HEALEY ALS Platform: Multi-Center, Multi-Regimen clinical trial evaluating investigational products for the treatment of ALS

Study on Investigational Medication for ALS, a Disease Affecting Nerve Cells Leading to Muscle Weakness and Paralysis

C
Cynthia Bodkin, MD

Primary Investigator

Enrolling By Invitation
18 years - 100 years
All
Phase N/A
15 participants needed
1 Location

Brief description of study

ALS is a degenerative disorder of the nerves controlling movement (“motor neurons”). ALS causes muscles to become weak, which leads to paralysis. ALS is a condition that gradually damages the nerve cells in the brain and spinal cord.  Currently, there are no treatments to prevent, reverse, or stop the development of ALS.  However, there are some treatments that may slow down the progression of the disease. We are doing this research to find out if DNL343 can help with Amyotrophic Lateral Sclerosis (ALS).  We also want to find out if DNL343 is safe to take without causing too many side effects.
 
THIS STUDY IS ENROLLING BY INVITATION ONLY

Detailed description of study

What is Invovled
  • In person at IUH Neuroscience Center
  • Daily oral medication with weekly appointments at IUH Neuroscience Center with home appointments available after first few weeks.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Amyotrophic Lateral Sclerosis, ALS
  • Age: 18 years - 100 years
  • Gender: All

Who is Eligible
  • Ages 18+
  • Diagnosis of ALS with onset of symptoms within the last 3 years
  • Able to swallow pills and liquids
  • No history of cancer within last 3 years
  • No diagnosis of diabetes

This study investigates Amyotrophic Lateral Sclerosis (ALS), a disease that affects nerve cells in the brain and spinal cord. ALS causes muscles to weaken and leads to paralysis. The purpose of this study is to find out if an investigational medication can be safe and effective for people with ALS.

Participants will take a daily oral medication and have weekly appointments at the IUH Neuroscience Center. Home appointments may be available after the first few weeks. The study aims to assess the safety and effects of the investigational medication.

  • Who can participate: Adults aged 18 and older who have been diagnosed with ALS within the last 3 years can participate. Participants must be able to swallow pills and liquids, and should not have had cancer within the last 3 years or have a diagnosis of diabetes.
  • Study details: Participants will take a daily oral medication. The study aims to assess the safety and effects of the investigational medication.
Updated on 12 May 2026. Study ID: NEUR-MGH-HEALEY-REG-I, 11493
Please visit our main page to search for other studies you may be interested in. If you need help finding a study or have any questions, please contact us at inhealth@iu.edu

Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only