Mucopolysaccharidosis VIPhase 2NCT00048711
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
A Phase 2 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 evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI) Read more about this study
- Who is it for?
- Ages 5 years and older
- 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
- Mar 2002to Jun 2006
- Phase
- Phase 2
- 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 5 years and older
- 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 biochemical or genetic proof of MPS VI
- Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 12-minute walk test
- If female of childbearing potential, patient must have a negative pregnancy test
Who cannot join
- Patient is under consideration for or has undergone a successful bone marrow transplant (BMT).
- 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
- Patient has a 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
The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (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.
12-minute walk test
Measured: weeks 6, 12, 24, 48, 96 and 144
urinary glycoaminoglycan(uGAG)levels
Measured: weeks 1, 4, 6, 8, 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2002 (estimated)
- Study startMar 2002
- Study completionJun 2006
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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Stuart J Sweidler, MD, Ph.D., Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00048711
- Sponsor's protocol ID
- ASB-01-04
- Status last verified
- Aug 2009
- First submitted
- 6 Nov 2002
- Last updated
- 25 Aug 2009
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
- ClinicalTrials.gov ID
- NCT00048711
- Acronym / sponsor ID
- ASB-01-04
- Conditions
- Mucopolysaccharidosis VI
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Mar 2002
- Study completion
- Jun 2006 (actual)
- First posted
- 8 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 evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI)
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patient consent
- Patient must be five years of age or older
- Patient must have documented biochemical or genetic proof of MPS VI
- Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 12-minute walk test
- If female of childbearing potential, patient must have a negative pregnancy test
Key exclusion criteria (7 listed)
- Patient is under consideration for or has undergone a successful bone marrow transplant (BMT).
- 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
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
12-minute walk test
Time frame: weeks 6, 12, 24, 48, 96 and 144
urinary glycoaminoglycan(uGAG)levels
Time frame: weeks 1, 4, 6, 8, 12
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 25 Aug 2009.
- Related InfoLink provided by the sponsor.
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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 Sweidler, 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 2 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 5 inclusion and 7 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 25 Aug 2009. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
12-minute walk test (time frame: weeks 6, 12, 24, 48, 96 and 144). The study lists 2 primary outcomes in all. 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 25 Aug 2009.