Trial · NCT00319046
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
NCT00319046, Phase 3, Completed, Actelion; results first posted 2012-05-23
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease. Trials, record NCT00319046. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00319046
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
- NCT number
- NCT00319046
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 42 participants, actual
- Lead sponsor
- Actelion
- Conditions
- Gaucher Disease Type 1
- Interventions
- Miglustat
- First posted
- 2006-04-27
- Last updated
- 2025-02-04
- Results first posted
- 2012-05-23
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Liver Volume at Baseline and at End of Treatment | Baseline and end of treatment (Month 24) | 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. |
| Primary | Mean Within-patient Percent Change From Baseline in Liver Volume | End of treatment (Month 24) | 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. |
| Secondary | Spleen Volume at Baseline and End of Treatment | Baseline and end of treatment (Month 24) | 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. |
| Secondary | Mean Percent Change From Baseline in Spleen Volume | End of treatment (Month 24) | 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. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Liver Volume at Baseline and at End of Treatment | Baseline and end of treatment (Month 24) | One patient was excluded from analysis as the baseline liver volume not available | cm^3 | MEAN |
| PRIMARY | Mean Within-patient Percent Change From Baseline in Liver Volume | End of treatment (Month 24) | One patient was excluded from analysis as the baseline liver volume not available | Percentage change | MEAN |
| SECONDARY | Spleen Volume at Baseline and End of Treatment | Baseline and end of treatment (Month 24) | The analysis was performed on those non-splenectomized patients who had a post-baseline assessment of spleen volume while on treatment with miglustat | cm^3 | MEAN |
| SECONDARY | Mean Percent Change From Baseline in Spleen Volume | End of treatment (Month 24) | The analysis was performed on those non-splenectomized patients who had a post-baseline assessment of spleen volume while on treatment with miglustat | Percentage change | MEAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Liver Volume at Baseline and at End of Treatment | Miglustat | Baseline | 1774.6 | Standard Deviation 484.07 | cm^3 |
| Liver Volume at Baseline and at End of Treatment | Miglustat | End of treatment | 1727.1 | Standard Deviation 381.73 | cm^3 |
| Mean Within-patient Percent Change From Baseline in Liver Volume | Miglustat | -1.1 | Standard Deviation 13.75 | Percentage change | |
| Spleen Volume at Baseline and End of Treatment | Miglustat | Baseline | 509.8 | Standard Deviation 371.77 | cm^3 |
| Spleen Volume at Baseline and End of Treatment | Miglustat | End of treatment | 611.9 | Standard Deviation 442.44 | cm^3 |
| Mean Percent Change From Baseline in Spleen Volume | Miglustat | 21.1 | Standard Deviation 25.37 | Percentage change |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Miglustat | 42 | |
| Overall Study | COMPLETED | Miglustat | 34 | |
| Overall Study | NOT COMPLETED | Miglustat | 8 |
Source ClinicalTrials.gov
Adverse events37
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | DIARRHOEA | Gastrointestinal disorders | Miglustat | 31 | 42 | |
| other | FLATULENCE | Gastrointestinal disorders | Miglustat | 21 | 42 | |
| other | ABDOMINAL DISTENSION | Gastrointestinal disorders | Miglustat | 4 | 42 | |
| other | ABDOMINAL PAIN | Gastrointestinal disorders | Miglustat | 4 | 42 | |
| other | NAUSEA | Gastrointestinal disorders | Miglustat | 4 | 42 | |
| other | ABDOMINAL PAIN UPPER | Gastrointestinal disorders | Miglustat | 3 | 42 | |
| other | TREMOR | Nervous system disorders | Miglustat | 15 | 42 | |
| other | HEADACHE | Nervous system disorders | Miglustat | 9 | 42 | |
| other | PARAESTHESIA | Nervous system disorders | Miglustat | 9 | 42 | |
| other | DIZZINESS | Nervous system disorders | Miglustat | 7 | 42 |
1 to 10 of 37Source ClinicalTrials.gov
See all 37 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Miglustat | ||
| Total, other adverse events | Miglustat | 40 | 42 |
| Total, serious adverse events | Miglustat | 5 | 42 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 23270487 | RESULT |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00319046
GET https://conductscience.org/api/trials/v1/NCT00319046/events?page=2