This trial is evaluating whether sepofarsen will improve 2 primary outcomes and 2 secondary outcomes in patients with Amaurosis. Measurement will happen over the course of 12 months.
This trial requires 15 total participants across 4 different treatment groups
This trial involves 4 different treatments. Sepofarsen is the primary treatment being studied. Participants will be divided into 4 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 & 3 and have had some early promising results.
"Neurologic problems cannot be cured. However, treatment can delay or prevent a worsening of these conditions in many patients. The best approach for neurological patients is to monitor the symptoms and work toward a cure after their diagnosis. For patients with severe neurological comorbidities, a cure is unlikely." - Anonymous Online Contributor
"Neoplasms may have neurologic sign(s) (e.g., headache, paresthesia, altered level of consciousness), but they are an indication of an underlying illness rather than a consequence of an occult brain tumour. Further research is needed to better understand the cause(s) of these signs." - Anonymous Online Contributor
"The most common signs of neurological dysfunction are the following: headaches, stiffness and/or weakness of arms, paralysis or weakness of legs and inability to control muscle pain. Weakness can be present as a vague feeling of feeling 'heavy', or that the limb muscles do not move as easily due to lack of coordination. There is typically not pain at the site of weakness. In general, weakness is a result of neurological dysfunction, but not usually the cause of the neurologic dysfunction. Weakness occurs most often in the hand and foot of those with CMT, also occurring in lower leg, lower back, shoulder/upper back and hip. The lower and upper back muscles are often the affected muscles, though leg weakness may also occur." - Anonymous Online Contributor
"Approximately 22,000 people may develop neurologic signs every year in the United States. The most commonly seen symptoms were headache (29.1%), sensory abnormalities, tremor (17.8%), and muscle weakness. Most often a neurologic evaluation was performed on those with neurological signs. The most common reasons for testing were to confirm or rule out subacute, acute, or chronic neurologic signs. The most commonly used tests included magnetic resonance imaging, electroencephalography, and electromyography. Neurologic signs were related to the presence of one or more symptoms. Neurologic signs were identified but not described by clinicians in 40 percent of all cases." - Anonymous Online Contributor
"There are many common treatments for neurologic signs. A combination of medications, counseling and a multidisciplinary approach should be the mainstay of treatment for neurological sign. Antisecretory medications are useful in cases of headache, dizziness and vertigo. Anticonvulsants must be used cautiously, as the risk of seizures must be kept at a minimum, but when used they are helpful in chronic headache and seizures caused by brain tumors. Medication for tremors, seizures, fatigue and ataxia are discussed. Treatment for visual field defects and motor symptoms depends on the underlying cause, such as stroke or brain tumors." - Anonymous Online Contributor
"Sepofensine increased QOL in patients with neurologic signs and may be an effective and well-tolerated alternative treatment option for persons with these life-threatening neuropathies in whom treatment with conventional therapies is not possible." - Anonymous Online Contributor
"Patients who develop any of these features should be referred to neurology to make the diagnosis. Neurology can then work to establish if they are truly related to their CVS or something else that requires urgent consideration. Neurology could also establish the presence of a CNS condition as well." - Anonymous Online Contributor
"In accordance with clinical trial data, we saw similar efficacy for SePO/PI and the placebo in the clinical assessment of a group of patients with SCC." - Anonymous Online Contributor
"Given the evidence of a neuropsychiatric effect on a large number of patients in a randomized study of patients with Alzheimer's disease, it is unclear whether the neurological abnormalities seen in our sepofarsen-treated patients are consistent with a putative neuropsychiatric effect. Further investigation are needed to see how this neuropsychiatric effect might differ from the therapeutic effect of sepofarsen." - Anonymous Online Contributor
"We recommend that surgeons in particular should be familiar with and consider the possibility of neurologic signs during resection of [neurofibromin 1 (NF1) gene]] mutation-associated neurofibromatosis." - Anonymous Online Contributor
"Recent findings to date do not support concerns that sepofarsen may cause neurological, neuromuscular, or neuromuscular junction impairment. In those persons who may be more susceptible to these rare adverse events, pre-admission and periodic monitoring of adverse events are recommended." - Anonymous Online Contributor