Male
2
  66.7%
2
  66.7%
4
  66.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 6 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
3
 100.0%
3
 100.0%
6
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Safety as Measured by Adverse Events Frequencies (by Type and Severity)
Hide Description Number of adverse events, by type and severity, from week 25 up to week 104
Time Frame From infusion week 25 up to week 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:
SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104 SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Measure Type: Number
Unit of Measure: events
Any adverse event 174 355
Any non-treatment emergent serious adverse event 0 1
Any treatment emergent adverse event (TEAE) 174 351
Any drug-related TEAE 69 202
Any non-serious TEAE 172 342
Any serious TEAE 2 9
Any serious drug-related TEAE 0 0
Any TEAE leading to study and/or treatment withdrawal 0 0
Any drug-related TEAE leading to study and/or treatment withdrawal 0 0
Any serious TEAE leading to study and/or treatment withdrawal 0 0
Any TEAE leading to death 0 0
Any Infusion Related Reaction 46 78
2.Secondary Outcome
Title The Observed SOBI003 Serum Concentration Immediately Before the Start of Infusion of SOBI003
Hide Description The observed SOBI003 serum concentration immediately before the start of infusion of SOBI003 (CPre-dose) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
The table report number of available pharmacokinetic (PK) samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Mean (Standard Deviation)
Unit of Measure: ng/mL
Week 38 - central serum Number Analyzed 0 participants 1 participants
120.0 [1]   (NA)
Week 52 - central serum Number Analyzed 0 participants 0 participants
Week 78 - central serum Number Analyzed 1 participants 1 participants
47.0 [1]   (NA) 75.0 [1]   (NA)
Week 104 - central serum Number Analyzed 1 participants 0 participants
2610 [1]   (NA)
Week 38 - peripheral serum Number Analyzed 1 participants 2 participants
49 [1]   (NA) 30  (14.142)
Week 52 - peripheral serum Number Analyzed 1 participants 2 participants
123 [1]   (NA) 11770  (16312)
Week 78 - peripheral serum Number Analyzed 2 participants 0 participants
208.5  (234.05)
Week 104 - peripheral serum Number Analyzed 2 participants 3 participants
765.5  (125.16) 66.33  (49.571)
[1]
Only one sample analyzed
3.Secondary Outcome
Title The Observed SOBI003 Serum Concentration at the End of Infusion of SOBI003
Hide Description The observed SOBI003 serum concentration at the end of infusion of SOBI003 (CEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
The table report number of available pharmacokinetic (PK) samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Mean (Standard Deviation)
Unit of Measure: ng/mL
Week 38 - central serum Number Analyzed 0 participants 1 participants
210000 [1]   (NA)
Week 38 - peripheral serum Number Analyzed 3 participants 2 participants
14370  (14583) 109500  (3535.5)
Week 52 - central serum Number Analyzed 1 participants 1 participants
7900 [1]   (NA) 203000 [1]   (NA)
Week 52 - peripheral serum Number Analyzed 2 participants 2 participants
72000  (37194) 86510  (122320)
Week 78 - central serum Number Analyzed 0 participants 0 participants
Week 78 - peripheral serum Number Analyzed 3 participants 2 participants
74450  (105310) 229500  (101120)
Week 104 - central serum Number Analyzed 1 participants 0 participants
79700 [1]   (NA)
Week 104 - peripheral serum Number Analyzed 2 participants 2 participants
292599  (37477) 195000  (72125)
[1]
Only one sample analyzed
4.Secondary Outcome
Title The Time of the End of the Infusion of SOBI003
Hide Description The time of the end of infusion of SOBI003 (tEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number of analysed are available samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: Hours
Week 38 - central serum Number Analyzed 0 participants 1 participants
6.20
(6.20 to 6.20)
Week 52 - central serum Number Analyzed 1 participants 1 participants
4.50
(4.50 to 4.50)
4.170
(4.17 to 4.17)
Week 78 - central serum Number Analyzed 0 participants 0 participants
Week 104 - central serum Number Analyzed 1 participants 0 participants
4.47
(4.47 to 4.47)
Week 38 - peripheral serum Number Analyzed 3 participants 2 participants
4.5
(4.08 to 6.25)
4.825
(4.33 to 5.32)
Week 52- peripheral serum Number Analyzed 2 participants 2 participants
4.775
(4.08 to 5.47)
5.845
(4.17 to 7.52)
Week 78- peripheral serum Number Analyzed 3 participants 2 participants
4.50
(4.18 to 5.88)
8.575
(4.73 to 12.4)
Week 104- peripheral serum Number Analyzed 2 participants 2 participants
5.030
(4.03 to 6.03)
8.540
(4.25 to 12.8)
5.Secondary Outcome
Title The Maximum Observed Serum Concentration of SOBI003
Hide Description The maximum observed serum concentration of SOBI003 (Cmax) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number of analysed are available samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: ng/mL
Week 38 - central serum Number Analyzed 0 participants 2 participants
105000
(32.0 to 210000)
Week 52 - central serum Number Analyzed 0 participants 1 participants
203000
(203000 to 203000)
Week 78 - central serum Number Analyzed 1 participants 1 participants
136.0
(136.0 to 136.0)
75.0
(75.0 to 75.0)
Week 104 - central serum Number Analyzed 1 participants 0 participants
79700
(79700 to 79700)
Week 38 - peripheral serum Number Analyzed 3 participants 2 participants
7690
(4330 to 31100)
109500
(107000 to 112000)
Week 52 - peripheral serum Number Analyzed 3 participants 3 participants
45700
(154 to 98300)
23300
(11600 to 173000)
Week 78 - peripheral serum Number Analyzed 3 participants 2 participants
195000
(28000 to 243000)
229500
(158000 to 301000)
Week 104 - peripheral serum Number Analyzed 2 participants 3 participants
292500
(266000 to 319000)
144000
(25000 to 246000)
6.Secondary Outcome
Title The Time at Which the Maximum Serum Concentration of SOBI003 is Observed
Hide Description The time after start of infusion at which the maximum serum concentration is observed (tmax) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number of analysed are available samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: Hours
Week 38 - central serum Number Analyzed 0 participants 2 participants
87.96
(8.17 to 168)
Week 52 - central serum Number Analyzed 1 participants 1 participants
4.50
(4.50 to 4.50)
4.170
(4.170 to 4.170)
Week 78 - central serum Number Analyzed 1 participants 1 participants
168
(168 to 168)
0
(0 to 0)
Week 104 - central serum Number Analyzed 1 participants 0 participants
4.47
(4.47 to 4.47)
Week 38 - peripheral serum Number Analyzed 3 participants 2 participants
4.5
(4.08 to 6.25)
4.825
(4.33 to 5.32)
Week 52 - peripheral serum Number Analyzed 3 participants 3 participants
5.47
(4.08 to 26.5)
7.52
(0 to 24.5)
Week 78 - peripheral serum Number Analyzed 3 participants 2 participants
5.88
(4.5 to 168)
8.575
(4.73 to 12.4)
Week 104 - peripheral serum Number Analyzed 2 participants 3 participants
5.03
(4.03 to 6.03)
12.83
(4.25 to 23.7)
7.Secondary Outcome
Title The Minimum Observed Serum Concentration of SOBI003
Hide Description The minimum observed serum concentration of SOBI003 (CTrough) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number of analysed are available samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: ng/mL
Week 38 - central serum Number Analyzed 0 participants 2 participants
68.0
(32.0 to 104.0)
Week 52 - central serum Number Analyzed 0 participants 0 participants
Week 78 - central serum Number Analyzed 1 participants 1 participants
47.0
(47.0 to 47.0)
75.0
(75.0 to 75.0)
Week 104 - central serum Number Analyzed 1 participants 0 participants
2610
(2610 to 2610)
Week 38 - peripheral serum Number Analyzed 1 participants 2 participants
34
(34 to 34)
30
(20 to 40)
Week 52 - peripheral serum Number Analyzed 2 participants 3 participants
138.5
(123 to 154)
25
(11 to 45)
Week 78 - peripheral serum Number Analyzed 3 participants 1 participants
362
(43 to 28000)
85
(85 to 85)
Week 104 - peripheral serum Number Analyzed 2 participants 3 participants
704.5
(555 to 854)
75
(13 to 111)
8.Secondary Outcome
Title Clearance
Hide Description Clearance (CL) of SOBI003 Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number of analysed are available samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: mL/min
Week 38 - central serum Number Analyzed 0 participants 0 participants
Week 52 - central serum Number Analyzed 0 participants 0 participants
Week 78 - central serum Number Analyzed 1 participants 0 participants
208
(208 to 208)
Week 104 - central serum Number Analyzed 1 participants 0 participants
1.6
(1.6 to 1.6)
Week 38 - peripheral serum Number Analyzed 0 participants 1 participants
0.661
(0.661 to 0.661)
Week 52 - peripheral serum Number Analyzed 1 participants 0 participants
3.51
(3.51 to 3.51)
Week 78 - peripheral serum Number Analyzed 0 participants 0 participants
Week 104 - peripheral serum Number Analyzed 1 participants 1 participants
2.21
(2.21 to 2.21)
3.61
(3.61 to 3.61)
9.Secondary Outcome
Title Area Under the SOBI003 Serum Concentration-time Curve From Time 0 to168 Hours
Hide Description Area under the SOBI003 serum concentration-time curve from time 0 to 168 hours (AUC 0-168h) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame 0,1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, 144, 168 hours post-dose on Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number of analysed are available samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: h*ng/mL
Week 38 - central serum Number Analyzed 0 participants 0 participants
Week 52 - central serum Number Analyzed 0 participants 0 participants
Week 78 - central serum Number Analyzed 1 participants 0 participants
15400
(15400 to 15400)
Week 104 - central serum Number Analyzed 1 participants 0 participants
2040000
(2040000 to 2040000)
Week 38 - peripheral serum Number Analyzed 0 participants 1 participants
2880000
(2880000 to 2880000)
Week 52 - peripheral serum Number Analyzed 1 participants 0 participants
1110000
(1110000 to 1110000)
Week 78 - peripheral serum Number Analyzed 0 participants 0 participants
Week 104 - peripheral serum Number Analyzed 1 participants 1 participants
4390000
(4390000 to 4390000)
2770000
(2770000 to 2770000)
10.Secondary Outcome
Title The Half-life
Hide Description The half-life of SOBI003 in serum (T1/2) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture.
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number of analysed are available samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
11.Secondary Outcome
Title SOBI003 Concentration in Cerebrospinal Fluid
Hide Description Concentration of SOBI003 in cerebrospinal fluid
Time Frame Weeks 52 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number analysed are available samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: ng/mL
Week 52 Number Analyzed 1 participants 2 participants
15.7
(15.7 to 15.7)
92.3
(73.6 to 115)
Week 104 Number Analyzed 2 participants 3 participants
118.35
(95.7 to 141.0)
57.1
(41.5 to 147)
12.Secondary Outcome
Title Number of Patients Having Anti-drug Antibodies in Serum
Hide Description Number of patients in each dose group having anti-drug antibodies in serum
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Measure Type: Count of Participants
Unit of Measure: Participants
Week 38
3
 100.0%
3
 100.0%
Week 52
3
 100.0%
3
 100.0%
Week 78
3
 100.0%
2
  66.7%
Week 104
3
 100.0%
3
 100.0%
13.Secondary Outcome
Title Number of Patients Having Anti-drug Antibodies in Cerebrospinal Fluid
Hide Description Number of patients in each dose group having anti-drug antibodies cerebrospinal fluid
Time Frame Weeks 52 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Measure Type: Count of Participants
Unit of Measure: Participants
Week 52
2
  66.7%
3
 100.0%
Week 104
3
 100.0%
3
 100.0%
14.Secondary Outcome
Title Change From Baseline in Heparan Sulfate Concentration in Cerebrospinal Fluid
Hide Description Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid
Time Frame Weeks 52 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: mg/L
Week 52
-1.19
(-4.91 to 0.1)
-3.47
(-23.2 to -2.579)
Week 104
-6.07
(-6.67 to -4.42)
-3.59
(-24.7 to -2.18)
15.Secondary Outcome
Title Change From Baseline in Heparan Sulfate Levels in Serum
Hide Description Change from baseline in Heparan sulfate, in mg/L, levels in serum
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: mg/L
Week 38
-1.811
(-2.168 to -1.793)
-1.88
(-3.721 to -1.854)
Week 52
-1.76
(-2.169 to -1.644)
-2.25
(-3.735 to -1.18)
Week 78
-1.729
(-2.41 to -1.686)
-2.01
(-3.05 to -0.97)
Week 104
-2.21
(-2.41 to 0.21)
-2.24
(-3.708 to -1.91)
16.Secondary Outcome
Title Change From Baseline in Heparan Sulfate Levels in Urine
Hide Description Change from baseline in Heparan sulfate levels, in g/mol, in urine
Time Frame Weeks 38, 52, 78 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number of analysed are available samples
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: g/mol
Week 38 Number Analyzed 3 participants 3 participants
-447.2
(-552.82 to -340.1)
-692.78
(-697.12 to -382.65)
Week 52 Number Analyzed 3 participants 3 participants
-464.4
(-559.02 to -411.67)
-698.79
(-701.99 to -388.86)
Week 78 Number Analyzed 2 participants 2 participants
-512.19
(-599.70 to -424.68)
-494.610
(-747.22 to -242.0)
Week 104 Number Analyzed 3 participants 3 participants
-578.89
(-709.47 to -489.68)
-726.24
(-753.97 to -381.45)
17.Secondary Outcome
Title Change From Baseline in Neurocognitive Development Quotient
Hide Description

Quotient between age equivalent score and age, 0 - 100%, where high values are desirable. The age equivalent score represent the age of the typical and normal individual who would achieve the same result as the one who was tested The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition.

The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor,social-emotional, and adaptive behavior.

The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.

These results are truly "unitless"/"dimensionless" because they represents quotients of values from the same scale, which means that the units in the denominator and

Time Frame Weeks 52 and 104
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Hide Analysis Population Description
Number of analysed are number of assessments
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: Unitless
Week 52 Number Analyzed 3 participants 2 participants
-8.97
(-18 to -6.9)
-31.74
(-48.9 to -14.6)
Week 104 Number Analyzed 3 participants 3 participants
-16.28
(-32.9 to -11.7)
-24.94
(-44.6 to -11.3)
18.Secondary Outcome
Title Change From Baseline in Age-equivalence Score
Hide Description The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development.
Time Frame Week 52 and 104
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Number of analyses are available assessments
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: Months
Week 52 Number Analyzed 3 participants 2 participants
-1.0
(-2 to 2)
-1.0
(-2 to 0)
Week 104 Number Analyzed 3 participants 3 participants
-3.0
(-4 to 1)
5.0
(-1 to 10)
19.Secondary Outcome
Title Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II.
Hide Description The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90. The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.
Time Frame Week 52 and 104
Hide Outcome Measure Data
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Number of analyses are available assessments
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: Months
Week 52 Number Analyzed 3 participants 2 participants
14.0
(10 to 31)
16.0
(12 to 20)
Week 104 Number Analyzed 3 participants 3 participants
13.0
(8 to 29)
19.0
(19 to 36)
20.Secondary Outcome
Title Change From Baseline in Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II.
Hide Description

The age equivalent score represents the age in months of the typical and normal individual who would achieve the same result as the one who was tested.

The age equivalent scores are assessed either by the Bayley Scales of Infant and Toddler Development®, third edition, (BSID-III) cognitive subtest or the Kaufman Assessment Battery for Children, Second edition (KABC-II) depending on chronological age of the subject. Quotient between age equivalent score and age, 0 - 100%, where high values are desirable.

The BSID-III is an individually administered test designed to assess developmental functioning of infants and toddlers. The BSID-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior.

The KABC-II is a clinical instrument for assessing cognitive development. The unit and measurement is the same in both scales (BSID-III and KABC-II): Age-equivalent score.

Time Frame Week 52 and 104
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Number of analysed are available assessments
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: Months
Week 52 Number Analyzed 3 participants 2 participants
-1.0
(-2 to 2)
-1.0
(-2 to 0)
Week 104 Number Analyzed 3 participants 3 participants
-3.0
(-4 to 1)
5.0
(-1 to 10)
21.Secondary Outcome
Title Age-equivalence Score as Assessed by VABS-II
Hide Description

The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested.

The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90.

The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.

Time Frame Week 52 and 104
Hide Outcome Measure Data
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[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: Months
Week 52
15
(15 to 26)
28
(13 to 37)
Week 104
16
(13 to 21)
23
(20 to 43)
22.Secondary Outcome
Title Change From Baseline in Age-equivalence Score as Assessed by VABS-II
Hide Description

The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested.

The age equivalent scores are assessed by Vineland™ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90.

The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior.

Time Frame Week 52 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: Months
Week 52
-1
(-12 to 2)
1
(0 to 4)
Week 104
-6
(-11 to 0)
8
(-5 to 10)
23.Secondary Outcome
Title Change From Baseline in Gray Matter Volume
Hide Description Grey matter contains most of the brain's neuronal cell bodies. The grey matter includes regions of the brain involved in muscle control, and sensory perception such as seeing and hearing, memory, emotions, speech, decision making, and self-control. The gray matter volume will be measured by volumetric magnetic resonance imaging (MRI) at weeks 52 and 104.
Time Frame Week 52 and 104
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Number analysed is available assessments
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Median (Full Range)
Unit of Measure: mL
Week 52 Number Analyzed 3 participants 2 participants
-24.629
(-34.32 to 66.87)
19.485
(-62.32 to 101.29)
Week 104 Number Analyzed 2 participants 2 participants
-53.584
(-161.03 to 53.86)
13.387
(-45.41 to 72.18)
24.Secondary Outcome
Title Pediatric Quality of Life Inventory (PedsQL™) Total Score
Hide Description Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. Lower scores indicate better functioning. Min score = 0, and max score = 144.
Time Frame Week 52 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Week 52 53.4  (3.9) 72.2  (11.6)
Week 104 55.9  (10.2) 76.5  (3.8)
25.Secondary Outcome
Title Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Total Score
Hide Description Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life (HRQOL) in healthy children and adolescents and those with acute and chronic health conditions. Higher scores indicate better functioning. Min score = 0, and max score = 144.
Time Frame Week 52 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Week 52 -17.0  (25.9) -3.7  (24.2)
Week 104 -14.5  (14.9) 0.6  (21.0)
26.Secondary Outcome
Title PedsQL™ Family Impact Module Total Score
Hide Description Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning. Min score = 0, and max score = 144.
Time Frame Week 52 and 104
Hide Outcome Measure Data
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[Not Specified]
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Week 52 66.7  (30.6) 70.0  (26.5)
Week 104 73.3  (34.0) 70.0  (26.5)
27.Secondary Outcome
Title Change From Baseline in PedsQL™ Family Impact Module Total Score
Hide Description Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning.
Time Frame Week 52 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
Number analysed is available assessments
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description:

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

Overall Number of Participants Analyzed 3 3
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Week 52 Number Analyzed 3 participants 2 participants
16.7  (12.6) 0.0  (0.0)
Week 104 Number Analyzed 3 participants 2 participants
23.3  (31.8) 0.0  (0.0)
Time Frame The period for recording adverse events, including Serious Adverse Events (SAEs), began Day 1 of week 25, when first dose in study SOBI003-002 was given, and ended at completion of week 104 visit. In addition SAEs was reported from the time of signing the informed consent form until 28 days past the last dose of SOBI003.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Dose Group 1 Dose Group 2
Hide Arm/Group Description

SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104

SOBI003: weekly i.v. infusion

All-Cause Mortality
Dose Group 1 Dose Group 2
Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)      0/3 (0.00%)    
Hide Serious Adverse Events
Dose Group 1 Dose Group 2
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/3 (66.67%)      2/3 (66.67%)    
General disorders     
Pyrexia  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Infections and infestations     
Upper respiratory tract infection  1  1/3 (33.33%)  1 1/3 (33.33%)  2
Device related infection  1  0/3 (0.00%)  1/3 (33.33%)  1
Endocarditis  1  0/3 (0.00%)  1/3 (33.33%)  1
Infection  1  0/3 (0.00%)  1/3 (33.33%)  1
Influenza  1  0/3 (0.00%)  1/3 (33.33%)  1
Pneumonia  1  0/3 (0.00%)  1/3 (33.33%)  1
Staphylococcal infection  1  0/3 (0.00%)  1/3 (33.33%)  1
Injury, poisoning and procedural complications     
Incision site haemorrhage  1  0/3 (0.00%)  1/3 (33.33%)  1
1
Term from vocabulary, MedDRA 23.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Dose Group 1 Dose Group 2
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/3 (100.00%)      3/3 (100.00%)    
Blood and lymphatic system disorders     
Iron deficiency anaemia  1  1/3 (33.33%)  2 1/3 (33.33%)  2
Cardiac disorders     
Aortic valve thickening  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Cyanosis  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Tachycardia  1  1/3 (33.33%)  20 3/3 (100.00%)  28
Ear and labyrinth disorders     
Auricular swelling  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Otorrhoea  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Eye disorders     
Eye swelling  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Gastrointestinal disorders     
Abdominal pain  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Abdominal pain upper  1  0/3 (0.00%)  0 2/3 (66.67%)  2
Abnormal faeces  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Aphthous ulcer  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Constipation  1  1/3 (33.33%)  4 0/3 (0.00%)  0
Dental caries  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Diarrhoea  1  1/3 (33.33%)  1 2/3 (66.67%)  10
Faeces discoloured  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Haematochezia  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Lip disorder  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Nausea  1  0/3 (0.00%)  0 2/3 (66.67%)  4
Oral contusion  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Retching  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Vomiting  1  3/3 (100.00%)  17 3/3 (100.00%)  22
General disorders     
Application site erythema  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Catheter site extravasation  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Catheter site inflammation  1  1/3 (33.33%)  1 1/3 (33.33%)  1
Catheter site injury  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Complication associated with device  1  0/3 (0.00%)  0 1/3 (33.33%)  6
Fatigue  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Infusion site bruising  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Infusion site irritation  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Medical device site haemorrhage  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Oedema  1  2/3 (66.67%)  2 0/3 (0.00%)  0
Pain  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Pyrexia  1  3/3 (100.00%)  8 3/3 (100.00%)  11
Immune system disorders     
Drug hypersensitivity  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Hypersensitivity  1  1/3 (33.33%)  2 1/3 (33.33%)  1
Seasonal allergy  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Infections and infestations     
Abscess  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Cellulitis  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Conjunctivitis  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Corona virus infection  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Device related infection  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Ear infection  1  1/3 (33.33%)  2 1/3 (33.33%)  1
Endocarditis  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Furuncle  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Gastroenteritis  1  2/3 (66.67%)  4 0/3 (0.00%)  0
Infection  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Influenza  1  1/3 (33.33%)  1 1/3 (33.33%)  1
Nasopharyngitis  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Otitis media acute  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Pharyngitis streptococcal  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Pneumonia  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Staphylococcal infection  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Streptococcal infection  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Tonsillitis  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Upper respiratory tract infection  1  3/3 (100.00%)  17 2/3 (66.67%)  12
Viral infection  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Injury, poisoning and procedural complications     
Arthropod bite  1  1/3 (33.33%)  1 1/3 (33.33%)  2
Contusion  1  1/3 (33.33%)  2 3/3 (100.00%)  8
Fall  1  1/3 (33.33%)  2 1/3 (33.33%)  8
Foreign body in gastrointestinal tract  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Human bite  1  1/3 (33.33%)  3 0/3 (0.00%)  0
Incision site haemorrhage  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Lip injury  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Scratch  1  1/3 (33.33%)  1 1/3 (33.33%)  6
Skin abrasion  1  0/3 (0.00%)  0 1/3 (33.33%)  10
Soft tissue injury  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Vascular access complication  1  0/3 (0.00%)  0 2/3 (66.67%)  4
Vascular access site occlusion  1  1/3 (33.33%)  4 2/3 (66.67%)  5
Investigations     
Activated partial thromboplastin time prolonged  1  0/3 (0.00%)  0 1/3 (33.33%)  3
Blood fibrinogen increased  1  0/3 (0.00%)  0 1/3 (33.33%)  2
Blood pressure diastolic increased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Blood pressure systolic increased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
C-reactive protein increased  1  0/3 (0.00%)  0 1/3 (33.33%)  2
CSF glucose decreased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
CSF protein increased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Cardiac murmur  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Eosinophil count increased  1  0/3 (0.00%)  0 1/3 (33.33%)  2
Haemoglobin increased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Heart rate increased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Monocyte count decreased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Neutrophil count increased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Oxygen saturation decreased  1  0/3 (0.00%)  0 2/3 (66.67%)  26
Platelet count decreased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Red blood cell count increased  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Vitamin D decreased  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Weight decreased  1  1/3 (33.33%)  1 0/3 (0.00%)  0
White blood cell count increased  1  0/3 (0.00%)  0 1/3 (33.33%)  2
Musculoskeletal and connective tissue disorders     
Muscle tightness  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Skin papilloma  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Nervous system disorders     
Ataxia  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Clonus  1  0/3 (0.00%)  0 1/3 (33.33%)  2
Drooling  1  0/3 (0.00%)  0 1/3 (33.33%)  2
Tremor  1  1/3 (33.33%)  1 2/3 (66.67%)  6
Product Issues     
Device damage  1  1/3 (33.33%)  2 0/3 (0.00%)  0
Device dislocation  1  1/3 (33.33%)  2 2/3 (66.67%)  2
Device malfunction  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Psychiatric disorders     
Abnormal behaviour  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Anxiety  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Irritability  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Restlessness  1  0/3 (0.00%)  0 2/3 (66.67%)  8
Sleep disorder  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Respiratory, thoracic and mediastinal disorders     
Cough  1  1/3 (33.33%)  1 2/3 (66.67%)  10
Hypoxia  1  0/3 (0.00%)  0 1/3 (33.33%)  2
Nasal congestion  1  0/3 (0.00%)  0 1/3 (33.33%)  3
Rhinorrhoea  1  2/3 (66.67%)  2 1/3 (33.33%)  1
Sneezing  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Tachypnoea  1  0/3 (0.00%)  0 1/3 (33.33%)  5
Wheezing  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Skin and subcutaneous tissue disorders     
Dermatitis  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Dermatitis allergic  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Dermatitis contact  1  1/3 (33.33%)  2 0/3 (0.00%)  0
Dermatitis diaper  1  1/3 (33.33%)  1 2/3 (66.67%)  5
Dry skin  1  0/3 (0.00%)  0 1/3 (33.33%)  2
Erythema  1  1/3 (33.33%)  1 2/3 (66.67%)  22
Generalised erythema  1  0/3 (0.00%)  0 1/3 (33.33%)  7
Hyperhidrosis  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Hyperkeratosis  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Livedo reticularis  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Rash  1  1/3 (33.33%)  13 2/3 (66.67%)  4
Rash erythematous  1  0/3 (0.00%)  0 1/3 (33.33%)  13
Rash maculo-papular  1  1/3 (33.33%)  2 0/3 (0.00%)  0
Skin discolouration  1  0/3 (0.00%)  0 1/3 (33.33%)  2
Skin fissures  1  1/3 (33.33%)  1 1/3 (33.33%)  1
Skin irritation  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Swelling face  1  0/3 (0.00%)  0 1/3 (33.33%)  1
Urticaria  1  2/3 (66.67%)  20 3/3 (100.00%)  20
Vascular disorders     
Flushing  1  0/3 (0.00%)  0 3/3 (100.00%)  5
Hyperaemia  1  1/3 (33.33%)  1 0/3 (0.00%)  0
Hypertension  1  0/3 (0.00%)  0 1/3 (33.33%)  4
1
Term from vocabulary, MedDRA 23.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Global Communication
Organization: Sobi
Phone: +4686972000
EMail: info@sobi.com
Layout table for additonal information
Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT03811028    
Other Study ID Numbers: SOBI003-002
First Submitted: December 17, 2018
First Posted: January 22, 2019
Results First Submitted: December 7, 2021
Results First Posted: February 25, 2022
Last Update Posted: February 25, 2022