Trial Outcomes & Findings for A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India (NCT NCT05058391)

NCT ID: NCT05058391

Last Updated: 2025-01-23

Results Overview

An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. A serious TEAE was defined as any untoward medical occurrence that at any dose results in: death; is life-threatening: requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; leads to a congenital anomaly/birth defect or is an important medical event. Number of participants with TEAEs, serious TEAEs, discontinuation due to TEAEs, and death are reported.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 5 participants
• Primary outcome timeframe: From start of the study drug administration up to Week 53
• Results posted on: 2025-01-23

Participant Flow

Participants took part in the study at investigative sites in India from 21 April 2022 to 18 April 2024.

Participants with a diagnosis of Hunters Syndrome were enrolled in this study to receive elaprase intravenous (IV) infusion.

Participant milestones

Measure	Elaprase 0.5 mg/kg Participants received a single dose of elaprase 0.5 milligrams per kilogram (mg/kg) IV infusion every week from Week 1 (Day 1) up to end of treatment (EOT) at Week 52.
Overall Study STARTED	5
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Elaprase 0.5 mg/kg Participants received a single dose of elaprase 0.5 milligrams per kilogram (mg/kg) IV infusion every week from Week 1 (Day 1) up to end of treatment (EOT) at Week 52.
Overall Study Adverse Event	1

Baseline Characteristics

A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India

Baseline characteristics by cohort

Measure	Elaprase 0.5 mg/kg n=5 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Age, Continuous	8.0 years STANDARD_DEVIATION 4.85 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	5 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: From start of the study drug administration up to Week 53

Population: The SAS included all participants who received at least one dose of study drug at any time during trial.

An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. A serious TEAE was defined as any untoward medical occurrence that at any dose results in: death; is life-threatening: requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; leads to a congenital anomaly/birth defect or is an important medical event. Number of participants with TEAEs, serious TEAEs, discontinuation due to TEAEs, and death are reported.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=5 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death TEAEs	5 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death Serious TEAEs	1 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death Discontinuation due to TEAEs	1 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death Death	0 Participants

PRIMARY outcome

Timeframe: From start of the study drug administration up to Week 53

Population: The SAS included all participants who received at least one dose of study drug at any time during trial.

An ADR was defined as a response to a drug which was noxious and unintended, and which occurred at doses normally used in humans for prophylaxis, diagnosis, or therapy of disease, or for the modification of physiological function.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=5 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Number of Participants With Adverse Drug Reactions (ADRs)	1 Participants

PRIMARY outcome

Timeframe: From start of the study drug administration up to Week 53

Population: The SAS included all participants who received at least one dose of study drug at any time during trial.

An IRR was defined as an AE that had been assessed as at least possibly related to treatment with elaprase and occurred during an infusion or up to 24 hours post-infusion.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=5 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Number of Participants With Infusion-related Reactions (IRRs)	1 Participants

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The Full Analysis Set (FAS) included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

FVC is the amount of air that can be forcibly exhaled from your lungs after taking the deepest breath possible, as measured by spirometry. FVC is a measure of respiratory function.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=2 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53 Week 27	-2.0 %FVC Standard Deviation 4.24
Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53 Week 53	1.0 %FVC Standard Deviation 2.83

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

6MWT is the distance covered over a time of 6 minutes and is a measure of physical functional capacity which is determined on a walking course.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=4 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53 Week 53	184.8 meters Standard Deviation 138.96
Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53 Week 27	24.3 meters Standard Deviation 24.31

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

Cardiac LVMI was measured by 2-dimensional (2D) echocardiography. Cardiac LVMI is the left ventricular mass (LVM) in grams indexed to body surface area (BSA), in square meters (m^2). Cardiac LVMI (in grams per square meter [g/m^2])=LVM divided by BSA.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=4 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in Cardiac Left Ventricular Mass Index (LVMI) at Weeks 27 and 53 Week 27	-12.0 g/m^2 Standard Deviation 20.91
Change From Baseline in Cardiac Left Ventricular Mass Index (LVMI) at Weeks 27 and 53 Week 53	-20.9 g/m^2 Standard Deviation 15.59

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

The LVEF was measured by 2D echocardiography and considered a sufficiently sensitive measure to evaluate any changes in cardiac function.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=4 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Weeks 27 and 53 Week 27	4.1 percentage of LVEF Standard Deviation 3.30
Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Weeks 27 and 53 Week 53	6.6 percentage of LVEF Standard Deviation 7.87

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

Liver volume was determined by Ultrasonography (USG).

Outcome measures

Measure	Elaprase 0.5 mg/kg n=4 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in Liver Volume at Weeks 27 and 53 Week 27	67.0 cubic centimeters (cm^3) Standard Deviation 245.27
Change From Baseline in Liver Volume at Weeks 27 and 53 Week 53	175.3 cubic centimeters (cm^3) Standard Deviation 255.56

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

Spleen volume was determined by USG.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=4 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in Spleen Volume at Weeks 27 and 53 Week 27	48.3 cm^3 Standard Deviation 129.60
Change From Baseline in Spleen Volume at Weeks 27 and 53 Week 53	123.7 cm^3 Standard Deviation 207.15

SECONDARY outcome

Timeframe: Baseline, Weeks 14, 27, 40, and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Number analyzed is the number of participants with data available for analysis for the specified category.

Normalized uGAG was analyzed using urine testing. The uGAG levels were normalized to urine creatinine and were reported as microgram glycosaminoglycan (GAG) per milligram creatinine (μg GAG/mg creatinine).

Outcome measures

Measure	Elaprase 0.5 mg/kg n=5 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53 Week 14	33.3 μg GAG/mg creatinine Standard Deviation 112.91
Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53 Week 27	-28.9 μg GAG/mg creatinine Standard Deviation 31.92
Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53 Week 40	123.4 μg GAG/mg creatinine Standard Deviation 222.41
Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53 Week 53	8.6 μg GAG/mg creatinine Standard Deviation 29.50

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

Passive joint mobility is defined as the range of motion of the shoulder, elbow, wrist, hip, knee, and ankle joints, as assessed by one expert physician using universal goniometry method. Global JROM (% of normal range of motion) is the average of 11 ratios multiplied by 100. Ratios are left/right means of passive range of motion in shoulder (flexion/extension, abduction, internal/external rotation), elbow (flexion/extension), wrist (flexion/extension), index finger (flexion/extension [combined metacarpophalangeal joint, proximal interphalangeal joint, distal interphalangeal joint motion]), hip (flexion/extension, abduction, internal/external rotation), knee (flexion/extension), and ankle (dorsiflexion) divided by the normal range (American Academy of Orthopedic Surgeons and American Medical Association).

Outcome measures

Measure	Elaprase 0.5 mg/kg n=2 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in Global Joint Range of Motion (JROM) Score at Weeks 27 and 53 Week 27	-8.0 percentage of normal range of motion Standard Deviation 7.14
Change From Baseline in Global Joint Range of Motion (JROM) Score at Weeks 27 and 53 Week 53	-14.0 percentage of normal range of motion Standard Deviation 0.00

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

Change from baseline in height (centimeters [cm]) was assessed in participants less than (<)18 years of age.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=4 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in Anthropometric Parameter: Height at Weeks 27 and 53 Week 27	3.0 cm Standard Deviation 2.34
Change From Baseline in Anthropometric Parameter: Height at Weeks 27 and 53 Week 53	3.7 cm Standard Deviation 3.08

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

Change from baseline in weight (kilograms[kg]) was assessed in all participants.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=4 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in Anthropometric Parameter: Weight at Weeks 27 and 53 Week 27	0.3 kg Standard Deviation 1.50
Change From Baseline in Anthropometric Parameter: Weight at Weeks 27 and 53 Week 53	1.6 kg Standard Deviation 1.11

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

HS-FOCUS is developed as disease-specific measure of the impact of Hunter syndrome on HRQL. The HS-FOCUS is designed to gather information on the participant's daily life and wellbeing, satisfaction with treatment, and hospitalizations, as well as on how Hunter syndrome impacts participant's general quality of life. HS-FOCUS includes 2 validated components: a parent version and a participant self-reported version for those over age 12 years. The HS-FOCUS (shortened version) questionnaire has 5 function domains (walking/standing, grip/reach, school/work, activities, and breathing). Items are scored using a response scale from 0 to 3, with 0 signifying an ability to complete the activity-related functions 'without any difficulty' and 3 denoting highest disability. Higher scores on each domain indicate greater disability.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=4 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores Walking/ Standing: Week 27	-0.3 score on a scale Standard Deviation 0.22
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores Walking/ Standing: Week 53	-0.5 score on a scale Standard Deviation 0.41
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores Grip/Reach: Week 27	-0.1 score on a scale Standard Deviation 0.49
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores Grip/Reach: Week 53	-0.1 score on a scale Standard Deviation 0.52
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores School/ Work: Week 27	0.3 score on a scale Standard Deviation 0.58
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores School/ Work: Week 53	-1.0 score on a scale Standard Deviation 1.73
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores Activities: Week 27	-0.1 score on a scale Standard Deviation 0.25
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores Activities: Week 53	-0.9 score on a scale Standard Deviation 1.03
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores Breathing: Week 27	-0.7 score on a scale Standard Deviation 0.80
Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores Breathing: Week 53	-0.8 score on a scale Standard Deviation 1.19

SECONDARY outcome

Timeframe: Baseline, Weeks 27 and 53

Population: The FAS included all enrolled participants who received at least one dose of study drug and with at least one post-baseline evaluation of any of the efficacy endpoints. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category.

The CHAQ was initially developed for assessing juvenile idiopathic arthritis, from the perspective of the parent or participant, and has been previously applied to other chronic disabling conditions such as Hunter syndrome. It is a 30-item instrument that measures functional capacity and independence in activities of daily life across eight domains: dressing and grooming, arising, eating, walking, reach, grip, hygiene, and activities. For each domain, there is a 4-level difficulty scale that is scored from 0 to 3, with 0 corresponding to 'without any difficulty' and 3 to 'unable to do'. Higher scores on each domain indicate greater disability.

Outcome measures

Measure	Elaprase 0.5 mg/kg n=4 Participants Participants received a single dose of elaprase 0.5 mg/kg IV infusion every week from Week 1 (Day 1) up to EOT at Week 52.
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Dressing/ Grooming: Week 27	0.0 score on a scale Standard Deviation 2.16
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Dressing/ Grooming: Week 53	-0.5 score on a scale Standard Deviation 2.65
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Arising: Week 27	-0.3 score on a scale Standard Deviation 0.50
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Arising: Week 53	-0.3 score on a scale Standard Deviation 0.50
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Eating: Week 27	-0.3 score on a scale Standard Deviation 0.50
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Eating: Week 53	-0.3 score on a scale Standard Deviation 0.50
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Walking: Week 27	-0.3 score on a scale Standard Deviation 0.50
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Walking: Week 53	-0.3 score on a scale Standard Deviation 0.50
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Hygiene: Week 27	-0.5 score on a scale Standard Deviation 0.58
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Hygiene: Week 53	-0.8 score on a scale Standard Deviation 0.96
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Reach: Week 27	-1.0 score on a scale Standard Deviation 1.41
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Reach: Week 53	-0.8 score on a scale Standard Deviation 0.96
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Grip: Week 27	-0.5 score on a scale Standard Deviation 1.00
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Grip: Week 53	-1.3 score on a scale Standard Deviation 1.50
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Activities: Week 27	0.0 score on a scale Standard Deviation 0.00
Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores Activities: Week 53	-0.5 score on a scale Standard Deviation 1.00

Adverse Events

Elaprase 0.5 mg/kg

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Measure	Elaprase 0.5 mg/kg n=5 participants at risk Participants received a single dose of elaprase 0.5 milligrams per kilogram (mg/kg) IV infusion every week from Week 1 (Day 1) up to end of treatment (EOT) at Week 52.
Infections and infestations Lower Respiratory Tract Infection	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.

Other adverse events

Measure	Elaprase 0.5 mg/kg n=5 participants at risk Participants received a single dose of elaprase 0.5 milligrams per kilogram (mg/kg) IV infusion every week from Week 1 (Day 1) up to end of treatment (EOT) at Week 52.
Ear and labyrinth disorders Ear Pain	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Gastrointestinal disorders Abdominal Pain Upper	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Gastrointestinal disorders Diarrhoea	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Gastrointestinal disorders Mouth Ulceration	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Gastrointestinal disorders Vomiting	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
General disorders Hernia Pain	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
General disorders Infusion Site Extravasation	20.0% 1/5 • Number of events 3 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
General disorders Pyrexia	60.0% 3/5 • Number of events 7 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
General disorders Swelling	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Infections and infestations Conjunctivitis	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Infections and infestations Ear Infection	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Infections and infestations Nasopharyngitis	80.0% 4/5 • Number of events 8 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Injury, poisoning and procedural complications Limb Injury	20.0% 1/5 • Number of events 2 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Respiratory, thoracic and mediastinal disorders Asthma	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Respiratory, thoracic and mediastinal disorders Cough	80.0% 4/5 • Number of events 6 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Respiratory, thoracic and mediastinal disorders Sneezing	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Skin and subcutaneous tissue disorders Dermatitis Allergic	20.0% 1/5 • Number of events 1 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.
Skin and subcutaneous tissue disorders Urticaria	20.0% 1/5 • Number of events 4 • From start of the study drug administration up to Week 53 The SAS included all participants who received at least one dose of study drug at any time during trial.

Additional Information

Study Director

Takeda

Phone: +1-877-825-3327

Email: TrialDisclosures@takeda.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place