Trial Outcomes & Findings for Combined Ruxolitinib and Enasidenib in Patients With Accelerated/Blast-phase Myeloproliferative Neoplasm or Chronic-phase Myelofibrosis With an IDH2 Mutation (NCT NCT04281498)
NCT ID: NCT04281498
Last Updated: 2025-02-04
Results Overview
The number of treated accelerated-phase and blast-phase MPN patients (primary cohort) that achieve a best response per 2013 International Working Group (IWG) criteria of either complete response (CR), Partial Response (PR), or complete response with incomplete recovery of counts (CRi), when treated with the combination of ruxolitinib with enasidenib within 6 cycles of combined therapy. Complete Response with incomplete recovery of counts (CRi) - complete remission (\<5% marrow blasts by morphology) with incomplete count recovery (platelet count \<100 x 10\^9\^/L and/or absolute neutrophil count \< 1 x 10\^9\^/L) Complete Response (CR) - full marrow recovery; full count recovery; resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH Partial Response (PR) - morphologic remission in the peripheral blood but not necessarily in the bone marrow; resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH
COMPLETED
PHASE2
6 participants
6 Months
2025-02-04
Participant Flow
Participant milestones
| Measure |
Patients With MPN
Ruxolitinib and Enasidenib combination therapy
Ruxolitinib: Patients who are on ruxolitinib will continue their current dose. Patients who are not on ruxolitinib will receive ruxolitinib dosing based on platelet count
Enasidenib: 50mg -100mg daily
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Patients With MPN
Ruxolitinib and Enasidenib combination therapy
Ruxolitinib: Patients who are on ruxolitinib will continue their current dose. Patients who are not on ruxolitinib will receive ruxolitinib dosing based on platelet count
Enasidenib: 50mg -100mg daily
|
|---|---|
|
Overall Study
Progression of disease
|
1
|
|
Overall Study
Planned SCT
|
1
|
|
Overall Study
Alternative Treatment
|
1
|
Baseline Characteristics
Combined Ruxolitinib and Enasidenib in Patients With Accelerated/Blast-phase Myeloproliferative Neoplasm or Chronic-phase Myelofibrosis With an IDH2 Mutation
Baseline characteristics by cohort
| Measure |
Patients With MPN
n=6 Participants
Ruxolitinib and Enasidenib combination therapy
Ruxolitinib: Patients who are on ruxolitinib will continue their current dose. Patients who are not on ruxolitinib will receive ruxolitinib dosing based on platelet count
Enasidenib: 50mg -100mg daily
|
|---|---|
|
Age, Continuous
|
74 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Prior Hydroxyurea
|
4 Participants
n=5 Participants
|
|
Prior Ruxolitinib
|
4 Participants
n=5 Participants
|
|
Prior Decitabine
Yes
|
1 Participants
n=5 Participants
|
|
Prior Decitabine
No
|
4 Participants
n=5 Participants
|
|
Prior Decitabine
Unknown
|
1 Participants
n=5 Participants
|
|
Total Symptom Score (TSS)
|
10 units on a scale
n=5 Participants
|
|
Platelets
|
111.0 cells*10^9/L
n=5 Participants
|
|
Bone marrow % blasts
|
12.0 % blast cells in bone marrow
n=5 Participants
|
|
Peripheral blood % blasts
|
14.0 % blast cells in peripheral blood
n=5 Participants
|
|
Fibrosis grade
0 None
|
1 Participants
n=5 Participants
|
|
Fibrosis grade
1 Mild
|
1 Participants
n=5 Participants
|
|
Fibrosis grade
2 Severe
|
3 Participants
n=5 Participants
|
|
Fibrosis grade
Not assessed
|
1 Participants
n=5 Participants
|
|
Patient is transfusion dependent: Red blood cells
Yes
|
3 Participants
n=5 Participants
|
|
Patient is transfusion dependent: Red blood cells
Missing
|
3 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 MonthsPopulation: Data for participants with MPN
The number of treated accelerated-phase and blast-phase MPN patients (primary cohort) that achieve a best response per 2013 International Working Group (IWG) criteria of either complete response (CR), Partial Response (PR), or complete response with incomplete recovery of counts (CRi), when treated with the combination of ruxolitinib with enasidenib within 6 cycles of combined therapy. Complete Response with incomplete recovery of counts (CRi) - complete remission (\<5% marrow blasts by morphology) with incomplete count recovery (platelet count \<100 x 10\^9\^/L and/or absolute neutrophil count \< 1 x 10\^9\^/L) Complete Response (CR) - full marrow recovery; full count recovery; resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH Partial Response (PR) - morphologic remission in the peripheral blood but not necessarily in the bone marrow; resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH
Outcome measures
| Measure |
Patients With MPN
n=5 Participants
Ruxolitinib and Enasidenib combination therapy
Ruxolitinib: Patients who are on ruxolitinib will continue their current dose. Patients who are not on ruxolitinib will receive ruxolitinib dosing based on platelet count
Enasidenib: 50mg -100mg daily
|
|---|---|
|
Number of MPN Participants With Response
|
2 Participants
|
SECONDARY outcome
Timeframe: 6 MonthsThe number of treated accelerated-phase and blast-phase MPN patients that achieve complete (CBR) and partial blast response (PBR).
Outcome measures
| Measure |
Patients With MPN
n=5 Participants
Ruxolitinib and Enasidenib combination therapy
Ruxolitinib: Patients who are on ruxolitinib will continue their current dose. Patients who are not on ruxolitinib will receive ruxolitinib dosing based on platelet count
Enasidenib: 50mg -100mg daily
|
|---|---|
|
Number of MPN Participants With Blast Response
|
3 Participants
|
SECONDARY outcome
Timeframe: 6 MonthsThe number of treated patients with MF-CP and 4%-9% circulating blasts that achieve complete response (CR) Partial Response (PR), clinical improvement (CI) with the combination of ruxolitinib and enasidenib within 6 cycles of combined therapy.
Outcome measures
| Measure |
Patients With MPN
n=1 Participants
Ruxolitinib and Enasidenib combination therapy
Ruxolitinib: Patients who are on ruxolitinib will continue their current dose. Patients who are not on ruxolitinib will receive ruxolitinib dosing based on platelet count
Enasidenib: 50mg -100mg daily
|
|---|---|
|
Number of MF-CP Participants With Any Response
|
0 Participants
|
Adverse Events
Patients With MPN
Serious adverse events
| Measure |
Patients With MPN
n=6 participants at risk
Ruxolitinib and Enasidenib combination therapy
Ruxolitinib: Patients who are on ruxolitinib will continue their current dose. Patients who are not on ruxolitinib will receive ruxolitinib dosing based on platelet count
Enasidenib: 50mg -100mg daily
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
1/6 • 6 months
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
16.7%
1/6 • 6 months
|
Other adverse events
| Measure |
Patients With MPN
n=6 participants at risk
Ruxolitinib and Enasidenib combination therapy
Ruxolitinib: Patients who are on ruxolitinib will continue their current dose. Patients who are not on ruxolitinib will receive ruxolitinib dosing based on platelet count
Enasidenib: 50mg -100mg daily
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
50.0%
3/6 • 6 months
|
|
Blood and lymphatic system disorders
Leukocytosis
|
33.3%
2/6 • 6 months
|
|
Blood and lymphatic system disorders
Neutrophil count decreased
|
33.3%
2/6 • 6 months
|
|
Blood and lymphatic system disorders
Platelet count decreased
|
50.0%
3/6 • 6 months
|
|
Blood and lymphatic system disorders
White blood cell decreased
|
50.0%
3/6 • 6 months
|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
1/6 • 6 months
|
|
Investigations
Alanine aminotransferase increased
|
16.7%
1/6 • 6 months
|
|
Investigations
Alkaline phosphatase increased
|
16.7%
1/6 • 6 months
|
|
Metabolism and nutrition disorders
Anorexia
|
16.7%
1/6 • 6 months
|
|
Investigations
Aspartate aminotransferase increased
|
50.0%
3/6 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
16.7%
1/6 • 6 months
|
|
Gastrointestinal disorders
Belching
|
16.7%
1/6 • 6 months
|
|
Gastrointestinal disorders
Bloating
|
16.7%
1/6 • 6 months
|
|
Investigations
Blood bilirubin increased
|
50.0%
3/6 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
33.3%
2/6 • 6 months
|
|
Injury, poisoning and procedural complications
Bruising
|
16.7%
1/6 • 6 months
|
|
Gastrointestinal disorders
Constipation
|
33.3%
2/6 • 6 months
|
|
Investigations
Creatinine increased
|
33.3%
2/6 • 6 months
|
|
Nervous system disorders
Dizziness
|
16.7%
1/6 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
33.3%
2/6 • 6 months
|
|
General disorders
Edema limbs
|
33.3%
2/6 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
50.0%
3/6 • 6 months
|
|
Injury, poisoning and procedural complications
Fall
|
16.7%
1/6 • 6 months
|
|
General disorders
Fatigue
|
16.7%
1/6 • 6 months
|
|
General disorders
Fever (Pyrexia)
|
33.3%
2/6 • 6 months
|
|
Nervous system disorders
Headache
|
50.0%
3/6 • 6 months
|
|
Cardiac disorders
Heart failure
|
16.7%
1/6 • 6 months
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
33.3%
2/6 • 6 months
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
16.7%
1/6 • 6 months
|
|
Metabolism and nutrition disorders
Hypernatremia
|
16.7%
1/6 • 6 months
|
|
Metabolism and nutrition disorders
Hyperphosphatemia
|
16.7%
1/6 • 6 months
|
|
Vascular disorders
Hypertension
|
16.7%
1/6 • 6 months
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
50.0%
3/6 • 6 months
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
33.3%
2/6 • 6 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
16.7%
1/6 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Muscle cramp
|
16.7%
1/6 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
16.7%
1/6 • 6 months
|
|
Infections and infestations
OTHER COVID-19
|
16.7%
1/6 • 6 months
|
|
Ear and labyrinth disorders
OTHER Ear lobe pain
|
16.7%
1/6 • 6 months
|
|
Gastrointestinal disorders
OTHER early satiety
|
16.7%
1/6 • 6 months
|
|
Gastrointestinal disorders
OTHER Heartburn
|
16.7%
1/6 • 6 months
|
|
General disorders
OTHER Night sweats
|
16.7%
1/6 • 6 months
|
|
Skin and subcutaneous tissue disorders
OTHER Petechial
|
16.7%
1/6 • 6 months
|
|
Skin and subcutaneous tissue disorders
OTHER Unspecified contact dermatitis
|
16.7%
1/6 • 6 months
|
|
Gastrointestinal disorders
Oral pain
|
16.7%
1/6 • 6 months
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
16.7%
1/6 • 6 months
|
|
Infections and infestations
Upper respiratory infection
|
16.7%
1/6 • 6 months
|
|
Ear and labyrinth disorders
Vertigo
|
16.7%
1/6 • 6 months
|
Additional Information
Dr. John Mascarenhas
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place