Clinical trial NCT00299000: A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
Official title: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Mucopolysaccharidosis VI
Maroteaux-Lamy Syndrome
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2006-03-06
Enrollment: 4
Sex: ALL
Maximum age (years): 1
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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 skeletal system and growth.

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.

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

Primary outcomes:
measure: Change in Height
timeFrame: 52 weeks
measure: Change in Weight
timeFrame: 52 weeks
measure: Change in Haed Circumference
timeFrame: 52 weeks
Secondary outcomes:
measure: Change in Urinary Glycosaminoglycan Levels
timeFrame: minimum 52 weeks of dosing
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Oakland
country: United States
facility: Children's Hospital Oakland
state: California
city: Lyon
country: France
facility: Hospital Femme Mere Enfant Centre
city: Coimbra
country: Portugal
facility: Hospital PediAtrico de Coimbra
Study officials:
affiliation: BioMarin Pharmaceutical
name: Celeste Decker, MD
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.