Investigating a new treatment for Amyotrophic Lateral Sclerosis using Pridopidine
Official title: An Intermediate-Size Expanded Access Protocol for Amyotrophic Lateral Sclerosis with Pridopidine
Funded at Massachusetts General Hospital · NIH-10934376 · Data updated 2026-11-12
This study is looking at how a medication called Pridopidine might help people with ALS by targeting a specific receptor in the brain, and if you join, you could get access to this treatment while researchers learn more about its safety and benefits.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 25, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $9,831,153 |
| NIH project number | 5U01NS136021-02 |
| Project ID | NIH-10934376 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Amyotrophic Lateral Sclerosis who are seeking new treatment options.
✕Less likely to help
Patients with other neurological disorders or those who do not have a diagnosis of ALS may not benefit from this research.
What this research studies
This research focuses on the Sigma 1 Receptor (S1R) as a potential therapeutic target for patients with Amyotrophic Lateral Sclerosis (ALS). The study will explore the effects of Pridopidine, a selective S1R agonist, on disease progression and symptoms in ALS patients. Participants may receive Pridopidine as part of an expanded access protocol, which allows them to access this treatment while ongoing clinical trials are being conducted. The research aims to gather data on the safety and efficacy of Pridopidine, potentially improving the quality of life for those affected by ALS.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option that may slow disease progression and improve symptoms for ALS patients.
How similar studies have gone: Previous research has shown promising trends in ALS treatment with S1R agonists, indicating potential for success with this approach.
Where this research is happening
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Paganoni, Sabrina — Massachusetts General Hospital
- Study coordinator: Paganoni, Sabrina
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.