Trial · NCT00146757

A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

NCT00146757, Phase 2, 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 Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old. Trials, record NCT00146757. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00146757

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
NCT number: NCT00146757
Status: Completed
Phases: Phase 2
Enrollment: 20 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), Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
First posted: 2005-09-07
Last updated: 2015-04-03
Results first posted: 2009-06-16
Limitations and caveats: Due to the rarity of the disease, this study is limited by its small sample size and lack of a control group.

Source ClinicalTrials.gov

Registered outcomes12

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Safety Evaluation	52 weeks	Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
Primary	Pharmacokinetics - Area Under the (Plasma Concentration-time) Curve (AUC∞)	52 weeks	AUC∞ is a measure of the total exposure to a drug.
Primary	Pharmacokinetics - Elimination Half Life (t1/2)	52 weeks	Half-life is the time it takes for the concentration of drug in plasma to decline by 50%.
Primary	Pharmacokinetics - Total Plasma Clearance (CL)	52 weeks	CL is volume of the body fluid cleared of the drug per unit of time.
Primary	Pharmacokinetics - Volume of Distribution (Vz)	52 weeks	Vz is the volume that relates the amount of drug in the body after absorption is complete to the concentration of drug in the plasma.
Other	Percent Change From Baseline to Week 52 in Urinary Glycosaminoglycan (uGAG) Level	Baseline to 52 weeks	Percentage change in the concentration of GAG relative to creatinine (ug GAG/mg creatinine) in urine from Baseline to Week 52; A greater decrease in percent change indicates a greater response.
Other	Percent Change From Baseline to Week 52 in Liver Size (Hepatomegaly)	Baseline to 52 weeks	Percent change in extent of Liver Edge Below Right Costal Margin (BRCM) measured in centimeters from Baseline to Week 52; A greater decrease in percent change indicates a greater response.
Other	Change From Baseline to Week 52 in Apnea/Hypopnea Index (AHI)	Baseline to 52 weeks	Number of absent (apnea) and shallow (hypopnea) breaths per hour of sleep. A greater decrease in events per hour indicates a greater response.
Other	Expert Global Assessment of Sleep Study Results at Week 52 Compared With Baseline	Baseline to 52 weeks	Independent experts provided a global assessment for each sleep study visit as well as the degree of clinically meaningful change over the course of the study. Assessment was based on AHI, severity and frequency of oxygen desaturations and sleep quality.
Other	Change From Baseline to Week 52 in Left Ventricular Mass (LVM) Z-Score	Baseline to 52 weeks	Change in LVM Z-scores as measured by echocardiography from Baseline to Week 52. Z-score=number of standard deviations from mean. Z-scores greater than +2 and less than -2 are abnormal. A greater decrease in abnormally high z-score indicates a greater response.

1 to 10 of 12Source ClinicalTrials.gov

See all 12 registered outcomes

Outcomes12

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Safety Evaluation	52 weeks	The number of participants was determined as adequate to assess the safety of Aldurazyme in young children with mucopolysaccharidosis I (MPS I). The analysis was intent-to-treat.	participants	NUMBER
PRIMARY	Pharmacokinetics - Area Under the (Plasma Concentration-time) Curve (AUC∞)	52 weeks	The analysis was intent-to-treat.	hours units/milliliters	MEAN
PRIMARY	Pharmacokinetics - Elimination Half Life (t1/2)	52 weeks	The analysis was intent-to-treat.	hours	MEAN
PRIMARY	Pharmacokinetics - Total Plasma Clearance (CL)	52 weeks	The analysis was intent-to-treat.	(milliliters/minute)/kilograms	MEAN
PRIMARY	Pharmacokinetics - Volume of Distribution (Vz)	52 weeks	The analysis was intent-to-treat.	liters/kilograms	MEAN
OTHER_PRE_SPECIFIED	Percent Change From Baseline to Week 52 in Urinary Glycosaminoglycan (uGAG) Level	Baseline to 52 weeks	The analysis was intent-to-treat.	Percentage of change	MEAN
OTHER_PRE_SPECIFIED	Percent Change From Baseline to Week 52 in Liver Size (Hepatomegaly)	Baseline to 52 weeks	The analysis was intent-to-treat.	Percentage of change	MEAN
OTHER_PRE_SPECIFIED	Change From Baseline to Week 52 in Apnea/Hypopnea Index (AHI)	Baseline to 52 weeks	The analysis was intent-to-treat.	Events per hour	MEAN
OTHER_PRE_SPECIFIED	Expert Global Assessment of Sleep Study Results at Week 52 Compared With Baseline	Baseline to 52 weeks	The analysis was intent-to-treat.	participants	NUMBER
OTHER_PRE_SPECIFIED	Change From Baseline to Week 52 in Left Ventricular Mass (LVM) Z-Score	Baseline to 52 weeks	The analysis was intent-to-treat.	Units on a scale	MEAN

1 to 10 of 12Source ClinicalTrials.gov

See all 12 outcomes

Results by group48

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Safety Evaluation	Aldurazyme (rhIDU) 100 U/kg Every Week	Having Adverse Events (AE)	16	21.41	participants
Safety Evaluation	Aldurazyme (rhIDU) 100-200 U/kg Every Week	Having Adverse Events (AE)	4	14.21	participants
Safety Evaluation	Aldurazyme (rhIDU) 100 U/kg Every Week	Having Drug-Related AEs	5		participants
Safety Evaluation	Aldurazyme (rhIDU) 100-200 U/kg Every Week	Having Drug-Related AEs	3		participants
Safety Evaluation	Aldurazyme (rhIDU) 100 U/kg Every Week	Deaths/Discontinuations Due to AEs	2		participants
Safety Evaluation	Aldurazyme (rhIDU) 100-200 U/kg Every Week	Deaths/Discontinuations Due to AEs	0		participants
Safety Evaluation	Aldurazyme (rhIDU) 100 U/kg Every Week	Having Serious AEs	12		participants
Safety Evaluation	Aldurazyme (rhIDU) 100-200 U/kg Every Week	Having Serious AEs	3		participants
Safety Evaluation	Aldurazyme (rhIDU) 100 U/kg Every Week	Having Severe AEs	5		participants
Safety Evaluation	Aldurazyme (rhIDU) 100-200 U/kg Every Week	Having Severe AEs	1		participants

1 to 10 of 48Source ClinicalTrials.gov

See all 48 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Aldurazyme (rhIDU) 100 U/kg Every Week	16
Overall Study	STARTED	Aldurazyme (rhIDU) 100-200 U/kg Every Week	4
Overall Study	COMPLETED	Aldurazyme (rhIDU) 100 U/kg Every Week	14
Overall Study	COMPLETED	Aldurazyme (rhIDU) 100-200 U/kg Every Week	4
Overall Study	NOT COMPLETED	Aldurazyme (rhIDU) 100 U/kg Every Week	2
Overall Study	NOT COMPLETED	Aldurazyme (rhIDU) 100-200 U/kg Every Week	0

Source ClinicalTrials.gov

Adverse events184

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
other	Iron deficiency anaemia	Blood and lymphatic system disorders	Aldurazyme *Check Title*	1	20
other	Lymphadenopathy	Blood and lymphatic system disorders	Aldurazyme *Check Title*	2	20
other	Angina pectoris	Cardiac disorders	Aldurazyme *Check Title*	1	20
other	Aortic valve disease	Cardiac disorders	Aldurazyme *Check Title*	1	20
other	Aortic valve incompetence	Cardiac disorders	Aldurazyme *Check Title*	2	20
other	Mitral valve incompetence	Cardiac disorders	Aldurazyme *Check Title*	1	20
other	Tachycardia	Cardiac disorders	Aldurazyme *Check Title*	1	20
other	Cerumen impaction	Ear and labyrinth disorders	Aldurazyme *Check Title*	2	20
other	Ear congestion	Ear and labyrinth disorders	Aldurazyme *Check Title*	1	20
other	Ear pain	Ear and labyrinth disorders	Aldurazyme *Check Title*	4	20

1 to 10 of 184Source ClinicalTrials.gov

See all 184 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Aldurazyme *Check Title*
Total, other adverse events	Aldurazyme *Check Title*	20	20
Total, serious adverse events	Aldurazyme *Check Title*	15	20

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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.

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GET https://conductscience.org/api/trials/v1/NCT00146757/results?page=2

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