Trial · NCT00701415

A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms

NCT00701415, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2016-06-29

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms. Trials, record NCT00701415. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00701415

On this page

Trial

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

Title: A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
NCT number: NCT00701415
Status: Completed
Phases: Phase 3
Enrollment: 31 participants, actual
Lead sponsor: Genzyme, a Sanofi Company
Conditions: Fabry Disease
Interventions: Agalsidase beta, Agalsidase beta
First posted: 2008-06-19
Last updated: 2016-06-29
Other registries' IDs: eudract:2007-005668-28
Results first posted: 2016-06-29

Source ClinicalTrials.gov

Registered outcomes3

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

List	Outcome	Time frame	Description
Primary	Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Baseline, Week 52, Week 156 and Week 260	Skin biopsies were taken at Baseline, Week 52, Week 156 and Week 260 or early withdrawal and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was scored for GL-3 accumulation on a severity score-scale of none, mild, moderate, severe (0-1-2-3). Scores are categorized as normal (score = 0) or abnormal (score = 1, 2 or 3). Data was summarized in terms of number of participants with none/trace, mild, moderate and severe biopsy scores.
Secondary	Percent Change From Baseline in GL-3 Clearance From Plasma	Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260	Plasma samples were assayed for GL-3 clearance using a validated tandem mass spectrometry with an upper limit of normal plasma GL-3 level of 7.0 μg/mL. Number of participants analyzed=participants with both baseline and post-baseline GL-3 plasma clearance assessment. Here 'n' signifies number of participants with available data for specified category.
Secondary	Percent Change From Baseline in GL-3 Clearance From Urine	Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260	Plasma samples were assayed for total urine GL-3 clearance using a validated tandem mass spectrometry with an upper limit of normal of \<0.030 mg/mmoL of creatinine. Number of participants analyzed=participants with both baseline and post-baseline GL-3 urine clearance assessment. Here 'n' signifies number of participants with available data for specified category.

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	Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Baseline, Week 52, Week 156 and Week 260	Analysis was performed on Full analysis set (FAS), which included all randomized participants who received at least 1 infusion of study treatment.	Percentage of participants	NUMBER
SECONDARY	Percent Change From Baseline in GL-3 Clearance From Plasma	Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260	Analysis was performed on FAS.	Percent change	MEAN
SECONDARY	Percent Change From Baseline in GL-3 Clearance From Urine	Baseline, Week 12, 28, 40, 52, 80, 104, 132, 156, 184, 208, 236 and 260	Analysis was performed on FAS.	Percent change	MEAN

Source ClinicalTrials.gov

Results by group88

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

Outcome	Group	Category	Value	Units
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 0.5 mg/kg	Zero (0) Skin GL-3 Score at Baseline	18.8	Percentage of participants
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 1.0 mg/kg	Zero (0) Skin GL-3 Score at Baseline	33.3	Percentage of participants
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 0.5 mg/kg	Zero (0) Skin GL-3 Score at Week 52	75	Percentage of participants
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 1.0 mg/kg	Zero (0) Skin GL-3 Score at Week 52	80	Percentage of participants
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 0.5 mg/kg	Zero (0) Skin GL-3 Score at Week 156	56.3	Percentage of participants
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 1.0 mg/kg	Zero (0) Skin GL-3 Score at Week 156	80	Percentage of participants
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 0.5 mg/kg	Zero (0) Skin GL-3 Score at Week 260	68.8	Percentage of participants
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 1.0 mg/kg	Zero (0) Skin GL-3 Score at Week 260	66.7	Percentage of participants
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 0.5 mg/kg	Mild (1) Skin GL-3 Score at Baseline	6.3	Percentage of participants
Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium	Fabrazyme 1.0 mg/kg	Mild (1) Skin GL-3 Score at Baseline	0	Percentage of participants

1 to 10 of 88Source ClinicalTrials.gov

See all 88 results by group

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Fabrazyme 0.5 mg/kg	16
Overall Study	STARTED	Fabrazyme 1.0 mg/kg	15
Overall Study	COMPLETED	Fabrazyme 0.5 mg/kg	15
Overall Study	COMPLETED	Fabrazyme 1.0 mg/kg	14
Overall Study	NOT COMPLETED	Fabrazyme 0.5 mg/kg	1
Overall Study	NOT COMPLETED	Fabrazyme 1.0 mg/kg	1

Source ClinicalTrials.gov

Adverse events534

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	ANAEMIA	Blood and lymphatic system disorders	Fabrazyme 0.5 mg/kg	1	16
other	ANAEMIA	Blood and lymphatic system disorders	Fabrazyme 1.0 mg/kg	1	15
other	EOSINOPHILIA	Blood and lymphatic system disorders	Fabrazyme 0.5 mg/kg	0	16
other	EOSINOPHILIA	Blood and lymphatic system disorders	Fabrazyme 1.0 mg/kg	1	15
other	LEUKOPENIA	Blood and lymphatic system disorders	Fabrazyme 0.5 mg/kg	0	16
other	LEUKOPENIA	Blood and lymphatic system disorders	Fabrazyme 1.0 mg/kg	2	15
other	LYMPHADENOPATHY	Blood and lymphatic system disorders	Fabrazyme 0.5 mg/kg	2	16
other	LYMPHADENOPATHY	Blood and lymphatic system disorders	Fabrazyme 1.0 mg/kg	0	15
other	MONOCYTOPENIA	Blood and lymphatic system disorders	Fabrazyme 0.5 mg/kg	0	16
other	MONOCYTOPENIA	Blood and lymphatic system disorders	Fabrazyme 1.0 mg/kg	1	15

1 to 10 of 534Source ClinicalTrials.gov

See all 534 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Fabrazyme 0.5 mg/kg
Total, all-cause mortality	Fabrazyme 1.0 mg/kg
Total, other adverse events	Fabrazyme 0.5 mg/kg	16	16
Total, other adverse events	Fabrazyme 1.0 mg/kg	15	15
Total, serious adverse events	Fabrazyme 0.5 mg/kg	6	16
Total, serious adverse events	Fabrazyme 1.0 mg/kg	5	15

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
23531228	DERIVED	PubMed
25955246	DERIVED	PubMed

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
Fabrazyme 0.5 mg/kg vs Fabrazyme 1.0 mg/kg: 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.

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.

GET https://conductscience.org/api/trials/v1/NCT00701415

GET https://conductscience.org/api/trials/v1/NCT00701415/results?page=2

API reference For AI assistants