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Clinical Trial Summary

The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme® (laronidase)] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.


Clinical Trial Description

n/a


Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT00146757
Study type Interventional
Source Sanofi
Contact
Status Completed
Phase Phase 2
Start date October 2002
Completion date May 2005

See also
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Active, not recruiting NCT04453085 - An Extension Study of JR-171-101 Study in Patients With MPS I Phase 1/Phase 2
Completed NCT05134571 - China Post-marketing Surveillance (PMS) Study of Aldurazyme® Phase 4
Terminated NCT00418821 - A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants Phase 4
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Completed NCT03053089 - Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I Phase 1/Phase 2
Active, not recruiting NCT04628871 - Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Completed NCT02597114 - Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181 Phase 1
Terminated NCT00748969 - Clinical Trial of Growth Hormone in MPS I, II, and VI Phase 2/Phase 3
Terminated NCT00215527 - Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I Phase 1
Recruiting NCT05619900 - Registry of Patients Diagnosed With Lysosomal Storage Diseases
Completed NCT00146770 - Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients Phase 3
Completed NCT00176917 - Stem Cell Transplantation for Hurler Phase 2
Completed NCT00176891 - Stem Cell Transplant w/Laronidase for Hurler Phase 2
Active, not recruiting NCT03153319 - Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI Phase 1/Phase 2
Completed NCT00852358 - A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I N/A
Enrolling by invitation NCT06103487 - Long Term Follow-Up for RGX-111