MPS INCT07361536
Cardiac Structure and Function in MPS
An observational study of MPS I and MPS IV A, run by Children's Hospital of Orange County at 3 sites in United States.
Trial summary
The purpose of this study is to better understand how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS). The investigators are looking at certain substances in the body called GAGs and proteoglycans to see how they affect the heart. Read more about this study
- Who is it for?
- Ages 0 to 99 yearsHealthy volunteers can take part
- Conditions the study is for
- MPS I, MPS IV A, MPS - Mucopolysaccharidosis
- How many people
- 240 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Sep 2025to 30 Apr 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 0 to 99 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Participants will be drawn from each participating site's existing patient population. Eligible individuals who meet protocol-defined criteria and are willing to take part in annual study visits will be invited to enroll. The study population will therefore consist of patients receiving care at the participating institutions who consent to ongoing observational follow-up.
Who may be able to join
- Any Participant with a molecularly confirmed diagnosis of mucopolysaccharidosis is eligible to enroll in this study
- Any healthy participant without a diagnosis of mucopolysaccharidosis whose age and biological sex can be matched with an enrolled mucopolysaccharidosis participant
- Parental / patient informed consent
Who cannot join
- Any reason that the investigators would deem a patient not eligible to participate in this study
- Inability to participate in the assessments required for this study
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Not yet recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
Donald Dengel, PhD, 612-626-9701, denge001@umn.edu
- Recruiting
Children'S Hospital of Orange County
Orange, California, 92868
Nina Movsesyan, 714-509-3008, Metabolic.CRC@choc.org
- Recruiting
Saint Louis University
St Louis, Missouri, 63104
Adriana M Montano, PhD, 314-977-9104, adriana.montano@health.slu.edu
About this study
Official title: Cardiovascular Structure and Function in Mucopolysaccharidosis Types I and IVA
The purpose of this study is to better understand how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS). The investigators are looking at certain substances in the body called GAGs and proteoglycans to see how they affect the heart. The investigators also want to find reliable blood and urine markers that can help us track heart health and guide future treatments.
This study aims to answer two main questions:
Read more
- Do people with MPS show faster changes in their blood vessels over time (such as thickening or stiffening of the carotid artery) compared to people without MPS?
- Do people with MPS have higher levels of certain proteins in their blood (such as clusterin and inflammatory markers) that are linked to blood vessel changes?
What participants will do?
Participants will complete the following tests once a year for 4 years:
- Carotid ultrasound: an imaging test that looks at the blood vessels in the neck.
- Echocardiogram: an ultrasound of the heart.
- Blood draw
- Urine collection
These tests help the investigators track changes in heart and blood vessel health over time.
- Conditions
- MPS I, MPS IV A, MPS - Mucopolysaccharidosis, MPS IVA
- Keywords
- mucopolysaccharidosis, carotid, cardiovascular, biomarker, observational
- Medical subject headings
- Mucopolysaccharidosis IV, Mucopolysaccharidoses
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 | Participants diagnosed with MPS I or MPS IVA | |
| Control | Participants who do not have an MPS diagnosis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cardiac and Carotid Structure and Function
- We aim to annually assess cardiac and carotid structure and function utilizing ultrasonography, in participants with mucopolysaccharidoses and matched control participants
Measured: Year 1, Year 2, Year 3, and Year 4
Secondary outcomes (1)
Measure biomarkers
- We aim to annually measure circulating levels of clusterin, elastin, cathepsin S, and cytokines in participants with mucopolysaccharidoses and matched control participants
Measured: Year 1, Year 2, Year 3, and Year 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2026
- Study start1 Sep 2025
- Primary completion30 Apr 2030 (estimated)
- Study completion30 Apr 2030 (estimated)
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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 240 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Orange County
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07361536
- Sponsor's protocol ID
- 13984001
- Other IDs
- 1R01HL173371-01A1
- Status last verified
- Jan 2026
- First submitted
- 21 Jan 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cardiovascular Structure and Function in Mucopolysaccharidosis Types I and IVA
- ClinicalTrials.gov ID
- NCT07361536
- Acronym / sponsor ID
- 13984001
- Conditions
- MPS I, MPS IV A, MPS - Mucopolysaccharidosis, MPS IVA
- Keywords
- mucopolysaccharidosis, carotid, cardiovascular, biomarker, observational
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 240 participants (estimated)
- Lead sponsor
- Children's Hospital of Orange CountyUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 1 Sep 2025 (actual)
- Primary completion
- 30 Apr 2030 (estimated)
- Study completion
- 30 Apr 2030 (estimated)
- First posted
- 23 Jan 2026 (actual)
- 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 better understand how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS). The investigators are looking at certain substances in the body called GAGs and proteoglycans to see how they affect the heart. The investigators also want to find reliable blood and urine markers that can help us track heart health and guide future treatments.
This study aims to answer two main questions:
- Do people with MPS show faster changes in their blood vessels over time (such as thickening or stiffening of the carotid artery) compared to people without MPS?
- Do people with MPS have higher levels of certain proteins in their blood (such as clusterin and inflammatory markers) that are linked to blood vessel changes?
What participants will do?
Participants will complete the following tests once a year for 4 years:
- Carotid ultrasound: an imaging test that looks at the blood vessels in the neck.
- Echocardiogram: an ultrasound of the heart.
- Blood draw
- Urine collection
These tests help the investigators track changes in heart and blood vessel health over time.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MPS | – | –Participants diagnosed with MPS I or MPS IVA |
| Control | – | –Participants who do not have an MPS diagnosis |
Who may qualify
- Ages
- 0 to 99 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants will be drawn from each participating site's existing patient population. Eligible individuals who meet protocol-defined criteria and are willing to take part in annual study visits will be invited to enroll. The study population will therefore consist of patients receiving care at the participating institutions who consent to ongoing observational follow-up.
Key inclusion criteria (3 listed)
- Any Participant with a molecularly confirmed diagnosis of mucopolysaccharidosis is eligible to enroll in this study
- Any healthy participant without a diagnosis of mucopolysaccharidosis whose age and biological sex can be matched with an enrolled mucopolysaccharidosis participant
- Parental / patient informed consent
Key exclusion criteria (2 listed)
- Any reason that the investigators would deem a patient not eligible to participate in this study
- Inability to participate in the assessments required for this study
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
Cardiac and Carotid Structure and Function
Time frame: Year 1, Year 2, Year 3, and Year 4
1) We aim to annually assess cardiac and carotid structure and function utilizing ultrasonography, in participants with mucopolysaccharidoses and matched control participants
1 secondary outcome
Measure biomarkers
Time frame: Year 1, Year 2, Year 3, and Year 4
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2026.
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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.
3 sites 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 A, run by Children's Hospital of Orange County at 3 sites 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 3 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: looking for participants now, as of the sponsor's last update on 21 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Nina Movsesyan (714-509-3008, Metabolic.CRC@choc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cardiac and Carotid Structure and Function (time frame: Year 1, Year 2, Year 3, and Year 4). 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 21 Oct 2026.