Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

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Trial Status Terminated

Trial Identifier

NCT02156674

Condition

MPS VI, Maroteaux-Lamy Syndrome

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

From 3 Years

Healthy Volunteers

No

Drug/Treatment:

Naglazyme®

Phase:

Na

Study Type:

Interventional

Number of Participants:

1

Study Started:

2016-01-26

Study Updated:

2020-12-23

Trial Locations

  • University of Minnesota Medical Center, Fairview

    Minneapolis, Minnesota, United States

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 stud

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