Clinical Studies


Clinical trials in Amyotrophic Lateral Sclerosis (ALS) are research studies conducted to evaluate the safety and effectiveness of new treatments, interventions, or therapeutic strategies for the disease. These trials play a crucial role in advancing our understanding of ALS, testing potential therapies, and improving patient care. Here is an overview of clinical trials in ALS:

Phases of Clinical Trials:

Phase I: Initial testing involves a small group of participants to assess the safety, dosage, and potential side effects of a new treatment.

Phase II: Expands the trial to a larger group to further evaluate safety and begin assessing effectiveness.

Phase III: Involves a larger population to confirm efficacy, monitor side effects, and compare the new treatment to existing standard treatments.

Phase IV: Conducted after the treatment is approved and available to the public, monitoring its long-term effects and gathering additional information.

Types of Clinical Trials:

Treatment Trials: Evaluate new medications, therapies, or interventions.

Prevention Trials: Investigate ways to prevent the development or recurrence of ALS.

Screening Trials: Focus on early detection or identification of biomarkers.

Quality of Life Trials: Assess strategies to improve the well-being and quality of life for individuals with ALS and their families.

Randomized Controlled Trials (RCTs):

  • Gold standard in clinical research.
  • Participants are randomly assigned to different treatment groups (experimental or control).
  • Minimizes bias and allows for a more robust assessment of treatment efficacy.
Endpoints in ALS Clinical Trials:

Primary Endpoint: The main outcome measure used to determine treatment effectiveness.

Secondary Endpoints: Additional measures providing more comprehensive insights into treatment effects.

Common endpoints include survival rates, changes in functional abilities, and quality of life assessments.

Challenges in ALS Clinical Trials:
  • ALS is a complex and rapidly progressing disease, posing challenges in trial design and duration.
  • Individuals with ALS who engage in clinical trials need to meet specific criteria related to the disease or its severity (referred to as "inclusion criteria"). Conversely, they may be deemed ineligible if they exhibit characteristics ("exclusion criteria").
  • Limited understanding of disease mechanisms hinders the identification of effective targets.
  • The need for sensitive and specific biomarkers to track disease progression and treatment response.
  • As trials involve a limited number of participants, statistical analysis is crucial to generalize findings to the broader patient population.
  • To ensure the efficacy of a new treatment, researchers must distinguish between the actual effects of the drug and the placebo effect. Control groups are employed to balance the impact of positive thinking, providing a clearer understanding of the true effects of the treatment.
Ongoing Research Areas:
  • Stem cell therapies, gene therapies, and neuroprotective agents are among the areas actively investigated.
  • Biomarker discovery to improve diagnosis, prognosis, and treatment monitoring.
  • Exploration of novel therapeutic targets based on evolving insights into the underlying biology of ALS.

It's important for individuals with ALS and their caregivers to stay informed about ongoing clinical trials and, if eligible, consider participating in these trials to contribute to advancements in ALS research and potentially access new treatments. Participation in clinical trials is always voluntary, and potential participants should carefully consider the risks and benefits in consultation with their healthcare providers.

Learn More

NEALS (Northeast Amyotrophic Lateral Sclerosis Consortium): NEALS is an academic research consortium, research organization and resource tool. They will have information about clinical trials.

US National Library of Medicine/Clinical Trials: This is a web-based resource that allows for easy access to gather information on clinical studies on a wide range of diseases and conditions. Enter Amyotrophic Lateral Sclerosis into Condition or Disease space.