Navigating ALS Clinical Trials: A Quick Overview of Ongoing ALS Research Studies, Participation, and Progress
- Hetvi Vashi
- 5 days ago
- 9 min read
Updated: 4 days ago
As ALS research continues to evolve further to offer different treatment options, the ALS clinical trials play an important role to present innovative choices for ALS therapy and patient-care. Let’s delve deeper into understanding ALS disease and clinical trials.
Understanding ALS & the Importance of Clinical Trials
What is ALS?
Amyotrophic Lateral Sclerosis, or ALS, is a brutal neurodegenerative disease which disrupts the functioning of nerve cells from the brain and spinal cord. ALS disease is very challenging to diagnose, as there are no specific biomarkers associated with the condition. It leads to severe muscle weakness, impacting mobility, and extreme difficulty in speech, leading to paralysis and eventually respiratory failure. The average survival times after ALS onset is approximately three years, with very few treatment options available. Raising awareness of the disease is critical to driving progress and increasing ALS life expectancy.
What are Clinical Trials?
Clinical trials, a type of clinical research study, are interventional and focus on assessing new treatments and the efficacy of drugs to determine their potential to cure or manage specific diseases. According to the U.S. FDA guidelines, any new drug or treatment must be evaluated through a series of large-scale clinical trials to ensure it meets established standards for safety and efficacy. The FDA reviews the data and outcomes from clinical trials to determine whether to approve or reject a drug or treatment.
Why do Clinical Trials Matter in ALS Research?
Clinical trials support the treatment of people living with ALS (pALS) by providing therapeutic options specific to the progression and stages of ALS. The key focus of researchers is to investigate the safety and efficacy of the drugs or treatment options, with the goal of extending and improving the quality of life for pALS. The discovery of new therapies through clinical trials can benefit people living with ALS by slowing the disease progression or extending survival. Through rigorous testing phases with patient-centric approaches, clinical trials accelerate the path from research to real-world treatment.
What are ALS Clinical Trials?
ALS clinical trials can be classified into two broader categories – novel approaches for ALS treatment and studies that help uncover ALS disease. Typically, clinical trials consist of several phases to determine the best dose, evaluate any potential side effects and analyze the drug’s efficacy. Clinical trials typically proceed through the following phases:
Pilot trial:
This is a preliminary study on a small scale to understand details concerning the safety and side effects of the ALS drug, find any biomarkers relevant to ALS disease or examine the design to be plotted for larger clinical trials. Pilot trials involve patients with ALS to test drugs priorly approved for other conditions.
Phase 1:
The objective of Phase 1 clinical trials is to initiate investigational treatments in humans. This involves administering the drug to a very small group, typically around 20 people. The researchers monitor all participants for any side effects or adverse reactions. During the study, if the drug appears to be too harmful, the trial is stopped immediately and the drug is withdrawn from any further clinical trials.
Phase 2:
Once the safety of the drug is tested on a small scale, the researchers look for the most effective dose, route of administration (oral, intravenous, intranasal, etc.), and the frequency and timing of doses. Phase 2 assesses the efficacy of the treatment to enhance the motor functioning for people living with ALS. This phase will help determine if the treatment can be effective for patients living with ALS. Phase 2 involves no more than 100 people living with ALS, but studies concerning investigational treatments need to include a larger group of people.
Phase 3:
Phase 3 of ALS clinical trials involves a large number of participants, which are adequate to confirm the treatment efficacy. With hundreds of participants, the study analyses the effect of the drug in pALS with different stages of ALS, and varying life expectancy for ALS. Once Phase 3 of ALS clinical trials is complete, the results are submitted to the FDA so they may review the data and consider whether to approve the treatment.
Phase 4:
This is the final phase of the ALS clinical trials and involves ongoing monitoring of the drug’s safety and efficacy of the ALS drug its approval by the FDA.
How to Find and Join ALS Clinical Trials
Navigating the world of ALS clinical trials begins with regularly checking the status of ongoing clinical trials and understanding the outcomes of those that have been completed. Several reliable platforms provide up-to-date information on trials, including eligibility criteria, locations, and enrollment status, which we’ll explore here.
Where to Search for ALS Clinical Trials:
This U.S. government database provides information on all registered clinical trials worldwide. The website can be easily searched for ALS clinical trials by entering the condition (ALS), or other details like location, phase, and by adding filters or other criteria.
ALS Trial Navigator:
This website is managed by the ALS Therapy Development Institute, and it provides up-to-date information on ALS clinical trials. This tool helps pALS and families find clinical trials suited to their personal medical history and disease progression.
National ALS Registry:
Managed by the Centers for Disease Control & Prevention (CDC), the National ALS Registry is another credible website with more information about ALS clinical trials. It connects the patients living with ALS in the U.S. with research opportunities and also offers valuable public health data.
In addition, EverythingALS also conducts several research studies to analyze the different stages of ALS using digital biomarkers to provide better clarity on the progression of ALS disease and life expectancy. Join our ALS research here and be a part of one or more ongoing ALS research studies.
Who can Participate in Clinical Trials?
Clinical trials for different diseases and conditions are designed with specific eligibility criteria to ensure patient safety and accurate data. Some of the common eligibility criteria, especially for ALS disease, are as follows:
Age:
Many clinical trials specify an age window, often between 18 and 65 years, to target appropriate populations and reduce age-related risks.
Disease characteristics:
Eligibility often varies according to the type and different stages of ALS. For instance, some clinical trials require a “definite” or “probable” ALS diagnosis, and may specify whether participants have sporadic or familial ALS. Other factors may include the duration of the disease and progression, and some trials may exclude patients with very advanced disease or slow progression.
Genetic markers:
Certain clinical trials focus on patients living with ALS with specific genetic mutations linked to ALS, such as SOD1 or C9orf72, to test targeted therapies.
Respiratory function:
Measures like forced vital capacity (FVC) or slow vital capacity (SVC) are often used to assess lung function, with minimum thresholds (typically 50-65%) required for participation.
Overall Health and Prior Treatments:
Participants generally must be in a healthy state to participate in clinical trials safely. They may be excluded if they have had a recent occurrence of certain cancers, tracheostomy, or use of assisted ventilation. Concurrent use of other investigational drugs is usually prohibited.
Steps to Enroll in a Clinical Trial
Eligibility Criteria and Screening:
ALS clinical trials’ enrollment begins with a thorough screening process to ensure that participants meet specific eligibility criteria designed to protect their safety and maintain the scientific integrity of the study. These criteria often include age, disease stage, genetic markers, respiratory function, and overall health status. During screening, study staff will review your ALS history, medical background, medications, and perform baseline assessments such as blood work, neurological exams, and respiratory tests. It’s important to know that many trials exclude a significant portion of ALS patients—on average, about 60%—but newer trials are working to broaden inclusion criteria to be more accessible.
Questions to Ask Before Participating:
Before agreeing to participate in a clinical trial, it is crucial to ask the research team key questions in order to fully understand what participation entails. Key questions include:
What is the purpose of the study?
Have similar studies been done, and what were their outcomes?
Who is funding the trial?
How will my privacy and medical information be protected?
What are the possible risks and benefits?
Will I receive a placebo, and if so, how is that managed?
How long will the study last, and what will be required of me?
Can I continue my current medications during the trial?
What support is available if I experience side effects or harm?
Discussing these questions with your doctor or ALS care team can help you make an informed decision about participation. It is important that all participants have informed consent by understanding the complete trial process and make a choice of participation aligning with the ALS clinical trials.
Support from ALS Care Teams and Networks
Enrolling in a clinical trial doesn't mean you're on your own. In fact, you’ll have support at every stage:
Neurologists and specialists can help evaluate your eligibility and explain how a trial fits into your care plan.
Trial coordinators will assist with paperwork, scheduling, and transportation logistics.
Patient advocacy organizations like EverythingALS provide community support, education, and guidance on trial selection and participation.
Current and Promising ALS Clinical Trials
ALS clinical trials are testing a range of innovative approaches to slow disease progression, manage symptoms, and work toward a cure. These include gene therapies targeting mutations like SOD1 and C9orf72, stem cell treatments to protect motor neurons, and drug trials using small molecules or repurposed medications. Observational studies collect real-world data to guide future research, while adaptive and platform trials allow for more flexible and efficient testing.
Notable Ongoing and Upcoming Trials
1. AP-101 (AL-S Pharma)
Type: Intravenous monoclonal antibody targeting SOD1 activation
Phase: IIa
Status: Active, recruiting (Primary completion June 2024)
Description: Evaluates safety, tolerability, pharmacodynamics, and pharmacokinetics in familial and sporadic ALS patients. Targets SOD1 mutations to reduce toxic protein effects.
2. RAPA-501 (Rapa Therapeutics)
Type: Autologous T cell therapy (epigenetically reprogrammed regulatory T cells)
Phase: II/III
Status: Actively recruiting (Primary completion July 2025)
Description: Designed to reduce neuroinflammation by enhancing anti-inflammatory T cell populations, potentially protecting motor neurons.
3. ALN-SOD (Regeneron Pharmaceuticals)
Type: RNA interference (RNAi) gene therapy targeting SOD1 mutation
Phase: I
Status: Actively recruiting (Started August 2024)
Description: Aims to reduce toxic SOD1 protein production in familial ALS patients with SOD1 mutations.
4. SNUG01 (Peking University Third Hospital)
Type: AAV vector-based gene therapy
Phase: Early Phase I
Status: Actively recruiting (Started October 2024)
Description: Uses viral vectors to deliver therapeutic genes to motor neurons to slow ALS progression.
5. Renzumestrocel (Corestemchemon)
Type: Autologous bone marrow-derived mesenchymal stem cell therapy
Phase: III
Status: Active but not recruiting
Description: Stem cell therapy aimed at slowing disease progression by delivering neuroprotective factors.
6. Healey ALS Platform Trial (Massachusetts General Hospital)
Type: Adaptive platform trial testing multiple investigational drugs
Phase: Ongoing
Status: Actively recruiting
Description: Tests multiple therapies simultaneously using shared placebo groups to accelerate drug development.
7. RESTORE-ALS (Cytokinetics)
Type: Small molecule drug CNM-AU8
Phase: III
Status: Ongoing
Description: Evaluates whether CNM-AU8 can improve survival and slow disease progression in ALS patients.
These trials represent a range of therapeutic approaches including gene therapies, cell therapies, monoclonal antibodies, and small molecules, reflecting the dynamic progress in ALS research. For detailed eligibility and enrollment information, visit ClinicalTrials.gov and the ALS Trial Navigator.
How Participation Advances ALS Research
Participation in ALS research is vital to advancing treatment options and improving patient outcomes. When pALS and their caregivers take part in clinical trials, observational studies, or data collection efforts, they directly contribute to the development of new treatments and diagnostic tools. Their involvement helps researchers better understand disease progression, identify patterns, and fine-tune therapies based on real-world experiences. Caregivers also provide essential insights into daily challenges and care needs, making research more patient-centered and impactful.
EverythingALS is leading several innovative, remote-friendly studies that anyone in the U.S. can join. These include voice and speech analysis to detect early signs of ALS, movement tracking through wearables and smartphones, digital biomarker development, and caregiver-focused surveys to improve support systems. Our studies are designed to be inclusive, accessible, and easy to participate in from home.
Join an ongoing study here or download the EverythingALS app to contribute to the future of ALS research.
CONCLUSION
ALS clinical trials are at the forefront of advancing new discoveries, offering hope to people living with ALS and families. By participating in research, individuals help accelerate treatments and bring us closer to a cure. From gene therapies to digital studies, opportunities are expanding across the U.S. Every contribution matters—whether through trials, data, or advocacy. Stay informed, get involved, and be part of the future of ALS research.
Frequently Asked Questions
Are ALS clinical trials safe?
Yes, clinical trials follow strict ethical guidelines and regulatory oversight to ensure participant safety. When participants are registered for the study, they are clearly informed about the risks during informed consent, with continuous monitoring throughout the study.
What are trusted websites to access updates on recent ALS clinical trials?
The most preferred website to access ALS clinical trials information is https://clinicaltrials.gov/. Other websites include https://www.alsnavigator.org/ (ALS Trial Navigator, ALS TDI), and https://www.cdc.gov/als (National ALS Registry, CDC)
What is the newest ALS treatment approved by the FDA?
The newest ALS treatment approved by the FDA is Tofersen (Qalsody). Tofersen is specifically designed for SOD1 gene related ALS.
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