ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 40 of 105 for:    Gaucher Disease

Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00319046
Recruitment Status : Completed
First Posted : April 27, 2006
Results First Posted : May 23, 2012
Last Update Posted : June 1, 2012
Sponsor:
Information provided by (Responsible Party):
Actelion

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Type 1 Gaucher Disease
Intervention Drug: miglustat
Enrollment 42

Recruitment Details 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  
Arm/Group Title Miglustat
Hide Arm/Group Description miglustat oral capsules 100mg three times a day
Period Title: Overall Study
Started 42 [1]
Completed 34
Not Completed 8
Reason Not Completed
withdrawal of subject's consent             7
administrative reason             1
[1]
One patient was excluded from analysis as the baseline liver volume not available
Arm/Group Title Miglustat
Hide Arm/Group Description miglustat oral capsules 100mg three times a day
Overall Number of Baseline Participants 42
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 42 participants
45.1  (12.7)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Between 22 and 70 years Number Analyzed 42 participants
42
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 42 participants
Female
20
  47.6%
Male
22
  52.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 42 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
1.Primary Outcome
Title Liver Volume
Hide Description Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging
Time Frame baseline to end of treatment (month 24 or imputed value)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One patient was excluded from analysis as the baseline liver volume not available
Arm/Group Title Miglustat
Hide Arm/Group Description:
miglustat oral capsules 100mg three times a day
Overall Number of Participants Analyzed 41
Mean (Standard Deviation)
Unit of Measure: cm^3
Baseline 1774.6  (484.07)
End of treatment 1727.1  (381.73)
2.Primary Outcome
Title Percent Change in Liver Volume
Hide Description Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging
Time Frame baseline to end of treatment (month 24 or imputed value)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
One patient was excluded from analysis as the baseline liver volume not available
Arm/Group Title Miglustat
Hide Arm/Group Description:
miglustat oral capsules 100mg three times a day
Overall Number of Participants Analyzed 41
Mean (Standard Deviation)
Unit of Measure: percentage change
-1.1  (13.75)
3.Secondary Outcome
Title Spleen Volume
Hide Description Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging
Time Frame baseline to end of treatment (month 24 or imputed value)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis was performed on those non-splenectomized patients who had a post-baseline assessment of spleen volume while on treatment with miglustat
Arm/Group Title Miglustat
Hide Arm/Group Description:
miglustat oral capsules 100mg three times a day
Overall Number of Participants Analyzed 22
Mean (Standard Deviation)
Unit of Measure: cm^3
Baseline 509.8  (371.77)
End of treatment 611.9  (442.44)
4.Secondary Outcome
Title Percent Change in Spleen Volume
Hide Description Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging
Time Frame baseline to end of treatment (month 24 or imputed value)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The analysis was performed on those non-splenectomized patients who had a post-baseline assessment of spleen volume while on treatment with miglustat
Arm/Group Title Miglustat
Hide Arm/Group Description:
miglustat oral capsules 100mg three times a day
Overall Number of Participants Analyzed 22
Mean (Standard Deviation)
Unit of Measure: percentage change
21.1  (25.37)
Time Frame From study treatment start to the study treatment end date plus 2 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Miglustat
Hide Arm/Group Description miglustat oral capsules 100mg three times a day
All-Cause Mortality
Miglustat
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Miglustat
Affected / at Risk (%)
Total   5/42 (11.90%) 
Gastrointestinal disorders   
ABDOMINAL DISCOMFORT  1  1/42 (2.38%) 
HAEMATOCHEZIA  1  1/42 (2.38%) 
Infections and infestations   
PNEUMONIA  1  1/42 (2.38%) 
Investigations   
BLOOD URINE PRESENT  1  1/42 (2.38%) 
Musculoskeletal and connective tissue disorders   
ARTHRALGIA  1  1/42 (2.38%) 
BACK PAIN  1  1/42 (2.38%) 
JOINT SWELLING  1  1/42 (2.38%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
COLON CANCER  1  1/42 (2.38%) 
CYST  1  1/42 (2.38%) 
TRANSITIONAL CELL CARCINOMA  1  1/42 (2.38%) 
Nervous system disorders   
CEREBELLAR SYNDROME  1  1/42 (2.38%) 
HYPERREFLEXIA  1  1/42 (2.38%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (13.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Miglustat
Affected / at Risk (%)
Total   40/42 (95.24%) 
Blood and lymphatic system disorders   
THROMBOCYTOPENIA  1  4/42 (9.52%) 
ANAEMIA  1  3/42 (7.14%) 
Gastrointestinal disorders   
DIARRHOEA  1  31/42 (73.81%) 
FLATULENCE  1  21/42 (50.00%) 
ABDOMINAL DISTENSION  1  4/42 (9.52%) 
ABDOMINAL PAIN  1  4/42 (9.52%) 
NAUSEA  1  4/42 (9.52%) 
ABDOMINAL PAIN UPPER  1  3/42 (7.14%) 
General disorders   
FATIGUE  1  8/42 (19.05%) 
Infections and infestations   
NASOPHARYNGITIS  1  4/42 (9.52%) 
UPPER RESPIRATORY TRACT INFECTION  1  4/42 (9.52%) 
Investigations   
CHITOTRIOSIDASE INCREASED  1  6/42 (14.29%) 
WEIGHT DECREASED  1  6/42 (14.29%) 
PLATELET COUNT DECREASED  1  5/42 (11.90%) 
HAEMOGLOBIN DECREASED  1  4/42 (9.52%) 
ANGIOTENSIN CONVERTING ENZYME INCREASED  1  3/42 (7.14%) 
BLOOD FOLATE DECREASED  1  3/42 (7.14%) 
Musculoskeletal and connective tissue disorders   
MUSCLE SPASMS  1  4/42 (9.52%) 
BONE PAIN  1  3/42 (7.14%) 
Nervous system disorders   
TREMOR  1  15/42 (35.71%) 
HEADACHE  1  9/42 (21.43%) 
PARAESTHESIA  1  9/42 (21.43%) 
DIZZINESS  1  7/42 (16.67%) 
HYPOAESTHESIA  1  5/42 (11.90%) 
Psychiatric disorders   
DEPRESSION  1  3/42 (7.14%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (13.0)
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.
Results Point of Contact
Name/Title: Cécile Luzy, MSc/Clinical Research Scientist
Organization: Actelion Pharmaceuticals Ltd
Phone: + 41 61 565 6386
Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT00319046     History of Changes
Other Study ID Numbers: OGT 918-011
First Submitted: April 26, 2006
First Posted: April 27, 2006
Results First Submitted: April 24, 2012
Results First Posted: May 23, 2012
Last Update Posted: June 1, 2012