Phase 4
N=4
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
Mucopolysaccharidosis VI · Maroteaux-Lamy Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00299000 ↗Enrolled (actual)
4
Serious AEs
100.0%
Results posted
Oct 2010
Primary outcome: Primary: Change in Height — 67.3; 80.3; 81.9; 91.3 centimeters
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Naglazyme (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- BioMarin Pharmaceutical
- Primary completion
- Apr 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Height |
67.3; 80.3; 81.9; 91.3; 14.6; 11.0 | — |
| PRIMARY Change in Weight |
7.1; 10.2; 11.3; 13.3; 4.2; 3.1 | — |
| PRIMARY Change in Haed Circumference |
43.5; 49.1; 48.5; 51.8; 5.0; 2.7 | — |
| SECONDARY Change in Urinary Glycosaminoglycan Levels |
1041.87; 698.95; 261.15; 178.10; -780.72; -520.85 | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT00299000). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.