Columbia–Weill Cornell NeuroNEXT clinical trial sites for neurological disorders
Clinical Research Sites for the Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT sites) (U24 Clinical Trial Not Allowed)
This program runs early-phase clinical trials at Columbia and Weill Cornell testing gene-targeting treatments like antisense oligonucleotides for adults with rare neurological conditions such as ALS, SMA, Huntington’s disease, and Duchenne.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11128358 on NIH RePORTER |
What this research studies
At Columbia and Weill Cornell a dedicated team runs NeuroNEXT trial sites that enroll adults with rare genetic neurologic diseases into early-phase trials. The site coordinates visits, safety monitoring, sample collection, and specialized genetic treatments such as antisense oligonucleotides and n-of-1 trials. Experienced investigators and recent NeuroNEXT fellows work together to open and run trials quickly and manage complex protocols. If you have a rare neurologic condition, you may be invited to participate in trials that offer experimental therapies not available elsewhere.
Who could benefit from this research
Good fit: Ideal candidates are adults with rare genetic neurological conditions (for example ALS, SMA, Huntington’s disease, or Duchenne muscular dystrophy) who are willing to travel to an academic center and participate in early-phase trials.
Not a fit: People who are not adults, who have non-genetic forms of neurological disease, or who are not eligible for early-phase trials may not gain direct benefit from this program.
Why it matters
Potential benefit: If successful, this network could give people with rare genetic neurological diseases earlier access to promising gene-targeted treatments and speed new therapies toward approval.
How similar studies have performed: Similar antisense oligonucleotide approaches have led to approved treatments for spinal muscular atrophy and shown promising early results in other genetic neurological diseases.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Marder, Karen S — Columbia University Health Sciences
- Study coordinator: Marder, Karen S
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 below.