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Compares treatment options for Venous Malformations
Official 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
ClinicalTrials.gov ID: NCT07285005
What this study is testing
What is KP-001?
KP-001 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for venous malformations.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older
You may be able to join if
- Patients aged 2 years or older at the time of consent or assent.
- Patients diagnosed with ISSVA classification of common VM, common (cystic) LM (including mixed type consisting mainly of either VM or LM), or...
- Patients who cannot be cured by resection, who are difficult to resect based on the assessment of the Investigator, or who are considered refractory...
- Patients with at least one target lesion at least 4 cm in the longest diameter.
- Patients with at least one MRI-volumetric target lesion at screening that is determined to be evaluable by the central imaging evaluator.
You likely can't join if
- Patients with the following diseases: Simple telangiectatic malformation, lymphangiomatosis, lymphangiectatic malformation associated with Gorham's...
- Patients with uncontrolled diabetes mellitus (HbA1c ≥ 7.0%) or diseases with abnormal glucose metabolism (glycogenic diseases, galactosemia, primary...
- Patients with ischemic heart disease, arrhythmia, or heart failure (NYHA III or IV).
- Patients with gastrointestinal disorders that affect drug absorption, as determined by the Investigator.
- Patients with concomitant or pre-existing serious drug hypersensitivity to PI3Kα inhibitors.
- Patients with allergy history of grade ≥ 3 and/or history of grade ≥ 3 allergic reactions to drug.
See the full eligibility criteria
Who can join
- Patients aged 2 years or older at the time of consent or assent.
- Patients diagnosed with ISSVA classification of common VM, common (cystic) LM (including mixed type consisting mainly of either VM or LM), or KTS/CLOVES syndrome.
- Patients who cannot be cured by resection, who are difficult to resect based on the assessment of the Investigator, or who are considered refractory to available treatment by the Investigator, or who have a...
- Patients with at least one target lesion at least 4 cm in the longest diameter.
- Patients with at least one MRI-volumetric target lesion at screening that is determined to be evaluable by the central imaging evaluator.
- 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...
- For patients ≥3 years old to \<8 years old: Wong-Baker FACES pain rating scale equal to or greater than 2 points at screening visit. Patients who are ≥3 years old to \<8 years old without qualifiable pain will be...
- For patients 2 years old: Symptomatic disease as judged in the opinion of the Investigator (ie, any visible lesion or lesion affecting activities of daily living), and patients will be recruited as far as they meet...
- Patients whose pain from vascular malformations has been stable for at least 30 days prior to screening and, if taking analgesic medication, does not require a change in the type of analgesic medication or its dosage...
- Patients who agree that they or their partner (if either of them is of childbearing potential) will use appropriate contraception (eg, condom and spermicide combination, low-dose pills or other appropriate contraceptive...
- Patients or their LAR who are able to give age-appropriate informed consent at the time of screening.
- Patients who are judged by the Investigator to be able to comply with the instructions of the Investigator and the study coordinator regarding the matters specified in the protocol, such as the use of study intervention...
What rules you out
- Patients with the following diseases: Simple telangiectatic malformation, lymphangiomatosis, lymphangiectatic malformation associated with Gorham's disease, lymphangiectasia, familial cutaneous mucocutaneous venous...
- Patients with uncontrolled diabetes mellitus (HbA1c ≥ 7.0%) or diseases with abnormal glucose metabolism (glycogenic diseases, galactosemia, primary lactose intolerance, etc).
- Patients with ischemic heart disease, arrhythmia, or heart failure (NYHA III or IV).
- Patients with gastrointestinal disorders that affect drug absorption, as determined by the Investigator.
- Patients with concomitant or pre-existing serious drug hypersensitivity to PI3Kα inhibitors.
- Patients with allergy history of grade ≥ 3 and/or history of grade ≥ 3 allergic reactions to drug.
- Patients with known hypersensitivity to quinine.
- Patients with concomitant or pre-existing alcohol or drug abuse.
- Patients with ANC of \<1.5×10\^9/L.
- Patients with acute or chronic kidney disease and/or dialysis dependence. Patients with screening eGFR \ 18 years of age will also be excluded.
- Patients who are judged by the Investigator to have hepatic impairment.
- Patients with total bilirubin ≥1.5×ULN for age (unless there is a history of Gilbert Syndrome), ALT ≥2×ULN for age, or AST ≥2×ULN for age will be excluded.
- Patients with target lesion infection that require treatment within 28 days prior to screening.
- Patients who have undergone invasive treatment, including sclerotherapy or laser therapy, for the target lesion within 84 days prior to screening.
- Patients who have used other PI3Kα inhibitors or sirolimus within 84 days prior to screening.
- Patients who have participated in other clinical studies within 90 days prior to the date of consent.
- Patients who have participated in a clinical study of KP-001 for any period and have received an investigational drug in the past year.
- Patients wearing orthodontic appliances, cochlear implants, etc, that may affect MRI, or patients in whom MRI is not feasible or, for example, patients who may have a contraindication to sedation and would require...
- Patients who are unable to take oral medications.
- Pregnant women, lactating female patients, female patients who may be pregnant, female patients who wish to become pregnant during the study period and up to 90 days after the last dose of study intervention, or male...
- Patients with any other illness or medical condition who are judged by the Investigator to be inappropriate as patients for this study.
- Patients who have received or plan to receive live vaccines during the study period.
The study team makes the final eligibility decision.
Where it's taking place
- Charleston, South Carolina, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Charleston, South Carolina, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.