Actively Recruiting
Phase 3 Study of KP-001 Compared to Placebo in Patients Aged 2 Years and Older with Venous Malformations, Lymphatic Malformations, or KTSCLOVES Syndrome
Led by Kaken Pharmaceutical · Updated on 2026-07-22
150
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a phase 3, double-blind, randomized, placebo-controlled study to evaluate the effectiveness and safety of KP-001 in patients aged 2 years and older with common venous malformations, common lymphatic malformations, or Klippel-TrenaunayCLOVES syndrome. This adaptive, multicenter trial will take place across sites in North America, Canada, Taiwan, and South Korea and includes oversight by an independent data monitoring committee that may adjust the study based on interim results. Participants will undergo a screening period up to 42 days before treatment begins. During the first 24 weeks, patients will receive either KP-001 oral daily doses 100 mg or lower based on body weight or placebo in a double-blind manner. After 24 weeks, all participants will enter an open-label extension phase where everyone receives KP-001 until 52 weeks. A follow-up visit is scheduled 30 days after the last dose, and patients who discontinue treatment are encouraged to continue study assessments. Throughout the study, vital signs and safety labs including blood counts, chemistry, coagulation tests, and urinalysis will be regularly monitored. Exploratory assessments will examine symptom changes using patient-reported scales such as the Numeric Rating Scale NRS for pain and Patient Global Impression scores. The primary measure is the change in volume of target lesions by MRI at 24 weeks. Secondary outcomes include symptom changes, lesion volume at other time points, clinical event monitoring, and adverse event tracking up to 52 weeks post-dose. Participants will be closely observed for safety and treatment effects during the entire study period.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 2 years or older at the time of consent or assent
- Diagnosed with ISSVA classification of common venous malformations, common (cystic) lymphatic malformations, or KTS/CLOVES syndrome
- Cannot be cured by resection, difficult to resect, refractory to available treatment, or have contraindication to available treatment
- Have at least one target lesion 4 cm or larger in longest diameter
- Have at least one MRI-volumetric target lesion evaluable by central imaging at screening
- Have symptomatic disease based on age-appropriate pain or symptom scales
- Pain from vascular malformations stable for at least 30 days prior to screening with no change in analgesic medication
- Agree to use appropriate contraception from consent until 90 days after last dose if of childbearing potential
- Able to give age-appropriate informed consent or have a legally authorized representative
- Able to comply with study instructions including use of study intervention and prohibited drugs
You will not qualify if you...
- Diagnosis of simple telangiectatic malformation, lymphangiomatosis, lymphangiectatic malformation tied to Gorham's disease, lymphangiectasia, familial cutaneous mucocutaneous venous malformation, blue rubber ball-like nevus syndrome, M-CM/MCAP, CLAPO syndrome, Proteus syndrome, Parkes Weber syndrome, Sturge-Weber syndrome, Mafucci syndrome, Osler's disease, Cowden's disease, or Adams-Oliver syndrome
- Uncontrolled diabetes mellitus (HbA1c ≥7.0%) or abnormal glucose metabolism diseases
- Ischemic heart disease, arrhythmia, or advanced heart failure (NYHA III or IV)
- Gastrointestinal disorders affecting drug absorption
- Serious hypersensitivity to PI3Kα inhibitors or quinine
- History of severe allergic reactions (grade ≥3)
- Alcohol or drug abuse
- Low absolute neutrophil count (<1.5×10^9/L)
- Acute or chronic kidney disease or dialysis dependence; low eGFR (<30 mL/min/1.73m²)
- Hepatic impairment or elevated liver enzymes outside allowed limits
- Infection in target lesion needing treatment within 28 days prior to screening
- Recent invasive treatment (sclerotherapy or laser) to target lesion within 84 days
- Recent use of PI3Kα inhibitors or sirolimus within 84 days
- Participation in other clinical studies within 90 days prior to consent
- Previous participation in KP-001 clinical study within past year
- Presence of MRI contraindications such as orthodontic appliances or implants
- Unable to take oral medications
- Pregnant or lactating women, or those wishing to become pregnant during the study period and 90 days after last dose
- Any other illness deemed inappropriate by the Investigator
- Planned or received live vaccines during the study period
Research Team
S
Sr. Clinical Trial Manager, Clinical Operations
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