Mucopolysaccharidosis VIPhase 1NCT00048620
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
A Phase 1 study testing N-acetylgalactosamine 4-sulfatase in people with Mucopolysaccharidosis VI, run by BioMarin Pharmaceutical at 1 site in United States.
Trial summary
The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis VI
- What is being tested?
- N-acetylgalactosamine 4-sulfatase
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Sep 2000to Nov 2005
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient consent
- Patient must be five years of age or older
- Patient must have documented diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI
- Leukocyte ASB enzyme activity level less than 20% of the normal range
- Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly.
- Ability to perform all protocol tests
- Ability to stand independently for six minutes
- Sexually active subjects must agree to use an adequate form of contraception
Who cannot join
- History of bone marrow transplantation
- Pregnant or lactating patient
- Use of an investigational drug or device within 30 days prior to study participation.
- A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up
- Known hypersensitivity to rhASB or to components of the study drug
- History of cancer (except low grade and fully resolved skin malignancy)
Trial locations and contacts
1 site in 1 country.
United States1 site
BioMarin Pharmaceutical Inc.
Novato, California, 94949
About this study
Official title: Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.
- Conditions
- Mucopolysaccharidosis VI
- Being tested
- N-acetylgalactosamine 4-sulfatase
- Medical subject headings
- Mucopolysaccharidosis VI
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-acetylgalactosamine 4-sulfataseDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Lysosomal storage disease
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2002 (estimated)
- Study startSep 2000
- Study completionNov 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis VIStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Stuart J Swiedler, MD, Ph.D., Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00048620
- Sponsor's protocol ID
- ASB-00-01
- Status last verified
- Nov 2006
- First submitted
- 4 Nov 2002
- Last updated
- 3 Nov 2006
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2006. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
- ClinicalTrials.gov ID
- NCT00048620
- Acronym / sponsor ID
- ASB-00-01
- Conditions
- Mucopolysaccharidosis VI
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Sep 2000
- Study completion
- Nov 2005
- First posted
- 6 Nov 2002 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Patient consent
- Patient must be five years of age or older
- Patient must have documented diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI
- Leukocyte ASB enzyme activity level less than 20% of the normal range
- Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly.
Key exclusion criteria (6 listed)
- History of bone marrow transplantation
- Pregnant or lactating patient
- Use of an investigational drug or device within 30 days prior to study participation.
- A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up
- Known hypersensitivity to rhASB or to components of the study drug
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| N-acetylgalactosamine 4-sulfatase | Drug | – |
Primary outcome
Lysosomal storage disease
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2006.
- Related InfoLink provided by the sponsor.
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Registry of Patients Diagnosed With Lysosomal Storage Diseases
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stuart J Swiedler, MD, Ph.D. study director
BioMarin Pharmaceutical
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing N-acetylgalactosamine 4-sulfatase in people with Mucopolysaccharidosis VI, run by BioMarin Pharmaceutical at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Nov 2006. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Lysosomal storage disease. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2006; AzTrials last refreshed its copy on 8 Nov 2026.