New pill could shrink abnormal vessels in rare childhood disorders

NCT ID NCT07285005

Recruiting now Disease control Sponsor: Kaken Pharmaceutical Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jul 23, 2026 · Updated 3 times

Summary

This Phase 3 trial tests an oral drug called KP-001 in 150 people aged 2 and older with venous malformations, lymphatic malformations, or KTS/CLOVES syndrome. Participants receive either KP-001 or a placebo for 24 weeks, then everyone gets the drug for another 28 weeks. The goal is to see if the drug can shrink the abnormal vessels and improve symptoms.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: KP-001 (an oral drug taken once daily)
What this could lead to: If successful, this could provide a new treatment option to shrink abnormal blood or lymph vessel growths and ease symptoms for people with these rare conditions.
What could go wrong: This is an early Phase 3 trial with only 150 participants, so results may not apply to everyone. The drug may not work better than placebo, and side effects are still being studied.

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Conditions

The condition(s) this trial relates to.

angioosteohypertrophic syndrome CLOVES syndrome Lymphatic Abnormalities lymphatic malformation venous hemangioma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CLOVES SYNDROME KLIPPEL TRENAUNAY SYNDROME LYMPHATIC MALFORMATIONS VENOUS MALFORMATIONS

Contacts and locations

Locations

MUSC Children's Health Primary Care
RECRUITING

Charleston, South Carolina, 29492, United States

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