Trial · NCT00069641
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
NCT00069641, Phase 2, Phase 3, Completed, Shire; results first posted 2015-05-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II). Trials, record NCT00069641. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00069641
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
- NCT number
- NCT00069641
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 96 participants, actual
- Lead sponsor
- Shire
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Iduronate-2-sulfatase enzyme replacement therapy, iduronate-2-sulfatase enzyme replacement therapy, Placebo
- First posted
- 2003-10-01
- Last updated
- 2021-06-10
- Results first posted
- 2015-05-13
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53 | Baseline, Week 53 | The 2-component composite variable consists of the sum of the ranked changes from baseline to Week 53 for percent predicted Forced Vital Capacity (FVC) and 6-Minute Walking Test (6MWT) total distance walked. For the 2 treatment groups being compared, ranking occurred within the comparison treatment groups combined (idursulfase weekly and placebo treatment groups). These comparison groups were pooled and ranked for each component separately. Within each component (% predicted FVC, 6MWT), the change from baseline was then ranked. The lowest change value was assigned a rank of 1, the next lowest a rank of 2, etc. The composite score for each participant was the sum of the 2 ranked scores corresponding to the 2 individual components (% predicted FVC and 6MWT) for each participant. Thus, the greater the composite score (greater the sum of the ranks of the changes from baseline, where the lowest change was ranked as 1), the greater the improvement. |
| Secondary | Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Baseline, Week 53 | Change was calculated at Week 53 from baseline. 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 \[Combined Metacarpophalangeal joint (MCP), Proximal interphalangeal joint (PIP), Distal interphalangeal joint (DIP) motion\]), 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). |
| Secondary | Mean Combined Liver and Spleen Volume at Baseline | Baseline | Liver and Spleen volume was determined by Magnetic Resonance Imaging (MRI). |
| Secondary | Percent Change From Baseline in Mean Combined Liver and Spleen Volume at Week 53 | Baseline, Week 53 | Liver and Spleen volume was determined by Magnetic Resonance Imaging (MRI). Change was calculated at Week 53 from baseline. |
| Secondary | Change From Baseline in Mean Normalized Urine Glycosaminoglycan (GAG) Levels at Week 53 | Baseline, Week 53 | Mean normalized urine GAG was analyzed using urine testing. Change was calculated at Week 53 from baseline. The urine GAG levels were normalized to urine creatinine and were reported as microgram GAG per milligram creatinine (mcg GAG/mg creatinine). |
| Secondary | Mean Cardiac Left Ventricular Mass Index (LVMI) at Baseline | Baseline | Cardiac LVMI was determined by echocardiography. LVMI is the left ventricular mass (LVM, in grams \[g\]) indexed to body surface area (BSA), in square meter \[m\^2\]. LVMI (in gram per square meter \[g/m\^2\]) = LVM divided by BSA. |
| Secondary | Percent Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 53 | Baseline, Week 53 | Cardiac LVMI was determined by echocardiography. Change was calculated at Week 53 from baseline. LVMI is the LVM, in grams indexed to BSA, in square meter \[m\^2\]. LVMI in g/m\^2 = LVM divided by BSA. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| SECONDARY | Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Baseline, Week 53 | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | percentage of normal range of motion | MEAN |
| SECONDARY | Mean Combined Liver and Spleen Volume at Baseline | Baseline | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | milliliter (mL) | MEAN |
| SECONDARY | Percent Change From Baseline in Mean Combined Liver and Spleen Volume at Week 53 | Baseline, Week 53 | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | percent change | MEAN |
| SECONDARY | Change From Baseline in Mean Normalized Urine Glycosaminoglycan (GAG) Levels at Week 53 | Baseline, Week 53 | ITT population. | mcg GAG/mg creatinine | MEAN |
| PRIMARY | Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53 | Baseline, Week 53 | Intent-to-treat (ITT) population, Only participants receiving Idursulfase Weekly or Placebo were to be analyzed for this outcome. | sum of the ranked scores | MEAN |
| SECONDARY | Mean Cardiac Left Ventricular Mass Index (LVMI) at Baseline | Baseline | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | gram per square meter (g/m^2) | MEAN |
| SECONDARY | Percent Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 53 | Baseline, Week 53 | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | percent change | MEAN |
Source ClinicalTrials.gov
Results by group26
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Idursulfase Weekly (0.5 mg/kg) | Baseline | 66.86 | Standard Error 1.85 | percentage of normal range of motion |
| Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Idursulfase EOW (0.5 mg/kg) | Baseline | 67.36 | Standard Error 1.43 | percentage of normal range of motion |
| Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Placebo | Baseline | 67.70 | Standard Error 1.59 | percentage of normal range of motion |
| Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Idursulfase Weekly (0.5 mg/kg) | Change at Week 53 | 0.89 | Standard Error 0.87 | percentage of normal range of motion |
| Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Idursulfase EOW (0.5 mg/kg) | Change at Week 53 | -0.61 | Standard Error 0.94 | percentage of normal range of motion |
| Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Placebo | Change at Week 53 | 0.70 | Standard Error 1.10 | percentage of normal range of motion |
| Mean Combined Liver and Spleen Volume at Baseline | Idursulfase Weekly (0.5 mg/kg) | 1578.48 | Standard Error 80.75 | milliliter (mL) | |
| Mean Combined Liver and Spleen Volume at Baseline | Idursulfase EOW (0.5 mg/kg) | 1442.2 | Standard Error 63.54 | milliliter (mL) | |
| Mean Combined Liver and Spleen Volume at Baseline | Placebo | 1485.28 | Standard Error 70.19 | milliliter (mL) | |
| Percent Change From Baseline in Mean Combined Liver and Spleen Volume at Week 53 | Idursulfase Weekly (0.5 mg/kg) | -25.81 | Standard Error 1.44 | percent change |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Idursulfase Weekly (0.5 mg/kg) | 32 | |
| Overall Study | STARTED | Idursulfase Every Other Week (EOW) (0.5 mg/kg) | 32 | |
| Overall Study | STARTED | Placebo | 32 | |
| Overall Study | COMPLETED | Idursulfase Weekly (0.5 mg/kg) | 31 | |
| Overall Study | COMPLETED | Idursulfase Every Other Week (EOW) (0.5 mg/kg) | 32 | |
| Overall Study | COMPLETED | Placebo | 31 | |
| Overall Study | NOT COMPLETED | Idursulfase Weekly (0.5 mg/kg) | 1 | |
| Overall Study | NOT COMPLETED | Idursulfase Every Other Week (EOW) (0.5 mg/kg) | 0 | |
| Overall Study | NOT COMPLETED | Placebo | 1 |
Source ClinicalTrials.gov
Adverse events591
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 | Lymphadenopathy | Blood and lymphatic system disorders | Idursulfase Weekly (0.5 mg/kg) | 0 | 32 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Idursulfase EOW (0.5 mg/kg) | 2 | 32 | 2 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Placebo | 0 | 32 | 0 |
| other | Atrial hypertrophy | Cardiac disorders | Idursulfase Weekly (0.5 mg/kg) | 2 | 32 | 2 |
| other | Atrial hypertrophy | Cardiac disorders | Idursulfase EOW (0.5 mg/kg) | 0 | 32 | 0 |
| other | Atrial hypertrophy | Cardiac disorders | Placebo | 3 | 32 | 3 |
| other | Tachycardia nos | Cardiac disorders | Idursulfase Weekly (0.5 mg/kg) | 1 | 32 | 1 |
| other | Tachycardia nos | Cardiac disorders | Idursulfase EOW (0.5 mg/kg) | 3 | 32 | 5 |
| other | Tachycardia nos | Cardiac disorders | Placebo | 2 | 32 | 10 |
| other | Ventricular hypertrophy | Cardiac disorders | Idursulfase Weekly (0.5 mg/kg) | 2 | 32 | 2 |
1 to 10 of 591Source ClinicalTrials.gov
See all 591 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Idursulfase Weekly (0.5 mg/kg) | ||
| Total, all-cause mortality | Idursulfase EOW (0.5 mg/kg) | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Idursulfase Weekly (0.5 mg/kg) | 32 | 32 |
| Total, other adverse events | Idursulfase EOW (0.5 mg/kg) | 32 | 32 |
| Total, other adverse events | Placebo | 32 | 32 |
| Total, serious adverse events | Idursulfase Weekly (0.5 mg/kg) | 9 | 32 |
| Total, serious adverse events | Idursulfase EOW (0.5 mg/kg) | 8 | 32 |
| Total, serious adverse events | Placebo | 9 | 32 |
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/NCT00069641
GET https://conductscience.org/api/trials/v1/NCT00069641/results?page=2