This trial is evaluating whether GZ402665 will improve 8 primary outcomes and 12 secondary outcomes in patients with Sphingomyelin Lipidosis. Measurement will happen over the course of Baseline (Day 1).
This trial requires 36 total participants across 2 different treatment groups
This trial involves 2 different treatments. GZ402665 is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 & 3 and have had some early promising results.
"Lipidoses is a group of disorders causing fatty deposition in body tissues. These disorders differ from classic lipidoses in that they can be caused by a range of factors both genetic and environmental. It is not uncommon for both conditions to be present at the same time. It is a rare disorder, seen usually only in children, which is characterised by the presence of accumulation of fat in the body on a particular part of the body. The location is most often assessed using MRI.\n\nWhat is an anxiety disorder?\nanswer: anxiety - the term loosely defined here and elsewhere - is a common mental health related problem." - Anonymous Online Contributor
"The signs and symptoms of lipidoses can be divided into five categories: visceral, neurologic and sensorineural, pulmonary, and cutaneous. Many patients present with non-specific symptoms with no definite explanation and may be managed by an internist or a rheumatologist." - Anonymous Online Contributor
"Estimates of age-standardized and cohort-derived lipoprotein associated mortality from the National Institute of Health/National Heart, Lung and Blood Institute (NHLBI) Lipid Action Plan Working Group Report imply an overall rate of lipidoses a year in the United States of 13.6/100,000 per year." - Anonymous Online Contributor
"Lipidoses are not curable. Lipidoses is an autosomal recessive disorder resulting from impaired activity of LIF. When a child inherits a mutated gene (either frameshift or missense) from the parent of an affected parent, they are at increased risk for developing lipidoses. A parent who knows that their child has lipidoses and/or who experiences a history suggestive of lipidoses, but who does not carry any of the known mutations, should seek genetic evaluation at early ages to provide early detection and proper medical management. When a child inherits the defective gene, its severity increases until the child develops lipoprotein lipase deficiency and lipidoses can develop in early childhood." - Anonymous Online Contributor
"Lipidoses is a disorder of carbohydrate metabolism which seems similar to Fabry's disease, although the lipidoses is much less severe. Genetic testing for Fabry disease might be a useful way of identifying individuals at risk." - Anonymous Online Contributor
"Several different drugs are used to treat lipidoses, but none are absolutely effective. Although most medications have only modest positive effects, CBT may help some patients to improve their quality of life. More rigorous studies in the area are required to develop specific recommendations." - Anonymous Online Contributor
"There is a large variety of diseases in which gz402665 shows potential. However, gz402665 is mainly administered in the treatment of patients with lipidoses, who are generally old and have few treatment options. The common adverse effects are dizziness, diarrhea and dizzy spells. No side effects have been reported so far and there are no reports of gz402665 causing or worsening of cardiovascular diseases. The dose-response relationship (in respect to cholesterol-control) between gz402665 and lipids is generally linear and the drug is not likely to show additive or antagonistic effects in combination with other lipid-modifying or statin drugs." - Anonymous Online Contributor
"For some conditions the mean and median age for the first observation is younger than for other diseases. For myocardial infarction, the mean age is closer to the median than for other diseases." - Anonymous Online Contributor
"Results from a recent clinical trial is based on a small sample. This was due to the fast and inexpensive development of this drug. It cannot be ruled out that side effects are less common than we thought before. It is also possible that gz402665 has different adverse effects in different people. However, we can confirm one main effect of gz402665 is an increase in weight in more than 50% of people. The weight gain is most common in those who have already been overweight before the treatment with gz402665. The weight gain may lead to a slight decrease in performance in some healthy people, so the increase in weight is not necessarily a sign of a side effect." - Anonymous Online Contributor
"In this pilot study, we found no advantage of a single dose from gz426665 over placebo regarding primary and secondary end points, but the study is too small to reveal the true effect of gz426665." - Anonymous Online Contributor
"The spectrum of lipidoses is extensive. Even for the more widely recognized familial diseases of GLUT1 deficiency, non-familial cases exist, and lipidoses may be mistaken for other disorders that have features of lipidosis as an underlying cause such as leukodystrophy." - Anonymous Online Contributor
"Gz402665 decreases triglycerides and cholesterol by targeting bile acid synthesis through the blockade of HMG-CoA reductase in liver. However, it also reduces insulin resistance through activating AMP kinase (AMPK) with a similar potency to pioglitazone. Thus, gz402665 provides a novel and powerful strategy to fight both central features of lipid disorders associated with T2D." - Anonymous Online Contributor