Trial Outcomes & Findings for Testing Nivolumab to Prevent Disease From Coming Back After Treatment in Patients With Acute Myeloid Leukemia, REMAIN Trial (NCT NCT02275533)

NCT ID: NCT02275533

Last Updated: 2025-12-08

Results Overview

Time from randomization to disease relapse or death from any cause

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

82 participants

Primary outcome timeframe

Assessed up to 5 years

Results posted on

2025-12-08

Participant Flow

Participant milestones

Participant milestones
Measure
Arm I (Nivolumab)
Patients receive nivolumab IV over 60 minutes once every 2 weeks. Treatment repeats every 2 weeks for 46 cycles in the absence of disease progression or unacceptable toxicity.
Arm II (Observation)
Patients undergo standard of care clinical observation for up to 2 years. Upon disease relapse, patients may cross-over to Arm I.
Overall Study
STARTED
41
41
Overall Study
COMPLETED
38
41
Overall Study
NOT COMPLETED
3
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Arm I (Nivolumab)
Patients receive nivolumab IV over 60 minutes once every 2 weeks. Treatment repeats every 2 weeks for 46 cycles in the absence of disease progression or unacceptable toxicity.
Arm II (Observation)
Patients undergo standard of care clinical observation for up to 2 years. Upon disease relapse, patients may cross-over to Arm I.
Overall Study
Protocol Violation
2
0
Overall Study
Never received study treatment
1
0

Baseline Characteristics

Testing Nivolumab to Prevent Disease From Coming Back After Treatment in Patients With Acute Myeloid Leukemia, REMAIN Trial

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Arm I (Nivolumab)
n=38 Participants
Patients receive nivolumab IV over 60 minutes once every 2 weeks. Treatment repeats every 2 weeks for 46 cycles in the absence of disease progression or unacceptable toxicity.
Arm II (Observation)
n=41 Participants
Patients undergo standard of care clinical observation for up to 2 years. Upon disease relapse, patients may cross-over to Arm I.
Total
n=79 Participants
Total of all reporting groups
Age, Continuous
63.2 years
n=37 Participants
65.5 years
n=37 Participants
64.4 years
n=74 Participants
Sex: Female, Male
Female
18 Participants
n=37 Participants
19 Participants
n=37 Participants
37 Participants
n=74 Participants
Sex: Female, Male
Male
20 Participants
n=37 Participants
22 Participants
n=37 Participants
42 Participants
n=74 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=37 Participants
0 Participants
n=37 Participants
0 Participants
n=74 Participants
Race (NIH/OMB)
Asian
2 Participants
n=37 Participants
0 Participants
n=37 Participants
2 Participants
n=74 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=37 Participants
0 Participants
n=37 Participants
0 Participants
n=74 Participants
Race (NIH/OMB)
Black or African American
2 Participants
n=37 Participants
5 Participants
n=37 Participants
7 Participants
n=74 Participants
Race (NIH/OMB)
White
32 Participants
n=37 Participants
32 Participants
n=37 Participants
64 Participants
n=74 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=37 Participants
0 Participants
n=37 Participants
0 Participants
n=74 Participants
Race (NIH/OMB)
Unknown or Not Reported
2 Participants
n=37 Participants
4 Participants
n=37 Participants
6 Participants
n=74 Participants
Region of Enrollment
United States
38 participants
n=37 Participants
41 participants
n=37 Participants
79 participants
n=74 Participants

PRIMARY outcome

Timeframe: Assessed up to 5 years

Time from randomization to disease relapse or death from any cause

Outcome measures

Outcome measures
Measure
Arm I (Nivolumab)
n=38 Participants
Patients receive nivolumab IV over 60 minutes once every 2 weeks. Treatment repeats every 2 weeks for 46 cycles in the absence of disease progression or unacceptable toxicity.
Arm II (Observation)
n=41 Participants
Patients undergo standard of care clinical observation for up to 2 years. Upon disease relapse, patients may cross-over to Arm I.
Progression Free Survival (PFS)
13.2 months
Interval 8.5 to 21.8
10.9 months
Interval 5.4 to 14.9

SECONDARY outcome

Timeframe: Assessed up to 5 years

Time from randomization to the date of death from any cause

Outcome measures

Outcome measures
Measure
Arm I (Nivolumab)
n=38 Participants
Patients receive nivolumab IV over 60 minutes once every 2 weeks. Treatment repeats every 2 weeks for 46 cycles in the absence of disease progression or unacceptable toxicity.
Arm II (Observation)
n=41 Participants
Patients undergo standard of care clinical observation for up to 2 years. Upon disease relapse, patients may cross-over to Arm I.
Overall Survival (OS)
53.9 Months
Interval 23.4 to
NA = Upper confidence limit cannot be estimated due to insufficient number of participants with events
30.9 Months
Interval 14.4 to
NA = Upper confidence limit cannot be estimated due to insufficient number of participants with events

SECONDARY outcome

Timeframe: Assessed up to 5 years

Patient death due to reasons other than relapse

Outcome measures

Outcome measures
Measure
Arm I (Nivolumab)
n=38 Participants
Patients receive nivolumab IV over 60 minutes once every 2 weeks. Treatment repeats every 2 weeks for 46 cycles in the absence of disease progression or unacceptable toxicity.
Arm II (Observation)
n=41 Participants
Patients undergo standard of care clinical observation for up to 2 years. Upon disease relapse, patients may cross-over to Arm I.
Non-relapse Mortality (NRM)
2 Participants
1 Participants

SECONDARY outcome

Timeframe: Up to 5 years

Will be assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Grade 3 or higher regardless of attribution.

Outcome measures

Outcome measures
Measure
Arm I (Nivolumab)
n=38 Participants
Patients receive nivolumab IV over 60 minutes once every 2 weeks. Treatment repeats every 2 weeks for 46 cycles in the absence of disease progression or unacceptable toxicity.
Arm II (Observation)
n=41 Participants
Patients undergo standard of care clinical observation for up to 2 years. Upon disease relapse, patients may cross-over to Arm I.
Incidence of Adverse Effects of Nivolumab
27 Participants
5 Participants

Adverse Events

Arm I (Nivolumab)

Serious events: 8 serious events
Other events: 37 other events
Deaths: 16 deaths

Arm II (Observation)

Serious events: 3 serious events
Other events: 8 other events
Deaths: 20 deaths

Serious adverse events

Serious adverse events
Measure
Arm I (Nivolumab)
n=38 participants at risk
Patients receive nivolumab IV over 60 minutes once every 2 weeks. Treatment repeats every 2 weeks for 46 cycles in the absence of disease progression or unacceptable toxicity.
Arm II (Observation)
n=41 participants at risk
Patients undergo standard of care clinical observation for up to 2 years. Upon disease relapse, patients may cross-over to Arm I.
Investigations
Alanine aminotransferase (ALT) increased
2.6%
1/38 • 5 years
0.00%
0/41 • 5 years
Investigations
Aspartate aminotransferase increase
2.6%
1/38 • 5 years
0.00%
0/41 • 5 years
Psychiatric disorders
Confusion
2.6%
1/38 • 5 years
0.00%
0/41 • 5 years
Infections and infestations
Lung infection
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Gastrointestinal disorders
Diverticulitis
2.6%
1/38 • 5 years
0.00%
0/41 • 5 years
Investigations
Neutrophil count decreased
2.6%
1/38 • 5 years
2.4%
1/41 • 5 years
Investigations
Platelet count decreased
2.6%
1/38 • 5 years
0.00%
0/41 • 5 years
Cardiac disorders
Sinus tachycardia
2.6%
1/38 • 5 years
0.00%
0/41 • 5 years
Gastrointestinal disorders
Upper gastrointestinal hemorrhage
2.6%
1/38 • 5 years
0.00%
0/41 • 5 years
Blood and lymphatic system disorders
Anemia
0.00%
0/38 • 5 years
2.4%
1/41 • 5 years
Gastrointestinal disorders
Colitis
0.00%
0/38 • 5 years
2.4%
1/41 • 5 years
General disorders
Fatigue
0.00%
0/38 • 5 years
2.4%
1/41 • 5 years
General disorders
Malaise
0.00%
0/38 • 5 years
2.4%
1/41 • 5 years
Infections and infestations
Sepsis
0.00%
0/38 • 5 years
2.4%
1/41 • 5 years

Other adverse events

Other adverse events
Measure
Arm I (Nivolumab)
n=38 participants at risk
Patients receive nivolumab IV over 60 minutes once every 2 weeks. Treatment repeats every 2 weeks for 46 cycles in the absence of disease progression or unacceptable toxicity.
Arm II (Observation)
n=41 participants at risk
Patients undergo standard of care clinical observation for up to 2 years. Upon disease relapse, patients may cross-over to Arm I.
Gastrointestinal disorders
Gastroesohpageal reflux
5.3%
2/38 • 5 years
2.4%
1/41 • 5 years
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
5.3%
2/38 • 5 years
2.4%
1/41 • 5 years
Nervous system disorders
Headache
21.1%
8/38 • 5 years
2.4%
1/41 • 5 years
Renal and urinary disorders
Hematuria
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Metabolism and nutrition disorders
Hypercalcemia
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Metabolism and nutrition disorders
Hyperglycemia
31.6%
12/38 • 5 years
2.4%
1/41 • 5 years
Metabolism and nutrition disorders
Hyperkalemia
7.9%
3/38 • 5 years
2.4%
1/41 • 5 years
Vascular disorders
Hypertension
36.8%
14/38 • 5 years
0.00%
0/41 • 5 years
Endocrine disorders
Hyperthyroidism
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Metabolism and nutrition disorders
Hyperuricemia
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Metabolism and nutrition disorders
Hypoalbuminemia
13.2%
5/38 • 5 years
2.4%
1/41 • 5 years
Metabolism and nutrition disorders
Hypocalcemia
10.5%
4/38 • 5 years
4.9%
2/41 • 5 years
Metabolism and nutrition disorders
Hypokalemia
5.3%
2/38 • 5 years
4.9%
2/41 • 5 years
Metabolism and nutrition disorders
Hypomagnesemia
10.5%
4/38 • 5 years
2.4%
1/41 • 5 years
Metabolism and nutrition disorders
Hyponatremia
7.9%
3/38 • 5 years
4.9%
2/41 • 5 years
Metabolism and nutrition disorders
Hypophosphatemia
18.4%
7/38 • 5 years
7.3%
3/41 • 5 years
Endocrine disorders
Hypothyroidism
18.4%
7/38 • 5 years
0.00%
0/41 • 5 years
General disorders
Infusion related reaction
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Psychiatric disorders
Insomnia
7.9%
3/38 • 5 years
4.9%
2/41 • 5 years
Respiratory, thoracic and mediastinal disorders
Laryngeal inflammation
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Investigations
Lipase increased
13.2%
5/38 • 5 years
0.00%
0/41 • 5 years
Investigations
Lymphocyte count decreased
21.1%
8/38 • 5 years
12.2%
5/41 • 5 years
Gastrointestinal disorders
Mucositis oral
10.5%
4/38 • 5 years
2.4%
1/41 • 5 years
Musculoskeletal and connective tissue disorders
Myalgia
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Respiratory, thoracic and mediastinal disorders
Nasal congestion
13.2%
5/38 • 5 years
0.00%
0/41 • 5 years
Gastrointestinal disorders
Nausea
28.9%
11/38 • 5 years
0.00%
0/41 • 5 years
Investigations
Neutrophil count decreased
31.6%
12/38 • 5 years
9.8%
4/41 • 5 years
General disorders
Pain
13.2%
5/38 • 5 years
0.00%
0/41 • 5 years
Musculoskeletal and connective tissue disorders
Pain in extremity
10.5%
4/38 • 5 years
2.4%
1/41 • 5 years
Nervous system disorders
Paresthesia
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Nervous system disorders
Peripheral sensory neuropathy
7.9%
3/38 • 5 years
4.9%
2/41 • 5 years
Investigations
Platelet count decreased
36.8%
14/38 • 5 years
17.1%
7/41 • 5 years
Respiratory, thoracic and mediastinal disorders
Productive cough
13.2%
5/38 • 5 years
0.00%
0/41 • 5 years
Skin and subcutaneous tissue disorders
Pruritis
18.4%
7/38 • 5 years
4.9%
2/41 • 5 years
Skin and subcutaneous tissue disorders
Rash maculo-papular
23.7%
9/38 • 5 years
4.9%
2/41 • 5 years
Investigations
Serum amylase increased
10.5%
4/38 • 5 years
0.00%
0/41 • 5 years
Gastrointestinal disorders
Toothache
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Infections and infestations
Upper respiratory infection
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Gastrointestinal disorders
Vomiting
13.2%
5/38 • 5 years
0.00%
0/41 • 5 years
Investigations
Weight gain
13.2%
5/38 • 5 years
0.00%
0/41 • 5 years
Investigations
White blood cell decreased
31.6%
12/38 • 5 years
17.1%
7/41 • 5 years
Musculoskeletal and connective tissue disorders
Shoulder pain
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Musculoskeletal and connective tissue disorders
Arthralgia
10.5%
4/38 • 5 years
2.4%
1/41 • 5 years
Investigations
Aspartate aminotransferase increased
34.2%
13/38 • 5 years
2.4%
1/41 • 5 years
Musculoskeletal and connective tissue disorders
Back pain
13.2%
5/38 • 5 years
0.00%
0/41 • 5 years
Investigations
Blood bilirubin increased
5.3%
2/38 • 5 years
4.9%
2/41 • 5 years
Injury, poisoning and procedural complications
Bruising
10.5%
4/38 • 5 years
2.4%
1/41 • 5 years
General disorders
Chills
7.9%
3/38 • 5 years
2.4%
1/41 • 5 years
Gastrointestinal disorders
Constipation
13.2%
5/38 • 5 years
2.4%
1/41 • 5 years
Respiratory, thoracic and mediastinal disorders
Cough
21.1%
8/38 • 5 years
4.9%
2/41 • 5 years
Investigations
Creatinine increased
13.2%
5/38 • 5 years
2.4%
1/41 • 5 years
Gastrointestinal disorders
Diarrhea
34.2%
13/38 • 5 years
4.9%
2/41 • 5 years
Nervous system disorders
Dizziness
13.2%
5/38 • 5 years
0.00%
0/41 • 5 years
Gastrointestinal disorders
Dry mouth
10.5%
4/38 • 5 years
2.4%
1/41 • 5 years
Skin and subcutaneous tissue disorders
Dry skin
7.9%
3/38 • 5 years
0.00%
0/41 • 5 years
Gastrointestinal disorders
Dyspepsia
5.3%
2/38 • 5 years
2.4%
1/41 • 5 years
Respiratory, thoracic and mediastinal disorders
Dyspnea
7.9%
3/38 • 5 years
7.3%
3/41 • 5 years
Ear and labyrinth disorders
Ear pain
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
General disorders
Edema limbs
13.2%
5/38 • 5 years
2.4%
1/41 • 5 years
Injury, poisoning and procedural complications
Fall
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
General disorders
Fatigue
42.1%
16/38 • 5 years
7.3%
3/41 • 5 years
General disorders
Fever
7.9%
3/38 • 5 years
2.4%
1/41 • 5 years
General disorders
Flu like symptoms
13.2%
5/38 • 5 years
0.00%
0/41 • 5 years
Vascular disorders
Flushing
7.9%
3/38 • 5 years
0.00%
0/41 • 5 years
Psychiatric disorders
Anxiety
7.9%
3/38 • 5 years
0.00%
0/41 • 5 years
Gastrointestinal disorders
Abdominal pain
13.2%
5/38 • 5 years
2.4%
1/41 • 5 years
Investigations
Activated partial thromboplastin time prolonged
5.3%
2/38 • 5 years
2.4%
1/41 • 5 years
Investigations
Alanine aminotransferase increased
28.9%
11/38 • 5 years
2.4%
1/41 • 5 years
Investigations
Alkaline phosphatase increased
13.2%
5/38 • 5 years
2.4%
1/41 • 5 years
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
5.3%
2/38 • 5 years
0.00%
0/41 • 5 years
Blood and lymphatic system disorders
Anemia
36.8%
14/38 • 5 years
9.8%
4/41 • 5 years
Metabolism and nutrition disorders
Anorexia
5.3%
2/38 • 5 years
4.9%
2/41 • 5 years

Additional Information

Theodore Karrison

University of Chicago

Phone: 708-925-6771

Results disclosure agreements

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

Restriction type: LTE60