Trial · NCT05058391
A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
NCT05058391, Phase 4, Completed, Takeda; results first posted 2025-01-23
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India. Trials, record NCT05058391. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05058391
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
- NCT number
- NCT05058391
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 5 participants, actual
- Lead sponsor
- Takeda
- Conditions
- Hunter Syndrome
- Interventions
- Elaprase
- First posted
- 2021-09-27
- Last updated
- 2025-01-23
- Other registries' IDs
- eudract:2022-004193-39
- Results first posted
- 2025-01-23
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death | 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 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. |
| Primary | Number of Participants With Adverse Drug Reactions (ADRs) | 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. |
| Primary | Number of Participants With Infusion-related Reactions (IRRs) | 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. |
| Secondary | Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53 | 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. |
| Secondary | Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53 | 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. |
| Secondary | Change From Baseline in Cardiac Left Ventricular Mass Index (LVMI) at Weeks 27 and 53 | 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. |
| Secondary | Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Weeks 27 and 53 | 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. |
| Secondary | Change From Baseline in Liver Volume at Weeks 27 and 53 | Baseline, Weeks 27 and 53 | Liver volume was determined by Ultrasonography (USG). |
| Secondary | Change From Baseline in Spleen Volume at Weeks 27 and 53 | Baseline, Weeks 27 and 53 | Spleen volume was determined by USG. |
| Secondary | Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53 | 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). |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death | 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. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Adverse Drug Reactions (ADRs) | 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. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Infusion-related Reactions (IRRs) | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53 | Baseline, Weeks 27 and 53 | 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 | MEAN |
| SECONDARY | Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53 | Baseline, Weeks 27 and 53 | 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. | meters | MEAN |
| SECONDARY | Change From Baseline in Cardiac Left Ventricular Mass Index (LVMI) at Weeks 27 and 53 | Baseline, Weeks 27 and 53 | 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. | g/m^2 | MEAN |
| SECONDARY | Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Weeks 27 and 53 | Baseline, Weeks 27 and 53 | 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. | percentage of LVEF | MEAN |
| SECONDARY | Change From Baseline in Liver Volume at Weeks 27 and 53 | Baseline, Weeks 27 and 53 | 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. | cubic centimeters (cm^3) | MEAN |
| SECONDARY | Change From Baseline in Spleen Volume at Weeks 27 and 53 | Baseline, Weeks 27 and 53 | 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. | cm^3 | MEAN |
| SECONDARY | Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53 | Baseline, Weeks 14, 27, 40, and 53 | 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. | μg GAG/mg creatinine | MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group54
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death | Elaprase 0.5 mg/kg | TEAEs | 5 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death | Elaprase 0.5 mg/kg | Serious TEAEs | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death | Elaprase 0.5 mg/kg | Discontinuation due to TEAEs | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death | Elaprase 0.5 mg/kg | Death | 0 | Participants | |
| Number of Participants With Adverse Drug Reactions (ADRs) | Elaprase 0.5 mg/kg | 1 | Participants | ||
| Number of Participants With Infusion-related Reactions (IRRs) | Elaprase 0.5 mg/kg | 1 | Participants | ||
| Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53 | Elaprase 0.5 mg/kg | Week 27 | -2.0 | Standard Deviation 4.24 | %FVC |
| Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53 | Elaprase 0.5 mg/kg | Week 53 | 1.0 | Standard Deviation 2.83 | %FVC |
| Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53 | Elaprase 0.5 mg/kg | Week 27 | 24.3 | Standard Deviation 24.31 | meters |
| Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53 | Elaprase 0.5 mg/kg | Week 53 | 184.8 | Standard Deviation 138.96 | meters |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Elaprase 0.5 mg/kg | 5 | |
| Overall Study | COMPLETED | Elaprase 0.5 mg/kg | 4 | |
| Overall Study | NOT COMPLETED | Elaprase 0.5 mg/kg | 1 |
Source ClinicalTrials.gov
Adverse events19
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Ear Pain | Ear and labyrinth disorders | Elaprase 0.5 mg/kg | 1 | 5 | 1 |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Elaprase 0.5 mg/kg | 1 | 5 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Elaprase 0.5 mg/kg | 1 | 5 | 1 |
| other | Mouth Ulceration | Gastrointestinal disorders | Elaprase 0.5 mg/kg | 1 | 5 | 1 |
| other | Vomiting | Gastrointestinal disorders | Elaprase 0.5 mg/kg | 1 | 5 | 1 |
| other | Hernia Pain | General disorders | Elaprase 0.5 mg/kg | 1 | 5 | 1 |
| other | Infusion Site Extravasation | General disorders | Elaprase 0.5 mg/kg | 1 | 5 | 3 |
| other | Pyrexia | General disorders | Elaprase 0.5 mg/kg | 3 | 5 | 7 |
| other | Swelling | General disorders | Elaprase 0.5 mg/kg | 1 | 5 | 1 |
| other | Conjunctivitis | Infections and infestations | Elaprase 0.5 mg/kg | 1 | 5 | 1 |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Elaprase 0.5 mg/kg | 0 | 5 |
| Total, other adverse events | Elaprase 0.5 mg/kg | 5 | 5 |
| Total, serious adverse events | Elaprase 0.5 mg/kg | 1 | 5 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05058391
GET https://conductscience.org/api/trials/v1/NCT05058391/results?page=2