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Chronic Myeloid LeukemiaPhase 2NCT04233346

The Study for CML Who Failed Prior TKIs or With T315I Mutation or Ph+ ALL Who Failed Prior TKIs or With T315I Mutation

A Phase 2 study testing Ponatinib 30mg OD and Ponatinib 45mg OD in people with Chronic Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Otsuka Beijing Research Institute at 14 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This protocol will allow ponatinib with refractory Chronic Myeloid Leukemia or Ph+ Acute Lymphoblastic Leukemia Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Ponatinib 30mg OD and Ponatinib 45mg OD
Could I receive a placebo?
No, everyone receives an active treatment
How many people
93 participants
Where
14 sitesChina
Study dates
9 Jul 2020to 8 Sep 2026
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • For CP-CML patients：
  • Patients with CP-CML
  • Patients must either meet criterion 2 or 3:
  • Be previously treated with and resistant or intolerant to either Dasatinib or Nilotinib:
  • Develop the T315I mutation after any TKI therapy;
  • Must be ≥18 years old.
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  • Provide written informed consent.
  • Eastern Cooperative Oncology Group performance status ≤ 2.
  • Minimum life expectancy of 3 months or more.
  • Adequate renal function
  • Adequate hepatic function
  • Normal pancreatic status
  • Normal QTc interval on screening electrocardiogram (ECG) evaluation under resting state, defined as QTc of ≤ 450 ms in males or ≤ 470 ms in females.
  • For AP/BP-CML and ALL patients：
  • Patients with AP-CML and BP-CML or Ph+ ALL
  • Other inclusions are the same with No.2-No.11 of CP-CML patients

Who cannot join

  • For CP-CML patients：
  • Received TKI therapy within 7 days prior to receiving the first dose of Ponatinib, or have not recovered (> grade 2 by NCI CTCAE v5.0) from AEs (except alopecia) due to agents previously administered.
  • Received other therapies (excluding hydroxyurea) as follows:
  • 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.
  • Underwent autologous or allogeneic stem cell transplant < 60 days prior to receiving the first dose of Ponatinib;
  • Take medications that are known to be associated with Torsades de Pointes or QT interval prolongation.
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  • Require concurrent treatment with immunosuppressive agents, other than corticosteroids prescribed for a short course of therapy.
  • Have previously been treated with Ponatinib or its analogues (including drug substance).
  • Patients with CP-CML are excluded if they are in CCyR.
  • Have active central nervous system (CNS) disease, as evidenced by cytology or pathology.
  • Have significant, uncontrolled, or active heart/brain/peripheral vascular diseases
  • Have a significant bleeding disorder unrelated to CML
  • Have a history of pancreatitis or alcohol abuse
  • Severely increased hypertriglyceridemia (triglycerides ≥ 5.6 mmol/L).
  • Have malabsorption syndrome or other gastrointestinal illness that could affect absorption of orally administered Ponatinib.
  • Have been diagnosed with another primary malignancy within the past 3 years (except for non-melanoma skin cancer, cervical cancer in situ, or controlled prostate cancer, which are allowed within 3 years).
  • Are pregnant or lactating.
  • Underwent major surgery (with the exception of minor surgical procedures, such as catheter placement or BM biopsy) within 14 days prior to first dose of Ponatinib.
  • Infectious diseases test showed anti-HIV (+) or anti-HCV (+) or HbsAg (+) or TP (+).
  • Suffer from any condition or illness that, in the opinion of the investigator, would compromise patient safety or interfere with the evaluation of the safety of the study drug.
  • Have hypertension (diastolic blood pressure ≥ 90 mmHg and/or systolic blood pressure ≥ 140 mm Hg).
  • Taken herbal preparations or related over-the-counter preparations containing Chinese herbal ingredients within 2 weeks prior to the first dose of Ponatinib.
  • Any subject who is not suitable for the study in the opinion of the investigator.
  • For AP/BP-CML and ALL patients：
  • Received TKI therapy within 7 days prior to receiving the first dose of Ponatinib, or have not recovered (> grade 2 by NCI CTCAE v.5.0) from AEs (except alopecia) due to agents previously administered.
  • Received other therapies (excluding hydroxyurea) as follows:
  • For AP-CML patients, 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.
  • For BP-CML patients, received chemotherapy within 7 days prior to the first dose of Ponatinib. Otherwise, 2a applies.
  • For Ph+ ALL patients, received corticosteroids within 24 hours before the first dose of Ponatinib; otherwise, 2a applies.
  • Underwent autologous or allogeneic stem cell transplant < 60 days prior to receiving the first dose of Ponatinib.
  • Take medications that are known to be associated with Torsades de Pointes or QT interval prolongation.
  • Require concurrent treatment with immunosuppressive agents, other than corticosteroids prescribed for a short course of therapy.
  • Have previously been treated with Ponatinib or its analogues (including drug substance).
  • Patients with AP-CML, BP-CML, or Ph+ ALL are excluded if they are in MaHR.
  • Patients with AP-CML, BP-CML, or Ph+ ALL are excluded if a baseline BM aspirate adequate for cell count and differential report is not available.
  • Have active central nervous system (CNS) disease as evidenced by cytology or pathology for AP-CML, BP-CML, or Ph+ ALL.
  • Have significant, uncontrolled, or active cardiovascular disease.
  • Have a significant bleeding disorder unrelated to CML or Ph+ ALL.
  • Other exclusions are the same with No.11-No.21 of CP-CML.

Trial locations and contacts

14 sites in 1 country.

14 sites in 1 country

China14 sites

  • 1st affiliated hospital, Peking University

    Beijing

  • Xiangya Hospital Central South University

    Changsha, Hunan

  • West China Hospital, Sichuan University

    Chengdu, Sichuan

  • Nanfang Hospital of Southern Medical University

    Guangzhou, Guangdong

  • The First Affiliated Hospital of Medical School of Zhejiang University

    Hangzhou, Zhejiang

  • Anhui Provincial Hospital

    Hefei, Anhui

  • The First Affiliated Hospital of Anhui Medical University

    Hefei, Anhui

  • Qilu Hospital of Shandong University

    Jinan, Shandong

  • Shengjing Hospital of China Medical University

    Shenyang, Liaoning

  • Shenzhen Second People's Hospital

    Shenzhen, Shenzhen

  • The First Affiliated Hospital of Soochow University

    Suzhou, Jiangsu

  • Second hospital of Shanxi Medical University

    Taiyuan, Shanxi

  • Hematology Hospital, Chinese Academy of Medical Sciences

    Tianjin

  • Tongji Hospital Affiliated to Tongji Medical College, Huazhong University of Science and Technology

    Wuhan, Hubei

Other trials in China

About this study

Official title: A Phase II Multi-center, Randomized, Open-label Study of Ponatinib in Chinese Patients With Chronic Myeloid Leukemia Who Have Failed Prior TKIs or With T315I Mutation, or Ph+ALL Who Have Failed Prior TKIs or With T315I Mutation

This protocol will allow ponatinib with refractory Chronic Myeloid Leukemia or Ph+ Acute Lymphoblastic Leukemia

Read the detailed description

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.

Being tested
Ponatinib 30mg OD, Ponatinib 45mg OD
Keywords
Acute Lymphoblastic Leukemia, Chronic Myeloid Leukemia, ponatinib
Medical subject headings
Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Precursor Cell Lymphoblastic Leukemia-Lymphoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ponatinib 30mg ODDrug

    Ponatinib 30mg OD

  • Ponatinib 45mg ODDrug

    Ponatinib 45mg OD

Study groups in NCT04233346
Study groupRoleWhat they receive
Ponatinib 30mgExperimental50% CML patients will be treated with 30mg/day ponatinib，the treatment will up to 60 months
Receives: Drug: Ponatinib 30mg OD
Ponatinib 45mgExperimental50% CP-CML patients will be randomized 45 mg dose group . Other patients (with AP-CML, BP-CML, Ph+ ALL) will only be assigned to 45 mg dose group(30 patients). The treatment will up to 60 months.
Receives: Drug: Ponatinib 45mg OD

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: 12 months

  2. 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

    Measured: 6 months

Secondary outcomes (7)

  1. Duration of response

    Assessment in the total patient population

    Measured: Up to 5 years

  2. Progression-free survival (PFS)

    Assessment in the total patient population

    Measured: Up to 5 years

  3. Overall survival (OS)

    Assessment in the total patient population

    Measured: Up to 5 years

  4. Time to response (TTR)

    Assessment in the total patient population

    Measured: Up to 5 years

  5. Adverse events

    Number of participants with adverse events as assessed by CTCAE v5.0

    Measured: Up to 5 years

  6. EORTC QLQ-C30 (version 3)

    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.

    Measured: Up to 5 years

  7. Maximum Plasma Concentration [Cmax]

    Plasma concentration-time data for the population PK study

    Measured: Up to 3 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jan 2020
  2. Study start9 Jul 2020
  3. Primary completion22 Jul 2022
  4. Study completion8 Sep 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
93 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04233346
Sponsor's protocol ID
297-403-00003
Status last verified
Oct 2026
First submitted
31 Dec 2019
Last updated
16 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ponatinib 30mg OD and Ponatinib 45mg OD in people with Chronic Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Otsuka Beijing Research Institute at 14 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 39 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

MCyR(Major Cytogenetic Response) of CP-CML patients (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04233346.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.

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