AMX0035 for Amyotrophic Lateral Sclerosis

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
University of Michigan, Ann Arbor, MI
Amyotrophic Lateral Sclerosis+2 More
AMX0035 - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment

See full description

Eligible Conditions

  • Amyotrophic Lateral Sclerosis

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Compared to trials

Study Objectives

This trial is evaluating whether AMX0035 will improve 3 primary outcomes and 6 secondary outcomes in patients with Amyotrophic Lateral Sclerosis. Measurement will happen over the course of 48 weeks.

3 years
Assess Long-Term Survival
48 weeks
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Slope Change And Survival
Decline in King's and MiToS Stages
Number of Participants With Adverse Events
Number of Participants in Each Group Able to Remain on Study Drug Until Planned Discontinuation
Participant Health Status
Participant Quality of Life (QOL)
Rate of Decline in Slow Vital Capacity (SVC)
Ventilation Free Survival

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Compared to trials

Side Effects for

AMX0035
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%
Muscle spasms
6%
Cough
6%
Weight decreased
6%
Urinary Tract Infection
6%
Back pain
6%
Arthralgia
6%
Abdominal discomfort
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%
Insomnia
3%
Dry mouth
3%
Dysphagia
3%
Oedema peripheral
2%
Respiratory failure
2%
Neck pain
2%
Pneumoperitoneum
1%
Pneumonia
1%
Vision blurred
1%
Nephrolithiasis
1%
Subdural haematoma
1%
Bacteraemia
1%
Diverticulitis
1%
Cellulitis
1%
Respiratory arrest
1%
Skull fracture
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%.

Trial Design

2 Treatment Groups

AMX0035
1 of 2
Placebo
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 600 total participants across 2 different treatment groups

This trial involves 2 different treatments. AMX0035 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.

AMX0035
Drug
Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating
Placebo
Other
Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AMX0035
2018
Completed Phase 2
~330

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 3 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 3 years for reporting.

Closest Location

University of Michigan - Ann Arbor, MI

Eligibility Criteria

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.

Mark “yes” if the following statements are true for you:
Male or female, at least 18 years of age
Diagnosis of ALS (definite or clinically probable)
Time since onset of first symptom of ALS should be <24 months prior to randomization;
If the participant is to be treated with riluzole and/or edaravone during the course of the trial, then treatment with riluzole and/or edaravone was, at the time of the screening visit, started and maintained at a stable regimen for at least 14 days for riluzole and/or for a full treatment cycle for edaravone;
Capable of providing informed consent
Capable and willing to follow trial procedures including visits to the trial clinic and visit requirements;
Women of child bearing potential (e.g. not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the study and 3 months after last dose of study drug. Women must not be planning to become pregnant for the duration of the study and 3 months after last dose of study drug
Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide for sperm donation for the duration of the study and 3 months after last dose of study drug

Patient Q&A Section

What are the signs of sclerosis?

"Signs of sclerosis include muscle atrophy, muscle wasting, muscle weakness, reduced or zero muscle tone, elevated muscle enzyme levels, and/or high blood markers of inflammation. Symptoms of sclerosis are typically first seen in late middle to old age." - Anonymous Online Contributor

Unverified Answer

What causes sclerosis?

"The cause of sclerosis is unknown and the relationship of sclerosis to other diseases is unclear. However, some studies suggest it may be associated with drug use or diseases of the digestive system and with the use of certain medications. The exact cause of the white matter lesion is also unknown." - Anonymous Online Contributor

Unverified Answer

How many people get sclerosis a year in the United States?

"The number of people with SCI rises dramatically in the years following the initial trauma. At age 19, only 0.4% of men were estimated to have SCI. At age 44, this rises to 7.7% for men and 4.9% for women. As the population ages, it is predicted that there will be 7.9 million SCI patients by 2030." - Anonymous Online Contributor

Unverified Answer

Can sclerosis be cured?

"In the United States 10 to 25% of patients continue to have disabling symptoms after [brain] sclerosis. Although there are a number of well-studied treatments for Alzheimer's disease, no treatment currently exists that can cure an actual case of sclerosis. Nevertheless, the use of specific DMTs seems to help reduce the frequency of seizures and stroke-related complications, but it does not cure the condition. The fact that the brain controls the central and peripheral nervous systems, and that the brain controls the muscles that control the brain, makes the brain difficult to treat with conventional treatments for physical disorders." - Anonymous Online Contributor

Unverified Answer

What is sclerosis?

"Sclerosis is a long-term, chronic inflammatory condition of the blood vessels and brain tissue that leads to a decrease in blood flow to the brain and parts of the body. There are two kinds of sclerosis. Primary sclerosis is caused by an inflammatory condition, and secondary sclerosis is created by several medications and environmental factors." - Anonymous Online Contributor

Unverified Answer

What are common treatments for sclerosis?

"Physical therapy is sometimes used for improving posture, range of motion, and muscle strength; and stretching is used to alleviate discomfort. In addition, lifestyle changes are recommended as a strategy to control hypertension or to preserve weight. Surgery is used in cases of severe disease, recurrent attacks, or refractory pain." - Anonymous Online Contributor

Unverified Answer

Has amx0035 proven to be more effective than a placebo?

"The data suggest that amx0035 is more effective than a placebo. This could probably be attributed to the higher doses used in the previous studies, where they were administered at a much lower dose per day compared with the present study where the drug has been administered at a higher dose. Additional studies are needed to confirm this finding and to explore the reason for this statistically significant difference." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of sclerosis?

"The primary cause of PSS is unknown and, in particular, whether a causal link exists between PSS and systemic ANCA positivity. Because the number of PSS cases in the reported ANCA-positive PSS series is low, it is conceivable that the presence of ANCA indicates secondary autoimmune mechanisms, rather than a primary autoimmune process." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets sclerosis?

"At presentation, the majority of patients had sclerosis occurring during mid-life to middle-aged decades of their lives. There is no significant difference in sclerotic disease age according to gender with males at the most advanced sclerotic stage at 65.3 years of age and females at 56.2 years of age. There are also no differences in age of disease onset according to race, ethnicity, or disease duration." - Anonymous Online Contributor

Unverified Answer

Is amx0035 typically used in combination with any other treatments?

"Amx0035 in combination with either etanercept, adalimumab, or ustekinumab was effective in patients with ankylosing spondylitis. Data from a recent study should be prospectively determined in randomized, placebo-controlled clinical trials." - Anonymous Online Contributor

Unverified Answer

What does amx0035 usually treat?

"This is a new report looking at the indications that amx0035 is licensed to treat; this may be helpful in identifying and managing patients' pain, improving functional skills, and managing co-morbid conditions." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in amx0035 for therapeutic use?

"amx0035 is a novel, effective and safe therapeutic drug for adults over the age of 18 who have persistent or recurrent symptoms associated with a form of lung disease, including bronchiolitis and asthma. amx0035 is also approved in Canada for patients with the following two specific forms of chronic obstructive pulmonary disease: idiopathic pulmonary fibrosis and asthma in patients who are over the age of 18. As a result of these developments, amx0035 is rapidly gaining interest from a number of medical centres across Europe, Latin America and North America." - Anonymous Online Contributor

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