Trial Outcomes & Findings for Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease (NCT NCT00319046)
NCT ID: NCT00319046
Last Updated: 2025-02-04
Results Overview
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.
COMPLETED
PHASE3
42 participants
Baseline and end of treatment (Month 24)
2025-02-04
Participant Flow
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.
Participant milestones
| Measure |
Miglustat
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
|
|---|---|
|
Overall Study
STARTED
|
42
|
|
Overall Study
COMPLETED
|
34
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Miglustat
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
|
|---|---|
|
Overall Study
withdrawal of subject's consent
|
7
|
|
Overall Study
administrative reason
|
1
|
Baseline Characteristics
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
Baseline characteristics by cohort
| Measure |
Miglustat
n=42 Participants
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
|
|---|---|
|
Age, Continuous
|
45.1 years
STANDARD_DEVIATION 12.7 • n=99 Participants
|
|
Age, Customized
Between 22 and 70 years
|
42 participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
20 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=99 Participants
|
|
Region of Enrollment
Australia
|
3 participants
n=99 Participants
|
|
Region of Enrollment
Brazil
|
1 participants
n=99 Participants
|
|
Region of Enrollment
Canada
|
5 participants
n=99 Participants
|
|
Region of Enrollment
Czech Republic
|
2 participants
n=99 Participants
|
|
Region of Enrollment
France
|
1 participants
n=99 Participants
|
|
Region of Enrollment
Netherlands
|
3 participants
n=99 Participants
|
|
Region of Enrollment
Spain
|
2 participants
n=99 Participants
|
|
Region of Enrollment
Taiwan
|
1 participants
n=99 Participants
|
|
Region of Enrollment
United Kingdom
|
6 participants
n=99 Participants
|
|
Region of Enrollment
United States
|
18 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Baseline and end of treatment (Month 24)Population: One patient was excluded from analysis as the baseline liver volume not available
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.
Outcome measures
| Measure |
Miglustat
n=41 Participants
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
|
|---|---|
|
Liver Volume at Baseline and at End of Treatment
Baseline
|
1774.6 cm^3
Standard Deviation 484.07
|
|
Liver Volume at Baseline and at End of Treatment
End of treatment
|
1727.1 cm^3
Standard Deviation 381.73
|
PRIMARY outcome
Timeframe: End of treatment (Month 24)Population: One patient was excluded from analysis as the baseline liver volume not available
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.
Outcome measures
| Measure |
Miglustat
n=41 Participants
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
|
|---|---|
|
Mean Within-patient Percent Change From Baseline in Liver Volume
|
-1.1 Percentage change
Standard Deviation 13.75
|
SECONDARY outcome
Timeframe: Baseline and end of treatment (Month 24)Population: The analysis was performed on those non-splenectomized patients who had a post-baseline assessment of spleen volume while on treatment with miglustat
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.
Outcome measures
| Measure |
Miglustat
n=22 Participants
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
|
|---|---|
|
Spleen Volume at Baseline and End of Treatment
Baseline
|
509.8 cm^3
Standard Deviation 371.77
|
|
Spleen Volume at Baseline and End of Treatment
End of treatment
|
611.9 cm^3
Standard Deviation 442.44
|
SECONDARY outcome
Timeframe: End of treatment (Month 24)Population: The analysis was performed on those non-splenectomized patients who had a post-baseline assessment of spleen volume while on treatment with miglustat
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.
Outcome measures
| Measure |
Miglustat
n=22 Participants
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
|
|---|---|
|
Mean Percent Change From Baseline in Spleen Volume
|
21.1 Percentage change
Standard Deviation 25.37
|
Adverse Events
Miglustat
Serious adverse events
| Measure |
Miglustat
n=42 participants at risk
Oral administration of miglustat 100 mg t.i.d. (median time exposure = 658 days)
|
|---|---|
|
Gastrointestinal disorders
ABDOMINAL DISCOMFORT
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Investigations
BLOOD URINE PRESENT
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Nervous system disorders
CEREBELLAR SYNDROME
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
COLON CANCER
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
CYST
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Gastrointestinal disorders
HAEMATOCHEZIA
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Nervous system disorders
HYPERREFLEXIA
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Musculoskeletal and connective tissue disorders
JOINT SWELLING
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Infections and infestations
PNEUMONIA
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
TRANSITIONAL CELL CARCINOMA
|
2.4%
1/42 • From study treatment start to the study treatment end date plus 2 days
|
Other adverse events
| Measure |
Miglustat
n=42 participants at risk
Oral administration of miglustat 100 mg t.i.d. (median time exposure = 658 days)
|
|---|---|
|
Gastrointestinal disorders
DIARRHOEA
|
73.8%
31/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Gastrointestinal disorders
FLATULENCE
|
50.0%
21/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Gastrointestinal disorders
ABDOMINAL DISTENSION
|
9.5%
4/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
9.5%
4/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Gastrointestinal disorders
NAUSEA
|
9.5%
4/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
|
7.1%
3/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Nervous system disorders
TREMOR
|
35.7%
15/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Nervous system disorders
HEADACHE
|
21.4%
9/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Nervous system disorders
PARAESTHESIA
|
21.4%
9/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Nervous system disorders
DIZZINESS
|
16.7%
7/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Nervous system disorders
HYPOAESTHESIA
|
11.9%
5/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Investigations
CHITOTRIOSIDASE INCREASED
|
14.3%
6/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Investigations
WEIGHT DECREASED
|
14.3%
6/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Investigations
PLATELET COUNT DECREASED
|
11.9%
5/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Investigations
HAEMOGLOBIN DECREASED
|
9.5%
4/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Investigations
ANGIOTENSIN CONVERTING ENZYME INCREASED
|
7.1%
3/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Investigations
BLOOD FOLATE DECREASED
|
7.1%
3/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Musculoskeletal and connective tissue disorders
MUSCLE SPASMS
|
9.5%
4/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Musculoskeletal and connective tissue disorders
BONE PAIN
|
7.1%
3/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Infections and infestations
NASOPHARYNGITIS
|
9.5%
4/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
9.5%
4/42 • From study treatment start to the study treatment end date plus 2 days
|
|
General disorders
FATIGUE
|
19.0%
8/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Psychiatric disorders
DEPRESSION
|
7.1%
3/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Blood and lymphatic system disorders
THROMBOCYTOPENIA
|
9.5%
4/42 • From study treatment start to the study treatment end date plus 2 days
|
|
Blood and lymphatic system disorders
ANAEMIA
|
7.1%
3/42 • From study treatment start to the study treatment end date plus 2 days
|
Additional Information
Cécile Luzy, MSc/Clinical Research Scientist
Actelion Pharmaceuticals Ltd
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60