ALID00601: Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

This study has been completed.

Sponsored By:	Genzyme BioMarin/Genzyme LLC
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00146770

Purpose

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.

Condition	Intervention	Phase
Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie Syndrome	Drug: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)	Phase IV

Study Type: Interventional

Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

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

Further Study Details:

Primary Outcomes: To collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) on patients previously enrolled in the phase 3 double-blind study.

Study Start: 2001-06; Study Completion: 2005-03

Eligibility

Genders Eligible for Study: Both

Criteria

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.

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 30days prior to the study enrollment.

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

Location Information

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

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

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

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

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

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

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

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

Brazil

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

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

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

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

Germany
Medizinishe Hochshule Hannover, Hannover, 30625, Germany
Children's Hospital at the University Hospital of Heidelberg, Heidelberg, 69120, Germany
Children's Hospital Klinikum Nord Heidberg, Hamburg, 22143, Germany
Children's Hospital Klinikum der F.S. Universitat, Jena, 07740, Germany

Italy
Catholic University Sacro Cuore, Rome, 00168, Italy

Netherlands
Academisch Ziekenhuis Rotterdam, Rotterdam, 3000 GR, Netherlands

United Kingdom
Birmingham Children's Hospital, Birmingham, B4 6NH, United Kingdom
Gartnavel Hospital, Glasgow, G11 6NT, United Kingdom
Bristol Royal Hospital for Children and Frenchay Hospital, Bristol, BS16 1LE, United Kingdom
Royal Victoria Hospital, Newcastle upon Tyre, NE1 4LB, United Kingdom
Belfast City Hospital, Belfast, BT9 78A, United Kingdom
Great Ormond Street Hospital for Sick Children and NHS Trust, London, WC1N 3GH, United Kingdom

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

More Information

US FDA Approved Full Prescribing Information for Aldurazyme®

Results synopsis for Clinical Trial Protocol ALID00601

Study ID Numbers: ALID00601
ClinicalTrials.gov Identifier: NCT00146770
Health Authority: United States: Food and Drug Administration