Trial Outcomes & Findings for Glutaminase Inhibitor CB-839 and Azacitidine in Treating Patients With Advanced Myelodysplastic Syndrome (NCT NCT03047993)

Last Updated: 2024-05-14

Results Overview

Response is Complete Remission (CR) + Partial Remission (PR) + Marrow CR = Complete Remission (CR) is \</=5% myeloblasts with normal maturation of all cell lines\* Persistent dysplasia will be noted, Peripheral blood; hemoglobin\>/=11 g/dL, Platelets \>/= 100x10\^9/L ,Neutrophils \>/=1.0 x 10\^9/L, Blasts = 0%. Partial Remission (PR) is all CR criteria if abnormal before treatment except: Bone marrow blasts decreased by \</=50% over pretreatment but still \>5% Cellularity and morphology not relevant. Marrow CR is Bone marrow: \</= 5% meyloblasts and decreased by \>/= 50% over pretreatment† Peripheral blood: if HI responses, they will be noted in addition to marrow CR.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

28 participants

Primary outcome timeframe

Up to 5 years, 4 months

Results posted on

2024-05-14

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Glutaminase Inhibitor CB-839, Azacitidine) Patients receive glutaminase inhibitor CB-839 PO BID on days 1-28 and azacitidine SC or IV over 10-40 minutes on days 1-7. Azacitidine: Given IV or SC Glutaminase Inhibitor CB-839: Given PO
Overall Study STARTED	28
Overall Study COMPLETED	28
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Glutaminase Inhibitor CB-839 and Azacitidine in Treating Patients With Advanced Myelodysplastic Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Glutaminase Inhibitor CB-839, Azacitidine) n=28 Participants Patients receive glutaminase inhibitor CB-839 PO BID on days 1-28 and azacitidine SC or IV over 10-40 minutes on days 1-7. Azacitidine: Given IV or SC Glutaminase Inhibitor CB-839: Given PO
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	18 Participants n=5 Participants
Age, Continuous	70 years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 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	0 Participants n=5 Participants
Race (NIH/OMB) White	24 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Region of Enrollment United States	28 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 5 years, 4 months

Outcome measures

Outcome measures
Measure	Treatment (Glutaminase Inhibitor CB-839, Azacitidine) n=28 Participants Patients receive glutaminase inhibitor CB-839 PO BID on days 1-28 and azacitidine SC or IV over 10-40 minutes on days 1-7. Azacitidine: Given IV or SC Glutaminase Inhibitor CB-839: Given PO
Number of Participants With a Response	11 Participants

Adverse Events

Treatment (Glutaminase Inhibitor CB-839, Azacitidine)

Serious events: 18 serious events

Other events: 0 other events

Deaths: 6 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Glutaminase Inhibitor CB-839, Azacitidine) n=28 participants at risk Patients receive glutaminase inhibitor CB-839 PO BID on days 1-28 and azacitidine SC or IV over 10-40 minutes on days 1-7. Azacitidine: Given IV or SC Glutaminase Inhibitor CB-839: Given PO
Renal and urinary disorders Acute Kidney Injury	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Blood and lymphatic system disorders Anemia	7.1% 2/28 • Number of events 2 • Up to 5 years, 4 months
Blood and lymphatic system disorders Blood and lymphatic disorders - Other, specify	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Cardiac disorders Cardiac Disorders, Other - Decompensated Heart Failure	7.1% 2/28 • Number of events 2 • Up to 5 years, 4 months
Gastrointestinal disorders Cholecystitis	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Psychiatric disorders Confusion	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Respiratory, thoracic and mediastinal disorders Dyspnea	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Respiratory, thoracic and mediastinal disorders Epistaxis	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
General disorders Fatigue	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Blood and lymphatic system disorders Febrile neutropenia	10.7% 3/28 • Number of events 3 • Up to 5 years, 4 months
General disorders Fever	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Injury, poisoning and procedural complications Fracture	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Gastrointestinal disorders Gastrointestinal Disorders, Other-Melena	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Gastrointestinal disorders Gastrointestinal Disorders-Hemorrhage	3.6% 1/28 • Number of events 2 • Up to 5 years, 4 months
General disorders General disorders and administration site conditions-Other, Neck Swelling	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Vascular disorders Hematoma	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Vascular disorders Hypotension	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Respiratory, thoracic and mediastinal disorders Hypoxia	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Immune system disorders Immune System Disorders, Other	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Infections and infestations Infection	14.3% 4/28 • Number of events 5 • Up to 5 years, 4 months
Nervous system disorders Intracranial hemorrhage	7.1% 2/28 • Number of events 2 • Up to 5 years, 4 months
Infections and infestations Lung infection	7.1% 2/28 • Number of events 2 • Up to 5 years, 4 months
Gastrointestinal disorders Mucositis	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified	7.1% 2/28 • Number of events 2 • Up to 5 years, 4 months
General disorders Non-cardiac chest pain	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Investigations Platelet count decreased	10.7% 3/28 • Number of events 4 • Up to 5 years, 4 months
Respiratory, thoracic and mediastinal disorders Respiratory failure	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Infections and infestations Scrotal infection	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Infections and infestations Soft tissue Infection	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Vascular disorders Stroke	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Vascular disorders Thromboembolic Event	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Infections and infestations Upper respiratory infection	10.7% 3/28 • Number of events 3 • Up to 5 years, 4 months
Infections and infestations Urinary tract infection	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Vascular disorders Vascular Disorders, Other-Hematoma	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
Investigations White blood cell decreased	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months
General disorders Death	7.1% 2/28 • Number of events 2 • Up to 5 years, 4 months
Infections and infestations Wound infection	3.6% 1/28 • Number of events 1 • Up to 5 years, 4 months

Other adverse events

Adverse event data not reported

Additional Information

Courtney DiNardo/Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-794-1141

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place