Safety and efficacy of ponatinib in PTTM

JRCT ID: jRCTs031210348

Registered date:28/09/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Pulmonary tumor thrombtic microangiopathy
• Date of first enrollment: 10/03/2022
• Target sample size: 5
• Study type: Interventional
• Intervention(s): Patients will receive 45mg of ponatinib once daily for 2 weeks.

Outcome(s)

• Primary Outcome: The primary outcome will be composite endpoint of all cause death and severe vascular occlusive event. We will also evaluate the prevalence of grade 2 or higher adverse event (CTCAE v4) associated with ponatinib.
• Secondary Outcome: The secondary outcome will be the change from baseline in right heart catheterization parameters (mean pulmonary artery pressure, cardiac output, pulmonary vascular resistance), echocardiographic parameters (tricuspid regurgitant velocity, estimated pulmonary artery pressure, right ventricular size), WHO functional class, vital sign, or laboratory markers (BNP, VEGF, PDGF). We will also evaluate the proportion of successful weaning from ECMO and pharmacokinetics of ponatinib.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) Patient diagnosed with pulmonary tumor thrombotic microangiopathy (PTTM)
-Histological diagnosis of PTTM will be made by presence of the tumor cells within the lumen of pulmonary pre-capillary vessels, with fibrocellular intimal hyperplasia in lung biopsy.
-Clinical diagnosis will be made when patient meet either (A)+(B) or (A)+(C) of following criteria.
(A) primary pulmonary hypertension.
(B) presence of malignant tumor.
(C) perfusion defects along the pleural margins on lung perfusion scan.
2) Patient aged 18 years or more.
3) Patient or representative person who received detailed explanation and understands the study well is willing to participate in this study.
4) Hospitalized patient.

Exclude criteria

1) Patient who will be considered as inappropriate to participate the study by principal investigator
2) Patient who received tyrosine kinase inhibitors.
3) Patient with fatal or severe adverse event within 24 hours after introduction of extracorporeal membrane oxygenation (ECMO).

Related Information

• Primary Sponsor: Sato Kimi
• Source(s) of Monetary Support: University of Tsukuba Hospital