CompletedNCT00067470Updated Dec 24, 2009Results posted

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

A Phase 3 interventional study of Placebo and N-acetylgalactosamine 4-sulfatase in Mucopolysaccharidosis VI, sponsored by BioMarin Pharmaceutical. Completed at 1 site in United States. Open to participants aged 7 Years and older. Per ClinicalTrials.gov, last updated 2009-12-24.

Sponsored by BioMarin Pharmaceutical · Phase 3, Interventional, and Treatment

Phase: Phase 3
Study type: Interventional
Enrollment: 39
Allocation: Randomized
Ages: 7 Years and older
Sex: All

Study summary

The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

Conditions studied

Mucopolysaccharidosis VI

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In context

Mucopolysaccharidoses

145 studies on the registry are indexed under Mucopolysaccharidoses; 11 are open to participants now.

This study's enrollment of 39 is above the median of 15 across 80 interventional studies indexed under Mucopolysaccharidoses.

Browse Mucopolysaccharidoses studies →

Lead sponsor

BioMarin Pharmaceutical is the lead sponsor of 110 studies on the registry; 13 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 7 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Patient consent
Patient must be seven years of age or older
Patient must have documented biochemical or genetic proof of MPS VI
The patient must be able to walk independently at least 5 meters and no more than 270 meters in the first 6 minutes, or no more then 400 meters total in 12 minutes, in the screening 12-minute walk test
If female of childbearing potential, patient must have a negative pregnancy test

Exclusion criteria

Exclusion Criteria

Patient is under consideration for or has undergone a successful bone marrow transplant (BMT)
Patient refuses or is unable to complete all screening evaluations
Pregnant or lactating patient
Patient has received an investigational drug within 30 days prior to study enrollment
Patient has been previously treated with rhASB
Patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly confound study results or decrease study compliance
The patient has clinically significant spinal cord compression
The patient has known hypersensitivity to rhASB or to components of the active or placebo test solutions

Study design

Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Double
Enrollment: 39 participants (actual)

Study arms

Placebo comparator
Placebo
Drug: Placebo
Active comparator
rhASB
Drug: N-acetylgalactosamine 4-sulfatase

Interventions

DrugPlacebo
DrugN-acetylgalactosamine 4-sulfatase
Also known as: rhASB

What researchers measure

Primary outcomes

Change From Baseline in 12-minute Walk Test at 24 Weeks
Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
Time frame: baseline and 24 weeks

Secondary outcomes

Change From Baseline in 3-minute Stair Climb at 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
Time frame: baseline and 24 weeks
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
Glycosaminoglycan (GAG) level measured in urine
Time frame: baseline and 24 weeks

Results

Posted Aug 17, 2009

Participant flow

Study Locations:Oakland, CA, USA Mainz, Germany Manchester, England Porto Alegre, Brazil Lyon, France Porto, Portugal Study Duration: 24 weeks First enrollment: 21 July 2003 Last Dose Given: 1 April 2004 Last Assessment: 8 April 2004

Participant flow — Overall Study
Milestone	rhASB	Placebo
Started	19	20
Completed	19	19
Not completed	0	1
Withdrew: Withdrawal by subject	0	1

Outcome measures

PrimaryChange From Baseline in 12-minute Walk Test at 24 Weeks

Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline

Time frame:: baseline and 24 weeks
Reported as:: Mean · meters

Change From Baseline in 12-minute Walk Test at 24 Weeks
meters	rhASB	Placebo
Change From Baseline in 12-minute Walk Test at 24 Weeks	109 ± 154	26 ± 122

Statistical analysis

rhASB vs Placebo · Regression, Linear · p = 0.025 · Mean difference (net): 92 · 95% CI 11 to 172

SecondaryChange From Baseline in 3-minute Stair Climb at 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

Time frame:: baseline and 24 weeks
Reported as:: Mean · stairs/min

Change From Baseline in 3-minute Stair Climb at 24 Weeks
stairs/min	rhASB	Placebo
Change From Baseline in 3-minute Stair Climb at 24 Weeks	7.4 ± 9.9	2.7 ± 6.9

Statistical analysis

rhASB vs Placebo · Regression, Linear · p = 0.053 · Mean difference (net): 5.7 · 95% CI -0.1 to 11.5The raw data were adjusted for the observed differences in baseline between the groups and fitted to a longitudinal repeated measures linear model.

SecondaryChange From Baseline in Urinary GAG (uGAG) at 24 Weeks

Glycosaminoglycan (GAG) level measured in urine

Time frame:: baseline and 24 weeks
Reported as:: Mean · micrograms per mg creatinine

Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
micrograms per mg creatinine	rhASB	Placebo
Baseline Urinary GAG Level	346 ± 128	330 ± 114
24-week Urinary GAG Level	85 ± 35	317 ± 80

Adverse events

Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
rhASB	—	3/19 (15.8%)	19/19 (100%)
Placebo	—	4/20 (20%)	20/20 (100%)

Most frequent serious events

Showing 10 of 12

Most frequent serious events
Event	rhASB	Placebo
PneumoniaInfections and infestations	0/19	3/20
Abdominal Strangulated HerniaGastrointestinal disorders	0/19	2/20
ApnoeaInfections and infestations	1/19	0/20
ConvulsionInfections and infestations	1/19	1/20
Corneal lesionNervous system disorders	1/19	0/20
Viral infectionEye disorders	1/19	0/20
Cardiac failure congestiveGastrointestinal disorders	0/19	1/20
HeadacheRespiratory, thoracic and mediastinal disorders	0/19	1/20
IleusRespiratory, thoracic and mediastinal disorders	0/19	1/20
Intracranial pressure increasedNervous system disorders	0/19	1/20

Most frequent other events

Showing 10 of 179

Most frequent other events
Event	rhASB	Placebo
HeadacheNervous system disorders	8/19	12/20
Abdominal painGastrointestinal disorders	8/19	6/20
ArthralgiaMusculoskeletal and connective tissue disorders	8/19	7/20
Ear painEar and labyrinth disorders	8/19	4/20
PyrexiaGeneral disorders	8/19	8/20
Pain in extremityMusculoskeletal and connective tissue disorders	2/19	8/20
Upper respiratory tract infectionInfections and infestations	4/19	7/20
VomitingGastrointestinal disorders	6/19	7/20
CoughRespiratory, thoracic and mediastinal disorders	5/19	6/20
DiarrheaGastrointestinal disorders	3/19	6/20

Baseline characteristics

Age Continuous
Age Continuous(years)	rhASB	Placebo	Total
Mean	13.7 ± 6.47	10.7 ± 4.35	12.2 ± 5.62

Sex: Female, Male
Sex: Female, Male(Participants)	rhASB	Placebo	Total
Female	12	14	26
Male	7	6	13

Race/Ethnicity, Customized
Race/Ethnicity, Customized(participants)	rhASB	Placebo	Total
White, non-Hispanic	15	9	24
Hispanic	1	3	4
Black	1	2	3
Asian	1	1	2
Indigenous	1	1	2
Other	0	4	4

Region of Enrollment
Region of Enrollment(participants)	rhASB	Placebo	Total
United States	2	4	6
Germany	4	4	8
United Kingdom	3	3	6
Brazil	4	4	8
France	2	3	5
Portugal	4	2	6

Study locations

1 site

BioMarin Pharmaceutical Inc.
Novato, California 94949, United States

References and documents

Publications

Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5. PubMed 33874971 ↗

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Dec 24, 2009, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT00067470
Lead sponsor: BioMarin Pharmaceutical
First posted: Aug 25, 2003
Start date: Sep 2003
Completion: Mar 2004
Results posted: Aug 17, 2009
Last update: Dec 24, 2009

View the source record on ClinicalTrials.gov ↗

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