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

This study has been completed.
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00146770
First received: September 2, 2005
Last updated: August 26, 2009
Last verified: April 2009
  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
Biological: Aldurazyme
Biological: placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I

Resource links provided by NLM:


Further study details as provided by Genzyme, a Sanofi Company:

Primary Outcome Measures:
  • Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) [ Time Frame: Baseline to Week 182 ] [ Designated as safety issue: No ]
  • Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) [ Time Frame: Baseline to Week 182 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI) [ Time Frame: Baseline to Week 182 ] [ Designated as safety issue: No ]
  • Change From Baseline to Week 182 in Liver Volume [ Time Frame: Baseline to Week 182 ] [ Designated as safety issue: No ]
  • Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score [ Time Frame: Baseline to Week 182 ] [ Designated as safety issue: No ]
  • Change From Baseline to Week 182 in Active Joint Range of Motion (ROM) [ Time Frame: Baseline to Week182 ] [ Designated as safety issue: No ]

Enrollment: 45
Study Start Date: May 2001
Study Completion Date: March 2005
Primary Completion Date: March 2005 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Placebo/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.
Biological: Aldurazyme
Placebo for 26 weeks then 0.58 mg/kg Aldurazyme every week for 182 weeks
Biological: placebo
Placebo for 26 weeks
Active Comparator: Aldurazyme/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.
Biological: Aldurazyme
0.58 mg/kg Aldurazyme every week for 208 weeks

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00146770

  Show 25 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Additional Information:
No publications provided by Genzyme, a Sanofi Company

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00146770     History of Changes
Other Study ID Numbers: ALID-006-01
Study First Received: September 2, 2005
Results First Received: January 7, 2009
Last Updated: August 26, 2009
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Mucopolysaccharidosis I
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on April 15, 2014