AMX0035 for Amyotrophic Lateral Sclerosis
This study is evaluating whether a drug called AMX0035 can help people with amyotrophic lateral sclerosis (ALS) live longer.
See full descriptionAbout the trial for Amyotrophic Lateral Sclerosis
Treatment Groups
This trial involves 2 different treatments. AMX0035 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 & 3 and have had some early promising results.
About The Treatment
Side Effect Profile for AMX0035
Eligibility
This trial is for patients born any sex aged 18 and older. There are 8 eligibility criteria to participate in this trial as listed below.
Approximate Timelines
Measurement Requirements
This trial is evaluating whether AMX0035 will improve 1 primary outcome in patients with Amyotrophic Lateral Sclerosis. Measurement will happen over the course of Through study completion an average of 1 year.
Patient Q & A Section
What is amyotrophic lateral sclerosis?
This condition is a disease that destroys nerve cells that are responsible for controlling muscles and joints; thus, paralysis of muscles and loss of voluntary muscle control are common symptoms. About half of individuals with ALS die within the first 8 years after symptom onset, but more than half of patients with ALS live longer than 5 years. ALS affects most older people (between the ages of 65 and 80 and over) and people over the age of 60 are nearly twice as prone to it as younger people. About 90% of the cases of ALS worldwide were reported in Japan and the United States.
How many people get amyotrophic lateral sclerosis a year in the United States?
The American Cancer Society estimates that there will be around 21,600 new cases of ALS in the year 2012, a rate of 2.8 cases per 100,000 US. At an annual mean population growth of 1.7%, the U.S. population will expand at a rate of 7% by 2012. Based on the current rate of 1.7 cases per 100,000, approximately 15,400 new cases will be diagnosed each year. This means that, with the average lifespan of 7.5 years, roughly one quarter of the people diagnosed with amyotrophic lateral sclerosis would be diagnosed in the year 2012. Although it is unlikely, given this rate of incidence, the number of new cases will exceed the number diagnosed.
Can amyotrophic lateral sclerosis be cured?
There is not yet a cure for amyotrophic lateral sclerosis. Many patients have a long and painful dying trajectory. Nevertheless, there are patients who have a very slow and very long course of the illness. On the basis of the findings of this study, we should be thinking about future therapy options.
What are the signs of amyotrophic lateral sclerosis?
Signs of ALS can range from subtle difficulties with movement, speaking, using the hands, and swallowing, to weakness which affects the whole body, and eventual muscle atrophy. They may eventually involve the hands, arms, or legs, or may be limited to one or some areas of the body.\n
What causes amyotrophic lateral sclerosis?
Current evidence strongly suggests that genetics plays an important role in the development of ALS. While no specific gene has been identified, genetic predisposition (e.g. autosomal dominant inheritance, mitochondrial abnormalities, etc.) plays a significant role in the development of ALS. A better understanding of the role of genetics in the development of ALS will help to elucidate its underlying pathophysiology.
What are common treatments for amyotrophic lateral sclerosis?
It is critical to discuss with patients with regard to the scope of potential treatments given the progression of the disease; there is a paucity of research investigating potential therapies that are potentially curative.
How serious can amyotrophic lateral sclerosis be?
Although patients with ALS have a very low life-expectancy, an increased BMI is associated with a prolonged survival in a cohort of patients with ALS.
What is the average age someone gets amyotrophic lateral sclerosis?
If the age of onset of ALS ranges from <40 years in a cohort with an incidence peak<60 years, the average age of the disease onset for the entire cohort is 35 years, whereas in cases with a peak of the incidence between 60 and <70 years the average date of onset is 44 years.
Does amyotrophic lateral sclerosis run in families?
There may be evidence for familial and/or autosomal dominant inheritance in the disease. The presence of family members with the disease and of the disease in those with no relatives, without family histories, suggests that a new mutation(s) occurred in the families. The presence of cases in both sexes, both young and old, and the absence of sex-specific inheritance of the disease strongly suggests that the genetic determinants of this disease are shared by both sexes and that they may be different. Further research is required to find out.
What is the primary cause of amyotrophic lateral sclerosis?
The current evidence does not support a single explanation for the pathogenesis of ALS and a pathogenesis that includes both genetic and environmental factors can be proposed. However, the possibility is not completely eliminated and other plausible hypotheses remain.
Has amx0035 proven to be more effective than a placebo?
This clinical study is the largest and longest study of amx0035 for ALS to date and demonstrates that this compound has shown efficacy when compared with a placebo. In a recent study, findings validate AMX0035 as a potential treatment for both the ALS and PDD subtypes in both young and older patients and provide evidence that the efficacy of a drug in one disease subset is transferrable to patient populations for which it is not yet approved. No serious side effects were observed.
Have there been other clinical trials involving amx0035?
There has only been a study of amx0035 in ALS, or other diseases with muscle atrophy such as COPD. There is another study in a myotonic disorder with muscle atrophy (MDP, dystrophinopathia myotonicasthenia), and an experimental treatment for ALS. There were also reports of Phase II clinical trials and two other studies with amx0035 in COPD for which reports have not been published.