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

Sponsor:	BioMarin Pharmaceutical
Information provided by:	BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:	NCT00299000

Purpose

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.

Condition	Intervention	Phase
Mucopolysaccharidosis VI Maroteaux-Lamy Syndrome	Drug: Naglazyme	Phase 4

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Prevention
Official Title:	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)

Resource links provided by NLM:

Genetics Home Reference related topics: mucopolysaccharidosis type VI

Drug Information available for: Galsulfase

U.S. FDA Resources

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:

Change in Height [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Change in Weight [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Change in Haed Circumference [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Change in Urinary Glycosaminoglycan Levels [ Time Frame: minimum 52 weeks of dosing ] [ Designated as safety issue: No ]
Change in urinary GAG levels was calculated from baseline to week 52 of treatment.

Enrollment:	4
Study Start Date:	May 2006
Study Completion Date:	April 2009
Primary Completion Date:	April 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Naglazyme, 1.0 mg/kg Dose comparison	Drug: Naglazyme Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution Other Names: recombinant human N-acetylgalactosamine 4-sulfatase rh-arylsulfatase B rhASB
Naglazyme, 2.0 mg/kg Dose Comparison	Drug: Naglazyme Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution Other Names: recombinant human N-acetylgalactosamine 4-sulfatase rh-arylsulfatase B rhASB

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

Ages Eligible for Study:	up to 1 Year
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00299000

Locations

United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Children's Hospital Oakland
Oakland, California, United States, 94609
France
Hospital Femme Mere Enfant Centre
Lyon, France, 69677
Portugal
Hospital PediAtrico de Coimbra
Coimbra, Portugal, 3000-076

Sponsors and Collaborators

BioMarin Pharmaceutical

Investigators

Study Director:

Celeste Decker, MD

BioMarin Pharmaceutical

More Information

Additional Information:

BioMarin Pharmaceutical Inc Website This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Celeste Decker, MD, BioMarin Pharmaceutical Inc.
ClinicalTrials.gov Identifier:	NCT00299000 History of Changes
Other Study ID Numbers:	ASB-008
Study First Received:	March 2, 2006
Results First Received:	May 7, 2010
Last Updated:	July 19, 2011
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Mucopolysaccharidoses
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn

Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 21, 2012