This trial is evaluating whether ION373 will improve 1 primary outcome and 16 secondary outcomes in patients with Alexander Disease. Measurement will happen over the course of Baseline and Week 61.
This trial requires 58 total participants across 2 different treatment groups
This trial involves 2 different treatments. ION373 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 3 and have had some early promising results.
"The name Alexander disease (AD) is used mostly for infants and young children presenting a progressive dystonia with bulbar function deficits. AD is not the common, long-cited variant of AD. The disease course is typically slowly progressive with poor response to therapy often requiring an early colectomy. A major question is whether the use of corticosteroids influences the short-term clinical evolution. Alexander disease can be diagnosed with magnetic resonance imaging but the only reliable way to confirm it is through genetic testing." - Anonymous Online Contributor
"Some treatments may include corticosteroids, baclofen, fludoclopirim and phenytoin. Other medications can be used for various complications. There is no cure for alexander disease. Treatment is also based on the specific patient and the severity of the disease." - Anonymous Online Contributor
"Alexander disease results in loss of coordination or muscle control. Patients also exhibit an unsteady gait, incoordination and an awkward gait.\nPatients may also develop difficulties in swallowing and speak in a hoarse whisper. Alexander disease patients may also develop problems with vision, hearing and face deformity.\n" - Anonymous Online Contributor
"The presence of alexander disease on clinical grounds is highly suggestive of dengue infection. The diagnosis can only be confirmed by obtaining a sample for dengue serology and other laboratory tests." - Anonymous Online Contributor
"About 36,000 people are diagnosed annually with alexander disease in the United States. Most cases arise at an early age and are clinically benign, whereas a small percentage progress to prostate cancer. The incidence of prostate cancer increases significantly from age 70 years and is a significant contributor to mortality." - Anonymous Online Contributor
"No cure can be found for alexander disease as it is caused by human herpes simplex virus 2. However, there are various treatments that are effective in controlling the disease and preventing recurrences. Nevertheless, it is still a progressive disease and will continue to keep deteriorating as more knowledge about the disease is gained." - Anonymous Online Contributor
"Ion373, a lysosomal protein, is overexpressed in a subset of pediatric ependymomas, particularly those with an infiltrative phenotype. The function of ion373 and possible therapeutic implications will be the subject of further investigation." - Anonymous Online Contributor
"Many of the ion373 side effects are commonly experienced by the elderly. Furthermore, ion373 prescribing patterns are highly variable among patients. The most prevalent side effects are typically mild and can be managed by regular medications used by the elderly to treat their long-term illnesses. The ion373 prescribing patterns are usually consistent among patients, and ion373 is a relatively safe prescription from a hospital for patients, as well as those with various types of ailments. [Power(http://http://www." - Anonymous Online Contributor
"We have seen the most recent advances in disease prevention and treatment of alexander disease, but we need to find a cure for the disease. In addition, more research is necessary to find a cure for alexander disease. For now, we have seen the most recent advances in disease prevention and treatment of alexander disease, but we need to find a cure for the disease. For now, we have seen the most recent discoveries in patient treatment and disease prevention." - Anonymous Online Contributor
"The recent article by Ojada et al, (1997) illustrates that the presence of the same mutation in different individuals with SSc is common. It is thought to be a consequence of the complex genetic basis. When considering inheritance of SSc, it is vital to ascertain the specific underlying mutation and to obtain appropriate family members. The more we can identify the risk factors, the better we can tailor appropriate and effective treatment for our patients." - Anonymous Online Contributor
"No randomized controlled trials of Ion373 were ever conducted. Data from a recent study presented in this article are a combination of published reports, observational studies, and unpublished abstracts of research conducted at IonAxon, which have been analysed by a qualified medical peer peer review group. These data do not include data from the IonAxon Clinical Trial Registry." - Anonymous Online Contributor
"Clinical trials for alexander disease are being considered but many patients do not consider clinical trials. Patient involvement in these trials is still a barrier despite the advent of clinical trials evaluating other diseases. Clinical trials are necessary and should be encouraged. There is no way to predict who will get alexander disease." - Anonymous Online Contributor