KP-001 for Venous Malformations, Lymphatic Malformations, KTS/CLOVES Syndrome

This study is testing KP-001, an oral medication, against a placebo (an inactive pill) for people aged 2 and older with common venous malformations (VM), common lymphatic malformations (LM), or Klippel-Trenaunay Syndrome (KTS)/CLOVES syndrome. The main goal is to see how well KP-001 works and if it's safe. Success will be measured by changes in the size of the affected areas on MRI scans after 24 weeks. You might be able to join if your condition can't be cured by surgery or is difficult to remove. The study is currently recruiting participants.

Study design
This is a Phase 3, double-blind, randomized study comparing KP-001 to placebo, involving about 150 participants across multiple international sites.
What's involved
You would have a screening period of up to 42 days, followed by regular visits where vital signs, blood tests, and urine tests will be monitored for safety.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint is measured at 24 weeks post-dose, indicating follow-up for at least that long.

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NCT07285005

A Study to Investigate Efficacy and Safety of KP-001 Compared With Placebo in Patients Aged ≥2 Years With Common VM, Common LM, or KTS/CLOVES Syndrome

Recruiting
PHASE3Ages 2+InterventionalTreatment
Kaken Pharmaceutical
~150 participants
Updated 2026-07-22 on ClinicalTrials.gov
What's tested:KP-001Placebo

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
The ratio of volume of target lesions based on MRI
Measured over pre-does and at 24weeks post-dose
Venous Malformations
Lymphatic Malformations
Klippel-Trenaunay Syndrome
CLOVES Syndrome
1 sites across 1 states
South Carolina1
Sr. Clinical Trial Manager, Clinical Operations
Email the study team

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  • The ratio of volume of target lesions based on MRIpre-does and at 24weeks post-dose

    To confirm the efficacy of KP-001 100 mg or lower based on body weight administered once daily in patients with vascular malformations (common VM, common LM, or KTS/CLOVES syndrome) in reducing volume of target lesions at 24 weeks