This clinical trial focuses on VEXAS syndrome, a rare genetic condition that causes inflammation in various parts of the body and can affect blood cell production. The study will test a medication called azacitidine, which will be given as an injection under the skin (subcutaneous injection). This medicine is typically used to treat blood disorders and works by modifying how genes function in the body.
The purpose of this research is to determine if azacitidine can reduce the amount of abnormal genetic material in patients with VEXAS syndrome. The treatment involves receiving azacitidine injections for six treatment cycles. During the study, participants will receive up to 200 milligrams of the medication per day, with a maximum total dose of 10,000 milligrams over a period of up to 10 months.
This is a Phase II study, which means researchers are testing the medication’s effectiveness in a group of patients with VEXAS syndrome. The study will specifically look at how well the treatment works in reducing certain genetic changes associated with the disease. Throughout the treatment period, participants will be monitored for both the medication’s effectiveness and any potential side effects.



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