Immune Tolerance Study With Aldurazyme® (Laronidase)

Phase 1/2 Completed 7 enrolled Genzyme, a Sanofi Company BioMarin/Genzyme LLC · collab

RandomizedSingle-groupOpen-labelTreatment

Summary

The purpose of this study is to see if treatment with an antigen-specific immunosuppressive can decrease or stop an antibody response to laronidase (Aldurazyme®) during enzyme replacement therapy with laronidase in severe Mucopolysaccharidosis I (MPS I) participants.

Timeline

Start: 2008-09
Primary completion: 2012-09
Completion: 2012-09

Publications

Giugliani R, Vieira TA, Carvalho CG, Munoz-Rojas MV, Semyachkina AN, Voinova VY, Richards S, Cox GF, Xue Y. Immune tolerance induction for laronidase treatment in mucopolysaccharidosis I. Mol Genet Metab Rep. 2017 Jan 13;10:61-66. doi: 10.1016/j.ymgmr.2017.01.004. eCollection 2017 Mar.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Azathioprine	Small molecule	2.5 mg/kg	Oral
Subject	Azathioprine	Small molecule	5 mg/kg	Oral
Subject	Cyclosporine	Peptide	5 mg/kg	Oral
Subject	Cyclosporine	Peptide	6.7 mg/kg	Oral
Subject	laronidase	Protein / enzyme biologic	0.58 mg/kg	Intravenous

Immune Tolerance Study With Aldurazyme® (Laronidase)

Summary

Timeline

Publications

Drugs

Indications