Plain-English translation of NCT04079179 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
This trial is testing whether a medication called can safely treat rare immune cell disorders—conditions where certain immune cells grow out of control and damage organs and tissues. works by blocking the incorrect growth signals that cause these immune cells to multiply. The medication may help patients whose diseases have not responded well to standard treatments.
Many patients with these rare immune cell disorders do not get better with standard treatments, and their disease continues to cause serious damage. This medication targets the specific genetic changes that drive these diseases, offering hope for patients who have run out of other options.
You likely qualify if…
You likely don't qualify if…
If you join, you will take by mouth (as a liquid or tablet) or through a feeding tube if needed. You will have regular clinic visits where your doctor will check how well the medication is working and monitor you for side effects through blood tests, scans, and physical exams. The study involves four different groups based on age and diagnosis type, so your specific schedule and treatment plan will depend on which group you're in.
AI-generated summary from trial data · Jun 9, 2026 · Not medical advice
United States