Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
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| ClinicalTrials.gov Identifier: NCT00146770 |
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Recruitment Status :
Completed
First Posted : September 7, 2005
Results First Posted : June 16, 2009
Last Update Posted : April 3, 2015
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Sponsor:
Genzyme, a Sanofi Company
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Sanofi
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Brief Summary:
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 or disease | Intervention/treatment | Phase |
|---|---|---|
| Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie Syndrome | Biological: Aldurazyme Biological: placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 45 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Single Group Assignment |
| Masking: | None (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 |
| Study Start Date : | May 2001 |
| Actual Primary Completion Date : | March 2005 |
| Actual Study Completion Date : | March 2005 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Mucopolysaccharidosis type I
Drug Information available for:
Laronidase
| Arm | Intervention/treatment |
|---|---|
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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.
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Biological: Aldurazyme
Placebo for 26 weeks then 0.58 mg/kg Aldurazyme every week for 182 weeks Biological: placebo Placebo for 26 weeks |
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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.
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Biological: Aldurazyme
0.58 mg/kg Aldurazyme every week for 208 weeks |
Primary Outcome Measures :
- Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC) [ Time Frame: Baseline to Week 182 ]Percent Predicted Forced Vital Capacity: the maximal exhaled breath volume following a maximal inhaled breath. Overall change from Baseline to Week 182 in percent predicted FVC = (observed value)/(predicted value) * 100%). A higher value indicates a greater response.
- Change From Baseline to Week 182 in Six Minute Walk Test (6MWT) [ Time Frame: Baseline to Week 182 ]Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.
Secondary Outcome Measures :
- Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI) [ Time Frame: Baseline to Week 182 ]Apnea/Hypopnea Index (AHI): Number of absent (apnea) and shallow (hypopnea) breaths per hour of sleep. A greater decrease in events indicates a greater response.
- Change From Baseline to Week 182 in Liver Volume [ Time Frame: Baseline to Week 182 ]Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response.
- 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 ]CHAQ/HAQ = Patient questionnaire that measures the degree of disability on a scale of 0 (no disability) to 3 (maximal disability). A lower score indicates a greater response.
- Change From Baseline to Week 182 in Active Joint Range of Motion (ROM) [ Time Frame: Baseline to Week182 ]Active Joint Range of Motion (ROM): Shoulder Flexion Ability to maximally raise one's arm overhead without assistance. Shoulder range of motion (mean of left and right arms) measured in degrees (0-180) by goniometry. Greater degree of flexion indicates greater response.
Other Outcome Measures:
- Change From Baseline to Week 182 in Urinary GAG Level [ Time Frame: Baseline to Week 182 ]Urinary Glycosaminoglycan (GAG) Levels: >> Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response.
Information from the National Library of Medicine
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| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00146770
Locations
Show 25 study locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Investigators
| Study Director: | Medical Monitor | Genzyme, a Sanofi Company |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Medical Monitor, Genzyme Corporation |
| ClinicalTrials.gov Identifier: | NCT00146770 |
| Other Study ID Numbers: |
ALID-006-01 |
| First Posted: | September 7, 2005 Key Record Dates |
| Results First Posted: | June 16, 2009 |
| Last Update Posted: | April 3, 2015 |
| Last Verified: | March 2015 |
Additional relevant MeSH terms:
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Mucopolysaccharidoses Mucopolysaccharidosis I Syndrome Disease Pathologic Processes Carbohydrate Metabolism, Inborn Errors |
Metabolism, Inborn Errors Genetic Diseases, Inborn Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |