A Long-term Study of KP-001 in patients with vascular malformation including venous malformation, lymphatic malformation, and Klippel-Trenaunay Syndrome (Phase III)

JRCT ID: jRCT2041230065

Registered date:03/08/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Vascular malformation including VM, LM, and KTS
• Date of first enrollment: 03/08/2023
• Target sample size: 50
• Study type: Interventional
• Intervention(s): KP-001 is given orally once after breakfast

Outcome(s)

Primary Outcome

Primary safety endpoints
(1) Adverse event
(2) Vital sign
(3) Laboratory test (hematology, blood biochemistry, and urinalysis)

Key inclusion & exclusion criteria

• Age minimum: >= 1month old
• Age maximum: Not applicable
• Gender: Both

Include criteria

(Cohort1)
1. Patients who complete the 52-week treatment period in the Phase III confirmatory study and are diagnosed as requiring continuation of investigational drug beyond 52 weeks.
(Cohort2)
1. Patient is at least 1 month of age at time of consent
2. Diagnosed as one of the following - ISSVA classification of Common VM, Common (cystic) LM (including combined type mainly consisting of VM or LM)
- Klippel-Trenaunay Syndrome
- Megalencephaly-capillary malformation-polymicrogyria
- Lymphangiomatosis
- Lymphangioleiomyomatosis in Gorham-Stout disease
- Lymphangiectasia
- Familial VM cutaneo-mucosal
- CLOVES syndrome
- CLAPO syndrome
- Proteus syndrome
- Diseases other than those listed above that are associated with PIK3CA-related overgrowth spectrum
- Blue rubber bleb nevus 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
(Cohort3)
1. Patients who complete the 24-week treatment period in the Phase II study and are diagnosed as requiring the administration of investigational drug and wish to resume the administration of it

Exclude criteria

(Cohort1)
1. Diagnosed as having diabetes mellitus (type I or II) or a disease with abnormal glucose metabolism (glycogen storage disease, hypergalactosemia, primary lactose intolerance, etc.) and poor control of the disease
2. Diagnosed as having hepatic or renal impairment
3. Patients with ischemic heart disease, arrhythmia, or heart failure (NYHA III or IV degree) diagnosed as inadequately controlled
4. Patients who wear orthodontic appliances, cochlear implants, etc., which may affect MRI imaging.
(Cohort2)
1. Diagnosed as having diabetes mellitus (type I or II) or a disease with abnormal glucose metabolism (glycogen storage disease, hypergalactosemia, primary lactose intolerance, etc.) and poor control of the disease
2. Diagnosed as having hepatic or renal impairment
3. Patients with ischemic heart disease, arrhythmia, or heart failure (NYHA III or IV degree) diagnosed as inadequately controlled
4. Patients who are unable to take drug orally
5. Patients who wear orthodontic appliances, cochlear implants, etc., which may affect MRI imaging. (Only for subjects whose target lesions are assessed by MRI imaging)
6. Patients with target lesion infection requiring treatment within 28 days prior to screening
7. Patients who have undergone invasive treatment, including sclerotherapy or laser therapy, for the target lesion within 84 days prior to screening
8. Patients who have used other PI3Ka inhibitors or Sirolimus within 84 days prior to screening
(Cohort3)
1. Diagnosed as having diabetes mellitus (type I or II) or a disease with abnormal glucose metabolism (glycogen storage disease, hypergalactosemia, primary lactose intolerance, etc.) and poor control of the disease
2. Diagnosed as having hepatic or renal impairment
3. Patients with ischemic heart disease, arrhythmia, or heart failure (NYHA III or IV degree) diagnosed as inadequately controlled
4. Patients who are unable to take drug orally
5. Patients who have undergone invasive treatment, including sclerotherapy or laser therapy, for the target lesion within 84 days prior to screening
6. Patients who have used other PI3Ka inhibitors or Sirolimus within 84 days prior to screening

Related Information

• Primary Sponsor: Hideki Kawabata