New AMPK-activating medicine for X-linked nephrogenic diabetes insipidus in children
Novel AMPK activator for treatment of congenital X-linked nephrogenic diabetes insipidus in pediatric patients
A new drug called NDI-5001 that activates AMPK is being developed to help children with X-linked nephrogenic diabetes insipidus produce less urine and avoid dehydration.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Nephrodi Therapeutics, INC. NIH-funded |
| Lab location | 1 site (Seattle, UNITED STATES) |
| Project ID | NIH-11183830 on NIH RePORTER |
What this research studies
This program is developing NDI-5001, a first-in-class medicine designed to bypass the faulty V2 vasopressin receptor by activating AMPK and downstream signaling. The company completed extensive lab and animal studies, formulation work, and IND-enabling toxicology to characterize safety and dosing. The plan is to move into human testing in pediatric patients with genetically confirmed X-linked NDI, with careful monitoring of urine output, hydration, and safety. If trials proceed, participants will have regular clinic visits for dosing, labs, and follow-up.
Who could benefit from this research
Good fit: Ideal candidates are children (typically boys) with genetically confirmed X-linked nephrogenic diabetes insipidus due to AVPR2 mutations.
Not a fit: Patients whose diabetes insipidus is due to central (pituitary) causes, non-AVPR2 genetic forms, adults outside the pediatric age range, or those with severe kidney failure may not benefit.
Why it matters
Potential benefit: If successful, NDI-5001 could meaningfully reduce excessive urine output, lower dehydration risk, and improve growth, development, and daily life for affected children.
How similar studies have performed: This is a novel, first-in-class approach; current care uses fluids, salt restriction, and off-label diuretics or NSAIDs to manage symptoms, and there is limited clinical precedent for an AMPK activator in this condition.
Where this research is happening
Seattle, UNITED STATES
- Nephrodi Therapeutics, INC. — Seattle, United States (Active)
Researchers
- Principal investigator: Sands, Jeff M — Nephrodi Therapeutics, INC.
- Study coordinator: Sands, Jeff M
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.