Trial · NCT00104234

Study of rhASB in Patients With Mucopolysaccharidosis VI

NCT00104234, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2009-03-06

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of rhASB in Patients With Mucopolysaccharidosis VI. Trials, record NCT00104234. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00104234

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Trial

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

Title: Study of rhASB in Patients With Mucopolysaccharidosis VI
NCT number: NCT00104234
Status: Completed
Phases: Phase 3
Enrollment: 39 participants, actual
Lead sponsor: BioMarin Pharmaceutical
Conditions: Mucopolysaccharidosis VI
Interventions: N-acetylgalactosamine 4-sulfatase, Placebo/rhASB
First posted: 2005-02-25
Last updated: 2010-02-02
Results first posted: 2009-03-06

Source ClinicalTrials.gov

Registered outcomes3

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

List	Outcome	Time frame	Description
Primary	12-Minute Walk Test	Baseline of ASB-03-05 through week 96 of ASB-03-06	Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.
Secondary	3-Minute Stair Climb	Baseline ASB-03-05 through week 96 of ASB-03-06.	Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group.
Secondary	Change in Urinary Glycosaminoglycans (GAG) Level	72 weeks	Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24.

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	12-Minute Walk Test	Baseline of ASB-03-05 through week 96 of ASB-03-06	The efficacy analysis included all subjects who continued in the extension study (ASB-03-06) except the 1 subject from the rhASB/rhASB group and 1 subject from the placebo/rhASB group who missed the Week 96 measurement.	meters	MEAN
SECONDARY	3-Minute Stair Climb	Baseline ASB-03-05 through week 96 of ASB-03-06.	The efficacy analysis included all subjects who continued in the extension study (ASB-03-06) except 1 subject from the placebo/rhASB group who missed the Week 96 measurement.	stairs/min	MEAN
SECONDARY	Change in Urinary Glycosaminoglycans (GAG) Level	72 weeks	rhASB/rhASB and placebo/rhASB groups were combined for the analysis, representing 72 weeks of rhASB treatment in each group.	microgram/mg creatinine	MEAN

Source ClinicalTrials.gov

Results by group5

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

Outcome	Group	Value	Spread	Units
12-Minute Walk Test	rhASB/rhASB	187	Standard Deviation 187	meters
12-Minute Walk Test	Placebo/rhASB	118	Standard Deviation 127	meters
3-Minute Stair Climb	rhASB/rhASB	13.1	Standard Deviation 16.1	stairs/min
3-Minute Stair Climb	Placebo/rhASB	11.1	Standard Deviation 10.0	stairs/min
Change in Urinary Glycosaminoglycans (GAG) Level	All Participants (N=37)	-231	Standard Deviation 91	microgram/mg creatinine

Source ClinicalTrials.gov

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	rhASB/rhASB	19
Overall Study	STARTED	Placebo/rhASB	20
Overall Study	COMPLETED	rhASB/rhASB	19
Overall Study	COMPLETED	Placebo/rhASB	19
Overall Study	NOT COMPLETED	rhASB/rhASB	0
Overall Study	NOT COMPLETED	Placebo/rhASB	1

Source ClinicalTrials.gov

Adverse events169

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	Ear infection	Infections and infestations	All Participants	17	38	17
other	Nasopharyngitis	Infections and infestations	All Participants	16	38	16
other	Upper respiratory tract infection	Infections and infestations	All Participants	15	38	15
other	Gastroenteritis	Infections and infestations	All Participants	12	38	12
other	Bronchitis	Infections and infestations	All Participants	9	38	9
other	Otitis externa	Infections and infestations	All Participants	8	38	8
other	Influenza	Infections and infestations	All Participants	7	38	7
other	Otitis media acute	Infections and infestations	All Participants	7	38	7
other	Sinusitis	Infections and infestations	All Participants	6	38	6
other	Hordeolum	Infections and infestations	All Participants	5	38	5

1 to 10 of 169Source ClinicalTrials.gov

See all 169 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	All Participants
Total, other adverse events	All Participants	38	38
Total, serious adverse events	All Participants	14	38

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
33874971	DERIVED

Source 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 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.

GET https://conductscience.org/api/trials/v1/NCT00104234

GET https://conductscience.org/api/trials/v1/NCT00104234/events?page=2

API reference For AI assistants