Trial Outcomes & Findings for Relapse Prophylaxis With N-803 for AML and MDS Pts Following Allo HSCT (NCT NCT02989844)
NCT ID: NCT02989844
Last Updated: 2023-11-03
Results Overview
Efficacy of N-803 as measured by the cumulative incidence of relapse between the 1st dose of N-803 and 2 years after a reduced intensity conditioning (RIC) allogeneic hematopoietic cell transplant (alloHCT)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
24 months
Results posted on
2023-11-03
Participant Flow
Participant milestones
| Measure |
N-803
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
20
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Relapse Prophylaxis With N-803 for AML and MDS Pts Following Allo HSCT
Baseline characteristics by cohort
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
13 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
7 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
19 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 24 monthsEfficacy of N-803 as measured by the cumulative incidence of relapse between the 1st dose of N-803 and 2 years after a reduced intensity conditioning (RIC) allogeneic hematopoietic cell transplant (alloHCT)
Outcome measures
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Incidence of Relapse
|
30 Percentage of participants
Interval 12.0 to 51.0
|
SECONDARY outcome
Timeframe: 12 monthsFrequency of all adverse
Outcome measures
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Incidence of Adverse Events
|
567 Count of events
|
SECONDARY outcome
Timeframe: Day 100Incidence of grade 2-4 and grade 3-4 acute graft-versus-host-disease (GVHD)
Outcome measures
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Incidence of Acute Graft-versus-host Disease
Grade 2-4 GVHD
|
10 Percentage of participants
Interval 2.0 to 28.0
|
|
Incidence of Acute Graft-versus-host Disease
Grade 3-4 GVHD
|
0 Percentage of participants
Interval 0.0 to 100.0
|
SECONDARY outcome
Timeframe: Day 180Incidence of grade 2-4 and grade 3-4 acute graft-versus-host-disease (GVHD)
Outcome measures
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Incidence of Acute Graft-versus-host Disease
Grade 2-4 GVHD
|
15 Percentage of participants
Interval 4.0 to 34.0
|
|
Incidence of Acute Graft-versus-host Disease
Grade 3-4 GVHD
|
5 Percentage of participants
Interval 0.0 to 21.0
|
SECONDARY outcome
Timeframe: 1 yearIncidence of acute graft-versus-host disease
Outcome measures
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Chronic GVHD
|
15 Percentage of participants
Interval 4.0 to 34.0
|
SECONDARY outcome
Timeframe: 1 yearIncidence of minimal residual disease (MRD) post-transplant
Outcome measures
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Minimal Residual Disease (MRD)
|
26 Percentage of participants
Interval 12.0 to 49.0
|
SECONDARY outcome
Timeframe: 1 year post transplantIncidence of overall survival at one year
Outcome measures
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Overall Survival
|
85 Percentage of participants
Interval 71.0 to 99.0
|
SECONDARY outcome
Timeframe: 1 yearIncidence of non-relapse mortality
Outcome measures
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Non-Relapse Mortality
|
5 Percentage of participants
Interval 0.0 to 21.0
|
SECONDARY outcome
Timeframe: 2 YearsPopulation: Intent is to compare the percentage of participants with relapse stratified according the number of doses of N-803 that were administered.
Incidence of relapse at 2 years after alloHCT stratified by number of doses of N-803 (1-3 or 4-10)
Outcome measures
| Measure |
N-803
n=20 Participants
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Relapse
1-3 doses of N-803
|
62 Percentage of participants
Interval 18.0 to 88.0
|
|
Relapse
4-10 doses of N-803
|
17 Percentage of participants
Interval 2.0 to 43.0
|
Adverse Events
N-803
Serious events: 7 serious events
Other events: 20 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
N-803
n=20 participants at risk
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Cardiac disorders
Atrial fibrillation
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Infections and infestations
Infections and infestations - Other,
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Immune system disorders
Immune system disorders - Other,
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Infections and infestations
Sepsis
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Injury, poisoning and procedural complications
Fall
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Infections and infestations
Lung infection
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Cardiac disorders
Pericardial effusion
|
5.0%
1/20 • Number of events 1 • 12 months
|
Other adverse events
| Measure |
N-803
n=20 participants at risk
N-803: N-803 at 6 mcg/kg SQ Day 1 of a 4 week (28 day) cycle with ± 1 week window Continue N-803 every 4 weeks for 10 doses or until relapse, unacceptable toxicity, or patient refusal, whichever comes earlier.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
100.0%
20/20 • Number of events 22 • 12 months
|
|
Cardiac disorders
Atrial Fibrillation
|
10.0%
2/20 • Number of events 2 • 12 months
|
|
Cardiac disorders
Pericardial effusion
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Eye disorders
Blurred Vision
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Eye disorders
Dry eye
|
10.0%
2/20 • Number of events 2 • 12 months
|
|
Ear and labyrinth disorders
Ear pain
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Abdominal pain
|
10.0%
2/20 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Diarrhea
|
25.0%
5/20 • Number of events 5 • 12 months
|
|
Gastrointestinal disorders
Dry mouth
|
10.0%
2/20 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Dysphagia
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Mucositis oral
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Nausea
|
35.0%
7/20 • Number of events 7 • 12 months
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
4/20 • Number of events 4 • 12 months
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Stomach pain
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
General disorders
Chills
|
85.0%
17/20 • Number of events 17 • 12 months
|
|
General disorders
Edema limbs
|
40.0%
8/20 • Number of events 8 • 12 months
|
|
General disorders
Fatigue
|
60.0%
12/20 • Number of events 12 • 12 months
|
|
General disorders
Fever
|
40.0%
8/20 • Number of events 8 • 12 months
|
|
General disorders
Injection site reaction
|
100.0%
20/20 • Number of events 48 • 12 months
|
|
General disorders
Gait disturbance
|
20.0%
4/20 • Number of events 4 • 12 months
|
|
General disorders
Pain
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
General disorders
Flu like symptoms
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
10.0%
2/20 • Number of events 2 • 12 months
|
|
Immune system disorders
Allergic reaction
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Infections and infestations
Infections and infestations - Other, specify
|
15.0%
3/20 • Number of events 3 • 12 months
|
|
Infections and infestations
Lung infection
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Infections and infestations
Paronychia
|
15.0%
3/20 • Number of events 3 • 12 months
|
|
Infections and infestations
Skin infection
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Infections and infestations
Upper respiratory infection
|
35.0%
7/20 • Number of events 7 • 12 months
|
|
Infections and infestations
Vulval infection
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Injury, poisoning and procedural complications
Fall
|
20.0%
4/20 • Number of events 4 • 12 months
|
|
Investigations
Alanine aminotransferase increased
|
25.0%
5/20 • Number of events 5 • 12 months
|
|
Investigations
Alkaline phosphatase increased
|
10.0%
2/20 • Number of events 2 • 12 months
|
|
Investigations
Aspartate aminotransferase increased
|
50.0%
10/20 • Number of events 10 • 12 months
|
|
Investigations
Blood bilirubin increased
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Investigations
Creatinine increased
|
40.0%
8/20 • Number of events 8 • 12 months
|
|
Investigations
Lymphocyte count decreased
|
25.0%
5/20 • Number of events 5 • 12 months
|
|
Investigations
Neutrophil count decreased
|
100.0%
20/20 • Number of events 21 • 12 months
|
|
Investigations
Platelet count decreased
|
100.0%
20/20 • Number of events 46 • 12 months
|
|
Investigations
Weight loss
|
10.0%
2/20 • Number of events 2 • 12 months
|
|
Investigations
White blood cell decreased
|
100.0%
20/20 • Number of events 39 • 12 months
|
|
Metabolism and nutrition disorders
Anorexia
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
75.0%
15/20 • Number of events 15 • 12 months
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
20.0%
4/20 • Number of events 4 • 12 months
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
20.0%
4/20 • Number of events 4 • 12 months
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
30.0%
6/20 • Number of events 6 • 12 months
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
10.0%
2/20 • Number of events 2 • 12 months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
25.0%
5/20 • Number of events 5 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, specify
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Nervous system disorders
Dizziness
|
15.0%
3/20 • Number of events 3 • 12 months
|
|
Nervous system disorders
Headache
|
45.0%
9/20 • Number of events 9 • 12 months
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Nervous system disorders
Vasovagal reaction
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Psychiatric disorders
Insomnia
|
10.0%
2/20 • Number of events 2 • 12 months
|
|
Renal and urinary disorders
Acute kidney injury
|
20.0%
4/20 • Number of events 4 • 12 months
|
|
Reproductive system and breast disorders
Erectile dysfunction
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Reproductive system and breast disorders
Breast pain
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial obstruction
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
4/20 • Number of events 4 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
75.0%
15/20 • Number of events 15 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
5.0%
1/20 • Number of events 1 • 12 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
50.0%
10/20 • Number of events 10 • 12 months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
40.0%
8/20 • Number of events 8 • 12 months
|
|
Vascular disorders
Hypertension
|
100.0%
20/20 • Number of events 76 • 12 months
|
|
Vascular disorders
Hypotension
|
30.0%
6/20 • Number of events 6 • 12 months
|
Additional Information
Jeffrey Miller
University of Minnesota, Masonic Cancer Center
Phone: 612-625-7409
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place