Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis I
Eligibility 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 (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 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
Sites / Locations
- University of South Alabama
- Emory University School of Medicine
- University of Rochester
- University of North Carolina at Chapel Hill
- Toledo Children's Hospital
- Merle West Medical Center
- The Childrens Hospital of Philadelphia
- Rhode Island Hospital
- Hospital Universatario de Universidade Federal de Santa Catarina
- Hospital Infantil Joana de Gusmao
- The Hospital for Sick Children
- Alberta Children's Hospital
- Children's Hospital Klinikum Nord Heidberg
- Medizinishe Hochshule Hannover
- Children's Hospital at the University Hospital of Heidelberg
- Children's Hospital Klinikum der F.S. Universitat
- Catholic University Sacro Cuore
- Academisch Ziekenhuis Rotterdam
- Blackpool Victoria Hospital
- Belfast City Hospital
- Birmingham Children's Hospital
- Bristol Royal Hospital for Children and Frenchay Hospital
- Gartnavel Hospital
- Great Ormond Street Hospital for Sick Children and NHS Trust
- Royal Victoria Hospital
Arms of the Study
Arm 1
Arm 2
Active Comparator
Active Comparator
Placebo/Aldurazyme
Aldurazyme/Aldurazyme
Patients received placebo for 26 weeks in the Double-Blind Study then received 182 weeks of Aldurazyme (0.58 mg/kg every week) in this Extension Study; patients received a total of 182 weeks of Aldurazyme.
Patients received 26 weeks of Aldurazyme in the Double-Blind Study and then received 182 weeks of Aldurazyme in this Extension Study; patients received a total of 208 weeks of Aldurazyme.