Trial · NCT02412787
Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
NCT02412787, Phase 2, Phase 3, Completed, Shire; results first posted 2025-06-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094. Trials, record NCT02412787. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02412787
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
- NCT number
- NCT02412787
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 56 participants, actual
- Lead sponsor
- Shire
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase-IT, Elaprase
- First posted
- 2015-04-09
- Last updated
- 2025-06-19
- Other registries' IDs
- eudract:2014-004143-13
- Results first posted
- 2025-06-19
Source ClinicalTrials.gov
Registered outcomes24
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AEs) | Up to 9 years | An AE is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product-related. |
| Primary | Number of Participants With Clinically Significant Changes in Vital Signs | Up to 9 years | Participants were assessed for clinically significant changes in vital signs like injection (IT) vital signs and regular vital signs (temperature, pulse, blood pressure \[systolic and diastolic\], oxygen saturation, and respiration rate). |
| Primary | Number of Participants With Clinically Significant Changes in Laboratory Parameters | Up to 9 years | Participants were assessed for clinically significant changes in laboratory parameters such as chemistry, hematology, urinalysis and cerebrospinal fluid (CSF) values. |
| Primary | Number of Participants With Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Findings | Up to 9 years | Participants were assessed for clinically significant changes in 12-lead ECG findings (such as heart rate, PR interval, QRS interval, QT interval, and the corrected QT interval). |
| Primary | Maximum Observed Serum Concentration (Cmax) of Idursulfase | Predose and at 30 min, 60 min, 120 min, 4 hours, 6 hours, 8 hours, 12 hours, 24 hours, 30 hours and 36 hours post-dose at Week 100 in relation to HGT-HIT-094 (Week 48 of this study) | Idursulfase concentrations in serum were determined using a validated Enzyme -Linked Immunosorbent Assay (ELISA) method. Concentration for Cmax is presented in this endpoint. |
| Primary | Percent Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in CSF at Month 67 | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. | The percent change in concentration of GAG in CSF was assessed. GAGs are long sugar chains that are like building blocks for the body's tissues, especially connective tissues like cartilage, skin, and tendons, and play a crucial role in cell signaling and interactions. |
| Primary | Percent Change From Baseline in the Concentration of GAG in Urine at Month 67 | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. | The percent change in concentration of GAG in urine was assessed. GAGs are long sugar chains that are like building blocks for the body's tissues, especially connective tissues like cartilage, skin, and tendons, and play a crucial role in cell signaling and interactions. |
| Primary | Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in CSF | Up to 9 years | |
| Primary | Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in Serum | Up to 9 years | |
| Secondary | Change From Baseline in Differential Ability Scales, Second Edition (DAS-II) Standard Cluster Scores at Month 67 | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. | DAS-II was used to assess all participants of age 2 years, 6 months or older. DAS-II comprises 2 overlapping batteries. Early Years battery (EYB) was designed for children ages 2 years, 6 months through 6 years, 11 months. The School Age Battery (SAB) was designed for children ages 7 years, 0 months through 17 years, 11months. These batteries are fully co-normed for ages 5 years, 0 months, through 8 years, 11 months. The cluster areas include general conceptual ability (GCA), verbal, nonverbal, spatial, and special nonverbal composite (SNC). The cluster area score represents a score (mean=100 and standard deviation=15) on which higher scores indicate higher level of cognitive ability. |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 registered outcomesOutcomes24
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 Adverse Events (AEs) | Up to 9 years | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Changes in Vital Signs | Up to 9 years | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Changes in Laboratory Parameters | Up to 9 years | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Findings | Up to 9 years | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Maximum Observed Serum Concentration (Cmax) of Idursulfase | Predose and at 30 min, 60 min, 120 min, 4 hours, 6 hours, 8 hours, 12 hours, 24 hours, 30 hours and 36 hours post-dose at Week 100 in relation to HGT-HIT-094 (Week 48 of this study) | The Pharmacokinetic Population included all the participants in Study SHP609-302 who received study drug and participated in the scheduled pharmacokinetic studies, and for whom at least 1 post-dose pharmacokinetic blood sample was collected. Overall number of participants analyzed is the number of participants with data available for analyses. Participants were divided into 3 groups for this outcome measure based on the time they received IT treatment in relation to prior study HGT-HIT-094. | nanograms per milliliter (ng/mL) | MEAN |
| OTHER_PRE_SPECIFIED | Maximum Observed Concentration (Cmax) of Idursulfase in Cerebrospinal Fluid (CSF) | Predose each week up to 9 years | |||
| PRIMARY | Percent Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in CSF at Month 67 | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | percent change | MEAN |
| PRIMARY | Percent Change From Baseline in the Concentration of GAG in Urine at Month 67 | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | percent change | MEAN |
| PRIMARY | Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in CSF | Up to 9 years | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in Serum | Up to 9 years | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 outcomesResults by group105
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 Adverse Events (AEs) | Idursulfase-IT | 56 | Participants | ||
| Number of Participants With Clinically Significant Changes in Vital Signs | Idursulfase-IT | 0 | Participants | ||
| Number of Participants With Clinically Significant Changes in Laboratory Parameters | Idursulfase-IT | 0 | Participants | ||
| Number of Participants With Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Findings | Idursulfase-IT | 0 | Participants | ||
| Maximum Observed Serum Concentration (Cmax) of Idursulfase | Idursulfase-IT 10 mg, Delayed IT | 128.19 | Standard Deviation 169.427 | nanograms per milliliter (ng/mL) | |
| Maximum Observed Serum Concentration (Cmax) of Idursulfase | Idursulfase-IT 10 mg, Early IT | 104.89 | Standard Deviation 66.879 | nanograms per milliliter (ng/mL) | |
| Maximum Observed Serum Concentration (Cmax) of Idursulfase | Idursulfase-IT 10 mg, Former Substudy | 120.23 | Standard Deviation 57.308 | nanograms per milliliter (ng/mL) | |
| Percent Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in CSF at Month 67 | Idursulfase-IT | -71.15 | Standard Deviation 17.842 | percent change | |
| Percent Change From Baseline in the Concentration of GAG in Urine at Month 67 | Idursulfase-IT | -32.44 | Standard Deviation 29.366 | percent change | |
| Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in CSF | Idursulfase-IT | 25 | Participants |
1 to 10 of 105Source ClinicalTrials.gov
See all 105 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Idursulfase-IT | 56 | |
| Overall Study | COMPLETED | Idursulfase-IT | 23 | |
| Overall Study | NOT COMPLETED | Idursulfase-IT | 33 |
Source ClinicalTrials.gov
Adverse events307
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 | Abdominal pain | Gastrointestinal disorders | Idursulfase-IT | 8 | 56 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Idursulfase-IT | 4 | 56 | |
| other | Abnormal behaviour | Psychiatric disorders | Idursulfase-IT | 8 | 56 | |
| other | Achilles tendon discomfort | Musculoskeletal and connective tissue disorders | Idursulfase-IT | 3 | 56 | |
| other | Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | Idursulfase-IT | 4 | 56 | |
| other | Adenovirus infection | Infections and infestations | Idursulfase-IT | 3 | 56 | |
| other | Aggression | Psychiatric disorders | Idursulfase-IT | 11 | 56 | |
| other | Agitation | Psychiatric disorders | Idursulfase-IT | 8 | 56 | |
| other | Alanine aminotransferase increased | Investigations | Idursulfase-IT | 11 | 56 | |
| other | Albumin CSF increased | Investigations | Idursulfase-IT | 12 | 56 |
1 to 10 of 307Source ClinicalTrials.gov
See all 307 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Idursulfase-IT | 0 | 56 |
| Total, other adverse events | Idursulfase-IT | 56 | 56 |
| Total, serious adverse events | Idursulfase-IT | 48 | 56 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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/NCT02412787
GET https://conductscience.org/api/trials/v1/NCT02412787/registered?page=2