Using rifampin to lower high calcium levels in patients with specific genetic mutations
Official title: Repurposing rifampin to reduce elevated levels of blood and urine calcium in patients with inactivating mutations of CYP24A1
Funded at Children's Hosp of Philadelphia · NIH-10991391 · Data updated 2026-11-12
This study is looking at whether the antibiotic rifampin can help babies with idiopathic infantile hypercalcemia, a condition that causes high calcium levels due to a gene mutation, by lowering their calcium levels and improving their health.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | December 23, 2022 |
| Project end | November 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $657,228 |
| NIH project number | 5R01DK112955-08 |
| Project ID | NIH-10991391 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are infants and children diagnosed with idiopathic infantile hypercalcemia linked to mutations in the CYP24A1 gene.
✕Less likely to help
Patients without CYP24A1 mutations or those with other forms of hypercalcemia may not benefit from this research.
What this research studies
This research investigates the use of rifampin, an FDA-approved antibiotic, to treat idiopathic infantile hypercalcemia (IIH) caused by mutations in the CYP24A1 gene. The study aims to assess the safety and effectiveness of rifampin in reducing elevated levels of calcium in the blood and urine of affected patients. By targeting the underlying metabolic defect that leads to excessive vitamin D levels, the research seeks to provide a new treatment option for patients who currently have limited management strategies. Participants will be monitored for changes in calcium levels and overall health throughout the trial.
Why it matters
Potential benefit: If successful, this research could offer a new long-term treatment option for patients suffering from elevated calcium levels due to CYP24A1 mutations.
How similar studies have gone: While the use of rifampin for this specific condition is novel, other studies have explored repurposing existing medications for metabolic disorders with varying degrees of success.
Where this research is happening
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Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Levine, Michael a. — Children's Hosp of Philadelphia
- Study coordinator: Levine, Michael a.
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.