Mucopolysaccharidosis VIPhase 3NCT00067470
Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
A Phase 3 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 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. Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Mucopolysaccharidosis VI
- What is being tested?
- N-acetylgalactosamine 4-sulfatase
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 39 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2003to Mar 2004
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 7 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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 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
- Mucopolysaccharidosis VI
- Being tested
- Placebo, 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.
PlaceboDrug
N-acetylgalactosamine 4-sulfataseDrug
Also called rhASB
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| rhASB | Active comparator | Not described Receives: Drug: N-acetylgalactosamine 4-sulfatase |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: baseline and 24 weeks
Secondary outcomes (2)
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
Measured: baseline and 24 weeks
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
Glycosaminoglycan (GAG) level measured in urine
Measured: baseline and 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Aug 2003 (estimated)
- Study startSep 2003
- Study completionMar 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00067470
- Sponsor's protocol ID
- ASB-03-05
- Status last verified
- Dec 2009
- First submitted
- 20 Aug 2003
- Last updated
- 24 Dec 2009
- Results posted
- Yes, on ClinicalTrials.gov
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
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
- ClinicalTrials.gov ID
- NCT00067470
- Acronym / sponsor ID
- ASB-03-05
- Conditions
- Mucopolysaccharidosis VI
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Sep 2003
- Study completion
- Mar 2004
- First posted
- 25 Aug 2003 (estimated)
- Results
- Posted 17 Aug 2009
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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| rhASB | Active comparator | Drug: N-acetylgalactosamine 4-sulfatase |
Who may qualify
- Ages
- 7 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 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
Key exclusion criteria (8 listed)
- 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
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 |
|---|---|---|
| Placebo | Drug | Placebo |
| N-acetylgalactosamine 4-sulfataseAlso: rhASB | Drug | rhASB |
Primary outcome
Change From Baseline in 12-minute Walk Test at 24 Weeks
Time frame: baseline and 24 weeks
Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
2 secondary outcomes
Change From Baseline in 3-minute Stair Climb at 24 Weeks
Time frame: baseline and 24 weeks
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
Time frame: baseline and 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Dec 2009.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33874971Tandon 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.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 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 8 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 24 Dec 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Aug 2009). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in 12-minute Walk Test at 24 Weeks (time frame: baseline and 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00067470, which also lists 1 linked publication.
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 24 Dec 2009.