Trial Outcomes & Findings for Testing the Addition of Ruxolitinib to the Usual Treatment (Tyrosine Kinase Inhibitors) for Chronic Myeloid Leukemia (NCT NCT03654768)

Last Updated: 2025-12-18

Results Overview

The participants BCR-ABL/BCR ratio must be at least 31,623 times (4.5 logs) smaller than100% IS, i.e., must demonstrate a 4.5-log reduction relative to 100% IS. When reported on the International Scale (IS), this response is equivalent to a value of ≤ 0.0032%. Values are reported as percentages with a higher rate being better then a lower rate.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

81 participants

Primary outcome timeframe

At 12 months

Results posted on

2025-12-18

Participant Flow

81 participants were assessed for eligibility, 6 were ineligible (3 had no bone marrow at diagnosis, 2 had TKI switch due to resistance, and 1 was on TKI for less than a year). 75 participants were randomized, 38 to the Single Agent TKI arm and 37 to the TKI + Ruxolitinib arm.

Participant milestones

Participant milestones
Measure	Single Agent TKI Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	38	37
Overall Study COMPLETED	33	22
Overall Study NOT COMPLETED	5	15

Reasons for withdrawal

Reasons for withdrawal
Measure	Single Agent TKI Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
Overall Study Adverse Event	1	6
Overall Study Loss of response	2	2
Overall Study Non-compliance	0	1
Overall Study Refusal not related to toxicity	1	5
Overall Study Did not receive protocol therapy	1	1

Baseline Characteristics

Testing the Addition of Ruxolitinib to the Usual Treatment (Tyrosine Kinase Inhibitors) for Chronic Myeloid Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Agent TKI n=38 Participants Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib n=37 Participants Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	Total n=75 Participants Total of all reporting groups
Age, Continuous	55 years n=47 Participants	47 years n=41 Participants	50 years n=88 Participants
Sex: Female, Male Female	9 Participants n=47 Participants	16 Participants n=41 Participants	25 Participants n=88 Participants
Sex: Female, Male Male	29 Participants n=47 Participants	21 Participants n=41 Participants	50 Participants n=88 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=47 Participants	1 Participants n=41 Participants	5 Participants n=88 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	24 Participants n=47 Participants	29 Participants n=41 Participants	53 Participants n=88 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	10 Participants n=47 Participants	7 Participants n=41 Participants	17 Participants n=88 Participants
Race/Ethnicity, Customized Race · Black	1 Participants n=47 Participants	1 Participants n=41 Participants	2 Participants n=88 Participants
Race/Ethnicity, Customized Race · Pacific Islander	1 Participants n=47 Participants	0 Participants n=41 Participants	1 Participants n=88 Participants
Race/Ethnicity, Customized Race · Asian	0 Participants n=47 Participants	1 Participants n=41 Participants	1 Participants n=88 Participants
Race/Ethnicity, Customized Race · White	33 Participants n=47 Participants	29 Participants n=41 Participants	62 Participants n=88 Participants
Race/Ethnicity, Customized Race · Unknown	3 Participants n=47 Participants	6 Participants n=41 Participants	9 Participants n=88 Participants
ECOG PS PS 0	26 Participants n=47 Participants	25 Participants n=41 Participants	51 Participants n=88 Participants
ECOG PS PS 1	12 Participants n=47 Participants	11 Participants n=41 Participants	23 Participants n=88 Participants
ECOG PS PS 2	0 Participants n=47 Participants	1 Participants n=41 Participants	1 Participants n=88 Participants
Length on TKI TKI: 1-4 years	24 Participants n=47 Participants	21 Participants n=41 Participants	45 Participants n=88 Participants
Length on TKI TKI: 4-10 years	14 Participants n=47 Participants	16 Participants n=41 Participants	30 Participants n=88 Participants
TKI and Randomization Bosutinib	4 Participants n=47 Participants	4 Participants n=41 Participants	8 Participants n=88 Participants
TKI and Randomization Dasatinib	22 Participants n=47 Participants	24 Participants n=41 Participants	46 Participants n=88 Participants
TKI and Randomization Imatinib	5 Participants n=47 Participants	2 Participants n=41 Participants	7 Participants n=88 Participants
TKI and Randomization Nilotinib	7 Participants n=47 Participants	7 Participants n=41 Participants	14 Participants n=88 Participants
Molecular Response at Randomization MMR	21 Participants n=47 Participants	20 Participants n=41 Participants	41 Participants n=88 Participants
Molecular Response at Randomization MR20	14 Participants n=47 Participants	11 Participants n=41 Participants	25 Participants n=88 Participants
Molecular Response at Randomization MR40	3 Participants n=47 Participants	6 Participants n=41 Participants	9 Participants n=88 Participants

PRIMARY outcome

Timeframe: At 12 months

Population: This population includes the 75 eligible and evaluable participants. 38 in the Single Agent TKI arm and 37 in the TKI + Ruxolitnib arm.

Outcome measures

Outcome measures
Measure	Single Agent TKI n=38 Participants Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib n=37 Participants Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
Rate of Molecular Response 4.5 (MR4.5)	3 percentage of participants	14 percentage of participants

SECONDARY outcome

Timeframe: From the date of randomization until death from any cause with observations censored at the date of last contact for patients last known to be alive, assessed up to 3 years

Population: This population includes the 75 eligible and evaluable participants. 38 in the Single Agent TKI arm and 37 in the TKI + Ruxolitnib arm.

Overall survival will be measured for all patients from the date of randomization until death from any cause with observations censored at the date of last contact for patients last known to be alive. The results were presented as 3-year OS estimate.

Outcome measures

Outcome measures
Measure	Single Agent TKI n=38 Participants Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib n=37 Participants Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
Overall Survival	100 percentage of participants Kaplan-Meier confidence intervals could not be estimated for the TKI alone arm because there were no events on that arm	97 percentage of participants Interval 80.0 to 100.0

SECONDARY outcome

Timeframe: From the date of randomization on study until the first of treatment failure/loss of response, progression, or death from any cause, assessed up to 5 years

Population: This population includes the 75 eligible and evaluable participants. 38 in the Single Agent TKI arm and 37 in the TKI + Ruxolitnib arm.

Progression-free survival is measured from the date of randomization on study until the first of treatment failure/loss of response, progression, or death from any cause. Observations are censored at the date of last follow-up for patients last known to be alive without report of treatment failure/loss of response or progression.

Outcome measures

Outcome measures
Measure	Single Agent TKI n=38 Participants Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib n=37 Participants Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
Progression-free Survival	89 percentage of participants Interval 74.0 to 96.0	89 percentage of participants Interval 73.0 to 96.0

SECONDARY outcome

Timeframe: Duration of treatment and follow-up until death or 5 years post registration.

Population: Participants who were eligible and received at least one dose of protocol treatment.

Only adverse events that are possibly, probably or definitely related to study drug are reported. CTCAE Version 5.0 was used for all AE reporting.

Outcome measures

Outcome measures
Measure	Single Agent TKI n=37 Participants Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib n=36 Participants Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Neutrophil count decreased	1 Participants	2 Participants
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Pancreatitis	1 Participants	0 Participants
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Pericardial effusion	1 Participants	0 Participants
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Respiratory failure	0 Participants	1 Participants
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Vomiting	0 Participants	1 Participants
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Constipation	0 Participants	1 Participants
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Fatigue	0 Participants	1 Participants
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Headache	0 Participants	1 Participants
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Hypertension	1 Participants	0 Participants
Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs Nausea	0 Participants	1 Participants

SECONDARY outcome

Timeframe: Up to 12 months

Population: This population includes the 75 eligible and evaluable participants. 38 in the Single Agent TKI arm and 37 in the TKI + Ruxolitnib arm.

Will be summarized descriptively

Outcome measures

Outcome measures
Measure	Single Agent TKI n=38 Participants Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib n=37 Participants Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
MR4.5 Attainment 3-Month Response	3 percentage of participants	8 percentage of participants
MR4.5 Attainment 6-Month Response	3 percentage of participants	5 percentage of participants
MR4.5 Attainment 9-Month Response	0 percentage of participants	14 percentage of participants
MR4.5 Attainment 12-Month Response	3 percentage of participants	14 percentage of participants

SECONDARY outcome

Timeframe: Up to 12 months

Population: This population includes the 75 eligible and evaluable participants. 38 in the Single Agent TKI arm and 37 in the TKI + Ruxolitnib arm.

Will be summarized descriptively

Outcome measures

Outcome measures
Measure	Single Agent TKI n=38 Participants Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib n=37 Participants Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
Molecular Rate 4.0 (MR 4.0) Attainment 3-Month Response	16 percentage of participants	24 percentage of participants
Molecular Rate 4.0 (MR 4.0) Attainment 6-Month Response	13 percentage of participants	24 percentage of participants
Molecular Rate 4.0 (MR 4.0) Attainment 9-Month Response	18 percentage of participants	22 percentage of participants
Molecular Rate 4.0 (MR 4.0) Attainment 12-Month Response	11 percentage of participants	19 percentage of participants

SECONDARY outcome

Timeframe: Up to 5 years

Population: This population includes the 73 participants who were eligible and received at least one dose of study treatment. 37 in the Single Agent TKI arm and 36 in the TKI + Ruxolitnib arm.

Will be summarized descriptively, results presented as number of participants that were fully compliant for all cycles that they received treatment.

Outcome measures

Outcome measures
Measure	Single Agent TKI n=37 Participants Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib n=36 Participants Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
Drug Compliance	36 Participants	31 Participants

SECONDARY outcome

Timeframe: Up to 5 years

Population: This population includes the 75 eligible and evaluable participants. 38 in the Single Agent TKI arm and 37 in the TKI + Ruxolitnib arm.

Quantitative RT-PCR measurement of BCR-ABL transcript levels on the International Scale (IS), expressed as log₁₀ ratios of BCR-ABL to ABL. Mean and standard deviation of log₁₀ BCR-ABL (IS) are reported at 3, 6, 9, and 12 months for each treatment arm. Lower values represent deeper molecular response. This scale ranges from 0 to -5, with a more negative number corresponding to a better outcome.

Outcome measures

Outcome measures
Measure	Single Agent TKI n=38 Participants Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.	TKI + Ruxolitinib n=37 Participants Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
BCR-ABL/BCR Comparison 3-months	-1.22 Log₁₀ BCR-ABL Ratio Standard Deviation 0.52	-1.37 Log₁₀ BCR-ABL Ratio Standard Deviation 0.60
BCR-ABL/BCR Comparison 6-months	-1.30 Log₁₀ BCR-ABL Ratio Standard Deviation 0.62	-1.48 Log₁₀ BCR-ABL Ratio Standard Deviation 0.64
BCR-ABL/BCR Comparison 9-months	-1.32 Log₁₀ BCR-ABL Ratio Standard Deviation 0.60	-1.47 Log₁₀ BCR-ABL Ratio Standard Deviation 0.68
BCR-ABL/BCR Comparison 12-months	-1.26 Log₁₀ BCR-ABL Ratio Standard Deviation 0.55	-1.52 Log₁₀ BCR-ABL Ratio Standard Deviation 0.68

Adverse Events

Single Agent TKI

Serious events: 0 serious events

Other events: 31 other events

Deaths: 0 deaths

TKI + Ruxolitinib

Serious events: 4 serious events

Other events: 35 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Single Agent TKI n=37 participants at risk Patients receive bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity. The number of participants at risk in All-cause Mortality are the number of participants who were randomized. The number of participants at risk in Serious Adverse Events are the number of participants who received at least one dose of protocol treatment.	TKI + Ruxolitinib n=36 participants at risk Patients receive ruxolitinib phosphate PO BID on days 1-90, and bosutinib PO daily or dasatinib PO daily or nilotinib PO BID or imatinib PO daily on days 1-90. Treatment repeats every 90 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity. The number of participants at risk in All-cause Mortality are the number of participants who were randomized. The number of participants at risk in Serious Adverse Events are the number of participants who received at least one dose of protocol treatment.
General disorders Fatigue	0.00% 0/37 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.	2.8% 1/36 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.
Renal and urinary disorders Renal calculi	0.00% 0/37 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.	2.8% 1/36 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/37 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.	5.6% 2/36 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.
Respiratory, thoracic and mediastinal disorders Pneumonitis	0.00% 0/37 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.	2.8% 1/36 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/37 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.	2.8% 1/36 • Duration of treatment and follow up until death or 5 years post randomization. CTCAE version 5.0 was used for toxicity and SAEs reporting. There were 37 participants on the Single Agent TKI arm that were assessed for AEs, and 36 participants on the TKI + Ruxolitinib arm that were assessed for AEs.

Other adverse events

Additional Information

Leukemia Committe Statistician

SWOG Statistics and Data Management Center

Phone: 2066674623

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60