NCT00319046

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

Completed Phase 3 Results posted Last updated 4 February 2025

What this trial tests

Phase 3 trial testing Miglustat in Gaucher Disease Type 1 in 42 participants. Completed in 1 July 2010.

Timeline

1 February 2006

Primary endpoint
1 June 2010

1 July 2010

Quick facts

Lead sponsor	Actelion
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	42
Start date	1 February 2006
Primary completion	1 June 2010
Estimated completion	1 July 2010

Drugs / interventions tested

• Miglustat (MIGLUSTAT) — full drug profile →

Conditions studied

• Gaucher Disease Type 1 — all drugs for Gaucher Disease Type 1 →

Sponsor

Actelion — full company profile →

Who can join

18 and older, any sex, with Gaucher Disease Type 1. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Liver Volume at Baseline and at End of Treatment Primary · 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.

Baseline

Group	Value	95% CI
Miglustat	1774.6	± 484.07

End of treatment

Group	Value	95% CI
Miglustat	1727.1	± 381.73

Mean Within-patient Percent Change From Baseline in Liver Volume Primary · 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.

Group	Value	95% CI
Miglustat	-1.1	± 13.75

Spleen Volume at Baseline and End of Treatment Secondary · 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.

Baseline

Group	Value	95% CI
Miglustat	509.8	± 371.77

End of treatment

Group	Value	95% CI
Miglustat	611.9	± 442.44

Mean Percent Change From Baseline in Spleen Volume Secondary · 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.

Group	Value	95% CI
Miglustat	21.1	± 25.37

Adverse events — posted to ClinicalTrials.gov

Time frame: From study treatment start to the study treatment end date plus 2 days. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Miglustat

Serious: 5/42 (12%)

Deaths: —

Serious adverse events (12 terms)

Reaction	System	Miglustat
ABDOMINAL DISCOMFORT	Gastrointestinal disorders	—
ARTHRALGIA	Musculoskeletal and connective tissue disorders	—
BACK PAIN	Musculoskeletal and connective tissue disorders	—
BLOOD URINE PRESENT	Investigations	—
CEREBELLAR SYNDROME	Nervous system disorders	—
COLON CANCER	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—
CYST	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—
HAEMATOCHEZIA	Gastrointestinal disorders	—
HYPERREFLEXIA	Nervous system disorders	—
JOINT SWELLING	Musculoskeletal and connective tissue disorders	—
PNEUMONIA	Infections and infestations	—
TRANSITIONAL CELL CARCINOMA	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—

Other adverse events (25 terms — click to expand)

Reaction	System	Miglustat
DIARRHOEA	Gastrointestinal disorders	—
FLATULENCE	Gastrointestinal disorders	—
TREMOR	Nervous system disorders	—
HEADACHE	Nervous system disorders	—
PARAESTHESIA	Nervous system disorders	—
FATIGUE	General disorders	—
DIZZINESS	Nervous system disorders	—
CHITOTRIOSIDASE INCREASED	Investigations	—
WEIGHT DECREASED	Investigations	—
HYPOAESTHESIA	Nervous system disorders	—
PLATELET COUNT DECREASED	Investigations	—
ABDOMINAL DISTENSION	Gastrointestinal disorders	—
ABDOMINAL PAIN	Gastrointestinal disorders	—
NAUSEA	Gastrointestinal disorders	—
HAEMOGLOBIN DECREASED	Investigations	—
MUSCLE SPASMS	Musculoskeletal and connective tissue disorders	—
NASOPHARYNGITIS	Infections and infestations	—
UPPER RESPIRATORY TRACT INFECTION	Infections and infestations	—
THROMBOCYTOPENIA	Blood and lymphatic system disorders	—
ABDOMINAL PAIN UPPER	Gastrointestinal disorders	—
ANGIOTENSIN CONVERTING ENZYME INCREASED	Investigations	—
BLOOD FOLATE DECREASED	Investigations	—
BONE PAIN	Musculoskeletal and connective tissue disorders	—
DEPRESSION	Psychiatric disorders	—
ANAEMIA	Blood and lymphatic system disorders	—

Most-reported serious reactions: ABDOMINAL DISCOMFORT, ARTHRALGIA, BACK PAIN, BLOOD URINE PRESENT, CEREBELLAR SYNDROME, COLON CANCER, CYST, HAEMATOCHEZIA.

Data from ClinicalTrials.gov NCT00319046 adverse events section.

Sponsor's own description

Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for enzyme replacement therapy (ERT), more data are required to establish the long term efficacy, safety and tolerability of miglustat in maintaining diseases stability after a switch from ERT.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study.
   Cox TM, Amato D, Hollak CE, Luzy C, et al · · 2012 · cited 31× · PMID 23270487 · DOI 10.1186/1750-1172-7-102
2. T-cell and natural killer-cell large granular lymphocyte leukemia neoplasias.
   Watters RJ, Liu X, Loughran TP. · · 2011 · cited 31× · PMID 21749307 · DOI 10.3109/10428194.2011.593276