TerminatedNot Applicable

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Stopped: Slow accrual

United States1 participantsStarted 2016-01-26

Plain-language summary

This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.

Who can participate

Age range

3 Years

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: * Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) treated with a prior allogeneic transplant \>2 years previously * Persons currently receiving Naglazyme may be accepted into the study * Age \> 2 years * \>10% engrafted based on most recent testing * Willing to commit to traveling to the University of Minnesota every 6 months * Written informed consent with parent/guardian consent for children \< 18 years of age or persons unable to consent with minor assent if appropriate Exclusion Criteria: * History of cardiac or pulmonary insufficiency or those requiring continuous supplemental oxygen * Pregnant or breastfeeding * Any condition that, in the view of the investigator, places the patient at high risk of poor treatment compliance or of not completing the study

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 in Urinary Glycosaminoglycan (GAG) Excretion

Timeframe: Baseline and 2 years

Change in Distance Traveled

Timeframe: Baseline and 2 years

Change in Neurocognitive Ability

Timeframe: Baseline and 2 years

Trial details

NCT IDNCT02156674

SponsorMasonic Cancer Center, University of Minnesota

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2019-12-01

Results submitted2020-12-01

View on ClinicalTrials.gov More Maroteaux-Lamy Syndrome trials

Who can participate

Age range

3 Years

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.