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Completed

NCT Number: NCT00299000

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

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.

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Key information

Conditions

Mucopolysaccharidosis VI Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Maroteaux-Lamy Syndrome Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

Up to 1 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Hospital Femme Mere Enfant Centre, Lyon, France

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Naglazyme

Drug

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

Primary outcomes

  1. Change in Height

    Time frame: 52 weeks

  2. Change in Weight

    Time frame: 52 weeks

  3. Change in Haed Circumference

    Time frame: 52 weeks

Secondary outcomes

  1. Change in Urinary Glycosaminoglycan Levels

    Time frame: minimum 52 weeks of dosing

    Change in urinary GAG levels was calculated from baseline to week 52 of treatment.

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study 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)

Important dates

Study start
2006
Primary completion
2009
Study completion
2009
First posted
Mar 6, 2006
Registry last updated
Jul 22, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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