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

Phase 2

• Conditions: Slow flow vascular malformations

• Registration Number: JPRN-jRCT2071210027
• Lead Sponsor: agabukuro Hiroshi

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-05-25
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 34

Inclusion 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).

Exclusion 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

Study & Design

• Study Type: Interventional
• Study Design: Not specified