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A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI | Clinical Trials | Clareo Health

COMPLETEDPHASE4

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With Maroteaux-Lamy Syndrome (MPS VI)

NCT00299000•BioMarin Pharmaceutical

View on ClinicalTrials.gov

Summary

The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.

Detailed Description

The primary objective of the study was to evaluate the efficacy of two dose levels of Naglazyme in preventing the progression of skeletal dysplasia in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth. The secondary objective of the study was to evaluate the efficacy of the two dose levels of Naglazyme in preventing several measures of disease progression in infants under the age of one year who have MPS VI by monitoring urinary GAGs, gross and fine motor function, cardiac function, vision, hearing, and use of health resources. The safety objective of the study was to evaluate the safety of two dose levels of Naglazyme in infants under the age of one year who have MPS VI.

Eligibility

Age

0 - 1 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Signed informed consent by a parent or legal guardian
•Parent or legal guardian willing and able to comply with all study procedures
•Equal to or greater than 36 weeks estimated gestational age by physical exam at birth
•Has a diagnosis of MPS VI based on a documented prenatal diagnosis or fibroblast or leukocyte N-acetylgalactosamine 4-sulfatase (ASB) enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory
•Is less than one year of age
•Has no evidence of skeletal dysplasia based on physical exam

Exclusion Criteria

•Parent of legal guardian perceived to be unreliable or unavailable for study participation
•Use of any investigational drug within 30 days prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments
•Has concurrent disease or condition that would interfere with study participation or safety (i.e., has previously undergone hematopoietic stem cell transplantation such as bone marrow or cord blood transplantation, or major organ transplantation)
•Any condition that, in the view of the principle investigator, renders the subject at high risk from treatment compliance and/or completing the study
•Has known hypersensitivity to Naglazyme
•Has previously received Naglazyme

Locations (4)

Children's Hospital Los Angeles

Los Angeles, California, United States

Children's Hospital Oakland

Oakland, California, United States

Hospital Femme Mere Enfant Centre

Lyon, France

Hospital PediAtrico de Coimbra

Coimbra, Portugal

Key Dates

Start Date

May 1, 2006

Primary Completion Date

April 1, 2009

Completion Date

April 1, 2009

Last Updated

July 22, 2011

Enrollment

ACTUAL participants

Conditions

Mucopolysaccharidosis VIMaroteaux-Lamy Syndrome

Interventions

Naglazyme

DRUG

Mucopolysaccharidosis VII Disease Monitoring Program

NCT03604835

Mucopolysaccharidosis VIIMPS VII+2 more

View study

UNKNOWNPHASE2

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

NCT03632213

Mucopolysaccharidosis IV AMucopolysaccharidosis VI+7 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: July 22, 2011Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

Inclusion Criteria

Exclusion Criteria

Mucopolysaccharidosis VII Disease Monitoring Program

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

A Study in MPS VI to Assess Safety and Efficacy of Odiparcil

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Stem Cell Transplantation for Hurler

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease