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

Primary Purpose

Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome

Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Naglazyme
Sponsored by
BioMarin Pharmaceutical
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Mucopolysaccharidosis VI

Eligibility Criteria

undefined - 1 Year (Child)All SexesDoes not accept 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

Sites / Locations

  • Children's Hospital Los Angeles
  • Children's Hospital Oakland
  • Hospital Femme Mere Enfant Centre
  • Hospital PediAtrico de Coimbra

Arms of the Study

Arm 1

Arm 2

Arm Type

Other

Other

Arm Label

Naglazyme, 1.0 mg/kg

Naglazyme, 2.0 mg/kg

Arm Description

Dose comparison

Dose Comparison

Outcomes

Primary Outcome Measures

Change in Height
Change in Weight
Change in Haed Circumference

Secondary Outcome Measures

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

Full Information

First Posted
March 2, 2006
Last Updated
July 19, 2011
Sponsor
BioMarin Pharmaceutical
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1. Study Identification

Unique Protocol Identification Number
NCT00299000
Brief Title
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With 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)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2011
Overall Recruitment Status
Completed
Study Start Date
May 2006 (undefined)
Primary Completion Date
April 2009 (Actual)
Study Completion Date
April 2009 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
BioMarin Pharmaceutical

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Naglazyme, 1.0 mg/kg
Arm Type
Other
Arm Description
Dose comparison
Arm Title
Naglazyme, 2.0 mg/kg
Arm Type
Other
Arm Description
Dose Comparison
Intervention Type
Drug
Intervention Name(s)
Naglazyme
Other Intervention Name(s)
recombinant human N-acetylgalactosamine 4-sulfatase, rh-arylsulfatase B, rhASB
Intervention Description
Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution
Primary Outcome Measure Information:
Title
Change in Height
Time Frame
52 weeks
Title
Change in Weight
Time Frame
52 weeks
Title
Change in Haed Circumference
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
Change in Urinary Glycosaminoglycan Levels
Description
Change in urinary GAG levels was calculated from baseline to week 52 of treatment.
Time Frame
minimum 52 weeks of dosing

10. Eligibility

Sex
All
Maximum Age & Unit of Time
1 Year
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Celeste Decker, MD
Organizational Affiliation
BioMarin Pharmaceutical
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Children's Hospital Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Hospital Femme Mere Enfant Centre
City
Lyon
ZIP/Postal Code
69677
Country
France
Facility Name
Hospital PediAtrico de Coimbra
City
Coimbra
ZIP/Postal Code
3000-076
Country
Portugal

12. IPD Sharing Statement

Citations:
PubMed Identifier
33775523
Citation
Garcia P, Phillips D, Johnson J, Martin K, Randolph LM, Rosenfeld H, Harmatz P. Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy. Mol Genet Metab. 2021 May;133(1):100-108. doi: 10.1016/j.ymgme.2021.03.006. Epub 2021 Mar 14.
Results Reference
derived
PubMed Identifier
24108527
Citation
Harmatz PR, Garcia P, Guffon N, Randolph LM, Shediac R, Braunlin E, Lachman RS, Decker C. Galsulfase (Naglazyme(R)) therapy in infants with mucopolysaccharidosis VI. J Inherit Metab Dis. 2014 Mar;37(2):277-87. doi: 10.1007/s10545-013-9654-7. Epub 2013 Oct 10.
Results Reference
derived
Links:
URL
http://www.BRMN.com
Description
BioMarin Pharmaceutical Inc Website

Learn more about this trial

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

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