A randomized, Open-label Phase II Trial of Ponatinib (OPTIC study)

JRCT ID: jRCT2080223237

Registered date:08/06/2016

Basic Information

• Recruitment status: completed
• Health condition(s) or Problem(s) studied: chronic phase chronic myeloid leukemia
• Date of first enrollment: 18/03/2016
• Target sample size: 276
• Countries of recruitment: Japan,Asia except Japan,North America,South America,Europe,Oceania
• Study type: Interventional

Intervention(s)

investigational material(s)
Generic name etc :
INN of investigational material : ponatinib
Therapeutic category code : 429 Other antitumor agents
Dosage and Administration for Investigational material :

control material(s)
Generic name etc :
INN of investigational material : -
Therapeutic category code :
Dosage and Administration for Investigational material :

Outcome(s)

• Primary Outcome: efficacy
• Secondary Outcome: efficacy

Key inclusion & exclusion criteria

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

Include criteria

1. Have CP-CML (No previous diagnosis of AP or BP) and have received at least two prior TKI therapies and have demonstrated resistance to treatment OR Have documented history of presence of T315I mutation after receiving any number of prior TKI.
2. Age >= 18 years old.
3. Have an ECOG performance status of 0, 1, or 2.
4. Have adequate renal function as defined by the following criterion:
a. Serum creatinine <= 1.5 x ULN for institution
b. Estimated creatinine clearance >= 30 mL/min (Cockcroft-Gault formula)
5. Have adequate hepatic function as defined by the following criteria:
a. Total serum bilirubin <= 1.5 x ULN, unless due to Gilbert's syndrome
b. ALT <= 2.5 x ULN, or <= 5 x ULN if leukemic infiltration of the liver is present
c. AST <= 2.5 x ULN, or <= 5 x ULN if leukemic infiltration of the liver is present
6. >1% BCR-ABL1IS as shown by real-time polymerase chain reaction

Exclude criteria

1. Have used any approved TKIs or investigational agents within 2 weeks or 6 half-lives of the agent, whichever is longer, prior to receiving study drug.
2. Received interferon, cytarabine or immunotherapy within 14 days, or any other cytotoxic chemotherapy, radiotherapy, or investigational therapy within 28 days prior to receiving the first dose of ponatinib, or have not recovered (> grade 1 by NCI CTCAE, v4.0) from AEs (except alopecia), due to agents previously administered.
3. Have undergone autologous or allogeneic SCT < 60 days prior to receiving the first dose of ponatinib; have any evidence of ongoing GVHD or GVHD requiring immunosuppressive therapy. Are being considered for HSCT within 6-12 months of enrollment (note: ponatinib is not to be used as a bridge to HSCT in this trial).
4. Are taking medications with a known risk of Torsades de Pointes.
5. Have clinically significant, uncontrolled, or active cardiovascular disease, specifically including, but not restricted to:
a. Any history of MI, unstable angina, cerebrovascular accident, or TIA
b. Any history of peripheral vascular infarction, including visceral infarction
c. Any revascularization procedure, including the placement of a stent
d. Congestive heart failure (CHF) (New York Heart Association [NYHA] class III or IV) within 6 months prior to enrollment, or left ventricular ejection fraction (LVEF) less than lower limit of normal, per local institutional standards, within 6 months prior to enrollment
e. History of clinically significant (as determined by the treating physician) atrial arrhythmia or any history of ventricular arrhythmia
f. Venous thromboembolism, including deep venous thrombosis or pulmonary embolism, within 6 months prior to enrollment

Related Information

• Primary Sponsor: Takeda Development Center Americas, Inc. / CMIC Co., Ltd.
• Secondary ID(s): NCT02467270,JapicCTI-163292