Media Articles Related to Zavesca (Miglustat)
Cerdelga Approved for Gaucher Disease
Source: MedicineNet Gaucher Disease Specialty [2014.08.21]
Title: Cerdelga Approved for Gaucher Disease
Category: Health News
Created: 8/20/2014 2:35:00 PM
Last Editorial Review: 8/21/2014 12:00:00 AM
Published Studies Related to Zavesca (Miglustat)
Evaluation of miglustat treatment in patients with type III mucopolysaccharidosis: a randomized, double-blind, placebo-controlled study. [2011.11]
OBJECTIVE: To evaluate the efficacy and safety of oral miglustat treatment in patients with mucopolysaccharidosis type III. The primary outcome was efficacy with improvement or stabilization in at least two domains of Vineland Adaptative Behavior Scales at 6 months. The secondary outcome measured the evolution of other cognitive tests at 12 months. The safety and tolerability were assessed throughout the study... CONCLUSION: Miglustat treatment was not associated with any improvement/stabilization in behavior problems in patients with mucopolysaccharidosis type III. Miglustat has an acceptable safety profile. However, the study has confirmed that miglustat is able to pass through the blood-brain barrier without significantly decreasing ganglioside levels. Copyright (c) 2011 Mosby, Inc. All rights reserved.
Miglustat in adult and juvenile patients with Niemann-Pick disease type C: long-term data from a clinical trial. [2010.04]
A randomized, controlled trial of miglustat indicated that miglustat (Zavesca) stabilized neurological disease over 12 months in adult and juvenile patients with Niemann-Pick disease type C (NP-C).Overall, these data suggest that long-term miglustat therapy stabilizes neurological disease and is well tolerated in adult and juvenile patients with NP-C.
Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment. [2009.06]
PURPOSE: To evaluate the safety and efficacy of miglustat in patients with GM2 gangliosidosis... CONCLUSION: Miglustat treatment was not shown to lead to measurable benefits in this cohort of patients with late-onset Tay-Sachs disease. The observed safety profile was consistent with that of the approved dose (100 mg TID) in type 1 Gaucher disease.
Randomized, controlled trial of miglustat in Gaucher's disease type 3. [2008.11]
OBJECTIVE: To evaluate the efficacy and safety of miglustat, concomitant with enzyme replacement therapy (ERT), in patients with Gaucher's disease type 3 (GD3)... INTERPRETATION: Miglustat does not appear to have significant benefits on the neurological manifestations of GD3. However, miglustat may have positive effects on systemic disease (pulmonary function and chitotriosidase activity) in addition to ERT in patients with GD3.
Miglustat: a review of its use in Niemann-Pick disease type C. 
Miglustat (Zavesca┬«, Brazaves┬«), a small iminosugar molecule that reversibly
inhibits glycosphingolipid synthesis, is the only disease-specific drug approved
for the treatment of progressive neurological manifestations of Niemann-Pick
disease type C (NP-C) in adult and paediatric patients... In the absence
of a cure, miglustat is a valuable agent to reduce the progression of clinically
relevant neurological symptoms in paediatric and adult patients with NP-C, which
is considered a significant achievement in the treatment of this disease.
Clinical Trials Related to Zavesca (Miglustat)
Pharmacokinetics and Tolerability of Zavesca« (Miglustat) In Patients With Juvenile GM2 Gangliosidosis [Active, not recruiting]
The purpose of the study is to investigate the pharmacokinetics of Zavesca (miglustat,
OGT918) when given as single and multiple doses in juvenile patients with GM2
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses [Completed]
We want to see if Zavesca (or miglustat) is safe and can be tolerated by patients with acute
infantile onset GM2 gangliosidosis - classical Tay-Sachs and infantile onset Sandhoff
disease. We know that miglustat inhibits the formation of GM2 ganglioside, the compound that
is stored in the brains of children with Tay-Sachs and Sandhoff disease. Since it inhibits
the synthesis of ganglioside, miglustat may be able to reduce or delay the onset of clinical
Miglustat in Niemann-Pick Type C Disease [Active, not recruiting]
This is a phase II randomized controlled study of miglustat in adult and juvenile patients
with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2: 1 ratio to
either treatment with miglustat or to a non-treatment group. Both groups will follow an
identical visit schedule.
Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ? [Recruiting]
The purpose of this study is to investigate within a short delay the effect of nasal
instillation of Miglustat on nasal potential difference in cystic fibrosis patients
homozygous for the F508del mutation.
OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease [Completed]
Gaucher disease is an inherited functional deficiency of glucocerebrosidase. This enzyme
breaks down a fatty substance (lipid) called glucocerebroside, which is present in all cells
of the body. When cells renew themselves, the lipids must be broken down and discarded.
Because the enzyme does not function well, the lipid builds up in certain tissues, such as
the liver and spleen. The nervous system is involved as well; memory is impaired and it is
difficult to move the eyes from side to side. It has been shown that repeated infusions of
glucocerebrosidase help break down the stored lipid. However, this treatment does not improve
any neurological symptoms.
A medicine called OGT 918 has been shown to slow the production of the lipid that builds up
in Gaucher disease. It also has been shown to enter the brain. It is hoped that taking OGT
918 will reduce the storage of glycolipids in cells and improve the neurological symptoms of
the disease. This clinical trial seeks to evaluate OGT 918 as a treatment for neuronopathic
Gaucher disease by assessing changes in eye movement velocity. A secondary goal is to assess
the clinical safety and tolerability of OGT 918 therapy.
Up to 30 patients from the National Institutes of Health and the Institute of Child Health
(London) will be randomly assigned to OGT 918 or no treatment for 12 months. Study
participants must be clinically diagnosed with neuronopathic Gaucher disease, 12 years of age
or older, and able to swallow capsules. They must have been stable on ERT for at least 6
months before the study.
Patients receiving OGT 918 will receive a dose of 200 mg OGT 918 three times daily. Data
analysis will be done after 12 months. The study will be extended up to 12 months to collect
safety and efficacy data. All patients who complete the main study and enter the extension
study will receive OGT 918.
During a 4-week screening period, eye movement velocity will be measured. These assessments
will be repeated at months 12 and 24. Also at screening and months 12 and 24, the following
tests will be done: MRI/CT, to measure spleen and liver volume; pulmonary imaging (by X-ray)
and function tests; nerve conduction velocity studies and neuropsychological assessments;
evoked response studies (to measure how the brain conducts electrical messages); and tremor
measurements. Additional assessments for tremor will be conducted at months 6 and 18.
Plasma samples will be obtained every 3 months to measure disease markers and safety
profiles. Proteasome samples will be taken at screening and month 6 to identify proteins that
may be associated with Gaucher disease. Blood will be obtained at month 1 from the first 6
consenting patients who have been randomly assigned to take OGT 918. These patients will also
have a cerebrospinal fluid sample taken by lumbar puncture at month 1. These samples will be
measured for how much OGT 918 is present.
All patients receiving OGT 918 will have an initial assessment 1 week after beginning
treatment to evaluate tolerance of the therapy. Clinic visits will be every 3 months. All
patients will be asked to keep a simple diary of adverse events and dietary information. Dose
levels may be reduced if a patient experiences severe gastrointestinal problems.
Reports of Suspected Zavesca (Miglustat) Side Effects
Weight Decreased (20),
Niemann-Pick Disease (14),
Condition Aggravated (13),
Disease Progression (8),
Growth Retardation (8), more >>