Trial · NCT00607386
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
NCT00607386, Phase 4, Completed, Shire; results first posted 2013-11-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy. Trials, record NCT00607386. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00607386
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
- NCT number
- NCT00607386
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 28 participants, actual
- Lead sponsor
- Shire
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II, MPS II
- Interventions
- Idursulfase
- First posted
- 2008-02-05
- Last updated
- 2021-06-08
- Other registries' IDs
- eudract:2007-006044-22
- Results first posted
- 2013-11-07
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety Evaluation | From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks | 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. |
| Secondary | Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels | Baseline, Weeks 18, 36 and 53 | 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). |
| Secondary | Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) | Weeks 1 and 27 | |
| Secondary | Single- and Repeat-Dose Pharmacokinetics - Time of Maximum Observed Serum Concentration (Tmax) | Weeks 1 and 27 | |
| Secondary | 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) | Weeks 1 and 27 | |
| Secondary | Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to Infinity (AUCinf) | Weeks 1 and 27 | |
| Secondary | Single- and Repeat-Dose Pharmacokinetics - Elimination Half-Life (t1/2) | Weeks 1 and 27 | 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. |
| Secondary | Single- and Repeat-Dose Pharmacokinetics - Mean Residence Time From Time 0 to Infinity (MRTinf) | Weeks 1 and 27 | MRTinf is an average duration of the drug in the body from time zero to infinity, and is expressed in minutes. |
| Secondary | Single- and Repeat-Dose Pharmacokinetics - Clearance (CL) | Weeks 1 and 27 | Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. |
| Secondary | Single- and Repeat-Dose Pharmacokinetics - Volume of Distribution at Steady State (Vss) | Weeks 1 and 27 | 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. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety Evaluation | From the start of study treatment until 30 days after the last infusion of idursulfase, up to 53 weeks | Safety population was defined as all enrolled participants who received at least one study dose (or any portion of a dose) of idursulfase. | participants | NUMBER |
| SECONDARY | Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels | Baseline, Weeks 18, 36 and 53 | Safety population. In the categories listed below, 'N' signifies the number of participants evaluable for the timepoint. | microgram/milligram creatinine | MEAN |
| SECONDARY | Single- and Repeat-Dose Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) | Weeks 1 and 27 | 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. | nanogram per milliliter | MEAN |
| SECONDARY | Single- and Repeat-Dose Pharmacokinetics - Time of Maximum Observed Serum Concentration (Tmax) | Weeks 1 and 27 | PK population. In the categories listed below, N signifies the number of participants evaluable for the timepoint. | minutes | MEAN |
| SECONDARY | 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) | Weeks 1 and 27 | PK population. In the categories listed below, N signifies the number of participants evaluable for the timepoint. | minute*nanogram per milliliter | MEAN |
| SECONDARY | Single- and Repeat-Dose Pharmacokinetics - Area Under the Serum Concentration-Time Curve From Time 0 to Infinity (AUCinf) | Weeks 1 and 27 | PK population with evaluable participants for this endpoint. In the categories listed below, N signifies the number of participants evaluable for the timepoint. | minute*nanogram per milliliter | MEAN |
| SECONDARY | Single- and Repeat-Dose Pharmacokinetics - Elimination Half-Life (t1/2) | Weeks 1 and 27 | PK population with evaluable participants for this endpoint. In the categories listed below, N signifies the number of participants evaluable for the timepoint. | minutes | MEAN |
| SECONDARY | Single- and Repeat-Dose Pharmacokinetics - Mean Residence Time From Time 0 to Infinity (MRTinf) | Weeks 1 and 27 | PK population with evaluable participants for this endpoint. In the categories listed below, N signifies the number of participants evaluable for the timepoint. | minutes | MEAN |
| SECONDARY | Single- and Repeat-Dose Pharmacokinetics - Clearance (CL) | Weeks 1 and 27 | PK population with evaluable participants for this endpoint. In the categories listed below, N signifies the number of participants evaluable for the timepoint. | milliliter/minute/kilogram | MEAN |
| SECONDARY | Single- and Repeat-Dose Pharmacokinetics - Volume of Distribution at Steady State (Vss) | Weeks 1 and 27 | PK population with evaluable participants for this endpoint. In the categories listed below, N signifies the number of participants evaluable for the timepoint. | milliliter per kilogram | MEAN |
Source ClinicalTrials.gov
Results by group27
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety Evaluation | Idursulfase | Experienced at least one adverse event (AE) | 28 | participants | |
| Safety Evaluation | Idursulfase | Deaths | 0 | participants | |
| Safety Evaluation | Idursulfase | Discontinued due to an AE | 0 | participants | |
| Safety Evaluation | Idursulfase | Experienced at least one drug-related AE | 16 | participants | |
| Safety Evaluation | Idursulfase | Experienced at least one serious AE (SAE) | 13 | participants | |
| Safety Evaluation | Idursulfase | Experienced at least one severe AE | 2 | participants | |
| Safety Evaluation | Idursulfase | Experienced at least one infusion-related AE | 16 | participants | |
| Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels | Idursulfase | Baseline (N=28) | 738.3 | Standard Deviation 165.21 | microgram/milligram creatinine |
| Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels | Idursulfase | Change at Week 18 (N=27) | -368.0 | Standard Deviation 165.44 | microgram/milligram creatinine |
| Mean Change From Baseline to Week 53 in Normalized Urinary Glycosaminoglycan (GAG) Levels | Idursulfase | Change at Week 36 (N=27) | -400.3 | Standard Deviation 180.27 | microgram/milligram creatinine |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 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 | 28 | |
| Overall Study | COMPLETED | Idursulfase | 27 | |
| Overall Study | NOT COMPLETED | Idursulfase | 1 |
Source ClinicalTrials.gov
Adverse events178
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 | Upper respiratory tract infection | Infections and infestations | Idursulfase | 18 | 28 | 42 |
| other | Respiratory tract infection | Infections and infestations | Idursulfase | 12 | 28 | 36 |
| other | Rhinitis | Infections and infestations | Idursulfase | 11 | 28 | 20 |
| other | Viral upper respiratory tract infection | Infections and infestations | Idursulfase | 9 | 28 | 17 |
| other | Otitis media acute | Infections and infestations | Idursulfase | 7 | 28 | 10 |
| other | Pharyngitis | Infections and infestations | Idursulfase | 7 | 28 | 18 |
| other | Gastroenteritis | Infections and infestations | Idursulfase | 6 | 28 | 7 |
| other | Upper respiratory tract infection bacterial | Infections and infestations | Idursulfase | 6 | 28 | 13 |
| other | Pneumonia | Infections and infestations | Idursulfase | 3 | 28 | 3 |
| other | Sinusitis | Infections and infestations | Idursulfase | 5 | 28 | 6 |
1 to 10 of 178Source ClinicalTrials.gov
See all 178 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Idursulfase | ||
| Total, other adverse events | Idursulfase | 28 | 28 |
| Total, serious adverse events | Idursulfase | 13 | 28 |
Source ClinicalTrials.gov
Papers3
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/NCT00607386
GET https://conductscience.org/api/trials/v1/NCT00607386/results?page=2