Trial Outcomes & Findings for Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy (NCT NCT00607386)
NCT ID: NCT00607386
Last Updated: 2021-06-08
Results Overview
An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. 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; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Number of participants with AEs occurred after start of study treatment until 30 days after the last infusion of idursulfase, were reported.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
28 participants
Primary outcome timeframe
From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
Results posted on
2021-06-08
Participant Flow
Participant milestones
| Measure |
Idursulfase
Open-label treatment with idursulfase
|
|---|---|
|
Overall Study
STARTED
|
28
|
|
Overall Study
COMPLETED
|
27
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Idursulfase
Open-label treatment with idursulfase
|
|---|---|
|
Overall Study
Physician Decision
|
1
|
Baseline Characteristics
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
Baseline characteristics by cohort
| Measure |
Idursulfase
n=28 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Age, Continuous
|
4.0 years
STANDARD_DEVIATION 1.62 • n=93 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=93 Participants
|
|
Baseline Normalized Urinary Glycosaminoglycan (GAG) Level
|
738.3 microgram per milligram creatinine
STANDARD_DEVIATION 165.21 • n=93 Participants
|
PRIMARY outcome
Timeframe: From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeksPopulation: Safety population was defined as all enrolled participants who received at least one study dose (or any portion of a dose) of idursulfase.
An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. 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; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Number of participants with AEs occurred after start of study treatment until 30 days after the last infusion of idursulfase, were reported.
Outcome measures
| Measure |
Idursulfase
n=28 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Safety Evaluation
Experienced at least one adverse event (AE)
|
28 participants
|
|
Safety Evaluation
Deaths
|
0 participants
|
|
Safety Evaluation
Discontinued due to an AE
|
0 participants
|
|
Safety Evaluation
Experienced at least one drug-related AE
|
16 participants
|
|
Safety Evaluation
Experienced at least one serious AE (SAE)
|
13 participants
|
|
Safety Evaluation
Experienced at least one severe AE
|
2 participants
|
|
Safety Evaluation
Experienced at least one infusion-related AE
|
16 participants
|
SECONDARY outcome
Timeframe: Baseline, Weeks 18, 36 and 53Population: Safety population. In the categories listed below, 'N' signifies the number of participants evaluable for the timepoint.
Analysis of urinary GAG levels was performed at baseline, Week 18, Week 36, and Week 53 as an assessment of the pharmacodynamic effects of Elaprase (idursulfase).
Outcome measures
| Measure |
Idursulfase
n=28 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels
Baseline (N=28)
|
738.3 microgram/milligram creatinine
Standard Deviation 165.21
|
|
Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels
Change at Week 18 (N=27)
|
-368.0 microgram/milligram creatinine
Standard Deviation 165.44
|
|
Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels
Change at Week 36 (N=27)
|
-400.3 microgram/milligram creatinine
Standard Deviation 180.27
|
|
Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels
Change at Week 53 (N=27)
|
-402.4 microgram/milligram creatinine
Standard Deviation 162.13
|
SECONDARY outcome
Timeframe: Weeks 1 and 27Population: Pharmacokinetic (PK) population was defined as all enrolled participants who had at least one serum concentration measurement available. In the categories listed below, "N" signifies the number of participants evaluable for the timepoint.
Outcome measures
| Measure |
Idursulfase
n=27 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax)
Week 1 (N=27)
|
1333 nanogram per milliliter
Standard Deviation 817
|
|
Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax)
Week 27 (N=19)
|
1032 nanogram per milliliter
Standard Deviation 590
|
SECONDARY outcome
Timeframe: Weeks 1 and 27Population: PK population. In the categories listed below, "N" signifies the number of participants evaluable for the timepoint.
Outcome measures
| Measure |
Idursulfase
n=27 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Single- and Repeat-Dose Pharmacokinetics - Time of Maximum Observed Serum Concentration (Tmax)
Week 1 (N=27)
|
163 minutes
Standard Deviation 28
|
|
Single- and Repeat-Dose Pharmacokinetics - Time of Maximum Observed Serum Concentration (Tmax)
Week 27 (N=19)
|
167 minutes
Standard Deviation 32
|
SECONDARY outcome
Timeframe: Weeks 1 and 27Population: PK population. In the categories listed below, "N" signifies the number of participants evaluable for the timepoint.
Outcome measures
| Measure |
Idursulfase
n=27 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to the Final Time Point With a Concentration of at Least Lower Limit of Quantitation (AUClast)
Week 1 (N=27)
|
196526 minute*nanogram per milliliter
Standard Deviation 71779
|
|
Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to the Final Time Point With a Concentration of at Least Lower Limit of Quantitation (AUClast)
Week 27 (N=19)
|
174869 minute*nanogram per milliliter
Standard Deviation 109118
|
SECONDARY outcome
Timeframe: Weeks 1 and 27Population: PK population with evaluable participants for this endpoint. In the categories listed below, "N" signifies the number of participants evaluable for the timepoint.
Outcome measures
| Measure |
Idursulfase
n=26 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to Infinity (AUCinf)
Week 1 (N=26)
|
224343 minute*nanogram per milliliter
Standard Deviation 76944
|
|
Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to Infinity (AUCinf)
Week 27 (N=18)
|
201130 minute*nanogram per milliliter
Standard Deviation 117575
|
SECONDARY outcome
Timeframe: Weeks 1 and 27Population: PK population with evaluable participants for this endpoint. In the categories listed below, "N" signifies the number of participants evaluable for the timepoint.
t1/2 refers to the elimination of the drug. It is the time taken for the blood plasma concentration to reach half the concentration in the terminal phase of elimination. It is expressed in minutes and derived from the terminal slope of the concentration versus time curve.
Outcome measures
| Measure |
Idursulfase
n=26 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Single- and Repeat-Dose Pharmacokinetics - Elimination Half-Life (t1/2)
Week 1 (N=26)
|
160 minutes
Standard Deviation 69
|
|
Single- and Repeat-Dose Pharmacokinetics - Elimination Half-Life (t1/2)
Week 27 (N=18)
|
109 minutes
Standard Deviation 43
|
SECONDARY outcome
Timeframe: Weeks 1 and 27Population: PK population with evaluable participants for this endpoint. In the categories listed below, "N" signifies the number of participants evaluable for the timepoint.
MRTinf is an average duration of the drug in the body from time zero to infinity, and is expressed in minutes.
Outcome measures
| Measure |
Idursulfase
n=26 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Single- and Repeat-Dose Pharmacokinetics - Mean Residence Time From Time 0 to Infinity (MRTinf)
Week 1 (N=26)
|
153 minutes
Standard Deviation 96
|
|
Single- and Repeat-Dose Pharmacokinetics - Mean Residence Time From Time 0 to Infinity (MRTinf)
Week 27 (N=18)
|
127 minutes
Standard Deviation 23
|
SECONDARY outcome
Timeframe: Weeks 1 and 27Population: PK population with evaluable participants for this endpoint. In the categories listed below, "N" signifies the number of participants evaluable for the timepoint.
Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.
Outcome measures
| Measure |
Idursulfase
n=26 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Single- and Repeat-Dose Pharmacokinetics - Clearance (CL)
Week 1 (N=26)
|
2.4 milliliter/minute/kilogram
Standard Deviation 0.7
|
|
Single- and Repeat-Dose Pharmacokinetics - Clearance (CL)
Week 27 (N=18)
|
4.7 milliliter/minute/kilogram
Standard Deviation 5.0
|
SECONDARY outcome
Timeframe: Weeks 1 and 27Population: PK population with evaluable participants for this endpoint. In the categories listed below, "N" signifies the number of participants evaluable for the timepoint.
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. Vss is the apparent volume of distribution at steadystate.
Outcome measures
| Measure |
Idursulfase
n=26 Participants
Open-label treatment with idursulfase
|
|---|---|
|
Single- and Repeat-Dose Pharmacokinetics - Volume of Distribution at Steady State (Vss)
Week 1 (N=26)
|
394 milliliter per kilogram
Standard Deviation 423
|
|
Single- and Repeat-Dose Pharmacokinetics - Volume of Distribution at Steady State (Vss)
Week 27 (N=18)
|
551 milliliter per kilogram
Standard Deviation 528
|
Adverse Events
Idursulfase
Serious events: 13 serious events
Other events: 28 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Idursulfase
n=28 participants at risk
Open-label treatment with idursulfase
|
|---|---|
|
Infections and infestations
Bronchopneumonia
|
10.7%
3/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Otitis media
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Pneumonia
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Ear infection
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Gastrointestinal infection
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Respiratory tract infection
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Viral pharyngitis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Pyrexia
|
10.7%
3/28 • Number of events 6 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Catheter site haematoma
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Irritability
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Oedema peripheral
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Skin hypertrophy
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
3.6%
1/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Blood and lymphatic system disorders
Microcytic anaemia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Food poisoning
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscle contracture
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Nervous system disorders
Atonic seizures
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Vascular disorders
Haematoma
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
Other adverse events
| Measure |
Idursulfase
n=28 participants at risk
Open-label treatment with idursulfase
|
|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
64.3%
18/28 • Number of events 42 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Respiratory tract infection
|
42.9%
12/28 • Number of events 36 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Rhinitis
|
39.3%
11/28 • Number of events 20 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Viral upper respiratory tract infection
|
32.1%
9/28 • Number of events 17 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Otitis media acute
|
25.0%
7/28 • Number of events 10 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Pharyngitis
|
25.0%
7/28 • Number of events 18 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Gastroenteritis
|
21.4%
6/28 • Number of events 7 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Upper respiratory tract infection bacterial
|
21.4%
6/28 • Number of events 13 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Pneumonia
|
10.7%
3/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Sinusitis
|
17.9%
5/28 • Number of events 6 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Bronchitis
|
14.3%
4/28 • Number of events 4 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Bronchopneumonia
|
10.7%
3/28 • Number of events 4 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Otitis media
|
7.1%
2/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Gastrointestinal infection
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Otitis externa
|
10.7%
3/28 • Number of events 5 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Viral infection
|
10.7%
3/28 • Number of events 4 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Viral pharyngitis
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Ear infection
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Gastroenteritis viral
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Herpes virus infection
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Influenza
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Laryngitis
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Nasopharyngitis
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Tonsillitis
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Abscess limb
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Body tinea
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Candidiasis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Catheter site infection
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Herpes zoster
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Lice infestation
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Oral candidiasis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Otitis media chronic
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Rash pustular
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Tinea versicolour
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Tonsillitis bacterial
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Varicella
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Infections and infestations
Viral diarrhoea
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Pyrexia
|
89.3%
25/28 • Number of events 59 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Fatigue
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Catheter site haematoma
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Catheter site haemorrhage
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Chest pain
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Irritability
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Malaise
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
General disorders
Oedema peripheral
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
57.1%
16/28 • Number of events 45 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
21.4%
6/28 • Number of events 9 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Allergic cough
|
10.7%
3/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
10.7%
3/28 • Number of events 4 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnea syndrome
|
10.7%
3/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
7.1%
2/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Choking
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Lung infiltration
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
21.4%
6/28 • Number of events 11 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
14.3%
4/28 • Number of events 7 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
7.1%
2/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Eczema
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Erythema
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Heat rash
|
7.1%
2/28 • Number of events 4 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis atopic
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Intertrigo
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysaesthesia syndrome
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Rash generalised
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Skin hypertrophy
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Skin and subcutaneous tissue disorders
Urticaria papular
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Vomiting
|
35.7%
10/28 • Number of events 12 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
25.0%
7/28 • Number of events 35 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Constipation
|
7.1%
2/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Dental caries
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Toothache
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Abdominal pain
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Anal fissure
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Faeces hard
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Food poisoning
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Gingivitis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Nausea
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Pruritus ani
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Stomatitis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Gastrointestinal disorders
Umbilical hernia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Ear and labyrinth disorders
Deafness
|
14.3%
4/28 • Number of events 4 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Ear and labyrinth disorders
Hypoacusis
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Ear and labyrinth disorders
Cerumen impaction
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Ear and labyrinth disorders
Conductive deafness
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Ear and labyrinth disorders
Ear pain
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Ear and labyrinth disorders
Hearing impaired
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Ear and labyrinth disorders
Otorrhoea
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Injury, poisoning and procedural complications
Burns second degree
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Injury, poisoning and procedural complications
Contusion
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Injury, poisoning and procedural complications
Head injury
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Injury, poisoning and procedural complications
Limb injury
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Injury, poisoning and procedural complications
Mouth injury
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Injury, poisoning and procedural complications
Procedural pain
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Injury, poisoning and procedural complications
Sunburn
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Psychiatric disorders
Agitation
|
14.3%
4/28 • Number of events 5 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Psychiatric disorders
Sleep disorder
|
10.7%
3/28 • Number of events 3 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Psychiatric disorders
Bipolar disorder
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Psychiatric disorders
Crying
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Psychiatric disorders
Insomnia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Psychiatric disorders
Learning disorder
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Cardiac disorders
Left ventricular hypertrophy
|
14.3%
4/28 • Number of events 4 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Cardiac disorders
Aortic valve incompetence
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Cardiac disorders
Cardiomyopathy
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Cardiac disorders
Cyanosis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Cardiac disorders
Right ventricular hypertrophy
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Cardiac disorders
Sinus bradycardia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Cardiac disorders
Ventricular extrasystoles
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Investigations
Hepatic enzyme abnormal
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Investigations
Alanine aminotransferase increased
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Investigations
Cardiac murmur
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Investigations
Electrocardiogram repolarisation abnormality
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Investigations
Gamma-glutamyltransferase increased
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Investigations
Lymphocyte morphology abnormal
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Investigations
Monocyte count decreased
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Nervous system disorders
Tremor
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Nervous system disorders
Atonic seizures
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Nervous system disorders
Epilepsy
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Nervous system disorders
Headache
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Nervous system disorders
Hypoaesthesia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Nervous system disorders
Somnolence
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Vascular disorders
Haematoma
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Vascular disorders
Haemorrhage
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Vascular disorders
Hyperaemia
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Vascular disorders
Hypertension
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Blood and lymphatic system disorders
Eosinophilia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Blood and lymphatic system disorders
Splenomegaly
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscle contracture
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
3.6%
1/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Musculoskeletal and connective tissue disorders
Lordosis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Metabolism and nutrition disorders
Obesity
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Eye disorders
Hypermetropia
|
7.1%
2/28 • Number of events 2 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Eye disorders
Conjunctivitis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Congenital, familial and genetic disorders
Hip dysplasia
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
|
Renal and urinary disorders
Enuresis
|
3.6%
1/28 • Number of events 1 • From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER