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Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Actelion
ClinicalTrials.gov Identifier:
NCT00319046
First received: April 26, 2006
Last updated: May 24, 2012
Last verified: May 2012
Results First Received: April 24, 2012  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Type 1 Gaucher Disease
Intervention: Drug: miglustat

  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
Patients were enrolled at 16 centers in 10 countries (Australia, Brazil, Canada, Czech Republic, France, Netherlands , Spain, Taiwan, UK, and USA. The first patient, first visit was 21 February 2006 and the last patient, last visit was 22 June 2010.

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
Miglustat miglustat oral capsules 100mg three times a day

Participant Flow:   Overall Study
    Miglustat  
STARTED     42 [1]
COMPLETED     34  
NOT COMPLETED     8  
withdrawal of subject's consent                 7  
administrative reason                 1  
[1] One patient was excluded from analysis as the baseline liver volume not available



  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
Miglustat miglustat oral capsules 100mg three times a day

Baseline Measures
    Miglustat  
Number of Participants  
[units: participants]
  42  
Age  
[units: years]
Mean ± Standard Deviation
  45.1  ± 12.7  
Age, Customized  
[units: participants]
 
Between 22 and 70 years     42  
Gender  
[units: participants]
 
Female     20  
Male     22  
Region of Enrollment  
[units: participants]
 
Australia     3  
Brazil     1  
Canada     5  
Czech Republic     2  
France     1  
Germany     0  
Hungary     0  
Italy     0  
Netherlands     3  
Spain     2  
Taiwan     1  
United Kingdom     6  
United States     18  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Liver Volume   [ Time Frame: baseline to end of treatment (month 24 or imputed value) ]

2.  Primary:   Percent Change in Liver Volume   [ Time Frame: baseline to end of treatment (month 24 or imputed value) ]

3.  Secondary:   Spleen Volume   [ Time Frame: baseline to end of treatment (month 24 or imputed value) ]

4.  Secondary:   Percent Change in Spleen Volume   [ Time Frame: baseline to end of treatment (month 24 or imputed value) ]


  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
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  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 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.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Cécile Luzy, MSc/Clinical Research Scientist
Organization: Actelion Pharmaceuticals Ltd
phone: + 41 61 565 6386


No publications provided by Actelion

Publications automatically indexed to this study:

Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT00319046     History of Changes
Other Study ID Numbers: OGT 918-011
Study First Received: April 26, 2006
Results First Received: April 24, 2012
Last Updated: May 24, 2012
Health Authority: United States: Food and Drug Administration