A Phase 3 interventional study of KP-001 and Placebo in Venous Malformations, Lymphatic Malformations and Klippel-Trenaunay Syndrome, sponsored by Kaken Pharmaceutical. Recruiting at 5 sites in 3 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2026-09-21.
Sponsored by Kaken Pharmaceutical · Phase 3, Interventional, and Treatment
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:
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.
42 studies on the registry are indexed under Lymphatic Abnormalities; 17 are open to participants now.
This study's planned enrollment of 150 is above the median of 46 across 28 interventional studies indexed under Lymphatic Abnormalities.
Browse Lymphatic Abnormalities studies →Kaken Pharmaceutical is the lead sponsor of 10 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patients with symptomatic disease, defined as:
・ For patients ≥8 years old: Pain NRS of ≥1 and ≤8 score at the screening visit will be eligible only if their daily pain NRS recorded via ePRO from screening to Day 1 (Week 0) does not show a maximum absolute change of ≥6 points. If patients are taking analgesic medication, there must be no change in the type or dosage of analgesic during the screening period. If patients do not have qualifiable pain, then Fatigue or Bleeding/Oozing NRS of ≥1 and ≤8 score at the screening visit is required. Patients with ≥6 points absolute change in pain NRS are eligible if at least one of either Fatigue OR Bleeding/Oozing NRS is ≥1 and ≤8.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Live vaccines
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: KP-001 · Drug: Placebo
Drug: KP-001 · Drug: Placebo
Oral repeated dose 100mg or lower
Oral repeated dose
The ratio of volume of target lesions based on MRI
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
Time frame: pre-does and at 24weeks post-dose
Change from baseline in NRS of symptom
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) on disease specific patient-reported outcomes from Week 20 through Week 24
Time frame: Week 20 through Week 24
The ratio of volume of target lesions based on MRI
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 12 and 52 weeks
Time frame: Pre-does and at 12 and 52 weeks post-dose
The proportion of patients defined as a responder at 12, 24, and 52 weeks
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) using response rate based on change in target lesion volume (MRI) at 12, 24, and 52 weeks
Time frame: pre-does and at 12, 24 and 52 weeks post-dose
Change from baseline of Brief Pain Inventory short form
To evaluate 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) on patient-reported outcomes at 4, 12, 24, 36, and 52 weeks
Time frame: pre-does and at 4, 12, 24, 36, and 52 weeks post-dose
Change from baseline of PGI-S (Patient Global Impression of Severity and PGI-I (Patient Global Impression of Improvement)
To evaluate 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) on patient-reported outcomes at 4, 12, 24, 36, and 52 weeks
Time frame: pre-does and at 4, 12, 24, 36, and 52 weeks post-dose
Change from baseline of EQ-5D
To evaluate 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) on patient-reported outcomes at 4, 12, 24, 36, and 52 weeks
Time frame: pre-does and at 4, 12, 24, 36, and 52 weeks post-dose
Change from baseline of NRS of symptom
To evaluate 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) on patient-reported outcomes at 4, 12, 24, 36, and 52 weeks
Time frame: pre-does and at 4, 12, 24, 36, and 52 weeks post-dose
Change from baseline of Wong-Baker Faces of symptom
To evaluate 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) on patient-reported outcomes at 4, 12, 24, 36, and 52 weeks
Time frame: pre-does and at 4, 12, 24, 36, and 52 weeks post-dose
Change from baseline of PedsQL Fatigue Scale
To evaluate 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) on patient-reported outcomes at 4, 12, 24, 36, and 52 weeks
Time frame: pre-does and at 4, 12, 24, 36, and 52 weeks post-dose
Change from baseline in CGI-S (Clinician Global Impression of Severity) and CGI-I (Clinician Global Impression of Improvemen) at 12, 24, 36, and 52 weeks
To evaluate 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 improving clinician-reported outcomes at 12, 24, 36, and 52 weeks
Time frame: pre-does and at 12, 24, 36, and 52 weeks post-dose
The proportion of patients achieving both ≥20% reduction in target lesion volume AND ≥1 score reduction in NRS of symptom
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) using response rate based on change in target lesion volume (MRI) AND disease specific patient-reported outcomes at 12, 24, and 52 weeks
Time frame: pre-does and at 12, 24 and 52 weeks post-dose
Change from baseline in non-target lesions by MRI
To evaluate 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 changing non-target lesion at 12, 24, and 52 weeks
Time frame: pre-does and at 12, 24 and 52 weeks post-dose
Presence of new lesions based on MRI
To evaluate 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 suppressing presence of new lesions at 12, 24, and 52 weeks
Time frame: pre-does and at 12, 24 and 52 weeks post-dose
Time to relapse
To evaluate 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) at 12, 24, and 52 weeks regarding time to relapse
Time frame: pre-does and at 12, 24 and 52 weeks post-dose
Time to disease progression
To evaluate 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) at 12, 24, and 52 weeks regarding time to disease progression
Time frame: pre-does and at 12, 24 and 52 weeks post-dose
Additional treatment for pain related to vascular malformation
To evaluate 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) for up to 24 weeks regarding concomitant medication for vascular malformation
Time frame: Up to 24 weeks post-dose
Plasma KP-001 concentration data obtained from sparse sampling for population PK analysis
To evaluate the pharmacokinetics 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) following multiple oral doses
Time frame: 8weeks, 16weeks, 20weeks and 32 weeks post-dose
Adverse events
To determine the safety 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) for up to 52 weeks
Time frame: up to 52 weeks post-dose
Clinical events relevant to the vascular malformations (bleeding, infection, thromboembolism, and others)
To determine the safety 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) for up to 52 weeks
Time frame: up to 52 weeks post-dose
Change in D-dimer From Baseline to Week 24
Serum D-dimer will be measured as a coagulation biomarker of disease activity in patients with vascular malformations. The change from baseline to Week 24 will be evaluated to explore its clinical relevance.
Time frame: up to 52 weeks post-dose
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Studies of the drug tested here: KP-001
Kaken Pharmaceutical