NIPH Clinical Trials Search

JRCT ID: jRCTs031210348

Registered date:28/09/2021

Safety and efficacy of ponatinib in PTTM

Basic Information

Recruitment status Recruiting
Health condition(s) or Problem(s) studiedPulmonary tumor thrombtic microangiopathy
Date of first enrollment10/03/2022
Target sample size5
Countries of recruitment
Study typeInterventional
Intervention(s)Patients will receive 45mg of ponatinib once daily for 2 weeks.


Primary OutcomeThe 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 OutcomeThe 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 maximumNot applicable
Include criteria1) 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 criteria1) 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


Public contact
Name Kimi Sato
Address 2-1-1 Amakubo, Tsukuba, Ibaraki Ibaraki Japan 3058576
Telephone +81-29-853-3143
Affiliation University of Tsukuba Hospital
Scientific contact
Name Naoto Kawamatsu
Address 2-1-1 Amakubo, Tsukuba, Ibaraki Ibaraki Japan 3058576
Telephone +81-29-853-3143
Affiliation University of Tsukukba Hospital