A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

BioMarin Pharmaceutical

Status and phase

Completed

Phase 4

Conditions

Maroteaux-Lamy Syndrome

Mucopolysaccharidosis VI

Treatments

Drug: Naglazyme

Study type

Interventional

Funder types

Industry

Identifiers

NCT00299000

ASB-008

Details and patient eligibility

About

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.

Full 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.

Enrollment

4 patients

Sex

All

Ages

Under 1 year old

Volunteers

No Healthy Volunteers

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