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

Mucopolysaccharidosis VI Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00299000
• Other study ID #: ASB-008
• Status: Completed
• Phase: Phase 4
• First received: March 2, 2006
• Last updated: July 19, 2011
• Start date: May 2006
• Est. completion date: April 2009

Study information

• Verified date: July 2011
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: April 2009
• Est. primary completion date: April 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 1 Year

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Prevention

Related Conditions & MeSH terms

• Maroteaux-Lamy Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis VI

Intervention

Drug:
• Naglazyme
Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution

Locations

Country	Name	City	State
France	Hospital Femme Mere Enfant Centre	Lyon
Portugal	Hospital PediAtrico de Coimbra	Coimbra
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	Children's Hospital Oakland	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  France,  Portugal, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Height		52 weeks	No
Primary	Change in Weight		52 weeks	No
Primary	Change in Haed Circumference		52 weeks	No
Secondary	Change in Urinary Glycosaminoglycan Levels	Change in urinary GAG levels was calculated from baseline to week 52 of treatment.	minimum 52 weeks of dosing	No

External Links

•   BioMarin Pharmaceutical Inc Website