A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
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)
1 other identifier
interventional
4
3 countries
4
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_4
Started May 2006
Typical duration for phase_4
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 2, 2006
CompletedFirst Posted
Study publicly available on registry
March 6, 2006
CompletedStudy Start
First participant enrolled
May 1, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2009
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2009
CompletedResults Posted
Study results publicly available
October 15, 2010
CompletedJuly 22, 2011
July 1, 2011
2.9 years
March 2, 2006
May 7, 2010
July 19, 2011
Conditions
Outcome Measures
Primary Outcomes (3)
Change in Height
52 weeks
Change in Weight
52 weeks
Change in Haed Circumference
52 weeks
Secondary Outcomes (1)
Change in Urinary Glycosaminoglycan Levels
minimum 52 weeks of dosing
Study Arms (2)
Naglazyme, 1.0 mg/kg
OTHERDose comparison
Naglazyme, 2.0 mg/kg
OTHERDose Comparison
Interventions
Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Children's Hospital Oakland
Oakland, California, 94609, United States
Hospital Femme Mere Enfant Centre
Lyon, 69677, France
Hospital PediAtrico de Coimbra
Coimbra, 3000-076, Portugal
Related Publications (2)
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.
PMID: 33775523DERIVEDHarmatz 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.
PMID: 24108527DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
Given the small number of patients (4) represented in this study, the outcomes observed in this study may not reflect or predict outcomes observed by physicians in clinical practice.
Results Point of Contact
- Title
- Medical Information Services
- Organization
- BioMarin Pharmaceutical Inc.
Study Officials
- STUDY DIRECTOR
Celeste Decker, MD
BioMarin Pharmaceutical
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
March 2, 2006
First Posted
March 6, 2006
Study Start
May 1, 2006
Primary Completion
April 1, 2009
Study Completion
April 1, 2009
Last Updated
July 22, 2011
Results First Posted
October 15, 2010
Record last verified: 2011-07