DNL343 for Amyotrophic Lateral Sclerosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called DNL343 for people with amyotrophic lateral sclerosis (ALS), a disease affecting nerve cells in the brain and spinal cord. The trial aims to determine if DNL343 is safe and how it behaves in the body. Participants will receive either a low dose, a high dose, or a placebo (a non-active substance) to compare effects. People with ALS who have had symptoms for 4 years or less, are on stable ALS medications, and can swallow pills might be a good fit for this trial. As a Phase 1 trial, this research focuses on understanding how DNL343 works in people, offering participants the opportunity to be among the first to receive this new treatment.
Is there any evidence suggesting that DNL343 is likely to be safe for humans?
Research shows that DNL343 is under study to determine its safety for people with amyotrophic lateral sclerosis (ALS). In earlier studies, researchers tested DNL343 on both healthy individuals and those with ALS. The results indicated that the treatment was generally well-tolerated. Some side effects occurred, but they were not serious and resembled those seen with other treatments.
This trial is in the early stages, so researchers are primarily focused on assessing the treatment's safety. While detailed information about all possible side effects is not yet available, these early results provide insight into the safety of DNL343.12345Why do researchers think this study treatment might be promising?
Researchers are excited about DNL343 for treating Amyotrophic Lateral Sclerosis (ALS) because it works differently from existing options like Riluzole and Edaravone. Most ALS treatments focus on slowing disease progression by targeting neurotransmitter pathways or reducing oxidative stress. However, DNL343 is unique because it targets protein synthesis pathways, potentially addressing the underlying causes of neuronal damage. This novel approach could offer a new way to slow or alter the disease course, providing hope for better outcomes in ALS management.
What evidence suggests that DNL343 might be an effective treatment for ALS?
Research suggests that DNL343, which participants in this trial may receive, might help treat amyotrophic lateral sclerosis (ALS). Early studies show that DNL343 can reach the brain and spinal fluid, the areas affected by ALS. It works by blocking a cellular process called the integrated stress response (ISR), which contributes to ALS development. Although it did not achieve the main goals of improving function and survival in some trials, it was safe and well-tolerated. DNL343 has also shown promise in possibly lowering a key blood marker linked to ALS. Overall, there is hope that DNL343 could help manage ALS symptoms in the future.24567
Who Is on the Research Team?
Linus Sun, MD, PhD
Principal Investigator
Denali Therapeutics Inc.
Are You a Good Fit for This Trial?
Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either DNL343 or placebo for 28 days to evaluate safety, pharmacokinetics, and pharmacodynamics
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants may opt into continuation of treatment with DNL343 for 18 months
What Are the Treatments Tested in This Trial?
Interventions
- DNL343
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Denali Therapeutics Inc.
Lead Sponsor