Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Endocrine |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | 3 - Any |
| Updated: | 9/28/2018 |
| Start Date: | January 26, 2016 |
| End Date: | July 1, 2019 |
Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome
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.
patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have
previously been treated with an allogeneic transplant.
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
We found this trial at
1
site
2450 Riverside Ave
Minneapolis, Minnesota 55454
Minneapolis, Minnesota 55454
(612) 273-3000
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