Trial Outcomes & Findings for Avelumab and Azacitidine in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia (NCT NCT02953561)

NCT ID: NCT02953561

Last Updated: 2020-10-22

Results Overview

Complete Response (CR) + Partial Remission (PR) + Complete Remission with incomplete recovery (CRi) + Clinical Benefit. CR = normalization of peripheral blood (PB) and bone marrow (BM) with \</= 5% BM blasts, PB granulocyte count \>/= (1.0 x 10\^9/L, and a platelet count \>/= 100 x 10\^9/L). PR = same as CR except presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. CRi meets all criteria for CR except for platelet recovery to \>100 x 10\^9/L and/or granulocyte count \> (1.0 x 10\^9/L). MLFS is BM with \</= 5% BM blasts with no PB recovery. Hematologic Improvement is platelets increase by \>/= 30 x 10\^9/L untransfused (if \<20 at pretherapy); or granulocytes increase by 100% and to \>0.5 x 10\^9/L (if lower than that pre-therapy); or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by \> 50%; or monocytosis reduction by \> 50% if pretreatment \> 5 x 109/L, or BM or PB Blasts decrease by \>/= 50%.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

19 participants

Primary outcome timeframe

Up to 2 years

Results posted on

2020-10-22

Participant Flow

Recruitment Period: February 2017 to February 2019

Participant milestones

Participant milestones
Measure
Treatment (Azacitidine, Avelumab)
Patients receive azacitidine SC or IV over 10-40 minutes on days 1-7 or on days 1-5 and 8-9. Patients also receive avelumab IV over 60 minutes on days 1 and 14 for 4 courses (or until complete response) and on day 1 for subsequent courses. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Avelumab: Given IV Azacitidine: Given subcutaneous or IV Laboratory Biomarker Analysis: Correlative studies
Overall Study
STARTED
19
Overall Study
COMPLETED
19
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Avelumab and Azacitidine in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Treatment (Azacitidine, Avelumab)
n=19 Participants
Patients receive azacitidine SC or IV over 10-40 minutes on days 1-7 or on days 1-5 and 8-9. Patients also receive avelumab IV over 60 minutes on days 1 and 14 for 4 courses (or until complete response) and on day 1 for subsequent courses. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Avelumab: Given IV Azacitidine: Given SC or IV Laboratory Biomarker Analysis: Correlative studies
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
8 Participants
n=5 Participants
Age, Categorical
>=65 years
11 Participants
n=5 Participants
Age, Continuous
69 years
n=5 Participants
Sex: Female, Male
Female
9 Participants
n=5 Participants
Sex: Female, Male
Male
10 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
3 Participants
n=5 Participants
Race (NIH/OMB)
White
15 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
Region of Enrollment
United States
19 participants
n=5 Participants

PRIMARY outcome

Timeframe: Up to 2 years

Population: Of the 19 participants registered, 17 participants were evaluable for response.

Complete Response (CR) + Partial Remission (PR) + Complete Remission with incomplete recovery (CRi) + Clinical Benefit. CR = normalization of peripheral blood (PB) and bone marrow (BM) with \</= 5% BM blasts, PB granulocyte count \>/= (1.0 x 10\^9/L, and a platelet count \>/= 100 x 10\^9/L). PR = same as CR except presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. CRi meets all criteria for CR except for platelet recovery to \>100 x 10\^9/L and/or granulocyte count \> (1.0 x 10\^9/L). MLFS is BM with \</= 5% BM blasts with no PB recovery. Hematologic Improvement is platelets increase by \>/= 30 x 10\^9/L untransfused (if \<20 at pretherapy); or granulocytes increase by 100% and to \>0.5 x 10\^9/L (if lower than that pre-therapy); or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by \> 50%; or monocytosis reduction by \> 50% if pretreatment \> 5 x 109/L, or BM or PB Blasts decrease by \>/= 50%.

Outcome measures

Outcome measures
Measure
Treatment (Azacitidine, Avelumab)
n=17 Participants
Patients receive azacitidine SC or IV over 10-40 minutes on days 1-7 or on days 1-5 and 8-9. Patients also receive avelumab IV over 60 minutes on days 1 and 14 for 4 courses (or until complete response) and on day 1 for subsequent courses. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Avelumab: Given IV Azacitidine: Given SC or IV Laboratory Biomarker Analysis: Correlative studies
Number of Participants With a Response
4 Participants

SECONDARY outcome

Timeframe: Up to 1 year

Time from date of treatment start until the date of first objective documentation of disease-relapse.

Outcome measures

Outcome measures
Measure
Treatment (Azacitidine, Avelumab)
n=17 Participants
Patients receive azacitidine SC or IV over 10-40 minutes on days 1-7 or on days 1-5 and 8-9. Patients also receive avelumab IV over 60 minutes on days 1 and 14 for 4 courses (or until complete response) and on day 1 for subsequent courses. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Avelumab: Given IV Azacitidine: Given SC or IV Laboratory Biomarker Analysis: Correlative studies
Disease-free Survival
8.2 Months
Interval 6.5 to 9.8

SECONDARY outcome

Timeframe: Up to 1 year

Distribution of OS will be estimated using the method of Kaplan and Meier. Comparisons of time-to-event endpoints by important subgroups will be made using the log-rank tests.

Outcome measures

Outcome measures
Measure
Treatment (Azacitidine, Avelumab)
n=17 Participants
Patients receive azacitidine SC or IV over 10-40 minutes on days 1-7 or on days 1-5 and 8-9. Patients also receive avelumab IV over 60 minutes on days 1 and 14 for 4 courses (or until complete response) and on day 1 for subsequent courses. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Avelumab: Given IV Azacitidine: Given SC or IV Laboratory Biomarker Analysis: Correlative studies
Overall Survival (OS)
4.7 Months
Interval 0.1 to 11.0

SECONDARY outcome

Timeframe: Up to 1 year

Distribution of PFS will be estimated using the method of Kaplan and Meier. Comparisons of time-to-event endpoints by important subgroups will be made using the log-rank tests.

Outcome measures

Outcome measures
Measure
Treatment (Azacitidine, Avelumab)
n=17 Participants
Patients receive azacitidine SC or IV over 10-40 minutes on days 1-7 or on days 1-5 and 8-9. Patients also receive avelumab IV over 60 minutes on days 1 and 14 for 4 courses (or until complete response) and on day 1 for subsequent courses. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Avelumab: Given IV Azacitidine: Given SC or IV Laboratory Biomarker Analysis: Correlative studies
Progression Free Survival (PFS)
3.2 Months
Interval 0.1 to 10.6

Adverse Events

Treatment (Azacitidine, Avelumab)

Serious events: 18 serious events
Other events: 16 other events
Deaths: 5 deaths

Serious adverse events

Serious adverse events
Measure
Treatment (Azacitidine, Avelumab)
n=19 participants at risk
Patients receive azacitidine SC or IV over 10-40 minutes on days 1-7 or on days 1-5 and 8-9. Patients also receive avelumab IV over 60 minutes on days 1 and 14 for 4 courses (or until complete response) and on day 1 for subsequent courses. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Avelumab: Given IV Azacitidine: Given SC or IV Laboratory Biomarker Analysis: Correlative studies
Infections and infestations
Anorectal Infection
10.5%
2/19 • Number of events 3 • Up to 1 year
Blood and lymphatic system disorders
Blood and Lymphatic System Disorders
5.3%
1/19 • Number of events 1 • Up to 1 year
General disorders
Death
5.3%
1/19 • Number of events 1 • Up to 1 year
Gastrointestinal disorders
Diarrhea
5.3%
1/19 • Number of events 1 • Up to 1 year
Gastrointestinal disorders
Dysphagia
5.3%
1/19 • Number of events 1 • Up to 1 year
Infections and infestations
Endocarditis
5.3%
1/19 • Number of events 1 • Up to 1 year
Infections and infestations
Neutropenic Fever
21.1%
4/19 • Number of events 4 • Up to 1 year
General disorders
Fever
5.3%
1/19 • Number of events 1 • Up to 1 year
Gastrointestinal disorders
Gastrointestinal disorders
5.3%
1/19 • Number of events 1 • Up to 1 year
Renal and urinary disorders
Hematuria
5.3%
1/19 • Number of events 1 • Up to 1 year
Metabolism and nutrition disorders
Hyponatremia
5.3%
1/19 • Number of events 1 • Up to 1 year
Vascular disorders
Hypotension
5.3%
1/19 • Number of events 1 • Up to 1 year
Infections and infestations
Infection
15.8%
3/19 • Number of events 6 • Up to 1 year
Infections and infestations
Lung Infection
31.6%
6/19 • Number of events 12 • Up to 1 year
Metabolism and nutrition disorders
Metabolism and Nutrition Disorder
5.3%
1/19 • Number of events 1 • Up to 1 year
General disorders
Pain
5.3%
1/19 • Number of events 1 • Up to 1 year
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
5.3%
1/19 • Number of events 2 • Up to 1 year
Infections and infestations
Pneumonitis
15.8%
3/19 • Number of events 3 • Up to 1 year
Infections and infestations
Sepsis
15.8%
3/19 • Number of events 4 • Up to 1 year
Infections and infestations
Skin Infection
5.3%
1/19 • Number of events 1 • Up to 1 year
Infections and infestations
Soft Tissue Infection
5.3%
1/19 • Number of events 1 • Up to 1 year
Nervous system disorders
Stroke
5.3%
1/19 • Number of events 1 • Up to 1 year
Nervous system disorders
Syncope
5.3%
1/19 • Number of events 1 • Up to 1 year

Other adverse events

Other adverse events
Measure
Treatment (Azacitidine, Avelumab)
n=19 participants at risk
Patients receive azacitidine SC or IV over 10-40 minutes on days 1-7 or on days 1-5 and 8-9. Patients also receive avelumab IV over 60 minutes on days 1 and 14 for 4 courses (or until complete response) and on day 1 for subsequent courses. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Avelumab: Given IV Azacitidine: Given SC or IV Laboratory Biomarker Analysis: Correlative studies
Investigations
Elevated Alanine Aminotransferase Increase
10.5%
2/19 • Number of events 2 • Up to 1 year
Infections and infestations
Alkaline Phosphatase Increased
5.3%
1/19 • Number of events 1 • Up to 1 year
Blood and lymphatic system disorders
Anemia
10.5%
2/19 • Number of events 2 • Up to 1 year
Gastrointestinal disorders
Anorexia
15.8%
3/19 • Number of events 3 • Up to 1 year
Blood and lymphatic system disorders
Blood and Lymphatic System Disorders
5.3%
1/19 • Number of events 1 • Up to 1 year
Investigations
Blood Bilirubin Increased
5.3%
1/19 • Number of events 1 • Up to 1 year
Investigations
Cardiac Troponin I Increase
5.3%
1/19 • Number of events 1 • Up to 1 year
General disorders
Cbills
5.3%
1/19 • Number of events 1 • Up to 1 year
Psychiatric disorders
Confusion
15.8%
3/19 • Number of events 3 • Up to 1 year
Gastrointestinal disorders
Diarrhea
31.6%
6/19 • Number of events 6 • Up to 1 year
General disorders
Fatigue
21.1%
4/19 • Number of events 4 • Up to 1 year
Musculoskeletal and connective tissue disorders
Generalized Muscle Weakness
10.5%
2/19 • Number of events 2 • Up to 1 year
Nervous system disorders
Headache
5.3%
1/19 • Number of events 1 • Up to 1 year
Metabolism and nutrition disorders
Hyperglycemia
5.3%
1/19 • Number of events 1 • Up to 1 year
Vascular disorders
Hypertension
10.5%
2/19 • Number of events 2 • Up to 1 year
Metabolism and nutrition disorders
Hypoalbuminemia
5.3%
1/19 • Number of events 1 • Up to 1 year
Metabolism and nutrition disorders
hyponatremia
5.3%
1/19 • Number of events 1 • Up to 1 year
Endocrine disorders
Hypothyroidism
5.3%
1/19 • Number of events 1 • Up to 1 year
General disorders
Infusion Related Reaction
21.1%
4/19 • Number of events 5 • Up to 1 year
Investigations
Lymphocyte Count Decreased
10.5%
2/19 • Number of events 2 • Up to 1 year
Infections and infestations
Mucosal Infection
5.3%
1/19 • Number of events 1 • Up to 1 year
Gastrointestinal disorders
Oral Mucositis
21.1%
4/19 • Number of events 4 • Up to 1 year
Gastrointestinal disorders
Nausea
31.6%
6/19 • Number of events 6 • Up to 1 year
Investigations
Neutropenia
10.5%
2/19 • Number of events 2 • Up to 1 year
Blood and lymphatic system disorders
Neutropenic Fever
5.3%
1/19 • Number of events 1 • Up to 1 year
Investigations
Platelet Count Decrease
5.3%
1/19 • Number of events 1 • Up to 1 year
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
10.5%
2/19 • Number of events 2 • Up to 1 year
Skin and subcutaneous tissue disorders
Rash
10.5%
2/19 • Number of events 2 • Up to 1 year
Gastrointestinal disorders
Vomiting
36.8%
7/19 • Number of events 8 • Up to 1 year
Investigations
White Blood Count Decreased
10.5%
2/19 • Number of events 3 • Up to 1 year

Additional Information

Naval Daver MD./Associate Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-794-4392

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place