MPS IVANCT01920828
Gait Analysis in MPS IVA
An observational study of MPS IVA and Morquio Syndrome, run by Oregon Health and Science University at 1 site in United States.
Trial summary
The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- MPS IVA, Morquio Syndrome
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2013to Jun 2017
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with a diagnosis of MPS IVA
Who may be able to join
- -Diagnosis of MPS IVA
Who cannot join
- Non-ambulatory
- Previous enrolled in clinical trial BMN-110
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health and Science University
Portland, Oregon, 97239
About this study
Official title: Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy
The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.
Read the detailed description
The purpose of this 3-year study is to conduct a longitudinal investigation using motion analysis of gait to objectively and quantitatively assess gait characteristics of MPS IVA patients. Data documenting gait characteristics and abnormalities will be gathered in patients initiating enzyme replacement therapy.
Inclusion criteria Individuals with MPS IVA diagnosed by enzyme or molecular studies Exclusion Criteria Individual with MPS IVA previously enrolled in BMN-110 Individual is non-ambulatory The individual is unable to complete longitudinal study visits at least yearly
- Conditions
- MPS IVA, Morquio Syndrome
- Medical subject headings
- Mucopolysaccharidosis IV
What is being measured
The primary outcome is the main question the study is designed to answer.
Measurable changes in gait
Data acquired on patients will be compared with age-matched standardized norms. The normal data is available from a pre-existing Gait Repository, a collection of gait parameters in children without a specific diagnosis studied at Oregon Health and Science University. The results will be converted to centile charts with the aid of a biostatistician. Centile charts will be used to track statistical changes over time in the following gait parameters including but not exclusive to: velocity, cadence, step length, and base of support.
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2013 (estimated)
- Study startAug 2013
- Primary completionJun 2017
- Study completionJun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MPS IVAStudies of the same condition, open and under way.
- 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
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oregon Health and Science University
- Sponsor type
- University, hospital or other
- Collaborators
- University of Utah, BioMarin Pharmaceutical
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01920828
- Sponsor's protocol ID
- OregonHSU-8833
- Status last verified
- Oct 2019
- First submitted
- 8 Aug 2013
- Last updated
- 17 Oct 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT01920828
- Acronym / sponsor ID
- OregonHSU-8833
- Conditions
- MPS IVA, Morquio Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Collaborators
- University of Utah, BioMarin Pharmaceutical
- Study start
- Aug 2013
- Primary completion
- Jun 2017 (actual)
- Study completion
- Jun 2017 (actual)
- First posted
- 12 Aug 2013 (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 this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.
Detailed description
The purpose of this 3-year study is to conduct a longitudinal investigation using motion analysis of gait to objectively and quantitatively assess gait characteristics of MPS IVA patients. Data documenting gait characteristics and abnormalities will be gathered in patients initiating enzyme replacement therapy.
Inclusion criteria Individuals with MPS IVA diagnosed by enzyme or molecular studies Exclusion Criteria Individual with MPS IVA previously enrolled in BMN-110 Individual is non-ambulatory The individual is unable to complete longitudinal study visits at least yearly
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with a diagnosis of MPS IVA
Key inclusion criteria (1 listed)
- -Diagnosis of MPS IVA
Key exclusion criteria (2 listed)
- Non-ambulatory
- Previous enrolled in clinical trial BMN-110
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
Measurable changes in gait
Time frame: 36 months
Data acquired on patients will be compared with age-matched standardized norms. The normal data is available from a pre-existing Gait Repository, a collection of gait parameters in children without a specific diagnosis studied at Oregon Health and Science University. The results will be converted to centile charts with the aid of a biostatistician. Centile charts will be used to track statistical changes over time in the following gait parameters including but not exclusive to: velocity, cadence, step length, and base of support.
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 17 Oct 2019.
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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?
An observational study of MPS IVA and Morquio Syndrome, run by Oregon Health and Science University 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 1 inclusion and 2 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 17 Oct 2019. 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.
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?
Measurable changes in gait (time frame: 36 months). 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 17 Oct 2019.