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Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease

This study has been completed.
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Sanofi
ClinicalTrials.gov Identifier:
NCT00258011
First received: November 22, 2005
Last updated: February 4, 2014
Last verified: February 2014
Results First Received: January 13, 2009  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Mucopolysaccharidosis I
Hurler Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

  Participant Flow
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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
Aldurazyme (Laronidase) Treatment Patients received weekly infusions of Aldurazyme (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight (labeled dose) for up to 73 weeks.

Participant Flow:   Overall Study
    Aldurazyme (Laronidase) Treatment  
STARTED     3  
COMPLETED     3  
NOT COMPLETED     0  



  Baseline Characteristics
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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
Aldurazyme (Laronidase) Treatment Patients received weekly infusions of Aldurazyme (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight (labeled dose) for up to 73 weeks.

Baseline Measures
    Aldurazyme (Laronidase) Treatment  
Number of Participants  
[units: participants]
  3  
Age  
[units: years]
Mean ± Standard Deviation
  12.2  ± 12.57  
Gender  
[units: participants]
 
Female     3  
Male     0  
Race/Ethnicity, Customized  
[units: participants]
 
Asian (Japanese)     3  



  Outcome Measures
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1.  Primary:   Safety Evaluation   [ Time Frame: Up to 73 Weeks ]

2.  Secondary:   Urinary Glycosaminoglycan (GAG) Excretion   [ Time Frame: Up to 73 Weeks ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
Due to the rarity of the disease, this study is limited by its small sample size, lack of a control group and patients had variable treatment durations.


  More Information
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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


No publications provided


Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00258011     History of Changes
Other Study ID Numbers: ALID02205
Study First Received: November 22, 2005
Results First Received: January 13, 2009
Last Updated: February 4, 2014
Health Authority: Japan: Ministry of Health, Labor and Welfare