Chronic Phase Chronic Myeloid LeukemiaPhase 2NCT03610971
Treatment Free Remission After Combination Therapy With Ruxolitinib Plus Tyrosine Kinase Inhibitors
A Phase 2 study testing Ruxolitinib and BCR-ABL Tyrosine Kinase Inhibitor (TKI) in people with Chronic Phase Chronic Myeloid Leukemia and Chronic Myeloid Leukemia, Chronic Phase, run by H. Lee Moffitt Cancer Center and Research Institute at 8 sites in United States.
Trial summary
The purpose of this study is to determine if adding Ruxolitinib to a Tyrone Kinase Inhibitor (TKI), prior to a second attempt at stopping a TKI will lead to prolonged treatment free remission (TFR). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Phase Chronic Myeloid Leukemia, Chronic Myeloid Leukemia, Chronic Phase
- What is being tested?
- Ruxolitinib and BCR-ABL Tyrosine Kinase Inhibitor (TKI)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 8 sitesUnited States
- Study dates
- 19 Nov 2019to Oct 2026
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Willing and able to give informed consent
- Diagnosed with CML in chronic phase and have either the b3a2 (e14a2) or b2a2 (e13a2) variants that give rise to the p210 BCR-ABL protein.
- Must have a documented history of attempting only one prior TKI discontinuation under the guidance of a treating physician
- Must have met ALL the following criteria prior to first attempt to discontinue their TKI:
- Stable molecular response (MR4; <0.01% IS) for > 2 years (allowance for a 2 week variance), as documented on at least 4 tests, performed at least 3 months apart. If any results are >0.05% IS, tests must have been repeated within 1 month and be less than 0.01% IS and stable.
- Treatment with one of the following FDA approved TKI; imatinib, dasatinib, nilotinib or bosutinib, at any dose for a minimum of approximately 3 years (allowance of a 4 week variance) prior to discontinuing TKIs
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- Has been on any number of TKIs, but has not been resistant to any TKI (changes made for intolerance are allowed)
- Must have relapsed (defined as loss of major molecular response (MMR), RQ-PCR for BCR-ABL > 0.1% IS after first attempted discontinuation of TKI
- After first failed TFR attempt, must have a minimum duration of 1 year on a TKI, and must plan to remain on this same TKI for a minimum of 12 months during the combination treatment phase
- Current TKI must be the same as the TKI being taken prior to the initial TFR attempt (e.g., if patient is on imatinib prior to first TFR attempt, they should be on imatinib at time of enrollment on this study)
- Eastern Cooperative Oncology Group (ECOG) performance status 0-3
- Must have a RQ-PCR for BCR-ABL less than 0.0032% IS (MR4.5) reported by the trial designated central lab at time of study enrollment
- Must adhere to all study contraception guidelines
Who cannot join
- History of accelerated or blast phase CML
- History of TKI resistance
- A second malignancy requiring active treatment
- Have previously received treatment with a JAK inhibitor.
- Platelet count less than 100 × 10^9/L or an absolute neutrophil count of less than 1 × 10^9/L or Hemoglobin less than 8 g/dL
- AST and ALT ≥ 3 times the institutional upper limit of normal (ULN)
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- Creatinine ≥ 2 times ULN
- Total bilirubin ≥ 1.5 times ULN or >3.0 x the ULN with Gilbert Syndrome (unless direct bilirubin is within normal limits)
- Pregnant or lactating
- Unable to comply with lab appointments schedule and patient response outcome assessments
- Another investigational drug within 4 weeks of enrollment
- Any serious medical or psychiatric illness that could, in the investigator's opinion, interfere with the completion of treatment according to this protocol
- Have undergone a prior allogeneic transplant
- Screening 12-lead ECG showing a baseline corrected QT interval >500msec (patients with a pacemaker will still be eligible with QTc>500msec)
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Emory -Winship Cancer Institute
Atlanta, Georgia, 30322
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263
Duke University Hospital
Durham, North Carolina, 27710
Froedtert Hospital & the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Memorial Sloan Kettering - Bergen
Montvale, New Jersey, 07645
David H. Koch Center for Cancer Care at Memorial Sloan Kettering
New York, New York, 10021
Oregon Health & Science University
Portland, Oregon, 97239
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612
About this study
Official title: Treatment Free Remission After Combination Therapy With Ruxolitinib Plus Tyrosine Kinase Inhibitors in Chronic Phase Chronic Myeloid Leukemia (CP-CML) Patients Who Relapsed After a Prior Attempt at TKI Discontinuation
The purpose of this study is to determine if adding Ruxolitinib to a Tyrone Kinase Inhibitor (TKI), prior to a second attempt at stopping a TKI will lead to prolonged treatment free remission (TFR).
Read the detailed description
All participants will have a confirmed diagnosis of chronic phase chronic myeloid leukemia (CML) and must have previously attempted to discontinue TKI therapy. All participants must be restarted on a TKI at the time of relapse in order to be eligible for this trial.
After completion of 12 cycles of combination therapy, eligible participants will remain in the TFR phase of the study for up to 36 months, and will have central polymerase chain reaction (PCR) testing during the first 24 months. Therefore, the total duration of the trial will be approximately 48 months (12 months on combination treatment phase + 36 months in the TFR phase).
- Being tested
- Ruxolitinib, BCR-ABL Tyrosine Kinase Inhibitor (TKI)
- Keywords
- CP-CML, Chronic Myeloid Leukemia, CML, combination therapy, BCR-ABL oncogene
- Medical subject headings
- Leukemia, Myeloid, Chronic-Phase, 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.
RuxolitinibDrug
Also called Jakafi®
Ruxolitinib: 15 mg by mouth (PO) twice a day (BID).
BCR-ABL Tyrosine Kinase Inhibitor (TKI)Drug
Also called TKI
The BCR-ABL TKIs that will be used include imatinib, dasatinib, nilotinib or bosutinib.
| Study group | Role | What they receive |
|---|---|---|
| Combination Therapy + Remission Phase | Experimental | Combination therapy followed by treatment free remission (TFR) phase.
Combination Therapy: Ruxolitinib plus BCR-ABL Tyrosine Kinase Inhibitors (TKIs).
All eligible patients will begin ruxolitinib in combination with their BCR-ABL TKI on cycle 1 day 1 of the combination phase. For cycle 2 and beyond, if day 1 of a cycle is delayed, day 1 procedures should be repeated if out of the specified window and day 1 of the cycle is considered the day study drug is restarted. They will continue combination therapy for a total of 12 cycles. Each cycle will be approximately 28 days.
At the end of 12 cycles ruxolitinib will be discontinued and any patient who has met the criteria for the treatment free remission (TFR) screening phase will enter into the TFR phase. Once in the TFR phase, participants will discontinue their BCR-ABL TKI and be monitored off treatment. Receives: Drug: Ruxolitinib; Drug: BCR-ABL Tyrosine Kinase Inhibitor (TKI) |
What is being measured
The primary outcome is the main question the study is designed to answer.
12 Month Treatment Free Remission (TFR)
TFR rate after completion of 12 cycles of combination therapy.
Measured: 12 months post 12 cycles of treatment
Secondary outcomes (1)
Adverse Events Possibly Related to Study Treatment
Establish the adverse event profile of ruxolitinib in combination with BCR-ABL TKIs: Adverse Events (AEs) and Serious Adverse Events (SAEs) according to Common Terminology Criteria for Adverse Events (CTCAE) V5.0, that are possibly, probably, or definitely related to study treatment.
Measured: Up to 30 days post treatment, approximately 13 months per participant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2018
- Study start19 Nov 2019
- Primary completion25 Sep 2025
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Phase Chronic Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- H. Jean Khoury Cure CML Consortium, Incyte Corporation
- Responsible party
- Sponsor
- Investigators
- Javier Pinilla-Ibarz, MD, PhD, Principal investigator, H. Lee Moffitt Cancer Center and Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03610971
- Sponsor's protocol ID
- MCC-19660
- Other IDs
- HJKC3-0002
- Status last verified
- Mar 2026
- First submitted
- 19 Jul 2018
- Last updated
- 2 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment Free Remission After Combination Therapy With Ruxolitinib Plus Tyrosine Kinase Inhibitors in Chronic Phase Chronic Myeloid Leukemia (CP-CML) Patients Who Relapsed After a Prior Attempt at TKI Discontinuation
- ClinicalTrials.gov ID
- NCT03610971
- Acronym / sponsor ID
- MCC-19660
- Keywords
- CP-CML, Chronic Myeloid Leukemia, CML, combination therapy, BCR-ABL oncogene
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 24 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- H. Jean Khoury Cure CML Consortium, Incyte Corporation
- Study start
- 19 Nov 2019 (actual)
- Primary completion
- 25 Sep 2025 (actual)
- Study completion
- Oct 2026 (estimated)
- First posted
- 1 Aug 2018 (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.
The purpose of this study is to determine if adding Ruxolitinib to a Tyrone Kinase Inhibitor (TKI), prior to a second attempt at stopping a TKI will lead to prolonged treatment free remission (TFR).
Detailed description
All participants will have a confirmed diagnosis of chronic phase chronic myeloid leukemia (CML) and must have previously attempted to discontinue TKI therapy. All participants must be restarted on a TKI at the time of relapse in order to be eligible for this trial.
After completion of 12 cycles of combination therapy, eligible participants will remain in the TFR phase of the study for up to 36 months, and will have central polymerase chain reaction (PCR) testing during the first 24 months. Therefore, the total duration of the trial will be approximately 48 months (12 months on combination treatment phase + 36 months in the TFR phase).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Combination Therapy + Remission Phase | Experimental | Drug: Ruxolitinib, Drug: BCR-ABL Tyrosine Kinase Inhibitor (TKI)Combination therapy followed by treatment free remission (TFR) phase. Combination Therapy: Ruxolitinib plus BCR-ABL Tyrosine Kinase Inhibitors (TKIs). All eligible patients will begin ruxolitinib in combination with their BCR-ABL TKI on cycle 1 day 1 of the combination phase. For cycle 2 and beyond, if day 1 of a cycle is delayed, day 1 procedures should be repeated if out of the specified window and day 1 of the cycle is considered the day study drug is restarted. They will continue combination therapy for a total of 12 cycles. Each cycle will be approximately 28 days. At the end of 12 cycles ruxolitinib will be discontinued and any patient who has met the criteria for the treatment free remission (TFR) screening phase will enter into the TFR phase. Once in the TFR phase, participants will discontinue their BCR-ABL TKI and be monitored off treatment. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Willing and able to give informed consent
- Diagnosed with CML in chronic phase and have either the b3a2 (e14a2) or b2a2 (e13a2) variants that give rise to the p210 BCR-ABL protein.
- Must have a documented history of attempting only one prior TKI discontinuation under the guidance of a treating physician
- Must have met ALL the following criteria prior to first attempt to discontinue their TKI:
- Must have relapsed (defined as loss of major molecular response (MMR), RQ-PCR for BCR-ABL > 0.1% IS after first attempted discontinuation of TKI
Key exclusion criteria (14 listed)
- History of accelerated or blast phase CML
- History of TKI resistance
- A second malignancy requiring active treatment
- Have previously received treatment with a JAK inhibitor.
- Platelet count less than 100 × 10^9/L or an absolute neutrophil count of less than 1 × 10^9/L or Hemoglobin less than 8 g/dL
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 |
|---|---|---|
| RuxolitinibAlso: Jakafi®Ruxolitinib: 15 mg by mouth (PO) twice a day (BID). | Drug | Combination Therapy + Remission Phase |
| BCR-ABL Tyrosine Kinase Inhibitor (TKI)Also: TKIThe BCR-ABL TKIs that will be used include imatinib, dasatinib, nilotinib or bosutinib. | Drug | Combination Therapy + Remission Phase |
Primary outcome
12 Month Treatment Free Remission (TFR)
Time frame: 12 months post 12 cycles of treatment
TFR rate after completion of 12 cycles of combination therapy.
1 secondary outcome
Adverse Events Possibly Related to Study Treatment
Time frame: Up to 30 days post treatment, approximately 13 months per participant
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 2 Apr 2026.
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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. This study is not recruiting at the moment.
Javier Pinilla-Ibarz, MD, PhD principal investigator
H. Lee Moffitt Cancer Center and Research Institute
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib and BCR-ABL Tyrosine Kinase Inhibitor (TKI) in people with Chronic Phase Chronic Myeloid Leukemia and Chronic Myeloid Leukemia, Chronic Phase, run by H. Lee Moffitt Cancer Center and Research Institute at 8 sites in United States.
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 13 inclusion and 14 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: under way, not looking for participants, as of the sponsor's last update on 2 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. 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?
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?
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?
12 Month Treatment Free Remission (TFR) (time frame: 12 months post 12 cycles of treatment). 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 2 Apr 2026.