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

A Phase 3, Randomized, Parallel, Multicenter, Double-blind, Placebo-controlled Study to Investigate Efficacy and Safety of KP 001 in Patients Aged ≥2 Years With Common Venous Malformations, Common Lymphatic Malformations, or KTS/CLOVES Syndrome

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 150 (estimated)
• Sponsor: Kaken Pharmaceutical · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 3, double-blind, randomized, placebo-controlled, parallel group, adaptive, multicenter study planned to be conducted at multiple sites in North America, Canada, Taiwan and South Korea. The purpose of this study is to measure the efficacy and safety of KP-001 compared with placebo in patients aged ≥2 years with common VM, common LM, or KTS/CLOVES syndrome. An independent data monitoring committee (DMC) will be established to determine whether to discontinue or continue the study. It will also determine the redesign of the number of cases based on the result of the interim analysis. The study will comprise the following: * Screening Period: Up to 42 days prior to the first dose of study intervention. * Treatment Period 1: This is a double-blind period in which KP-001 100 mg (or lower dose depending on their body weight) or placebo will be administered to patients once daily after breakfast until Week 24. * Treatment Period 2: After 24 weeks of double blind treatment, all patients will switch to the KP-001 open label extension and treated up to Week 52. * Follow-up Visit: This visit will occur 30 days after the last dose of study intervention, and assessments will be performed per the SoA. * Discontinuation Visit: Patients who discontinue study intervention will be requested to continue participating in the study and assessments will be performed per the SoA. If the patients request to withdraw from the study, all tests and evaluations when possible will be performed at Discontinuation visit.

Detailed description

Safety and Exploratory Assessments Vital signs (blood pressure, heart rate, respiratory rate, and body temperature) will be monitored at each scheduled visit for safety. Safety laboratory assessments will include hematology, serum chemistry, coagulation parameters (e.g., PT/INR, aPTT, fibrinogen), and urinalysis. Coagulation biomarkers other than D-dimer will be collected for exploratory/safety purposes only and will not be included in the reported outcome measures. Exploratory analyses may include the relationship between changes in Symptom Numeric Rating Scale (NRS) scores and Patient Global Impression of Severity (PGI-S) scores.

Conditions

• Venous Malformations
• Lymphatic Malformations
• Klippel-Trenaunay Syndrome
• CLOVES Syndrome

Interventions

Type	Name	Description
DRUG	KP-001	Oral repeated dose 100mg or lower
DRUG	Placebo	Oral repeated dose

Timeline

• Start date: 2026-02-26
• Primary completion: 2028-02-01
• Completion: 2028-02-01
• First posted: 2025-12-16
• Last updated: 2025-12-19

Regulatory

• FDA-regulated drug study