Trial · NCT00144781
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
NCT00144781, Phase 4, Completed, Genzyme, a Sanofi Company; results first posted 2009-06-16
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease. Trials, record NCT00144781. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00144781
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
- NCT number
- NCT00144781
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 34 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Interventions
Aldurazyme (Recombinant Human Alpha-L-Iduronidase), Aldurazyme (Recombinant Human Alpha-L-Iduronidase), Aldurazyme (Recombinant Human Alpha-L-Iduronidase), Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- First posted
- 2005-09-05
- Last updated
- 2015-04-03
- Results first posted
- 2009-06-16
- Limitations and caveats
- This study is limited by its small sample size and ability to detect small differences in urinary GAG levels between groups.
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level | Baseline to 26 Weeks | Urinary GAG Level - Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response. |
| Secondary | Percent Change From Baseline to Week 26 in Liver Organ Volume | Baseline to 26 Weeks | A greater decrease in liver volume indicates a greater response. |
| Secondary | Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) | Baseline to 26 Weeks | Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level | Baseline to 26 Weeks | Based on the changes in urinary GAG levels observed in the Phase 3 double-blind study, a sample size of 8 patients per group would have sufficient power to detect a 29 percentage point difference between groups in the mean change in urinary GAG levels as being statistically significant. The analysis was intention to treat. | Percentage of Change in GAG Level | MEAN |
| SECONDARY | Percent Change From Baseline to Week 26 in Liver Organ Volume | Baseline to 26 Weeks | The analysis was intention to treat. | Percentage of Change in Liver Volume | MEAN |
| SECONDARY | Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) | Baseline to 26 Weeks | The analysis was intention to treat. | meters | MEAN |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level | 0.58 mg/kg Aldurazyme Every Week | -57.57 | 95% Confidence Interval 18.789 | Percentage of Change in GAG Level | |
| Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level | 1.2 mg/kg Aldurazyme Every Week | -66.63 | 95% Confidence Interval 14.917 | Percentage of Change in GAG Level | |
| Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level | 1.2 mg/kg Aldurazyme Every 2 Weeks | -66.15 | 95% Confidence Interval 8.043 | Percentage of Change in GAG Level | |
| Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level | 1.8 mg/kg Aldurazyme Every 2 Weeks | -62.66 | 95% Confidence Interval 6.506 | Percentage of Change in GAG Level | |
| Percent Change From Baseline to Week 26 in Liver Organ Volume | 0.58 mg/kg Aldurazyme Every Week | -26.27 | 95% Confidence Interval 7.85 | Percentage of Change in Liver Volume | |
| Percent Change From Baseline to Week 26 in Liver Organ Volume | 1.2 mg/kg Aldurazyme Every Week | -30.67 | 95% Confidence Interval 8.13 | Percentage of Change in Liver Volume | |
| Percent Change From Baseline to Week 26 in Liver Organ Volume | 1.2 mg/kg Aldurazyme Every 2 Weeks | -30.74 | 95% Confidence Interval 18.24 | Percentage of Change in Liver Volume | |
| Percent Change From Baseline to Week 26 in Liver Organ Volume | 1.8 mg/kg Aldurazyme Every 2 Weeks | -32.14 | 95% Confidence Interval 10.10 | Percentage of Change in Liver Volume | |
| Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) | 0.58 mg/kg Aldurazyme Every Week | 7.00 | 95% Confidence Interval 83.355 | meters | |
| Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) | 1.2 mg/kg Aldurazyme Every Week | -12.17 | 95% Confidence Interval 58.043 | meters |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 0.58 mg/kg Aldurazyme Every Week | 8 | |
| Overall Study | STARTED | 1.2 mg/kg Aldurazyme Every Week | 9 | |
| Overall Study | STARTED | 1.2 mg/kg Aldurazyme Every 2 Weeks | 8 | |
| Overall Study | STARTED | 1.8 mg/kg Aldurazyme Every 2 Weeks | 9 | |
| Overall Study | COMPLETED | 0.58 mg/kg Aldurazyme Every Week | 8 | |
| Overall Study | COMPLETED | 1.2 mg/kg Aldurazyme Every Week | 7 | |
| Overall Study | COMPLETED | 1.2 mg/kg Aldurazyme Every 2 Weeks | 8 | |
| Overall Study | COMPLETED | 1.8 mg/kg Aldurazyme Every 2 Weeks | 9 | |
| Overall Study | NOT COMPLETED | 0.58 mg/kg Aldurazyme Every Week | 0 | |
| Overall Study | NOT COMPLETED | 1.2 mg/kg Aldurazyme Every Week | 2 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events660
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 | Anaemia | Blood and lymphatic system disorders | Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** | 0 | 8 | |
| other | Anaemia | Blood and lymphatic system disorders | Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** | 1 | 9 | |
| other | Anaemia | Blood and lymphatic system disorders | Aldurazyme|Weekly|0.58 mg/kg***Check Title*** | 0 | 8 | |
| other | Anaemia | Blood and lymphatic system disorders | Aldurazyme|Weekly|1.2 mg/kg***Check Title*** | 0 | 8 | |
| other | Eosinophilia | Blood and lymphatic system disorders | Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** | 1 | 8 | |
| other | Eosinophilia | Blood and lymphatic system disorders | Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** | 0 | 9 | |
| other | Eosinophilia | Blood and lymphatic system disorders | Aldurazyme|Weekly|0.58 mg/kg***Check Title*** | 0 | 8 | |
| other | Eosinophilia | Blood and lymphatic system disorders | Aldurazyme|Weekly|1.2 mg/kg***Check Title*** | 0 | 8 | |
| other | White blood cell disorder | Blood and lymphatic system disorders | Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** | 1 | 8 | |
| other | White blood cell disorder | Blood and lymphatic system disorders | Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** | 0 | 9 |
1 to 10 of 660Source ClinicalTrials.gov
See all 660 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** | ||
| Total, all-cause mortality | Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** | ||
| Total, all-cause mortality | Aldurazyme|Weekly|0.58 mg/kg***Check Title*** | ||
| Total, all-cause mortality | Aldurazyme|Weekly|1.2 mg/kg***Check Title*** | ||
| Total, other adverse events | Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** | 8 | 8 |
| Total, other adverse events | Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** | 9 | 9 |
| Total, other adverse events | Aldurazyme|Weekly|0.58 mg/kg***Check Title*** | 8 | 8 |
| Total, other adverse events | Aldurazyme|Weekly|1.2 mg/kg***Check Title*** | 7 | 8 |
| Total, serious adverse events | Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** | 2 | 8 |
| Total, serious adverse events | Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** | 1 | 9 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
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/NCT00144781
GET https://conductscience.org/api/trials/v1/NCT00144781/events?page=2