Trial · NCT00258011
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
NCT00258011, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2009-02-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease. Trials, record NCT00258011. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00258011
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
- NCT number
- NCT00258011
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 3 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Mucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Interventions
- Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- First posted
- 2005-11-24
- Last updated
- 2014-03-07
- Results first posted
- 2009-02-12
- Limitations and caveats
- Due to the rarity of the disease, this study is limited by its small sample size, lack of a control group and patients had variable treatment durations.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety Evaluation | Up to 73 Weeks | Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports. |
| Secondary | Urinary Glycosaminoglycan (GAG) Excretion | Up to 73 Weeks | Percentage change in the concentration of GAG relative to creatinine in urine (ug GAG/mg creatinine) from baseline to last study visit. Greater decrease indicates greater response. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety Evaluation | Up to 73 Weeks | The study enrolled eligible MPS I patients prior to marketing authorization in Japan. The analysis was intention to treat. | participants | NUMBER |
| SECONDARY | Urinary Glycosaminoglycan (GAG) Excretion | Up to 73 Weeks | The study enrolled eligible MPS I patients prior to marketing authorization in Japan. The analysis was intention to treat.\>~\* 1 patient final visit = Week 73; 1 patient final visit = Week 32; 1 patient final visit = Week 10 | percent change in concentration of GAG | MEAN |
Source ClinicalTrials.gov
Results by group22
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety Evaluation | Aldurazyme (Laronidase) Treatment | Having Adverse Events (AEs) | 3 | -81.1 to -60.6 | participants |
| Safety Evaluation | Aldurazyme (Laronidase) Treatment | Having drug related AEs | 0 | participants | |
| Safety Evaluation | Aldurazyme (Laronidase) Treatment | Discontinuations due to AEs | 0 | participants | |
| Safety Evaluation | Aldurazyme (Laronidase) Treatment | Having Serious AEs | 1 | participants | |
| Safety Evaluation | Aldurazyme (Laronidase) Treatment | Having Severe AEs | 1 | participants | |
| Safety Evaluation | Aldurazyme (Laronidase) Treatment | Deaths | 0 | participants | |
| Safety Evaluation | Aldurazyme (Laronidase) Treatment | Having infusion-associated reactions | 0 | participants | |
| Urinary Glycosaminoglycan (GAG) Excretion | Aldurazyme (Laronidase) Treatment | Baseline (3 patients analyzed) | 0 | Standard Deviation 0 | percent change in concentration of GAG |
| Urinary Glycosaminoglycan (GAG) Excretion | Aldurazyme (Laronidase) Treatment | Week 4 (3 patients analyzed) | -68.0 | Standard Deviation 12.76 | percent change in concentration of GAG |
| Urinary Glycosaminoglycan (GAG) Excretion | Aldurazyme (Laronidase) Treatment | Week 8 (3 patients analyzed) | -69.9 | Standard Deviation 7.98 | percent change in concentration of GAG |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Aldurazyme (Laronidase) Treatment | 3 | |
| Overall Study | COMPLETED | Aldurazyme (Laronidase) Treatment | 3 | |
| Overall Study | NOT COMPLETED | Aldurazyme (Laronidase) Treatment | 0 |
Source ClinicalTrials.gov
Adverse events44
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 | Conjunctivitis | Eye disorders | Aldurazyme | 1 | 3 | |
| other | Ocular hyperaemia | Eye disorders | Aldurazyme | 1 | 3 | |
| other | Diarrhoea | Gastrointestinal disorders | Aldurazyme | 1 | 3 | |
| other | Catheter related complication | General disorders | Aldurazyme | 1 | 3 | |
| other | Catheter site erythema | General disorders | Aldurazyme | 1 | 3 | |
| other | Catheter site pruritus | General disorders | Aldurazyme | 1 | 3 | |
| other | Pyrexia | General disorders | Aldurazyme | 1 | 3 | |
| other | Bronchitis | Infections and infestations | Aldurazyme | 1 | 3 | |
| other | Catheter site infection | Infections and infestations | Aldurazyme | 1 | 3 | |
| other | Gastroenteritis | Infections and infestations | Aldurazyme | 1 | 3 |
1 to 10 of 44Source ClinicalTrials.gov
See all 44 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Aldurazyme | ||
| Total, other adverse events | Aldurazyme | 3 | 3 |
| Total, serious adverse events | Aldurazyme | 1 | 3 |
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/NCT00258011
GET https://conductscience.org/api/trials/v1/NCT00258011/results?page=2