Phase 2 clinical trial evaluating the efficacy and safety of ART-001 in patients with slow-flow vascular malformations

JRCT ID: jRCT2071210027

Registered date:25/05/2021

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Slow flow vascular malformations
• Date of first enrollment: 12/08/2021
• Target sample size: 34
• Study type: Interventional
• Intervention(s): Perticipants will be orally taking 50 mg or 100 mg of ART-001 once daily after breakfast

Outcome(s)

• Primary Outcome: Proportion of participants with response defined by MRI volumetric change of target lesion at Week 24

Secondary Outcome

Efficacy
Proportion of participants with response defined by MRI volumetric change of target lesion at Week 12
Change from baseline to week 12 and 24 in MRI volume of target lesion
Change from baseline to week 4, 12 and 24 in pain score (VAS)
Change from baseline to week 12 and 24 in QOL score
Change from baseline to week 12 and 24 in performance status

Safety
Frequency and severity of adverse events

Pharmacokinetics
Plasma ART-001 concentrations

Key inclusion & exclusion criteria

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

Include criteria

1) All sexes, 2 years old and older
2) Patients with a diagonisis of venous malformation, lymphatic malformation (lymphangioma) or Klippel-Trenone syndrome.
3) Patients must have symptoms caused by slow-flow vascular malformation (i.e. pain, inflammation such as repeated cellulitis, bleeding, disfigurement, etc.)
4) Patients who have a intractable slow-flow vascular malformation (i.e. poor response or recurrence for standard treatment, multiple lesions, inoperable, thrombotic, etc.)
5) Patients with at least one lesions whose volume is measurableby MRI
6) Written consent to participate in this clinical trial has been given by the patient, or by the parent or a legal guardian (for pediatric patients).

Exclude criteria

1) Patients with diabetes (type I or type II) or diseases that cause abnormal glucose metabolism
2) Patients with renal dysfunction
3) Patients with liver dysfunction
4) Patients with poorly controlled ischemic heart disease, arrhythmia, or heart failure (NYHA III or IV degree)
5) Patients taking drugs that have CYP3A4 inhibitory or inducing effects
6) Patients with gastrointestinal disorders that affect drug absorption
7) Patients who cannot take the drug orally
8) Patients with orthodontic appliances, cochlear implants, etc. that may affect MRI image evaluation
9) Patients with inflammatory infections requiring treatment for target lesions within 4 weeks at time of informed consent
10) Patients who received invasive treatment including sclerotherapy or laser therapy for target lesions within 12 weeks at time of informed consent
11) Patients who have used other PI3Kalpha inhibitors or sirolimus within 12 weeks at time of informed consent.
12) Patients who participated in other clinical trials (excluding observational studies) within 12 weeks at time of informed consent
13) Patients who disagree with contraception
14) Pregnant or lactating female patients
15) Patients who are positive for immunological tests (HBs antigen, HCV antibody, HIV antibody, syphilis serum test) by screening test
16) Patients who are judged by the investigator to be not eligible for the clinical trial

Related Information

• Primary Sponsor: Nagabukuro Hiroshi