A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
July 19, 2011 updated by: BioMarin Pharmaceutical
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)
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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.
Study Type
Interventional
Enrollment (Actual)
4
Phase
- Phase 4
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Lyon, France, 69677
- Hospital Femme Mere Enfant Centre
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Coimbra, Portugal, 3000-076
- Hospital Pediátrico de Coimbra
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California
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Los Angeles, California, United States, 90027
- Children's Hospital Los Angeles
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Oakland, California, United States, 94609
- Children's Hospital Oakland
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 1 year (CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: PREVENTION
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
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OTHER: Naglazyme, 1.0 mg/kg
Dose comparison
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Weekly infusion for minimum of 52 weeks.
Naglazyme is diluted in sterile 0.9% sodium chloride solution
Other Names:
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OTHER: Naglazyme, 2.0 mg/kg
Dose Comparison
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Weekly infusion for minimum of 52 weeks.
Naglazyme is diluted in sterile 0.9% sodium chloride solution
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
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Change in Height
Time Frame: 52 weeks
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52 weeks
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Change in Weight
Time Frame: 52 weeks
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52 weeks
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Change in Haed Circumference
Time Frame: 52 weeks
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52 weeks
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
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Change in Urinary Glycosaminoglycan Levels
Time Frame: minimum 52 weeks of dosing
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Change in urinary GAG levels was calculated from baseline to week 52 of treatment.
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minimum 52 weeks of dosing
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- 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.
- 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.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
May 1, 2006
Primary Completion (ACTUAL)
April 1, 2009
Study Completion (ACTUAL)
April 1, 2009
Study Registration Dates
First Submitted
March 2, 2006
First Submitted That Met QC Criteria
March 2, 2006
First Posted (ESTIMATE)
March 6, 2006
Study Record Updates
Last Update Posted (ESTIMATE)
July 22, 2011
Last Update Submitted That Met QC Criteria
July 19, 2011
Last Verified
July 1, 2011
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ASB-008
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.