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

Study Overview
=================
Brief 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.

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)

Conditions
-----------------
Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome

Intervention / Treatment
-----------------
* Drug: Naglazyme


Participation Criteria
=================
Eligibility 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

Ages Eligible for Study
-----------------
Maximum Age: 1 Year

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Naglazyme, 1.0 mg/kg<br>Dose comparison | Drug: Naglazyme<br>* Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution<br>* Other names: rhASB;|
| Other: Naglazyme, 2.0 mg/kg<br>Dose Comparison | Drug: Naglazyme<br>* Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution<br>* Other names: rhASB;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Height |  | 52 weeks | 
| Change in Weight |  | 52 weeks | 
| Change in Haed Circumference |  | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Urinary Glycosaminoglycan Levels | Change in urinary GAG levels was calculated from baseline to week 52 of treatment. | minimum 52 weeks of dosing |