Awhi ALS Study

ALS (Amyotrophic Lateral Sclerosis), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder that affects nerve cells in the brain and spinal cord. It leads to loss of muscle control, eventually affecting speech, swallowing, movement, and breathing.

There is currently no cure for ALS, but some treatments are available to help slow the progression of the disease and manage symptoms. Medications such as Riluzole are approved and have shown modest benefits in extending survival and preserving physical function. Supportive care is a critical part of managing ALS. A multidisciplinary team, including neurologists, physical and occupational therapists, speech-language pathologists, and respiratory specialists can work together to improve one’s quality of life.

Despite its challenges, many people living with ALS benefit from coordinated care, support networks, and ongoing medical advances. As research continues to uncover more about the causes and potential treatments for ALS, there is growing hope for improved outcomes and, one day, a cure.

About the investigational treatment V1
The investigational treatment being studied is a new approach for people living with ALS (Amyotrophic Lateral Sclerosis). It’s made from a combination of naturally occurring compounds called short-chain fatty acids (SCFAs). These SCFAs are important for gut health and reducing inflammation in the body.

In this treatment, the SCFAs are attached to a carrier molecule called poly-L-lysine, which helps deliver them more effectively in the body. Together, this combination is designed to target gut health and immune balance; two areas that researchers believe may influence how ALS progresses.

By supporting the gut microbiome and helping to reduce harmful inflammation, the investigational treatment may help slow down the progression of ALS symptoms and improve quality of life. This study is testing whether it’s safe and if it can make a difference for people with ALS.

About the investigational medication V2
This is a Phase 1/2 clinical trial testing PLL001, an investigational treatment for ALS. PLL001 is a combination of naturally occurring molecules (short-chain fatty acids) designed to potentially improve the gut microbiota and reduce intestinal permeability, which may play a role in ALS progression.

Main Aim/Purpose:
• Evaluate the safety, tolerability, and effectiveness of PLL001 in people with ALS
• Assess whether PLL001 can slow disease progression and improve quality of life
• Study the impact on ALS functional rating scores and survival

Study Aim
The main purpose of this study is to learn about the study drug and evaluate its safety, tolerability and effectiveness in treating ALS. Researchers are also assessing whether the study drug can slow disease progression and improve quality of life.

Reason to Participate
The study addresses the significant unmet medical need in ALS treatment, as current options are limited. Your involvement in this study will help advance scientific knowledge that could contribute to the development of a new medication and better treatment for people with ALS. All participants will receive close monitoring by specialised medical teams.

About the Study
Around 150 people worldwide are taking part in this study. Taking part in the study involves a first meeting with the research Doctor to find out more about your health and to see if the study is right for you. If you wish to take part and you are eligible, we would like to see you for 5 to 46 weeks with regular clinic visits (depending on which part of the study you join). There are 3 parts to this study. Please see details below:

Part 1 (Single Dose): Approximately 5 weeks with an overnight hospital stay

Part 2 (Main Study): 30 weeks total
- 24 weeks of daily treatment (self-administered at home)
- Up to 6 clinic visits (Days 1, 15, 57, 113, 169, and 183)
- Weekly phone check-ins

Part 3 (Optional Extension): Additional 26 weeks for those who wish to continue
- Clinic visits every 8 weeks
- Weekly phone check-ins

The treatment involves daily subcutaneous injections that participants or their caregivers can administer at home after initial training.

At the visits, we undertake health checks which can include physical examinations and blood tests. We would like to ask you questions about your ALS throughout the study. Other procedures include: (but aren’t limited to)
• ALS functional rating scale questionnaires
• Daily study drug administration
• Injection site monitoring
• Biochemistry and organ function tests
• Physical and Neurological exams
• Slow vital capacity tests
• Hand-held dynamometry
• Gut microbial analysis
• Quality of life assessments  

The study medication is taken as subcutaneous injection, meaning that it is injected just under the skin. The study medication is required to be administered daily.

The study aims to investigate the investigational treatment and to show that it is safe and effective in improving the gut microbiome, reduce intestinal permeability which may play a role in ALS progression.