Trial Outcomes & Findings for Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease (NCT NCT02060526)
NCT ID: NCT02060526
Last Updated: 2021-06-08
Results Overview
The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. The development quotient (DQ) is a means to express a neurodevelopmental/cognitive delay which was computed as a ratio and expressed as a percentage using the age equivalent score divided by the age at testing (\[age-equivalent score/chronological age\] × 100; range: 0, 100). The BSID--III DQ score is based on the cognitive domain. A positive value indicates improvement in health and cognition. Overall response was the maximum decline in the DQ of 10 points or less over 48 weeks. Number of participants with the overall response were reported here.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
Baseline (Week 0) up to Week 48
Results posted on
2021-06-08
Participant Flow
A total of 24 participants were screened, of them 21 participants were enrolled in the study.
Participant milestones
| Measure |
No HGT-1410
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
Participants received HGT-1410 45 milligram (mg) intrathecally once every two weeks (Q2W) using surgically implanted intrathecal drug delivery device (IDDD) or lumbar puncture (LP) for 48 weeks.
|
HGT-1410 45 mg Q4W
Participants received HGT-1410 45 mg intrathecally once every four weeks (Q4W) using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Overall Study
STARTED
|
7
|
7
|
7
|
|
Overall Study
COMPLETED
|
7
|
7
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Baseline characteristics by cohort
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
32.42 months
STANDARD_DEVIATION 9.548 • n=5 Participants
|
29.64 months
STANDARD_DEVIATION 9.989 • n=7 Participants
|
33.53 months
STANDARD_DEVIATION 9.336 • n=5 Participants
|
31.87 months
STANDARD_DEVIATION 9.287 • n=4 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline (Week 0) up to Week 48Population: ITT population included all randomized participants.
The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. The development quotient (DQ) is a means to express a neurodevelopmental/cognitive delay which was computed as a ratio and expressed as a percentage using the age equivalent score divided by the age at testing (\[age-equivalent score/chronological age\] × 100; range: 0, 100). The BSID--III DQ score is based on the cognitive domain. A positive value indicates improvement in health and cognition. Overall response was the maximum decline in the DQ of 10 points or less over 48 weeks. Number of participants with the overall response were reported here.
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III)
|
0 participants
|
2 participants
|
1 participants
|
SECONDARY outcome
Timeframe: Baseline (Week 0) up to Week 52Population: Safety population included all participants who received a dose of HGT-1410 either using IDDD implantation or LP; underwent the IDDD surgical implant procedure without receiving a dose of HGT-1410; were randomly assigned to the untreated group and had any safety follow-up data.
An adverse event (AE) was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product related. This included an exacerbation of a pre-existing condition. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; lifethreatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Number of Participants With Serious Adverse Events (SAE)
|
3 participants
|
5 participants
|
6 participants
|
SECONDARY outcome
Timeframe: Baseline (Week 0) up to Week 52Population: Safety population included all participants who received a dose of HGT-1410 using the IDDD implantation or LP; underwent the IDDD surgical implant procedure without receiving a dose of HGT-1410; were randomly assigned to the untreated group and had any safety follow-up data.
An adverse event (AE) was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product related. This included an exacerbation of a pre-existing condition. TEAEs were defined as AE occurring on or after the time of first IDDD implantation or LP procedure to the end of study (EOS) visit (+30 days).
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
|
6 participants
|
7 participants
|
7 participants
|
SECONDARY outcome
Timeframe: Baseline (Week 0) up to Week 48Population: Safety population included all participants who received a dose of HGT-1410 using either the IDDD implantation or LP; underwent the IDDD surgical implant procedure without receiving a dose of HGT-1410; were randomly assigned to the untreated group and had any safety followup data.
A participant was considered positive if they had at least 1 positive result during the study. Once a participant reported antibody positive, they were considered positive for the remainder of the study.
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Number of Participants With Positive Anti-recombinant Human Heparan-N-Sulfatase (rhHNS) Antibody in Serum at Week 48
|
1 participants
|
7 participants
|
6 participants
|
SECONDARY outcome
Timeframe: Baseline (Week 0), Week 48Population: ITT population included all randomized participants.
The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. The DQ is a means to express a neurodevelopmental/cognitive delay. The DQ was computed as a ratio and expressed as a percentage using the age-equivalent score divided by the age at testing (\[age-equivalent score/chronological age\] × 100; range, 0, 100). The overall DQ score is calculated from the mean age-equivalent score obtained by averaging out the age-equivalent scores for the all the sub-domains except for Gross and Fine motor skills. This test measures the following 5 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite (a composite of the other 4 domains). A positive value indicates improvement in health and cognition.
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Change From Baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II) Development Quotient (DQ) Score at Week 48
Communication Domain
|
-5.12 percentage of chronological age
Standard Deviation 10.981
|
-20.83 percentage of chronological age
Standard Deviation 23.475
|
-4.97 percentage of chronological age
Standard Deviation 17.253
|
|
Change From Baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II) Development Quotient (DQ) Score at Week 48
Daily Living Skills Domain
|
-4.20 percentage of chronological age
Standard Deviation 29.005
|
-27.09 percentage of chronological age
Standard Deviation 21.444
|
-11.74 percentage of chronological age
Standard Deviation 26.713
|
|
Change From Baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II) Development Quotient (DQ) Score at Week 48
Socialization Domain
|
-16.12 percentage of chronological age
Standard Deviation 23.320
|
-24.24 percentage of chronological age
Standard Deviation 40.617
|
-29.34 percentage of chronological age
Standard Deviation 29.425
|
|
Change From Baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II) Development Quotient (DQ) Score at Week 48
Motor Skills Domain
|
-5.37 percentage of chronological age
Standard Deviation 24.741
|
-27.01 percentage of chronological age
Standard Deviation 20.820
|
-17.88 percentage of chronological age
Standard Deviation 12.007
|
SECONDARY outcome
Timeframe: Baseline (Week 0), Week 48Population: ITT population included all randomized participants.
The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. The DQ is a means to express a neurodevelopmental/cognitive delay which was computed as a ratio and expressed as a percentage using the age-equivalent score divided by the age at testing (\[age-equivalent score/chronological age\] × 100; range: 0, 100). The BSID--III DQ score is based on the cognitive domain. A positive value indicates improvement in health and cognition.
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Change From Baseline in Development Quotient (DQ) Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) at Week 48
Cognitive
|
-19.81 percentage of chronological age
Standard Deviation 3.974
|
-23.82 percentage of chronological age
Standard Deviation 14.684
|
-19.87 percentage of chronological age
Standard Deviation 12.724
|
|
Change From Baseline in Development Quotient (DQ) Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) at Week 48
Receptive Communication
|
-13.15 percentage of chronological age
Standard Deviation 13.318
|
-16.33 percentage of chronological age
Standard Deviation 25.373
|
-12.41 percentage of chronological age
Standard Deviation 14.981
|
|
Change From Baseline in Development Quotient (DQ) Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) at Week 48
Expressive Communication
|
-14.77 percentage of chronological age
Standard Deviation 10.055
|
-19.71 percentage of chronological age
Standard Deviation 22.878
|
-10.01 percentage of chronological age
Standard Deviation 15.573
|
|
Change From Baseline in Development Quotient (DQ) Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) at Week 48
Fine Motor
|
-23.72 percentage of chronological age
Standard Deviation 14.752
|
-18.59 percentage of chronological age
Standard Deviation 18.879
|
-18.86 percentage of chronological age
Standard Deviation 16.308
|
|
Change From Baseline in Development Quotient (DQ) Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) at Week 48
Gross Motor
|
-11.60 percentage of chronological age
Standard Deviation 13.668
|
-12.52 percentage of chronological age
Standard Deviation 21.203
|
-2.77 percentage of chronological age
Standard Deviation 18.947
|
SECONDARY outcome
Timeframe: Baseline (Week 0), Week 48Population: ITT population included all randomized participants.
The change from baseline in grey matter volume at Week 48 was assessed by magnetic resonance imaging (MRI).
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Change From Baseline in Total Cortical Grey Matter Volume at Week 48
|
-45.1 cubic centimeter (cc)
Standard Deviation 23.35
|
-102.0 cubic centimeter (cc)
Standard Deviation 68.12
|
-58.8 cubic centimeter (cc)
Standard Deviation 66.24
|
SECONDARY outcome
Timeframe: Baseline (Week 0), Week 48Population: ITT population included all randomized participants.
Change from baseline in concentration of GAG in CSF at Week 48 was reported.
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Change From Baseline in Concentration of Glycosaminoglycan (GAG) in Cerebrospinal Fluid (CSF) at Week 48
|
-0.193 micromolar
Standard Deviation 1.318
|
-6.888 micromolar
Standard Deviation 5.388
|
-5.924 micromolar
Standard Deviation 2.470
|
SECONDARY outcome
Timeframe: Baseline (Week 0), Week 48Population: ITT population included all randomized participants.
The concentration of GAG in urine was normalized to the urine creatinine value and reported as milligram (mg) GAG per millimole (mmol) creatinine.
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Change From Baseline in Concentration of GAG in Urine at Week 48
|
-7.18 mg GAG/mmol creatinine
Standard Deviation 39.175
|
-31.81 mg GAG/mmol creatinine
Standard Deviation 22.887
|
-42.46 mg GAG/mmol creatinine
Standard Deviation 29.861
|
SECONDARY outcome
Timeframe: Pre-dose, 4, 48 hours on Week 0 and Week 48Population: Pharmacokinetic (PK) population included all participants who received HGT-1410, participated in the scheduled PK studies, and had sufficient samples available for analysis.
Concentration of rhHNS in CSF was assessed using validated enzyme-linked immunosorbent assay (ELISA) method.
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Concentration of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF)
Week 0: Predose
|
0 nanogram per milliliter (ng/ml)
Standard Deviation 0
|
0 nanogram per milliliter (ng/ml)
Standard Deviation 0
|
—
|
|
Concentration of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF)
Week 0: 4h
|
313392.49 nanogram per milliliter (ng/ml)
Standard Deviation 142748.885
|
266021.60 nanogram per milliliter (ng/ml)
Standard Deviation 113340.887
|
—
|
|
Concentration of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF)
Week 0: 48h
|
366.05 nanogram per milliliter (ng/ml)
Standard Deviation 571.528
|
2119.59 nanogram per milliliter (ng/ml)
Standard Deviation 2068.093
|
—
|
|
Concentration of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF)
Week 48: Predose
|
869.67 nanogram per milliliter (ng/ml)
Standard Deviation 1863.989
|
365.04 nanogram per milliliter (ng/ml)
Standard Deviation 965.797
|
—
|
|
Concentration of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF)
Week 48: 4h
|
374544.38 nanogram per milliliter (ng/ml)
Standard Deviation 135047.700
|
335203.84 nanogram per milliliter (ng/ml)
Standard Deviation 82918.537
|
—
|
|
Concentration of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Cerebrospinal Fluid (CSF)
Week 48: 48h
|
104.49 nanogram per milliliter (ng/ml)
Standard Deviation 65.881
|
8892.93 nanogram per milliliter (ng/ml)
Standard Deviation 19519.717
|
—
|
SECONDARY outcome
Timeframe: Predose, 0.5 h, 1 h, 2 h, 4 h, 8 h, 12 h, 24 h, and 48 h post-dose on Week 0 and Week 48Population: Pharmacokinetic (PK) population included all participants who received HGT-1410, participated in the scheduled PK studies, and had sufficient samples available for analysis.
Cmax of rhHNS in serum was evaluated using enzyme-linked immunosorbent assay (ELISA) method and liquid chromatography tandem mass spectrometry (LC-MS) method.
Outcome measures
| Measure |
No HGT-1410
n=7 Participants
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 Participants
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Maximum Observed Drug Concentration (Cmax) of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Serum
ELISA Method: Week 0
|
120.9 ng/ml
Standard Deviation 84.77
|
237.0 ng/ml
Standard Deviation 159.68
|
—
|
|
Maximum Observed Drug Concentration (Cmax) of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Serum
ELISA Method: Week 48
|
63.5 ng/ml
Standard Deviation 149.76
|
118.8 ng/ml
Standard Deviation 161.98
|
—
|
|
Maximum Observed Drug Concentration (Cmax) of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Serum
LC-MS Method: Week 0
|
102.4 ng/ml
Standard Deviation 63.00
|
191.0 ng/ml
Standard Deviation 125.84
|
—
|
|
Maximum Observed Drug Concentration (Cmax) of Recombinant Human Heparan-N-Sulfatase (rhHNS) in Serum
LC-MS Method: Week 48
|
188.1 ng/ml
Standard Deviation 127.40
|
119.7 ng/ml
Standard Deviation 133.18
|
—
|
Adverse Events
No HGT-1410
Serious events: 3 serious events
Other events: 6 other events
Deaths: 0 deaths
HGT-1410 45 mg Q2W
Serious events: 5 serious events
Other events: 7 other events
Deaths: 0 deaths
HGT-1410 45 mg Q4W
Serious events: 6 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
No HGT-1410
n=7 participants at risk
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 participants at risk
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 participants at risk
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Gastrointestinal disorders
Haematemesis
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
General disorders
Adverse drug reaction
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 8 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
General disorders
Asthenia
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
General disorders
Device breakage
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
General disorders
Device failure
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
General disorders
Implant site extravasation
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 3 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
General disorders
Implant site pain
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Central nervous system infection
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Gastrointestinal infection
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Implant site infection
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Influenza
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Pneumonia
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Postoperative wound infection
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Skin infection
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Viral upper respiratory tract infection
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Wound infection
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Incision site swelling
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Tooth injury
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Cerebrospinal fluid leakage
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Somnolence
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Psychiatric disorders
Irritability
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
Other adverse events
| Measure |
No HGT-1410
n=7 participants at risk
Participants received no treatment (HGT-1410).
|
HGT-1410 45 mg Q2W
n=7 participants at risk
Participants received HGT-1410 45 mg intrathecally Q2W using surgically implanted IDDD or LP for 48 weeks.
|
HGT-1410 45 mg Q4W
n=7 participants at risk
Participants received HGT-1410 45 mg intrathecally Q4W using surgically implanted IDDD or LP for 48 weeks.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Leukopenia
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Musculoskeletal and connective tissue disorders
Rickets
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Vascular disorders
Diastolic hypertension
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Vascular disorders
Diastolic hypotension
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Vascular disorders
Haematoma
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Vascular disorders
Systolic hypertension
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Ear and labyrinth disorders
Motion sickness
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Ear and labyrinth disorders
Otorrhoea
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Abdominal pain
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Diarrhoea
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Enteritis
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 4 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Gastrointestinal disorder
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Salivary gland enlargement
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
71.4%
5/7 • Number of events 12 • From start of study drug administration up to Week 52
|
42.9%
3/7 • Number of events 4 • From start of study drug administration up to Week 52
|
|
General disorders
Adverse drug reaction
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
General disorders
Catheter site haemorrhage
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
General disorders
Developmental delay
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
General disorders
Impaired healing
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
General disorders
Implant site erythema
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
General disorders
Implant site extravasation
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 4 • From start of study drug administration up to Week 52
|
|
General disorders
Implant site swelling
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
General disorders
Local swelling
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
General disorders
Pyrexia
|
57.1%
4/7 • Number of events 10 • From start of study drug administration up to Week 52
|
57.1%
4/7 • Number of events 22 • From start of study drug administration up to Week 52
|
57.1%
4/7 • Number of events 5 • From start of study drug administration up to Week 52
|
|
Cardiac disorders
Bradycardia
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Cardiac disorders
Left ventricular hypertrophy
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Cardiac disorders
Sinus tachycardia
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Immune system disorders
Drug hypersensitivity
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Immune system disorders
Seasonal allergy
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Acute tonsillitis
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Bronchitis
|
14.3%
1/7 • Number of events 4 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Conjunctivitis
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Ear infection
|
28.6%
2/7 • Number of events 3 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
57.1%
4/7 • Number of events 6 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Gastroenteritis
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 3 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Gastrointestinal infection
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 2 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Hand-Foot-And-Mouth disease
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Implant site infection
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Influenza
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Meningitis aseptic
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Nasopharyngitis
|
42.9%
3/7 • Number of events 6 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 4 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Oral candidiasis
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Otitis media
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
42.9%
3/7 • Number of events 3 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Otitis media acute
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Pharyngitis
|
28.6%
2/7 • Number of events 4 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 3 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Respiratory syncytial virus infection
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Rhinitis
|
28.6%
2/7 • Number of events 3 • From start of study drug administration up to Week 52
|
42.9%
3/7 • Number of events 4 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Scarlet fever
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Sinusitis
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Stitch abscess
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Tinea pedis
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Tonsillitis bacterial
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Tonsillitis streptococcal
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Tooth abscess
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Upper respiratory tract infection
|
28.6%
2/7 • Number of events 6 • From start of study drug administration up to Week 52
|
71.4%
5/7 • Number of events 18 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Viral infection
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Infections and infestations
Viral upper respiratory tract infection
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Airway complication of anaesthesia
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Craniocerebral injury
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Fall
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
42.9%
3/7 • Number of events 3 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Incision site complication
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Limb injury
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Postoperative fever
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Procedural dizziness
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Procedural headache
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Procedural hypotension
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Procedural pain
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
42.9%
3/7 • Number of events 4 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Stab wound
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Tooth fracture
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Tooth injury
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Injury, poisoning and procedural complications
Wound complication
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Investigations
Blood alkaline phosphatase increased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Investigations
Blood pressure decreased
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Investigations
Blood pressure diastolic decreased
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 5 • From start of study drug administration up to Week 52
|
|
Investigations
Blood pressure diastolic increased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 3 • From start of study drug administration up to Week 52
|
|
Investigations
Blood pressure increased
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Investigations
Blood pressure systolic decreased
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 10 • From start of study drug administration up to Week 52
|
|
Investigations
Blood pressure systolic increased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Investigations
Body temperature decreased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 3 • From start of study drug administration up to Week 52
|
|
Investigations
Crystal urine present
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Investigations
Csf protein increased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Investigations
Csf test abnormal
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Investigations
Csf white blood cell count increased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
42.9%
3/7 • Number of events 3 • From start of study drug administration up to Week 52
|
|
Investigations
Haematocrit decreased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Investigations
Haemoglobin decreased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Investigations
Heart rate decreased
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Investigations
Heart rate increased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 8 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Cognitive disorder
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Investigations
Hepatic enzyme increased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Investigations
Oxygen saturation decreased
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 5 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Investigations
Platelet count decreased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Investigations
Protein total increased
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Investigations
Red blood cell count decreased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Investigations
Respiratory rate decreased
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Investigations
Urine uric acid
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Hepatobiliary disorders
Liver disorder
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Metabolism and nutrition disorders
Iron deficiency
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Metabolism and nutrition disorders
Vitamin d deficiency
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 2 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Aphasia
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Cerebral atrophy
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Cerebrospinal fluid leakage
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Demyelination
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Drooling
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Headache
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Motor dysfunction
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Nervous system disorders
Psychomotor hyperactivity
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 2 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Psychiatric disorders
Agitation
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Psychiatric disorders
Attention deficit/hyperactivity disorder
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Psychiatric disorders
Irritability
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Psychiatric disorders
Sleep disorder
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Psychiatric disorders
Sleep terror
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Reproductive system and breast disorders
Vulvovaginal erythema
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
28.6%
2/7 • Number of events 2 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Respiratory, thoracic and mediastinal disorders
Sinus disorder
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
|
Skin and subcutaneous tissue disorders
Mucocutaneous rash
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 2 • From start of study drug administration up to Week 52
|
|
Skin and subcutaneous tissue disorders
Pityriasis rosea
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
28.6%
2/7 • Number of events 2 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
|
Skin and subcutaneous tissue disorders
Rash macular
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
0.00%
0/7 • From start of study drug administration up to Week 52
|
14.3%
1/7 • Number of events 1 • From start of study drug administration up to Week 52
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER