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N-acetylgalactosamine 4-sulfatase

Phase 3

Mucopolysaccharidosis VI | Small molecule | Rare Disease |BioMarin Pharmaceutical Inc.|Last Updated: Feb 2, 2010

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials4
Total Enrollment78

FDA Designations

No designations recorded

Clinical trial landscape

N-acetylgalactosamine 4-sulfatase · 4 trials · 1 indication

Phase 3 2Phase 2 1Phase 1 1
NCT00104234Study of rhASB in Patients With Mucopolysaccharidosis VIMucopolysaccharidosis VI
COMPLETED39 Analytics
NCT00067470Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VIMucopolysaccharidosis VI
COMPLETED39 Analytics
PHASE3COMPLETED
Study of rhASB in Patients With Mucopolysaccharidosis VI
Mucopolysaccharidosis VI
NCT0010423439
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PHASE3COMPLETED
Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
Mucopolysaccharidosis VI
NCT0006747039
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Study Endpoints

Primary Endpoints

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.

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

urinary glycoaminoglycan(uGAG)levels
weeks 1, 4, 6, 8, 12
Lysosomal storage disease

Secondary Endpoints

3-Minute Stair Climb
Baseline ASB-03-05 through week 96 of ASB-03-06.
Change in Urinary Glycosaminoglycans (GAG) Level
72 weeks
Change From Baseline in 3-minute Stair Climb at 24 Weeks
baseline and 24 weeks
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
rhASB/rhASBOTHERN-acetylgalactosamine 4-sulfatase
Placebo/rhASBOTHER -
PlaceboPLACEBO_COMPARATOR -
rhASBACTIVE_COMPARATOR -

Interventions

NameTypeDescription
N-acetylgalactosamine 4-sulfataseDRUG -
Placebo/rhASBDRUG -
PlaceboDRUG -
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Eligibility Criteria

Age Range7 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Patient consent * Patient must have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions * If female of childbearing potential, patient must have a negative pregn...

Countries:United States
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Frequently asked questions about N-acetylgalactosamine 4-sulfatase

What is N-acetylgalactosamine 4-sulfatase used for?

N-acetylgalactosamine 4-sulfatase is used for the treatment of Mucopolysaccharidosis VI (MPS VI), a rare genetic disorder. It is an investigational enzyme replacement therapy being developed by BioMarin Pharmaceutical Inc. (BMRN). The drug is currently in Phase 3 clinical development and has completed four clinical trials.

What does N-acetylgalactosamine 4-sulfatase target?

N-acetylgalactosamine 4-sulfatase targets the enzyme deficiency that causes Mucopolysaccharidosis VI. It is a recombinant form of the human enzyme N-acetylgalactosamine 4-sulfatase, which is deficient in patients with MPS VI. By replacing this enzyme, the drug aims to address the underlying metabolic defect in the condition.

Who makes N-acetylgalactosamine 4-sulfatase?

N-acetylgalactosamine 4-sulfatase is developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded under the ticker symbol BMRN. BioMarin is conducting the clinical development of this drug for the treatment of Mucopolysaccharidosis VI.

What phase is N-acetylgalactosamine 4-sulfatase in?

N-acetylgalactosamine 4-sulfatase is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The drug has completed four clinical trials, including two Phase 3 studies, but it remains in the clinical development stage.

What clinical trials is N-acetylgalactosamine 4-sulfatase in?

N-acetylgalactosamine 4-sulfatase has completed four clinical trials, all in the United States. These include NCT00048620 (Phase 1), NCT00048711 (Phase 2), NCT00067470 (Phase 3), and NCT00104234 (Phase 3). All trials studied the drug in patients with Mucopolysaccharidosis VI, with the Phase 3 trials enrolling 39 patients each.

Is N-acetylgalactosamine 4-sulfatase the same as galsulfase?

N-acetylgalactosamine 4-sulfatase is the same as galsulfase, which is the recombinant form of the enzyme. The drug is also known by the name rhASB (recombinant human N-acetylgalactosamine 4-sulfatase). It is being developed by BioMarin Pharmaceutical Inc. for the treatment of Mucopolysaccharidosis VI.