Mucopolysaccharidosis Type VI (MPS VI
Conditions
Interventions
Trade Name: Naglazyme
Product Name: Naglazyme
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: galsulfase
CAS Number: 552858-79-4
Other descriptive name: recombinant hum
Sponsors
BioMarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: · In the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian after the nature of the study has been explained and prior to any research-related procedures; · 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. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: · Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable; · 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; · 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 PI, renders the subject at high risk from treatment compliance and/or completing the study; · Has known hypersensitivity to Naglazyme; · Has previously received Naglazyme.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is 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.;Secondary Objective: The secondary objective of the study is 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 is to evaluate the safety of two dose levels of Naglazyme in infants under the age of one year who have MPS VI.;Primary end point(s): The primary efficacy endpoint will be assessed by changes in dysmorphic features as assessed by photographs, changes in skeletal dysplasia as assessed by skeletal survey, and growth as measured by height, weight and head circumference. Other potential benefits of treatment will be periodically assessed by evaluation of gross and fine motor skills as part of the Denver II Development Test, cardiac evaluation by echocardiography, complete eye and visual exam, hearing test by audiometry, Health Resource Utilization Questionnaire, and urinary GAGs. | — |
Countries
France, Portugal
Outcome results
None listed