Status:
COMPLETED
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Mucopolysaccharidosis VI
Maroteaux-Lamy Syndrome
Eligibility:
All Genders
Up to 1 years
Phase:
PHASE4
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 ske...
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...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
May 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2009
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT00299000
Start Date
May 1 2006
End Date
April 1 2009
Last Update
July 22 2011
Active Locations (4)
Enter a location and click search to find clinical trials sorted by distance.
1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
2
Children's Hospital Oakland
Oakland, California, United States, 94609
3
Hospital Femme Mere Enfant Centre
Lyon, France, 69677
4
Hospital PediAtrico de Coimbra
Coimbra, Portugal, 3000-076