MPS IV ANCT00787995
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
An observational study of MPS IV A and Mucopolysaccharidosis IVA, run by BioMarin Pharmaceutical at 15 sites in 10 countries.
Trial summary
This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- How many people
- 353 participants
- Where
- 15 sitesin 10 countries
- Study dates
- Oct 2008to Jul 2014
- Phase
- Observational
- 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
Study population: Subjects diagnosed with MPS IVA
Who may be able to join
- Willing and able to provide written, signed informed consent, or in the case of subjects age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
- Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
- Willing and able to comply with all study procedures.
Who cannot join
- Use of any investigational product or investigational medical device within 30 days prior to screening.
- Previous hematopoietic stem cell transplant (HSCT).
- Concurrent disease or condition that would interfere with study participation or pose a safety concern.
Trial locations and contacts
15 sites in 10 countries.
15 sites in 10 countries
United Kingdom3 sites
Study site
Birmingham
Study site
London
Study site
Manchester
United States2 sites
Study site
Chicago, Illinois
Study site
Oakland, California
About this study
Official title: A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.
- Conditions
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Keywords
- Mucopolysaccharidosis IV type A, MPS IV Type A, Mucopolysaccharidosis IVA, MPS IVA, Morquio A Syndrome, Lysosomal Storage Disorder, LSD, N-acetylgalactosamine-6-sulfatase, galactose-6-sulfatase, GALNS, enzyme replacement therapy, ERT
- Medical subject headings
- Mucopolysaccharidosis IV, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| MPS IVA | Subjects with mucopolysaccharidosis IVA (Morquio Syndrome) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Endurance
Measured: Study Visit
Respiratory Function
Measured: Study Visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2008 (estimated)
- Study startOct 2008
- Primary completionJul 2014
- Study completionJul 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for MPS IV AStudies 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
- Observational
- Observational model
- Case only
- Enrolment
- 353 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Celeste Decker, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00787995
- Sponsor's protocol ID
- MOR-001
- Status last verified
- Dec 2014
- First submitted
- 6 Nov 2008
- Last updated
- 8 Dec 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED. Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome. Bioanalysis. 2011 Aug;3(16):1855-66. doi: 10.4155/bio.11.172.
PubMed 21877895
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
- ClinicalTrials.gov ID
- NCT00787995
- Acronym / sponsor ID
- MOR-001
- Conditions
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Keywords
- Mucopolysaccharidosis IV type A, MPS IV Type A, Mucopolysaccharidosis IVA, MPS IVA, Morquio A Syndrome, Lysosomal Storage Disorder, LSD, N-acetylgalactosamine-6-sulfatase
- Study type
- Observational
- Recruitment status
- Terminated
- Enrolment
- 353 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Oct 2008
- Primary completion
- Jul 2014 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 10 Nov 2008 (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.
This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.
Study design
- Observational model
- Case only
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MPS IVA | – | –Subjects with mucopolysaccharidosis IVA (Morquio Syndrome) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Subjects diagnosed with MPS IVA
Key inclusion criteria (3 listed)
- Willing and able to provide written, signed informed consent, or in the case of subjects age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
- Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
- Willing and able to comply with all study procedures.
Key exclusion criteria (3 listed)
- Use of any investigational product or investigational medical device within 30 days prior to screening.
- Previous hematopoietic stem cell transplant (HSCT).
- Concurrent disease or condition that would interfere with study participation or pose a safety concern.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Endurance
Time frame: Study Visit
Respiratory Function
Time frame: Study Visit
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2014.
- Derived from this study: PubMed 21877895Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED. Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome. Bioanalysis. 2011 Aug;3(16):1855-66. doi: 10.4155/bio.11.172.
- BioMarin Pharmaceutical Inc. websiteLink provided by the sponsor.
Related trials for MPS IV A
RecruitingObservational study
Cardiac Structure and Function in MPS
MPS I, MPS IV A
Enrolling by invitationObservational study
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
RecruitingObservational study
Non-invasive Functional Assessment and Pathogenesis of Morquio A
Mucopolysaccharidosis IV Type A, Morquio A Syndrome
RecruitingObservational study
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Mucopolysaccharidosis I, Mucopolysaccharidosis II
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.
Celeste Decker, MD study director
BioMarin Pharmaceutical
15 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of MPS IV A and Mucopolysaccharidosis IVA, run by BioMarin Pharmaceutical at 15 sites in 10 countries.
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 3 inclusion and 3 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: stopped early, as of the sponsor's last update on 8 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United Kingdom, United States, Brazil, France, Argentina and 5 more countries. 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.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
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?
Endurance (time frame: Study Visit). 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?
On the ClinicalTrials.gov record for NCT00787995, 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 8 Dec 2014.