Chronic Myeloid LeukemiaNCT05440747
Treatment Free Remission (TFR) in CML Patients （CML-CP）Study
An observational study of Chronic Myeloid Leukemia and Treatment-free Remission, run by Guangdong Provincial People's Hospital.
Trial summary
Improving the quality of life and achieving Treatment-Free Remission(TFR) is a long-term goal of treatment in CML-CP patients, and deep molecular response (DMR) is necessary to achieve TFR. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Chronic Myeloid Leukemia, Treatment-free Remission
- What is being tested?
- TFR(Treatment-Free Remission)
- How many people
- 203 participants
- Where
- No sites listed yet
- Study dates
- 31 Jul 2022to 31 Oct 2031
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The participants should older than 14 years old, and Newly diagnosed with CML-CP or had been treated with TKI (Imatinib, Nilotinib, Dasatinib and Flumatinib) are not achieved optimal (BCR-ABLIS>10% in 3 months, BCR-ABLIS>1% in 6 months, BCR-ABLIS>0.1% in 12 months or BCR-ABLIS>0.01% in 24 months) or intolerance to these TKIs.
Who may be able to join
- 1) Aged >=14 years old male and female;
- 2) Patients with Ph+ CML-CP should meet any of the following conditions;
- Newly diagnosed Patients with CML-CP;
- The CML-CP patients who treated with TKI (Imatinib, Nilotinib, Dasatinib and Flumatinib) are not achieved optimal （BCR-ABLIS>10% in 3 months, BCR-ABLIS>1% in 6 months, BCR-ABLIS>0.1% in 12 months or BCR-ABLIS>0.01% in 24 months ）or intolerance to these TKIs; The definition of the confirmed diagnosis: Bone marrow cytogenetics Ph chromosome t(9;22) positive and/or BCR-ABL fusion gene positive by FISH, and/or BCR-ABL fusion gene positive(>10%) by Q-PCR ;
- 3) Never received stem cell transplantation before enrollment;
- 4) Female patients with fertility have a negative pregnancy test (within 7 days before enrollment).
- All Patients with TFR requeirement should provid written informed consent before enrollment.
Who cannot join
- T315I mutation is known;
- Received stem cell transplantation before enrollment;
- With other malignant tumors and need active intervention;
- Those who are unable to follow the protocol steps or follow up on time;
- Eastern Cooperative Oncology Group physical performance score (ECOG PS) >=3;
- Other situations deemed unsuitable by the researcher.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Multicenter, Observational Register Study of Treatment Free Remission in Patients With Chronic Myeloid Leukemia (CML-CP)
Improving the quality of life and achieving Treatment-Free Remission(TFR) is a long-term goal of treatment in CML-CP patients, and deep molecular response (DMR) is necessary to achieve TFR. Cording to the historical literature, it is reported that patients with CML-CP take MMR as the therapeutic target, and the acquisition rate of DMR under long-term TKI treatment is 50%. The 2-year success rate of TFR patients was 50%. Therefore, maybe only 25% of patients with CML can successfully stop the drug for a long time. It cannot meet the withdrawal needs of patients with long-term drug survival.
This study is to design a real-world observational registration study for optimal effect. On the premise of taking DMR as the target decision, through initial treatment intervention, improve the DMR rate, which will promote clinical practice, so as to improve the 2-year TFR rate of cml-cp patients. This study is a multicenter, observational, prospective registry to identify the optimal treatment for achieving TFR in CML patients. In this study, the investigators will assess the deep molecular response after 12 months of treatment and the 2-year treatment-free remission rate (TFR 2y) after drug discontinuation.
Eligible participants with CML-CP can be enrolled. The observation period of all participants is at least 60 months, of which the first 36 months is the shortest treatment period, and the last 24 months is the TFR observation period after TKIs (Imatinib/Flumatinib/Nilotinb/ Dasatinib) withdrawal. During the treatment phase, participants can receive TKIs ± IFN (or other treatments) as first-line/second-line treatment, and the treatment plan will be adjusted according to the molecular response. Patients should accept TKI treatment for at least 3 years or more, and MR4/MR4.5 should achieve at least 2 years before discontinuation.
- Conditions
- Chronic Myeloid Leukemia, Treatment-free Remission
- Being tested
- TFR(Treatment-Free Remission)
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TFR(Treatment-Free Remission)Other
Withdrawal TKIs(Tyrosine kinase inhibitors: Imatinib or Flumatinib or Nilotinb or Dasatinib)
| Study group | Role | What they receive |
|---|---|---|
| Newly diagnosed CML-CP patients | Treat with TKI (Imatinib or Flumatinib or Nilotinb or Dasatinib). Receives: Other: TFR(Treatment-Free Remission) | |
| Patients with suboptimal response | 1. Treat with original TKI
2. Treat with original TKI combined with interferon/thymosin;
3. Replace other TKI ;
4. Replace other TKI and combined with interferon/thymosin. Receives: Other: TFR(Treatment-Free Remission) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Deep molecular response(DMR) rate of 12 months
DMR is defined by IS BCR-ABL/ABL achieve MR4.0(≤0.01%)
Measured: The 12th month
Treatment-Free Remission(TFR) rate of 2 years
The proportion of drug withdrawal
Measured: The second year
Secondary outcomes (8)
TFR rate
TFR rate of 6/12months，Median duration of TFR
Measured: The 6months、12months
DMR rate
DMR rate of 24/36/48/60 months, Median duration of DMR, DMR is defined by IS BCR-ABL/ABL achieve MR4.0
Measured: The 24/36/48/60 months
DMR persistence rate in 2 years
DMR is defined by IS BCR-ABL/ABL achieve MR4.0
Measured: 2 years
Disease progression rate , time to progression
Time and proportion from diagnosis to disease progression to AP or BC
Measured: The whole study, up to 7 years
Loss of Major Molecular Remission(MMR-loss IS BCR-ABL≥0.1%）
Time and proportion from discontinuation to loss of MMR in TFR phase up to 2 years.
Measured: TFR phase in the study, up to 2 years
Re-Major Molecular Remission
Time and proportion from restarting therapy after MMR-lose to obtain MMR through TFR phase.
Measured: The TFR phase, up to 2 years
Treatment-related safety
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
Measured: The whole study , up to 7 years
Patient Report Outcome（PRO）
EORTCQLQ-CML-24 survey every 3 months in the whole enrollment period
Measured: Every 3 months in the whole enrollment period, up to 7 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2022
- Study start31 Jul 2022 (estimated)
- Primary completion31 Oct 2024 (estimated)
- Study completion31 Oct 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 203 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangdong Provincial People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, WENG Jian yu, Guangdong Provincial People's Hospital
- Investigators
- Jianyu Jianyu, PhD, Study chair, Sponsor GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05440747
- Sponsor's protocol ID
- GDPH-CML-TFR-2020-12
- Status last verified
- Jun 2022
- First submitted
- 2 Apr 2022
- Last updated
- 1 Jul 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: After the study was complete, IPD will be uploaded as an supplementary data of the research paper. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Observational Register Study of Treatment Free Remission in Patients With Chronic Myeloid Leukemia (CML-CP)
- ClinicalTrials.gov ID
- NCT05440747
- Acronym / sponsor ID
- GDPH-CML-TFR-2020-12
- Conditions
- Chronic Myeloid Leukemia, Treatment-free Remission
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 203 participants (estimated)
- Lead sponsor
- Guangdong Provincial People's HospitalUniversity, hospital or other
- Study start
- 31 Jul 2022 (estimated)
- Primary completion
- 31 Oct 2024 (estimated)
- Study completion
- 31 Oct 2031 (estimated)
- First posted
- 1 Jul 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Improving the quality of life and achieving Treatment-Free Remission(TFR) is a long-term goal of treatment in CML-CP patients, and deep molecular response (DMR) is necessary to achieve TFR. Cording to the historical literature, it is reported that patients with CML-CP take MMR as the therapeutic target, and the acquisition rate of DMR under long-term TKI treatment is 50%. The 2-year success rate of TFR patients was 50%. Therefore, maybe only 25% of patients with CML can successfully stop the drug for a long time. It cannot meet the withdrawal needs of patients with long-term drug survival.
This study is to design a real-world observational registration study for optimal effect. On the premise of taking DMR as the target decision, through initial treatment intervention, improve the DMR rate, which will promote clinical practice, so as to improve the 2-year TFR rate of cml-cp patients. This study is a multicenter, observational, prospective registry to identify the optimal treatment for achieving TFR in CML patients. In this study, the investigators will assess the deep molecular response after 12 months of treatment and the 2-year treatment-free remission rate (TFR 2y) after drug discontinuation.
Eligible participants with CML-CP can be enrolled. The observation period of all participants is at least 60 months, of which the first 36 months is the shortest treatment period, and the last 24 months is the TFR observation period after TKIs (Imatinib/Flumatinib/Nilotinb/ Dasatinib) withdrawal. During the treatment phase, participants can receive TKIs ± IFN (or other treatments) as first-line/second-line treatment, and the treatment plan will be adjusted according to the molecular response. Patients should accept TKI treatment for at least 3 years or more, and MR4/MR4.5 should achieve at least 2 years before discontinuation.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Newly diagnosed CML-CP patients | – | Other: TFR(Treatment-Free Remission)Treat with TKI (Imatinib or Flumatinib or Nilotinb or Dasatinib). |
| Patients with suboptimal response | – | Other: TFR(Treatment-Free Remission)1. Treat with original TKI 2. Treat with original TKI combined with interferon/thymosin; 3. Replace other TKI ; 4. Replace other TKI and combined with interferon/thymosin. |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The participants should older than 14 years old, and Newly diagnosed with CML-CP or had been treated with TKI (Imatinib, Nilotinib, Dasatinib and Flumatinib) are not achieved optimal (BCR-ABLIS>10% in 3 months, BCR-ABLIS>1% in 6 months, BCR-ABLIS>0.1% in 12 months or BCR-ABLIS>0.01% in 24 months) or intolerance to these TKIs.
Key inclusion criteria (7 listed)
- 1) Aged >=14 years old male and female;
- 2) Patients with Ph+ CML-CP should meet any of the following conditions;
- 3) Never received stem cell transplantation before enrollment;
- 4) Female patients with fertility have a negative pregnancy test (within 7 days before enrollment).
- All Patients with TFR requeirement should provid written informed consent before enrollment.
Key exclusion criteria (6 listed)
- T315I mutation is known;
- Received stem cell transplantation before enrollment;
- With other malignant tumors and need active intervention;
- Those who are unable to follow the protocol steps or follow up on time;
- Eastern Cooperative Oncology Group physical performance score (ECOG PS) >=3;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TFR(Treatment-Free Remission)Withdrawal TKIs(Tyrosine kinase inhibitors: Imatinib or Flumatinib or Nilotinb or Dasatinib) | Other | Newly diagnosed CML-CP patients, Patients with suboptimal response |
Primary outcome
Deep molecular response(DMR) rate of 12 months
Time frame: The 12th month
DMR is defined by IS BCR-ABL/ABL achieve MR4.0(≤0.01%)
Treatment-Free Remission(TFR) rate of 2 years
Time frame: The second year
The proportion of drug withdrawal
8 secondary outcomes
TFR rate
Time frame: The 6months、12months
DMR rate
Time frame: The 24/36/48/60 months
DMR persistence rate in 2 years
Time frame: 2 years
Disease progression rate , time to progression
Time frame: The whole study, up to 7 years
Loss of Major Molecular Remission(MMR-loss IS BCR-ABL≥0.1%）
Time frame: TFR phase in the study, up to 2 years
Re-Major Molecular Remission
Time frame: The TFR phase, up to 2 years
Treatment-related safety
Time frame: The whole study , up to 7 years
Patient Report Outcome（PRO）
Time frame: Every 3 months in the whole enrollment period, up to 7 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2022.
- Individual participant dataSharing planned: Yes. After the study was complete, IPD will be uploaded as an supplementary data of the research paper. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jianyu Jianyu, PhD study chair
Sponsor GmbH
Questions about this trial
What is this clinical trial studying?
An observational study of Chronic Myeloid Leukemia and Treatment-free Remission, run by Guangdong Provincial People's Hospital.
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 7 inclusion and 6 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: opening soon, as of the sponsor's last update on 1 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jianyu Weng, PhD (+86-020-83827812, gdph_wjy21@gd.gov.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Deep molecular response(DMR) rate of 12 months (time frame: The 12th month). 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?
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 1 Jul 2022; AzTrials last refreshed its copy on 26 Oct 2026.