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

1. Patients with the following diseases: Simple telangiectatic malformation, lymphangiomatosis, lymphangiectatic malformation associated with 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.

2. Patients with uncontrolled diabetes mellitus (HbA1c ≥ 7.0%) or diseases with abnormal glucose metabolism (glycogenic diseases, galactosemia, primary lactose intolerance, etc).

3. Patients with ischemic heart disease, arrhythmia, or heart failure (NYHA III or IV).

4. Patients with gastrointestinal disorders that affect drug absorption, as determined by the Investigator.

5. Patients with concomitant or pre-existing serious drug hypersensitivity to PI3Kα inhibitors.

6. Patients with allergy history of grade ≥ 3 and/or history of grade ≥ 3 allergic reactions to drug.

7. Patients with known hypersensitivity to quinine.

8. Patients with concomitant or pre-existing alcohol or drug abuse.

9. Patients with ANC of \<1.5×10\^9/L.

10. Patients with acute or chronic kidney disease and/or dialysis dependence. Patients with screening eGFR \<30 mL/min/1.73m\^2 using the beside Schwartz equation for patients aged \<18 years of age or CKD-EPI formula for \>18 years of age will also be excluded.

11. Patients who are judged by the Investigator to have hepatic impairment.

12. 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.

13. Patients with target lesion infection that require treatment within 28 days prior to screening.

14. Patients who have undergone invasive treatment, including sclerotherapy or laser therapy, for the target lesion within 84 days prior to screening.

15. Patients who have used other PI3Kα inhibitors or sirolimus within 84 days prior to screening.

16. Patients who have participated in other clinical studies within 90 days prior to the date of consent.

17. Patients who have participated in a clinical study of KP-001 for any period and have received an investigational drug in the past year.

18. 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 sedation in order to have MRI completed.

19. Patients who are unable to take oral medications.

20. 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 who have partners who wish to become pregnant.

21. Patients with any other illness or medical condition who are judged by the Investigator to be inappropriate as patients for this study.

22. Patients who have received or plan to receive live vaccines during the study period.

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

What this study is about

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

What this study is about