Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Completed

Conditions studied: Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

In brief

The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals; to provide a resource to physicians and patients by providing information for optimizing patient care based on aggregate data; to characterize the clinical response to long-term Naglazyme® (galsulfase) treatment; to further characterize the long-term safety of Naglazyme® treatment.

Key facts

Study ID

NCT00214773

Run by

BioMarin Pharmaceutical

People needed

237

Starts

2005-07-01

Expected to finish

2020-05-01

Last updated by the study team

2021-04-05

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• All patients must meet the following criteria to qualify for enrollment in the CSP:
• Patient or patient's parent or legal guardian, if child is under 18 year old or is unable to consent, has provided a signed Patient Information and Authorization Form.
• Patient has laboratory results confirming a diagnosis of MPS VI disease based on detection of deficient ARSB activity (on fibroblasts, leucocytes or dried blood spots)and/or abnormality on the ARSB gene.
• Patient is willing to undergo general assessments to establish baseline data or permits physician to enter assessment data recorded prior to CSP entry if available in the patient's medical records. General assessments include: urinary GAG level, urinary protein level, serum sample for antibody levels, height, weight, and patient history.

Where it is running

• University of California, Irvine — Irvine, California, United States
• Children's Hospital of Los Angeles — Los Angeles, California, United States
• Children's Hospital and Research Center Oakland — Oakland, California, United States
• Emory University — Decatur, Georgia, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Indiana University School of Medicine — Indianapolis, Indiana, United States
• Baton Rouge Clinic — Baton Rouge, Louisiana, United States
• Tulane University Medical Center — New Orleans, Louisiana, United States
• Johns Hopkins Univeristy School of Medicine — Baltimore, Maryland, United States
• Children's Health Care — Minneapolis, Minnesota, United States
• University of Minnesota - Fairview University Medical Center — Minneapolis, Minnesota, United States
• Children's Mercy Hospital — Kansas City, Missouri, United States
• St. Joseph's Healthcare — Paterson, New Jersey, United States
• New York University — New York, New York, United States
• Fullerton Genetic Center — Asheville, North Carolina, United States
• Carolinas Medical Center — Charlotte, North Carolina, United States
• Oregon Health and Science University — Portland, Oregon, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Sioux Valey Children's Speciality Clinics — Sioux Falls, South Dakota, United States
• University of Utah Medical Center — Salt Lake City, Utah, United States
• Children's Hospital of the King's Daughters — Norfolk, Virginia, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Westmead Hospital — Wentworthville, New South Wales, Australia
• Royal Children's Hospital, Brisbane — Brisbane, Queensland, Australia

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.