Eligibility, any sex, with Hunter Syndrome. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and DeathPrimary· From start of the study drug administration up to Week 53
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 hospitalizat
TEAEs
Group
Value
95% CI
Elaprase 0.5 mg/kg
5
Serious TEAEs
Group
Value
95% CI
Elaprase 0.5 mg/kg
1
Discontinuation due to TEAEs
Group
Value
95% CI
Elaprase 0.5 mg/kg
1
Death
Group
Value
95% CI
Elaprase 0.5 mg/kg
0
Number of Participants With Adverse Drug Reactions (ADRs)Primary· From start of the study drug administration up to Week 53
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.
Group
Value
95% CI
Elaprase 0.5 mg/kg
1
Number of Participants With Infusion-related Reactions (IRRs)Primary· From start of the study drug administration up to Week 53
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.
Group
Value
95% CI
Elaprase 0.5 mg/kg
1
Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53Secondary· Baseline, Weeks 27 and 53
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.
Week 27
Group
Value
95% CI
Elaprase 0.5 mg/kg
-2.0
± 4.24
Week 53
Group
Value
95% CI
Elaprase 0.5 mg/kg
1.0
± 2.83
Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53Secondary· Baseline, Weeks 27 and 53
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.
Week 27
Group
Value
95% CI
Elaprase 0.5 mg/kg
24.3
± 24.31
Week 53
Group
Value
95% CI
Elaprase 0.5 mg/kg
184.8
± 138.96
Change From Baseline in Cardiac Left Ventricular Mass Index (LVMI) at Weeks 27 and 53Secondary· Baseline, Weeks 27 and 53
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.
Week 27
Group
Value
95% CI
Elaprase 0.5 mg/kg
-12.0
± 20.91
Week 53
Group
Value
95% CI
Elaprase 0.5 mg/kg
-20.9
± 15.59
Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Weeks 27 and 53Secondary· Baseline, Weeks 27 and 53
The LVEF was measured by 2D echocardiography and considered a sufficiently sensitive measure to evaluate any changes in cardiac function.
Week 27
Group
Value
95% CI
Elaprase 0.5 mg/kg
4.1
± 3.30
Week 53
Group
Value
95% CI
Elaprase 0.5 mg/kg
6.6
± 7.87
Change From Baseline in Liver Volume at Weeks 27 and 53Secondary· Baseline, Weeks 27 and 53
Liver volume was determined by Ultrasonography (USG).
Week 27
Group
Value
95% CI
Elaprase 0.5 mg/kg
67.0
± 245.27
Week 53
Group
Value
95% CI
Elaprase 0.5 mg/kg
175.3
± 255.56
Change From Baseline in Spleen Volume at Weeks 27 and 53Secondary· Baseline, Weeks 27 and 53
Spleen volume was determined by USG.
Week 27
Group
Value
95% CI
Elaprase 0.5 mg/kg
48.3
± 129.60
Week 53
Group
Value
95% CI
Elaprase 0.5 mg/kg
123.7
± 207.15
Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53Secondary· Baseline, Weeks 14, 27, 40, and 53
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).
Week 14
Group
Value
95% CI
Elaprase 0.5 mg/kg
33.3
± 112.91
Week 27
Group
Value
95% CI
Elaprase 0.5 mg/kg
-28.9
± 31.92
Week 40
Group
Value
95% CI
Elaprase 0.5 mg/kg
123.4
± 222.41
Week 53
Group
Value
95% CI
Elaprase 0.5 mg/kg
8.6
± 29.50
Change From Baseline in Global Joint Range of Motion (JROM) Score at Weeks 27 and 53Secondary· Baseline, Weeks 27 and 53
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 \[combined metacarpophalangeal joint, proximal interphalangeal joint, distal interphalangeal joint motion\]),
Week 27
Group
Value
95% CI
Elaprase 0.5 mg/kg
-8.0
± 7.14
Week 53
Group
Value
95% CI
Elaprase 0.5 mg/kg
-14.0
± 0.00
Change From Baseline in Anthropometric Parameter: Height at Weeks 27 and 53Secondary· Baseline, Weeks 27 and 53
Change from baseline in height (centimeters \[cm\]) was assessed in participants less than (\<)18 years of age.
Week 27
Group
Value
95% CI
Elaprase 0.5 mg/kg
3.0
± 2.34
Week 53
Group
Value
95% CI
Elaprase 0.5 mg/kg
3.7
± 3.08
Adverse events — posted to ClinicalTrials.gov
Time frame: From start of the study drug administration up to Week 53.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The main aim of this study is to learn more about the safety profile of Elaprase in Indian children and adults with hunter syndrome.
Participants will receive Elaprase once per week over a 3-hour period which can be reduced to 1 hour as determined by the study doctor. Participants will need to visit the clinic weekly during the duration of the study.
Publications & conference data
No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.
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Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Takeda
Last refreshed: 23 January 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05058391.