Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Miglustat · 3 trials · 3 indications
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.
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.
| Arm | Type | Description |
|---|---|---|
| Open-label miglustat | EXPERIMENTAL | Oral administration of miglustat 100 mg t.i.d. for a period of 2 years |
| 1 | EXPERIMENTAL | miglustat |
| 2 | PLACEBO_COMPARATOR | placebo |
| Name | Type | Description |
|---|---|---|
| Miglustat | DRUG | Oral capsules containing miglustat 100 mg, administered three times daily (t.i.d.) |
| placebo | DRUG | Oral placebo capsules matching in appearance miglustat capsules given t.i.d. (three times a day) for 7 days and a single dose on Day 8 |
Inclusion Criteria: 1. Males or females aged 18 years or older 2. Type 1 Gaucher disease, diagnosed by glucocerebrosidase assay or molecular analysis of the glucocerebrosidase gene. 3. Treatment with ERT for at least 3 years, with a stable dose regimen for at least the last 6 months. 4. Clinically ...
Miglustat is a small molecule being studied for the treatment of Cystic Fibrosis, Gaucher Disease Type 1, and Niemann-Pick Type C Disease. It is developed by Johnson & Johnson and is currently in Phase 3 clinical development for these rare diseases.
Miglustat is a small molecule that targets the enzyme glucosylceramide synthase, which is involved in the production of glycosphingolipids. By inhibiting this enzyme, it reduces the accumulation of these lipids in cells, which is relevant to the pathology of Gaucher Disease and Niemann-Pick Type C Disease.
Miglustat is developed by Johnson & Johnson, a multinational pharmaceutical company listed on the New York Stock Exchange under the ticker symbol JNJ. The company is conducting clinical trials to evaluate the drug's efficacy and safety in multiple rare disease indications.
Miglustat is in Phase 3 clinical development. It has completed a Phase 3 trial in Gaucher Disease Type 1, as well as Phase 2 trials in Niemann-Pick Type C Disease and Cystic Fibrosis. The drug is investigational and not yet approved by regulatory authorities.
Miglustat has been studied in three completed clinical trials. NCT00319046 is a Phase 3 trial in Gaucher Disease Type 1 with 42 participants. NCT00517153 is a Phase 2 trial in Niemann-Pick Type C Disease with 29 participants. NCT00742092 is a Phase 2 trial in Cystic Fibrosis with 11 participants.
Miglustat is also known by the brand name Zavesca. It is a small molecule drug developed by Johnson & Johnson for the treatment of rare diseases such as Gaucher Disease Type 1 and Niemann-Pick Type C Disease.