CLINICAL TRIAL

AMX0035 for Amyotrophic Lateral Sclerosis

EnrollingByInvitation · 18+ · All Sexes · Ann Arbor, MI

This study is evaluating whether a drug called AMX0035 can help people with amyotrophic lateral sclerosis (ALS) live longer.

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About the trial for Amyotrophic Lateral Sclerosis

Eligible Conditions
Motor Neuron Disease · Sclerosis · 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.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
AMX0035
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AMX0035
2018
Completed Phase 2
~330

Side Effect Profile for AMX0035

AMX0035
Show all side effects
Fall
28%
Diarrhoea
21%
Muscular weakness
20%
Nausea
18%
Headache
15%
Constipation
13%
Viral Upper Respiratory Tract Infection
11%
Salivary hypersecretion
11%
Dizziness
10%
Dyspnoea
10%
Abdominal pain
8%
Decreased appetite
8%
Fatigue
8%
Laceration
7%
Weight decreased
6%
Urinary Tract Infection
6%
Back pain
6%
Cough
6%
Arthralgia
6%
Abdominal discomfort
6%
Muscle spasms
6%
Musculoskeletal pain
6%
Rash
6%
Asthenia
6%
Contusion
6%
Aspartate aminotransferase increased
4%
Upper Respiratory Tract Infection
4%
Musculoskeletal chest pain
4%
Alanine aminotransferase increased
4%
Abdominal pain upper
4%
Dry mouth
3%
Insomnia
3%
Dysphagia
3%
Oedema peripheral
2%
Respiratory failure
2%
Neck pain
2%
Nephrolithiasis
1%
Pneumoperitoneum
1%
Vision blurred
1%
Subdural haematoma
1%
Bacteraemia
1%
Diverticulitis
1%
Cellulitis
1%
Respiratory arrest
1%
Skull fracture
1%
Pneumonia
1%
Stoma site haemorrhage
1%
Pulmonary Embolism
0%
Device dislocation
0%
Catheter Site Infection
0%
Pelvic fracture
0%
This histogram enumerates side effects from a completed 2019 Phase 2 trial (NCT03127514) in the AMX0035 ARM group. Side effects include: Fall with 28%, Diarrhoea with 21%, Muscular weakness with 20%, Nausea with 18%, Headache with 15%.

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.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
People who have been seeing a neurologist at a center for ALS patients and will continue doing so for the duration of the study. show original
Women must not lactate while taking the study drug or for 3 months after the last dose. show original
Is able to visit the study site frequently for short visits, and can also attend remote study visits through telemedicine if that is currently being used at the site. show original
, The subject is capable of and willing to undergo the procedures involved in a clinical trial. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Through study completion an average of 1 year
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Through study completion an average of 1 year.
View detailed reporting requirements
Trial Expert
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- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

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.

Treatment emergent Adverse Events
THROUGH STUDY COMPLETION AN AVERAGE OF 1 YEAR
Rate of treatment emergent adverse events during AMX0035 therapy

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer
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