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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
Recruitment Status : Completed
First Posted : September 7, 2005
Results First Posted : June 16, 2009
Last Update Posted : April 3, 2015
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Sanofi

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Mucopolysaccharidosis I
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Interventions: Biological: Aldurazyme
Biological: placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Placebo/Aldurazyme Patients received placebo for 26 weeks in the Double-Blind Study then received 182 weeks of Aldurazyme treatment in the Extension Study; patients received a total of 182 weeks of Aldurazyme treatment. “Baseline” for this group is the last measurement in the Double-Bind Study prior to enrollment in this Extension Study.
Aldurazyme/Aldurazyme Patients received 26 weeks of Aldurazyme treatment in the Double-Blind Study and then received 182 weeks of Aldurazyme treatment in the Extension Study; patients received a total of 208 weeks of Aldurazyme treatment. “Baseline” for this group is the last measurement prior to randomization in the Double-Blind Study.

Participant Flow:   Overall Study
    Placebo/Aldurazyme   Aldurazyme/Aldurazyme
STARTED   23   22 
COMPLETED   18   22 
NOT COMPLETED   5   0 
Adverse Event                2                0 
Pregnancy                1                0 
Withdrawal by Subject                2                0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Placebo/Aldurazyme Patients received placebo for 26 weeks in the Double-Blind Study then received 182 weeks of Aldurazyme treatment in the Extension Study; patients received a total of 182 weeks of Aldurazyme treatment.
Aldurazyme/Aldurazyme Patients received 26 weeks of Aldurazyme treatment in the Double-Blind Study and then received 182 weeks of Aldurazyme treatment in the Extension Study; patients received a total of 208 weeks of Aldurazyme treatment.
Total Total of all reporting groups

Baseline Measures
   Placebo/Aldurazyme   Aldurazyme/Aldurazyme   Total 
Overall Participants Analyzed 
[Units: Participants]
 23   22   45 
Age, Customized 
[Units: Participants]
     
<=12 years   10   12   22 
13 to <= 18 years   8   3   11 
19 to <= 65 years   5   7   12 
Age [1] 
[Units: Years]
Mean (Standard Deviation)
 15.4  (7.63)   15.6  (8.63)   15.5  (8.04) 
[1] Age characteristics presented were recorded at Baseline in the Double-Blind Study.
Gender [1] 
[Units: Participants]
     
Female   12   11   23 
Male   11   11   22 
[1] Gender characteristics presented were recorded at Baseline in the Double-Blind Study.
Race/Ethnicity [1] 
[Units: Participants]
     
Caucasian   21   16   37 
Black   0   0   0 
Hispanic   0   4   4 
Asian   1   1   2 
Other   1   1   2 
[1] Race characteristics presented were recorded at Baseline in the Double-Blind Study.


  Outcome Measures

1.  Primary:   Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)   [ Time Frame: Baseline to Week 182 ]

2.  Primary:   Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)   [ Time Frame: Baseline to Week 182 ]

3.  Secondary:   Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI)   [ Time Frame: Baseline to Week 182 ]

4.  Secondary:   Change From Baseline to Week 182 in Liver Volume   [ Time Frame: Baseline to Week 182 ]

5.  Secondary:   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 ]

6.  Secondary:   Change From Baseline to Week 182 in Active Joint Range of Motion (ROM)   [ Time Frame: Baseline to Week182 ]

7.  Other Pre-specified:   Change From Baseline to Week 182 in Urinary GAG Level   [ Time Frame: Baseline to Week 182 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Rare disease with limited sample size, lack of control group.FVC calculations less reliable for patients whose height is below the 3rdpercent of general population.Height term used to calculate FVC may be influenced by joint contractures and posture.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Genzyme Medical Information
Organization: Genzyme Corporation
phone: 800-745-4447


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
First Submitted: September 2, 2005
First Posted: September 7, 2005
Results First Submitted: January 7, 2009
Results First Posted: June 16, 2009
Last Update Posted: April 3, 2015