MPS INCT02067650
Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis
An observational study of MPS I and MPS IV, run by Children's Hospital of Eastern Ontario at 1 site in Canada.
Trial summary
Hardly any imaging studies specifically addressing the articular and periarticular structures in MPS patients are available at this point. The investigators propose to do for the first time a pilot study using musculoskeletal ultrasound in patients with various types of MPS. Read more about this study
- Who is it for?
- Ages 2 to 99 years
- How many people
- 18 participants
- Where
- 1 siteCanada
- Study dates
- Sep 2013to Dec 2016
- 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 2 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with MPS I and IV
Who may be able to join
- Diagnosis of Mucopolysaccharidosis
- Ability to collaborate with the ultrasound assessment
- Written informed consent from parents and patients over 14 years of age, assent from children 7 to 14 years
Who cannot join
- Inability to collaborate with the ultrasound assessment
- Acute trauma to a joint that will be assessed in the 3 months preceding the study
- Coexistence of other pathology that is known to affect the respective joints
Trial locations and contacts
1 site in 1 country.
About this study
Hardly any imaging studies specifically addressing the articular and periarticular structures in MPS patients are available at this point. The investigators propose to do for the first time a pilot study using musculoskeletal ultrasound in patients with various types of MPS.
- Medical subject headings
- Mucopolysaccharidosis IV
What is being measured
The primary outcome is the main question the study is designed to answer.
Power Doppler
The primary objective is to determine whether patients with various types of MPS and at various stages of the disease exhibit abnormalities of the finger, wrist and knee joints as assessed by grey scale and Power Doppler ultrasonography.
Measured: one time exam
Secondary outcomes (5)
Ultrasound scanning technique
To determine the optimal ultrasound scanning technique for MPS patients given the potentially advanced joint contractures
Measured: one time scan
Findings of synovitis or tenosynovitis
To determine whether there are ultrasound findings indicative of synovitis or tenosynovitis
Measured: one time scan
Differences between MPS I and IV
To determine whether differences exist between MPS I and MPS IV
Measured: one time scan
Differences between ERT and BMT
To determine whether differences exist between patients with or without Enzyme Replacement Therapy (ERT) or Bone Marrow Transplants
Measured: one time scan
Differences between severe and attenuated phenotypes
To determine whether differences exist between severe and attenuated phenotypes
Measured: one time scan
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2014 (estimated)
- Study startSep 2013
- Primary completionMar 2016
- Study completionDec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MPS IStudies 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
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Eastern Ontario
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Johannes Roth, Children's Hospital of Eastern Ontario
- Investigators
- Johannes Roth, MD, Principal investigator, Children's Hospital of Eastern Ontario
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02067650
- Sponsor's protocol ID
- MPSUS
- Status last verified
- Jan 2018
- First submitted
- 13 Aug 2013
- Last updated
- 24 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis
- ClinicalTrials.gov ID
- NCT02067650
- Acronym / sponsor ID
- MPSUS
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Children's Hospital of Eastern OntarioUniversity, hospital or other
- Study start
- Sep 2013 (actual)
- Primary completion
- Mar 2016 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 20 Feb 2014 (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.
Hardly any imaging studies specifically addressing the articular and periarticular structures in MPS patients are available at this point. The investigators propose to do for the first time a pilot study using musculoskeletal ultrasound in patients with various types of MPS.
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 2 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with MPS I and IV
Key inclusion criteria (3 listed)
- Diagnosis of Mucopolysaccharidosis
- Ability to collaborate with the ultrasound assessment
- Written informed consent from parents and patients over 14 years of age, assent from children 7 to 14 years
Key exclusion criteria (3 listed)
- Inability to collaborate with the ultrasound assessment
- Acute trauma to a joint that will be assessed in the 3 months preceding the study
- Coexistence of other pathology that is known to affect the respective joints
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
Power Doppler
Time frame: one time exam
The primary objective is to determine whether patients with various types of MPS and at various stages of the disease exhibit abnormalities of the finger, wrist and knee joints as assessed by grey scale and Power Doppler ultrasonography.
5 secondary outcomes
Ultrasound scanning technique
Time frame: one time scan
Findings of synovitis or tenosynovitis
Time frame: one time scan
Differences between MPS I and IV
Time frame: one time scan
Differences between ERT and BMT
Time frame: one time scan
Differences between severe and attenuated phenotypes
Time frame: one time scan
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 24 Jan 2018.
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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.
Johannes Roth, MD principal investigator
Children's Hospital of Eastern Ontario
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 I and MPS IV, run by Children's Hospital of Eastern Ontario at 1 site in Canada.
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: finished, as of the sponsor's last update on 24 Jan 2018. 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 Canada. 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?
Power Doppler (time frame: one time exam). 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 24 Jan 2018.