This trial is evaluating whether BIIB067 (Tofersen) will improve 1 primary outcome and 9 secondary outcomes in patients with Amyotrophic Lateral Sclerosis. Measurement will happen over the course of Up to 12 months.
This trial requires 150 total participants across 5 different treatment groups
This trial involves 5 different treatments. BIIB067 (Tofersen) is the primary treatment being studied. Participants will be divided into 4 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.
"L-citrulline, lysine and myelin phospholipids are all elevated in the serum of patients who have amyotrophic lateral sclerosis. The findings illustrate that the metabolism of these biomolecules is altered in the brain of patients with this disorder and may be relevant to determining the genesis of the disorder. A detailed biochemical definition of the metabolism of these biomolecules may be useful for the diagnosis and management of neurodegenerative disorders such as amyotrophic lateral sclerosis." - Anonymous Online Contributor
"Because of its relatively recent discovery, the optimal management of ALS remains unsettled. We discuss the current management as a means of defining optimum management strategies." - Anonymous Online Contributor
"The symptoms of ALS can vary depending upon the severity of motor symptomatology. There are often associated disturbances of consciousness, and extrapyramidal symptoms. Patients also may develop sensory symptoms and changes in pain or temperature perception. The age of onset of ALS is typically within the fifth decade of life, and with a male to female ratio of 3:2. It affects mainly motor and lower limbs. About 10 percent of cases of ALS occur with a definite diagnosis." - Anonymous Online Contributor
"ALS is a very rare but progressive motor neurodegenerative condition characterized by slow, progressive weakness and wasting of muscles, leading to reduced quality of life and eventual respiratory failure. Overall, less than 1-3% of all motor neuron disorders are ALS. The epidemiological characteristics and the underlying cause of ALS are currently being explored with much need.\n" - Anonymous Online Contributor
"Around 100 new cases of ALS of all subtypes and the prevalence of fatal cases have been ascertained to be around 100 per 100,000 a year in the U.S. of any ethnic origins. The incidence of ALS in the U.S. is highest among non-Hispanic whites and those with presymptomatic myotonic degeneration, but the rates of incidence in other groups are similar." - Anonymous Online Contributor
"The majority of treatments for amyotrophic lateral sclerosis have been tested only with animal models of the disease, and only two treatments have been tested for the treatment of amyotrophic lateral sclerosis in humans; both are supportive therapy. The treatment for amyotrophic lateral sclerosis, either by a specific or supportive approach, is highly dependent on a patient's functional level, age, and motivation to treat." - Anonymous Online Contributor
"Findings from recent clinical trials have brought up a new possible treatment. A phase III trial concluded that baclofen may reduce the progression of non-focal ALS by slowing down the worsening of the disease. However, additional studies are still needed to see if this therapy will bring significant relief to patients." - Anonymous Online Contributor
"Only 5% of patients receiving tofersen alone or in combination with other anti-tumor agents to treat patients with stage III NSCLC used the recommended dose. It is unknown as to why patients would not be treated according to the dosing guidelines." - Anonymous Online Contributor
"Recent findings supports a heritable component in the disease, since we found that the siblings of these patients who died without any signs of a definite neurological disease are also afflicted with ALS. The family history is the major risk factor as only 10 of the 18 patients who did not have a family history of ALS died without any neurological disease. There is no evidence of a genetic linkage between ALS and SOD1 locus." - Anonymous Online Contributor
"The study results suggest that BIB067 does not improve QoL for people with ALS. Future clinical trials should investigate a range of quality of life measures." - Anonymous Online Contributor
"Clinical trials of disease modifying drugs (SOD1 gene and ALZ05001, GMDC-0196, LNA-1425, LNA-1501) targeting the protein misfolding pathway have resulted in new insights in the treatment of these rare disorders. Thus, the new discoveries for these therapies are potential for future trials and studies." - Anonymous Online Contributor
"Biib067 is a novel drug candidate for treating several forms of dementia-related neurodegenerative diseases including Alzheimer's disease and vascular dementia. Recently, the U.S. FDA granted priority review to the drug application for the potential therapeutic use of Biib067. In accordance with the FDA’s guidelines, we are evaluating Biib067 as a potential treatment for patients with dementia and tofersen is under investigation in a Phase IIb, double-blind, randomized study to evaluate the cognitive symptoms of patients with Alzheimer’s disease. This is expected to complete in early 2014." - Anonymous Online Contributor