RecruitingNot Applicable

Non-invasive Functional Assessment and Pathogenesis of Morquio A

United States60 participantsStarted 2021-05-01

Plain-language summary

Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all ages of patients to better understand the progress of their disease and the most serious clinical problems (cervical instability and stenosis, tracheal obstruction, hyperlaxity of joints, hip dysplasia, and small lung capacity). The outcome of this project will lead to a more precise understanding of the skeletal/pulmonary compromise and defining clinical endpoints in this disease for future clinical trials of current or developing therapies.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Patients affected by MPS IVA. The diagnosis of MPS IVA is confirmed by deficient enzyme activity of \< 5% of normal activity level as measured in plasma or leukocytes. Exclusion Criteria: * No

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change of Total body length

Timeframe: baseline, 18 months, 36 months, 48 months

Change of Body mass index (BMI)

Timeframe: baseline, 18 months, 36 months, 48 months

Change of Height velocity

Timeframe: baseline, 18 months, 36 months, 48 months

Change of QOL questionnaire

Timeframe: baseline, 18 months, 36 months, 48 months

Change of Joint Mobility

Timeframe: baseline, 18 months, 36 months, 48 months

Change of Skeletal survey by x-rays

Timeframe: baseline, 36 months

Change of MRI in cervical spine (distance)

Timeframe: baseline, 18 months, 36 months, 48 months

Trial details

NCT IDNCT05284006

SponsorNemours Children's Clinic

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2027-04-30

Contact for this trial

Shunji Tomatsu, MD PhD

3022987336 stomatsu@nemours.org

View on ClinicalTrials.gov More Mucopolysaccharidosis IV Type A trials

Plain-language summary

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Change of Total body length

Timeframe: baseline, 18 months, 36 months, 48 months

Change of Body mass index (BMI)

Timeframe: baseline, 18 months, 36 months, 48 months

Change of Height velocity

Timeframe: baseline, 18 months, 36 months, 48 months

Change of QOL questionnaire

Timeframe: baseline, 18 months, 36 months, 48 months

Change of Joint Mobility

Timeframe: baseline, 18 months, 36 months, 48 months

Change of Skeletal survey by x-rays

Timeframe: baseline, 36 months

Change of MRI in cervical spine (distance)

Timeframe: baseline, 18 months, 36 months, 48 months