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Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

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ClinicalTrials.gov Identifier: NCT00319046
Recruitment Status : Completed
First Posted : April 27, 2006
Results First Posted : May 23, 2012
Last Update Posted : November 21, 2018
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 Gaucher Disease Type 1
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 Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
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 Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
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
Netherlands 3
Spain 2
Taiwan 1
United Kingdom 6
United States 18
1.Primary Outcome
Title Liver Volume at Baseline and at End of Treatment
Hide Description Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging. Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the ‘worst’ within-patient value not more than 2 days after the end of treatment was used to impute the missing value.
Time Frame Baseline and end of treatment (Month 24)
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:
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
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 Mean Within-patient Percent Change From Baseline in Liver Volume
Hide Description Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging. Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the ‘worst’ within-patient value not more than 2 days after the end of treatment was used to impute the missing value.
Time Frame End of treatment (Month 24)
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:
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
Overall Number of Participants Analyzed 41
Mean (Standard Deviation)
Unit of Measure: Percentage change
-1.1  (13.75)
3.Secondary Outcome
Title Spleen Volume at Baseline and End of Treatment
Hide Description

Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging.

Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the ‘worst’ within-patient value not more than 2 days after the end of treatment was used to impute the missing value.

Time Frame Baseline and end of treatment (Month 24)
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:
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
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 Mean Percent Change From Baseline in Spleen Volume
Hide Description

Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging.

Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the ‘worst’ within-patient value not more than 2 days after the end of treatment was used to impute the missing value.

Time Frame End of treatment (Month 24)
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:
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
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 Oral administration of miglustat 100 mg t.i.d. (median time exposure = 658 days)
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: November 21, 2018