Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
Stopped early · Not applicable
Conditions studied: Maroteaux-Lamy Syndrome
In brief
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.
Key facts
- Study ID
- NCT02156674
- Run by
- Masonic Cancer Center, University of Minnesota
- People needed
- 1
- Starts
- 2016-01-26
- Expected to finish
- 2019-12-01
- Last updated by the study team
- 2020-12-23
Who can join
Age: 3 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- University of Minnesota Medical Center, Fairview — Minneapolis, Minnesota, United States
Full record on ClinicalTrials.gov
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