Trial · NCT00146770
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
NCT00146770, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2009-06-16
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients. Trials, record NCT00146770. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00146770
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
- NCT number
- NCT00146770
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 45 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Interventions
- Aldurazyme, Aldurazyme, placebo
- First posted
- 2005-09-07
- Last updated
- 2015-04-03
- Results first posted
- 2009-06-16
- Limitations and caveats
Rare disease with limited sample size, lack of control group.FVC calculations less reliable for patients whose height is below the 3rdpercent of general population.Height term used to calculate FVC may be influenced by joint contractures and posture.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) | Baseline to Week 182 | Percent Predicted Forced Vital Capacity: the maximal exhaled breath volume following a maximal inhaled breath. Overall change from Baseline to Week 182 in percent predicted FVC = (observed value)/(predicted value) \* 100%). A higher value indicates a greater response. |
| Primary | Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) | Baseline to Week 182 | Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response. |
| Secondary | Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI) | Baseline to Week 182 | Apnea/Hypopnea Index (AHI): Number of absent (apnea) and shallow (hypopnea) breaths per hour of sleep. A greater decrease in events indicates a greater response. |
| Secondary | Change From Baseline to Week 182 in Liver Volume | Baseline to Week 182 | Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response. |
| Secondary | Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score | Baseline to Week 182 | CHAQ/HAQ = Patient questionnaire that measures the degree of disability on a scale of 0 (no disability) to 3 (maximal disability). A lower score indicates a greater response. |
| Secondary | Change From Baseline to Week 182 in Active Joint Range of Motion (ROM) | Baseline to Week182 | Active Joint Range of Motion (ROM): Shoulder Flexion Ability to maximally raise one's arm overhead without assistance. Shoulder range of motion (mean of left and right arms) measured in degrees (0-180) by goniometry. Greater degree of flexion indicates greater response. |
| Other | Change From Baseline to Week 182 in Urinary GAG Level | Baseline to Week 182 | Urinary Glycosaminoglycan (GAG) Levels: \>\> Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) | Baseline to Week 182 | This study enrolled patients who completed the Phase 3 Double-Blind Study and wished to receive open-label treatment with Aldurazyme. The analysis method was intention to treat with last value carried forward. | percent predicted FVC | MEAN |
| PRIMARY | Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) | Baseline to Week 182 | The study enrolled patients who completed the Phase 3 double-blind study and wished to receive open-label treatment with Aldurazyme. The analysis method was intention to treat with last value carried forward. | Meters | MEAN |
| SECONDARY | Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI) | Baseline to Week 182 | The study enrolled patients who completed the Phase 3 double-blind study and wished to receive open-label treatment with Aldurazyme. The analysis method was intention to treat. | Events per Hour | MEAN |
| SECONDARY | Change From Baseline to Week 182 in Liver Volume | Baseline to Week 182 | The study enrolled patients who completed the Phase 3 double-blind study and wished to receive open-label treatment with Aldurazyme. The analysis method was intention to treat. | Cubic centimeters (cm3) | MEAN |
| SECONDARY | Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score | Baseline to Week 182 | The study enrolled patients who completed the Phase 3 double-blind study and wished to receive open-label treatment with Aldurazyme. The analysis method was intention to treat. | Units on a scale | MEAN |
| SECONDARY | Change From Baseline to Week 182 in Active Joint Range of Motion (ROM) | Baseline to Week182 | The study enrolled patients who completed the Phase 3 double-blind study and wished to receive open-label treatment with Aldurazyme. The analysis method was intention to treat. | Degrees | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline to Week 182 in Urinary GAG Level | Baseline to Week 182 | The study enrolled patients who completed the Phase 3 double-blind study and wished to receive open-label treatment with Aldurazyme. The analysis method was intention to treat. | ug GAG/mg Creatinine | MEAN |
Source ClinicalTrials.gov
Results by group42
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) | Placebo/Aldurazyme | Baseline percentage points FVC | 51.0 | Standard Deviation 13.15 | percent predicted FVC |
| Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) | Aldurazyme/Aldurazyme | Baseline percentage points FVC | 48.4 | Standard Deviation 14.85 | percent predicted FVC |
| Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) | Placebo/Aldurazyme | Week 182 percentage points FVC | 47.7 | Standard Deviation 13.69 | percent predicted FVC |
| Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) | Aldurazyme/Aldurazyme | Week 182 percentage points FVC | 47.2 | Standard Deviation 15.24 | percent predicted FVC |
| Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) | Placebo/Aldurazyme | Overall Change from Baseline to Week 182 | -3.3 | Standard Deviation 9.07 | percent predicted FVC |
| Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) | Aldurazyme/Aldurazyme | Overall Change from Baseline to Week 182 | -1.2 | Standard Deviation 6.52 | percent predicted FVC |
| Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) | Placebo/Aldurazyme | Baseline distance walked | 348.3 | Standard Deviation 128.81 | Meters |
| Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) | Aldurazyme/Aldurazyme | Baseline distance walked | 319.0 | Standard Deviation 131.41 | Meters |
| Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) | Placebo/Aldurazyme | Week 182 distance walked | 367.7 | Standard Deviation 151.25 | Meters |
| Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) | Aldurazyme/Aldurazyme | Week 182 distance walked | 358.3 | Standard Deviation 126.40 | Meters |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo/Aldurazyme | 23 | |
| Overall Study | STARTED | Aldurazyme/Aldurazyme | 22 | |
| Overall Study | COMPLETED | Placebo/Aldurazyme | 18 | |
| Overall Study | COMPLETED | Aldurazyme/Aldurazyme | 22 | |
| Overall Study | NOT COMPLETED | Placebo/Aldurazyme | 5 | |
| Overall Study | NOT COMPLETED | Aldurazyme/Aldurazyme | 0 |
Source ClinicalTrials.gov
Adverse events1,128
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 | Placebo/|Aldurazyme***Check Title*** | 5 | 23 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Aldurazyme/|Aldurazyme***Check Title*** | 2 | 22 | |
| other | Splenomegaly | Blood and lymphatic system disorders | Placebo/|Aldurazyme***Check Title*** | 2 | 23 | |
| other | Splenomegaly | Blood and lymphatic system disorders | Aldurazyme/|Aldurazyme***Check Title*** | 4 | 22 | |
| other | Angina pectoris | Cardiac disorders | Placebo/|Aldurazyme***Check Title*** | 0 | 23 | |
| other | Angina pectoris | Cardiac disorders | Aldurazyme/|Aldurazyme***Check Title*** | 1 | 22 | |
| other | Aortic valve incompetence | Cardiac disorders | Placebo/|Aldurazyme***Check Title*** | 3 | 23 | |
| other | Aortic valve incompetence | Cardiac disorders | Aldurazyme/|Aldurazyme***Check Title*** | 2 | 22 | |
| other | Arrhythmia | Cardiac disorders | Placebo/|Aldurazyme***Check Title*** | 1 | 23 | |
| other | Arrhythmia | Cardiac disorders | Aldurazyme/|Aldurazyme***Check Title*** | 2 | 22 |
1 to 10 of 1,128Source ClinicalTrials.gov
See all 1,128 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo/|Aldurazyme***Check Title*** | ||
| Total, all-cause mortality | Aldurazyme/|Aldurazyme***Check Title*** | ||
| Total, other adverse events | Placebo/|Aldurazyme***Check Title*** | 23 | 23 |
| Total, other adverse events | Aldurazyme/|Aldurazyme***Check Title*** | 22 | 22 |
| Total, serious adverse events | Placebo/|Aldurazyme***Check Title*** | 11 | 23 |
| Total, serious adverse events | Aldurazyme/|Aldurazyme***Check Title*** | 14 | 22 |
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Aldurazyme/|Aldurazyme***Check Title*** vs Placebo/|Aldurazyme***Check Title***: serious adverse events |
Source Trial comparisons
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/NCT00146770
GET https://conductscience.org/api/trials/v1/NCT00146770/results?page=2