Trial · NCT00067470

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

NCT00067470, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2009-08-17

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI. Trials, record NCT00067470. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00067470

On this page

Trial

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

Title: Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
NCT number: NCT00067470
Status: Completed
Phases: Phase 3
Enrollment: 39 participants, actual
Lead sponsor: BioMarin Pharmaceutical
Conditions: Mucopolysaccharidosis VI
Interventions: Placebo, N-acetylgalactosamine 4-sulfatase
First posted: 2003-08-25
Last updated: 2009-12-24
Results first posted: 2009-08-17

Source ClinicalTrials.gov

Registered outcomes3

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

List	Outcome	Time frame	Description
Primary	Change From Baseline in 12-minute Walk Test at 24 Weeks	baseline and 24 weeks	Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
Secondary	Change From Baseline in 3-minute Stair Climb at 24 Weeks	baseline and 24 weeks	Number of stairs climbed per minute in 3 minutes at 24 weeks minus number of stairs climbed per minute in 3 minutes at baseline
Secondary	Change From Baseline in Urinary GAG (uGAG) at 24 Weeks	baseline and 24 weeks	Glycosaminoglycan (GAG) level measured in urine

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	Change From Baseline in 12-minute Walk Test at 24 Weeks	baseline and 24 weeks	The efficacy analysis included all subjects except one from the placebo group who withdrew from the study prior to the week 24 assessment.	meters	MEAN
SECONDARY	Change From Baseline in 3-minute Stair Climb at 24 Weeks	baseline and 24 weeks	The efficacy analysis included all subjects except one from the placbo group who withdrew from the study prior to the week 24 assessment.	stairs/min	MEAN
SECONDARY	Change From Baseline in Urinary GAG (uGAG) at 24 Weeks	baseline and 24 weeks	Baseline uGAG level was measured for 19 rhASB-treated subjects and 20 placebo-treated subjects. However, uGAG levels at 24 weeks were measured for 19 rhASB-treated subjects and 19 placebo-treated subjects, because 1 placebo-treated subject withdrew from the study before week 24.	micrograms per mg creatinine	MEAN

Source ClinicalTrials.gov

Results by group8

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

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in 12-minute Walk Test at 24 Weeks	rhASB		109	Standard Deviation 154	meters
Change From Baseline in 12-minute Walk Test at 24 Weeks	Placebo		26	Standard Deviation 122	meters
Change From Baseline in 3-minute Stair Climb at 24 Weeks	rhASB		7.4	Standard Deviation 9.9	stairs/min
Change From Baseline in 3-minute Stair Climb at 24 Weeks	Placebo		2.7	Standard Deviation 6.9	stairs/min
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks	rhASB	Baseline Urinary GAG Level	346	Standard Deviation 128	micrograms per mg creatinine
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks	Placebo	Baseline Urinary GAG Level	330	Standard Deviation 114	micrograms per mg creatinine
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks	rhASB	24-week Urinary GAG Level	85	Standard Deviation 35	micrograms per mg creatinine
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks	Placebo	24-week Urinary GAG Level	317	Standard Deviation 80	micrograms per 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	19
Overall Study	STARTED	Placebo	20
Overall Study	COMPLETED	rhASB	19
Overall Study	COMPLETED	Placebo	19
Overall Study	NOT COMPLETED	rhASB	0
Overall Study	NOT COMPLETED	Placebo	1

Source ClinicalTrials.gov

Adverse events382

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	Abdominal distension	Gastrointestinal disorders	rhASB	1	19	1
other	Abdominal distension	Gastrointestinal disorders	Placebo	2	20	2
other	Abdominal pain	Gastrointestinal disorders	rhASB	8	19	8
other	Abdominal pain	Gastrointestinal disorders	Placebo	6	20	6
other	Abdominal pain lower	Gastrointestinal disorders	rhASB	1	19	1
other	Abdominal pain lower	Gastrointestinal disorders	Placebo	0	20	0
other	Abdominal pain upper	Gastrointestinal disorders	rhASB	2	19	2
other	Abdominal pain upper	Gastrointestinal disorders	Placebo	0	20	0
other	Abdominal strangulated hernia	Gastrointestinal disorders	rhASB	0	19	0
other	Abdominal strangulated hernia	Gastrointestinal disorders	Placebo	1	20	1

1 to 10 of 382Source ClinicalTrials.gov

See all 382 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	rhASB
Total, all-cause mortality	Placebo
Total, other adverse events	rhASB	19	19
Total, other adverse events	Placebo	20	20
Total, serious adverse events	rhASB	3	19
Total, serious adverse events	Placebo	4	20

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 comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
rhASB vs Placebo: 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/NCT00067470

GET https://conductscience.org/api/trials/v1/NCT00067470/events?page=2

API reference For AI assistants