A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Completed
BioMarin/Genzyme LLC
Phase 4
2004-07-01
The purpose of this study is to determine whether the development of antibodies to laronidase
in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Completed
Genzyme, a Sanofi Company
Phase 4
2004-07-01
The purpose of this study is to determine whether the development of antibodies to laronidase
in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
The investigators hypothesize that weekly infusions of Laronidase ERT for 10-12 weeks prior
to transplant and 8 weeks following transplant will result in a reduction of
glycosaminoglycans (GAG) burden that is associated with decreased complications following
transplant.
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Terminated
FDA Office of Orphan Products Development
Phase 1
2005-11-01
The investigators are studying the use of enzyme replacement therapy into the spinal fluid
for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of
mucopolysaccharidosis I (MPS I). Funding source -- FDA OOPD
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Terminated
The Ryan Foundation
Phase 1
2005-11-01
The investigators are studying the use of enzyme replacement therapy into the spinal fluid
for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of
mucopolysaccharidosis I (MPS I). Funding source -- FDA OOPD
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