This protocol will allow ponatinib with refractory Chronic Myeloid Leukemia or Ph+ Acute Lymphoblastic Leukemia
The purpose of this study is to determine the safety and efficacy of ponatinib in patients with chronic myeloid leukemia (CML) in chronic phase (CP), accelerated phase (AP) or blast phase (BP) or with Ph positive (Ph+) acute lymphoblastic leukemia (ALL) who either are resistant or intolerant to either dasatinib or nilotinib, or have the T315I mutation.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
93
Ponatinib 30mg OD
Ponatinib 45mg OD
Anhui Provincial Hospital
Hefei, Anhui, China
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, China
Nanfang Hospital of Southern Medical University
MCyR(Major Cytogenetic Response) of CP-CML patients
To confirm the efficacy of Ponatinib in Chinese patients with CML who have failed Dasatinib or Nilotinib or with T315I mutation, or Ph+ ALL who have failed prior TKIs or with T315I mutation as evidenced by cytogenetic responses
Time frame: 12 months
MaHR(Major Hematologic Response) of AP-CML, BP-CML and Ph+ ALL patients by 6 months
To confirm the efficacy of Ponatinib in Chinese patients with CML who have failed Dasatinib or Nilotinib or with T315I mutation, or Ph+ ALL who have failed prior TKIs or with T315I mutation as evidenced by hematology responses
Time frame: 6 months
Duration of response
Assessment in the total patient population
Time frame: Up to 5 years
Progression-free survival (PFS)
Assessment in the total patient population
Time frame: Up to 5 years
Overall survival (OS)
Assessment in the total patient population
Time frame: Up to 5 years
Time to response (TTR)
Assessment in the total patient population
Time frame: Up to 5 years
Adverse events
Number of participants with adverse events as assessed by CTCAE v5.0
Time frame: Up to 5 years
EORTC QLQ-C30 (version 3)
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Guangzhou, Guangdong, China
Tongji Hospital Affiliated to Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, China
Xiangya Hospital Central South University
Changsha, Hunan, China
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
Shengjing Hospital of China Medical University
Shenyang, Liaoning, China
Qilu Hospital of Shandong University
Jinan, Shandong, China
Second hospital of Shanxi Medical University
Taiyuan, Shanxi, China
Shenzhen Second People's Hospital
Shenzhen, Shenzhen, China
...and 4 more locations
EORTC QLQ-C30 (version 3) score ranges from 1 to 4 or 1 to 7, A higher score represents a severer impressions or a best applies of patients.
Time frame: Up to 5 years
Maximum Plasma Concentration [Cmax]
Plasma concentration-time data for the population PK study
Time frame: Up to 3 months