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Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme� (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Information source: Sanofi
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie Syndrome

Intervention: Aldurazyme (Biological); Aldurazyme (Biological); placebo (Biological)

Phase: Phase 3

Status: Completed

Sponsored by: Genzyme, a Sanofi Company

Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme, a Sanofi Company


This study is being conducted to collect additional long-term efficacy and safety data of Aldurazyme (laronidase) patients with MPS I disease. Patients who were previously enrolled in the Phase 3 Double-Blind Study will be enrolled in this study.

Clinical Details

Official title: A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme (Laronidase) in Patients With Mucopolysaccharidosis I

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

Primary outcome:

Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)

Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)

Secondary outcome:

Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI)

Change From Baseline to Week 182 in Liver Volume

Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score

Change From Baseline to Week 182 in Active Joint Range of Motion (ROM)


Minimum age: N/A. Maximum age: N/A. Gender(s): Both.


Inclusion Criteria:

- The patient or patient's legal guardian must provide written informed consent prior

to any protocol-related procedures being performed.

- The patient must have successfully completed Study ALID-003-99 (who received 21 of 26

consecutive weekly infusions).

- The patient has not experienced any safety issues that would contraindicate

participation in the Extension study.

- A female patient of childbearing potential must have a negative pregnancy test at

entry Exclusion Criteria:

- The patient is pregnant or lactating.

- The patient has received an investigational drug within 30 days prior to the study


- The patient has a medical condition, serious intercurrent illness, or other

extenuating circumstance that may significantly interfere with study compliance including all prescribed evaluations and follow-up activities

Locations and Contacts

Hospital Infantil Joana de Gusmao, Florianopolis CEP 88025-301, Brazil

Hospital Universatario de Universidade Federal de Santa Catarina, Florianopolis 88040-500, Brazil

Alberta Children's Hospital, Calgary T2T 5C7, Canada

Children's Hospital Klinikum Nord Heidberg, Hamburg 22143, Germany

Medizinishe Hochshule Hannover, Hannover 30625, Germany

Children's Hospital at the University Hospital of Heidelberg, Heidelberg 69120, Germany

Children's Hospital Klinikum der F.S. Universitat, Jena 07740, Germany

Catholic University Sacro Cuore, Rome 00168, Italy

Academisch Ziekenhuis Rotterdam, Rotterdam 3000 GR, Netherlands

Belfast City Hospital, Belfast BT9 78A, United Kingdom

Birmingham Children's Hospital, Birmingham B4 6NH, United Kingdom

Bristol Royal Hospital for Children and Frenchay Hospital, Bristol BS16 1LE, United Kingdom

Gartnavel Hospital, Glasgow G11 6NT, United Kingdom

Great Ormond Street Hospital for Sick Children and NHS Trust, London WC1N 3GH, United Kingdom

Royal Victoria Hospital, Newcastle upon Tyne NE1 4LB, United Kingdom

University of South Alabama, Mobile, Alabama 36604, United States

Emory University School of Medicine, Atlanta, Georgia 30322, United States

Blackpool Victoria Hospital, Blackpool, Lancashire FY3 8NR, United Kingdom

University of Rochester, Rochester, New York 14542, United States

University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, United States

Toledo Children's Hospital, Toledo, Ohio 43606, United States

The Hospital for Sick Children, Toronto, Ontario M5G 1X8, Canada

Merle West Medical Center, Klamath Falls, Oregon 97601, United States

The Childrens Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States

Rhode Island Hospital, Providence, Rhode Island 02903, United States

Additional Information

Link to Results Synopsis for ALID-006-01

Starting date: May 2001
Last updated: March 17, 2015

Page last updated: August 20, 2015

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