This trial is evaluating whether GZ402665 will improve 8 primary outcomes and 8 secondary outcomes in patients with Sphingomyelin Lipidosis. Measurement will happen over the course of Baseline to after at least 3 years in the study.
This trial requires 25 total participants across 1 different treatment group
This trial involves a single treatment. GZ402665 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.
"There are no cases of total recovery in our series. In all patients, medical and surgical treatment with a long-term follow-up proved to be effective and safe." - Anonymous Online Contributor
"The aim of treatment is to keep as many of the symptoms of lipodystrophy in remission. Treatments are varied and differ between individuals and include diet and regular exercise, medications, insulin regimen and the use of insulin for the people who are not able to control their blood sugar level which the doctors will prescribe insulin depending on the severity of symptoms.\n" - Anonymous Online Contributor
"About 100,000 to 140,000 people per year are diagnosed with liposes in the Unites States. These amounts are probably more frequent than estimated because many patients are asymptomatic during their lifetime and may be under-diagnosed." - Anonymous Online Contributor
"Focal lipidoses may cause local edema and atrophy of the fat-filled foci in lean tissues. In cases of systemic or disseminated lipidoses, localized edema and atrophy may lead to secondary fluid retention, peripheral edema, and generalized fluid overload, potentially mimicking lipoid myopathy and cardiac lipohypertrophy (see figure)." - Anonymous Online Contributor
"Signs of lipidoses may include weight loss, abdominal pain, enlarged lymph nodes, enlarged spleen, or rashes. In certain rare conditions, symptoms such as an enlarged liver may indicate a lipidosis, while in others, the presence of lipiduria may indicate a disorder related to lipid metabolism." - Anonymous Online Contributor
"There are many possible causes of lipidoses, which can be further divided by the type of lesion, whether it be infectious or genetic, and even by type of organism. Most commonly, lipidoses are associated with endocrine imbalance. Other possible causes include exposure to radiation, as for hyperlipoproteinemia, metabolic imbalance, and the accumulation of lipids in the body due to lipodystrophy, with or without lipomatosis. As of yet, no causal relationship exists." - Anonymous Online Contributor
"In a recent study, findings suggests the existence of LPO2 mutations in families and the need to set up a more detailed survey to ascertain whether LPO2 mutations are a prerequisite of LPO1 or LPO3 in the families studied here." - Anonymous Online Contributor
"Overall, gz402665 had a low overall adverse event rate. Mild but clinically relevant (as per protocol) adverse event were reported for 25% of subjects. The frequency of adverse events was similar (approximately 1/10 subjects) regardless of age at onset or gender. No significant differences were observed between people with lipomyeloedema and people without lipodystrophy receiving gz402665 or placebo. Adverse events did not appear to be dose-dependent. However, the safety of gz402665 had not been investigated in people with lipodystrophies receiving concomitant orlistat therapy. Data from a recent study support the safety of gz402665 in lipodystrophies." - Anonymous Online Contributor
"Gz402665 was well tolerated in the study population, showing no evidence of toxicity in either the short-term, Phase 1a, or long-term, Phase 1b, studies. It has been granted Orphan Drug Status by the Food and Drug Administration (FDA) for use as a potential treatment for lipidoses. Results from a Phase 1c clinical trial were announced in October 2010. A larger Phase 2a study is planned and will evaluate safety and tolerability in patients with lysosomal storage diseases. A new study was initiated in 2013. Gz402665 has yet to complete clinical development for lysosomal storage diseases, but Phase 1b studies have proven promising results." - Anonymous Online Contributor
"Lipidoses are a disease of aging of both whites and persons of African descent. If these two groups can make adjustments in lifestyle, a low rate of lipidoses would likely be achieved." - Anonymous Online Contributor
"Most of the recent developments have focused on new treatments for patients with Gaucher disease (GD), Morquio syndrome or the Tay–Sachs disease. There has also been an increase of research related to other lipidoses. Patients suffering from the three main forms of lipidoses can be treated with current treatments, [hepatitis C] (https://www.med.umich.edu/news/ncbi/2013/11/ncbi." - Anonymous Online Contributor
"[The FDA-approved (2007) treatment of gz402665 for inflammatory eye disease has been followed up in clinical trials with an expanded treatment schedule and more frequent follow-up visits (24 weeks rather than 4 weeks for the placebo group). The FDA label also highlights the need for long-term trials of gz402665 as, at the current rate of progression of disease (nausea and dizziness experienced in 1/3 of patients at this dose), gz402665 could be in effect for many years to come." - Anonymous Online Contributor