Plain-English translation of NCT05983159 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.
TARGET-VM is testing two different oral medications — for slow-flow vascular malformations and for fast-flow vascular malformations — in patients whose condition hasn't improved with standard treatments. Before enrolling, you'll have genetic testing to identify which specific genetic change is causing your vascular malformation, which determines which medication you would receive. The medication is designed to target and block the exact genetic pathway driving your condition.
Many patients with vascular malformations don't respond well to current standard treatments, leaving them with few options. This trial exists to test whether these newer, targeted medications — which work by blocking the specific genetic changes causing the abnormal blood vessels — can help patients who have run out of other treatment choices.
You likely qualify if…
You likely don't qualify if…
If you qualify, you'll take the oral medication once or twice daily for 48 weeks. You'll have regular clinic visits for blood tests to check how you're responding and monitor for side effects, and your vascular malformation will be assessed during these visits. After the 48-week treatment period ends, you'll have a 24-week follow-up period with additional check-ins to see how long the benefits last and ensure you're doing well. The entire study spans about 18 months from start to finish.
AI-generated summary from trial data · Aug 20, 2026 · Not medical advice
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