MedPath

Laronidase

Generic Name
Laronidase
Brand Names
Aldurazyme
Drug Type
Biotech
Chemical Formula
-
CAS Number
210589-09-6
Unique Ingredient Identifier
WP58SVM6R4
Background

Human recombinant alpha-L-iduronidase, 628 residues (mature form), produced by recombinant DNAtechnology in a Chinese hamster ovary cell line. Laronidase is a glycoprotein with a molecular weight of approximately 83 kD. The predicted amino acid sequence of the recombinant form, as well as the nucleotide sequence that encodes it, are identical to a polymorphic form of human a-L-iduronidase. It contains 6 N-linked oligosaccharide modification sites.

Indication

For the treatment of mucopolysaccharidosis

Associated Conditions
Mucopolysaccharidosis Type I (MPS I)
Associated Therapies
-

China Post-marketing Surveillance (PMS) Study of Aldurazyme®

Phase 4
Completed
Conditions
Mucopolysaccharidosis I
Interventions
First Posted Date
2021-11-26
Last Posted Date
2023-09-28
Lead Sponsor
Genzyme, a Sanofi Company
Target Recruit Count
12
Registration Number
NCT05134571
Locations
🇨🇳

Investigational Site Number :1560001, Wuhan, China

🇨🇳

Investigational Site Number :1560004, Guangzhou, China

🇨🇳

Investigational Site Number :1560006, Hangzhou, China

and more 2 locations

Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Terminated
Conditions
Hurler Syndrome
MPS I
Mucopolysaccharidosis Type IH
Interventions
First Posted Date
2012-04-06
Last Posted Date
2018-09-27
Lead Sponsor
Masonic Cancer Center, University of Minnesota
Target Recruit Count
20
Registration Number
NCT01572636
Locations
🇺🇸

Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Phase 1
Completed
Conditions
Hurler Syndrome
Interventions
First Posted Date
2010-07-30
Last Posted Date
2020-03-20
Lead Sponsor
Masonic Cancer Center, University of Minnesota
Target Recruit Count
11
Registration Number
NCT01173016
Locations
🇺🇸

Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States

A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

Not Applicable
Completed
Conditions
Cognitive Decline
Hurler-Scheie Syndrome
Scheie Syndrome
Lysosomal Storage Disease
Mucopolysaccharidosis I
Interventions
First Posted Date
2009-02-27
Last Posted Date
2016-01-26
Lead Sponsor
Patricia I. Dickson, M.D.
Target Recruit Count
9
Registration Number
NCT00852358
Locations
🇺🇸

Los Angeles Biomedical Institute at Harbor-UCLA, Torrance, California, United States

🇺🇸

Children's Hospital & Research Center Oakland, Oakland, California, United States

🇺🇸

University of Minnesota, Minneapolis, Minnesota, United States

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Phase 1
Terminated
Conditions
Spinal Cord Compression
Mucopolysaccharidosis I
Scheie Syndrome
Hurler-Scheie Syndrome
Lysosomal Storage Disease
Interventions
First Posted Date
2008-11-06
Last Posted Date
2013-02-21
Lead Sponsor
Patricia I. Dickson, M.D.
Target Recruit Count
3
Registration Number
NCT00786968
Locations
🇫🇮

Helsinki University Central Hospital, Helsinki, Finland

🇺🇸

Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, California, United States

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

Phase 1
Terminated
Conditions
Mucopolysaccharidosis I
Lysosomal Storage Diseases
Spinal Cord Compression
Interventions
First Posted Date
2005-09-22
Last Posted Date
2013-02-21
Lead Sponsor
Patricia I. Dickson, M.D.
Target Recruit Count
4
Registration Number
NCT00215527
Locations
🇺🇸

Los Angeles Biomedical Research Institute at Harbor-UCLA ( LA BioMed ), Torrance, California, United States

🇫🇮

Helsinki University Central Hospital, Helsinki, Finland

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

Phase 4
Completed
Conditions
Mucopolysaccharidosis I
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie's Syndrome
First Posted Date
2005-09-05
Last Posted Date
2014-05-05
Lead Sponsor
Genzyme, a Sanofi Company
Target Recruit Count
25
Registration Number
NCT00144768
Locations
🇺🇸

Connecticut Children's Medical Center, Hartford, Connecticut, United States

🇺🇸

Children's Hospital of Wisconsin, Milwaukee, Wisconsin, United States

🇺🇸

Childrens Hospital Los Angeles, Los Angles, California, United States

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