Phase III confirmatory study of in patients with venous malformation, lymphatic malformation and Klippel-Trenaunay Syndrome

JRCT ID: jRCT2041230064

Registered date:03/08/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Venous malformation, Lymphatic malformation, Klippel-Trenaunay Syndrome
• Date of first enrollment: 03/08/2023
• Target sample size: 32
• Study type: Interventional
• Intervention(s): KP-001 is given orally once after breakfast

Outcome(s)

• Primary Outcome: Target lesion response based on MRI at the last evaluation after 24 weeks of treatment

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Patient is at least 2 years of age at time of consent
2. Diagnosed as ISSVA classification of Common VM, Common (cystic) LM (including combined type mainly consisting of VM or LM), or Klippel-Trenaunay Syndrome
3. Diagnosed as pain, bleeding, disfigurement, inflammation such as cellulitis, etc., and judged to be symptomatic
4. Diagnosed as refractory because resection is not curative, resection is difficult, or for other reasons
5. Patients with at least one target lesion that can be volumetrically measured by MRI at screening

Exclude criteria

1. Patients with the following diseases: simple capillary malformation, lymphangiomatosis, lymphangioleiomyomatosis in Gorham-Stout disease, lymphangiectasia, familial VM cutaneo-mucosal, blue rubber bleb nevus syndrome, M-CM/MCAP, CLOVE(S) syndrome, CLAPO syndrome, proteus syndrome, Parkes Weber syndrome, Sturge-Weber syndrome, maffucci syndrome, Rendu-Osler-Weber syndrome, Cowden's disease, Adams-Oliver syndrome
2. Patients with diabetes mellitus (type I or II) or diseases with abnormal glucose metabolism (glycogen storage disease, hypergalactosemia, primary lactose intolerance, etc.)
3. Diagnosed as having hepatic or renal impairment
4. Patients with ischemic heart disease, arrhythmia, or heart failure (NYHA III or IV degree) diagnosed as inadequately controlled
5. Diagnosed as having a gastrointestinal disorder that affects drug absorption
6. Patients who are unable to take the drug orally
7. Patients with orthodontic appliances, cochlear implants, etc. which may affect MRI imaging
8. Patients with target lesion infection requiring treatment within 28 days prior to the date of consent
9. Patients who have undergone invasive treatment, including sclerotherapy or laser therapy, for the target lesion within 84 days prior to the date of consent
10. Patients who have used other PI3Ka inhibitors or Sirolimus within 84 days prior to the date of consent

Related Information

• Primary Sponsor: Hideki Kawabata