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sodium valproate + sirolimus + calcitriol
- Development stage
- Phase 2
- Lead developer
- Chinese PLA General Hospital
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
sodium valproate + sirolimus + calcitriol is an experimental combination therapy being investigated for the treatment of severe hemophilia A and B. Developed by Xue-chun Lu at the PLA General Hospital, the regimen consists of oral sodium valproate (an extended-release histone deacetylase inhibitor), sirolimus (an mTOR inhibitor and immunosuppressant), and calcitriol (a Vitamin D receptor agonist). The combination is hypothesized to improve clinical outcomes in severe hemophilia patients, potentially by modulating the immune system to prevent inhibitor formation or by epigenetically upregulating endogenous clotting factor production. It is currently being evaluated in a Phase 1/2 clinical trial to determine its efficacy and safety as an adjunct to primary treatment.
- Other names
- Combination Regimen for Severe Hemophilia
02
Targets
ABAT (4-aminobutyrate aminotransferase)HDAC (HDAC family)mTOR (Mammalian target of rapamycin kinase)VDR (Vitamin D receptor)NaV (Voltage-gated sodium channels)
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