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

Part of paid clinical trials in Charleston, South Carolina.

Sponsor
Kaken Pharmaceutical
Study ID
NCT07285005
Phase
PHASE3
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
2 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • KP-001 — DRUG
    Oral repeated dose 100mg or lower
  • Placebo — DRUG
    Oral repeated dose

Study Details

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.

Key Dates

First listed
Dec 16, 2025
Start date
May 13, 2026
Status verified
May 2026
Primary completion
Feb 29, 2028
Completion
Feb 29, 2028

Study Design

Enrollment
150 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: KP-001
  • Placebo Comparator: placebo

Primary Outcome Measure

The ratio of volume of target lesions based on MRI [ Time Frame: pre-does and at 24weeks post-dose ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
MUSC Children's Health Primary CareCharlestonSouth Carolina29492
Kennedy Mcleod
[email protected]
843-792-4091
Lara Wine Lee (PRINCIPAL_INVESTIGATOR)

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By research site
MUSC Children's Health Primary Care· Charleston, SC

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