# Clinical Trial Readiness for K channel inhibitors in Cantu Syndrome

> **NIH NIH R21** · WASHINGTON UNIVERSITY · 2020 · $196,875

## Abstract

PROJECT SUMMARY
Cantu syndrome (CS) is a rare genetic condition for which there is currently no directed therapy. CS patients suffer
from multiple pathologies, but cardiovascular complications, and neurological features are major concerns. CS
results from gain-of-function mutations in two specific genes that encode cardiovascular ATP-sensitive (KATP)
channels. FDA-approved blockers of these channels are potential treatments. In preparation for a clinical trial, this
project will validate cardiovascular and neurological features as markers of disease in a unique cohort of CS
patients, and will validate KATP channel inhibitors as appropriate therapy in unique animal models of CS and in
patient-derived cells. Successful accomplishment of the project will complete clinical trial readiness for the
proposed therapeutic approach.

## Key facts

- **NIH application ID:** 10053408
- **Project number:** 1R21HD103347-01
- **Recipient organization:** WASHINGTON UNIVERSITY
- **Principal Investigator:** Dorothy Katherine Grange
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $196,875
- **Award type:** 1
- **Project period:** 2020-08-01 → 2022-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10053408

## Citation

> US National Institutes of Health, RePORTER application 10053408, Clinical Trial Readiness for K channel inhibitors in Cantu Syndrome (1R21HD103347-01). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10053408. Licensed CC0.

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