Trial Outcomes & Findings for Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay (NCT NCT01872819)
NCT ID: NCT01872819
Last Updated: 2018-07-11
Results Overview
Whether treatment was administered in the time frame based on the high throughput drug screen. Time from sample procurement to assay results.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
16 participants
Primary outcome timeframe
Up to 21 days
Results posted on
2018-07-11
Participant Flow
Participant milestones
| Measure |
Treatment (Chemotherapy, Biological Therapy)
Patients receive 1 of 160 possible interventions based on high throughput drug sensitivity assay.
antitumor drug screening assay: Undergo high throughput drug sensitivity assay
chemotherapy: Patients receive 1 of 160 possible interventions
biological therapy: Patients receive 1 of 160 possible interventions
16 patients were enrolled. 14 patients were treated.
|
|---|---|
|
Overall Study
STARTED
|
14
|
|
Overall Study
COMPLETED
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay
Baseline characteristics by cohort
| Measure |
Treatment (Chemotherapy, Biological Therapy)
n=16 Participants
Patients receive 1 of 160 possible interventions based on high throughput drug sensitivity assay.
antitumor drug screening assay: Undergo high throughput drug sensitivity assay
chemotherapy: Patients receive 1 of 160 possible interventions
biological therapy: Patients receive 1 of 160 possible interventions
|
|---|---|
|
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
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 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
|
2 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
|
14 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
|
|
Region of Enrollment
United States
|
16 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 21 daysPopulation: 14 patients were treated.
Whether treatment was administered in the time frame based on the high throughput drug screen. Time from sample procurement to assay results.
Outcome measures
| Measure |
Treated Patients
n=14 Participants
14 patients were treated.
|
|---|---|
|
Achievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AML
|
5.1 days
Interval 4.0 to 8.0
|
SECONDARY outcome
Timeframe: Baseline up to 2 yearsPopulation: 14 patients received therapy. Out of 14 patients treated, 9 were evaluable (4 patients died prior to D14-21 marrow and 1 patient refused the D14-21 marrow).
Number of patients who achieved a Complete Response (CR) with Minimal Residual Disease (MRD), a Complete Response with incomplete hematologic recovery (CRi), or showed reduced blasts in their bone marrow by flow cytometry (Cytoreduction). Cheson et al. defines a CR as: Bone Marrow blasts \<5%, absence of circulating blasts and blasts with Auer rods, absence of extramedullary disease, absolute neutrophil count \>1.0 x 10\^9/L, and platelet count \>100 x 10\^9/L. Cheson et al. defines a CRi as: all CR criteria except for residual neutropenia (\<1.0 x 10\^9/L) or thrombocytopenia (\<100 x 10\^9/L).
Outcome measures
| Measure |
Treated Patients
n=9 Participants
14 patients were treated.
|
|---|---|
|
Rate of Complete Response, Defined by Criteria of Cheson et al.
CR with MRD
|
1 Patients
|
|
Rate of Complete Response, Defined by Criteria of Cheson et al.
CRi
|
2 Patients
|
|
Rate of Complete Response, Defined by Criteria of Cheson et al.
Cytoreduction
|
6 Patients
|
Adverse Events
Treatment (Chemotherapy, Biological Therapy)
Serious events: 11 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Chemotherapy, Biological Therapy)
n=14 participants at risk
Patients receive 1 of 160 possible interventions based on high throughput drug sensitivity assay.
antitumor drug screening assay: Undergo high throughput drug sensitivity assay
chemotherapy: Patients receive 1 of 160 possible interventions
biological therapy: Patients receive 1 of 160 possible interventions
|
|---|---|
|
Nervous system disorders
seizure
|
14.3%
2/14 • Number of events 2 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Infections and infestations
pneumonia
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Nervous system disorders
headache
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
malignant neoplasm progression
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Infections and infestations
febrile neutropenia
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Investigations
hypokalemia
|
14.3%
2/14 • Number of events 2 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Psychiatric disorders
confusion
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Investigations
hyperbilirubinemia
|
14.3%
2/14 • Number of events 2 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Investigations
Hypoalbuminemia
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Skin and subcutaneous tissue disorders
drug rash
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Metabolism and nutrition disorders
hypoalbuminemia
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Gastrointestinal disorders
mucositis
|
7.1%
1/14 • Number of events 2 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Metabolism and nutrition disorders
hypophosphatemia
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Investigations
Alanine aminotransferase increased
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Investigations
blood bilirubin increased
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Infections and infestations
bacteremia-GPC
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Infections and infestations
sepsis
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Vascular disorders
hypotension
|
14.3%
2/14 • Number of events 2 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Metabolism and nutrition disorders
hypokalemia
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Metabolism and nutrition disorders
tumor lysis syndrome
|
14.3%
2/14 • Number of events 2 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Respiratory, thoracic and mediastinal disorders
pleural effusions
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Gastrointestinal disorders
worsening abdominal pain
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Musculoskeletal and connective tissue disorders
severe bone pain
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Infections and infestations
c-diff colitis infection
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
General disorders
LE edema
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Respiratory, thoracic and mediastinal disorders
hypoxia
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Blood and lymphatic system disorders
coagulopathy
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Investigations
Elevated AST
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Blood and lymphatic system disorders
DIC
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Metabolism and nutrition disorders
uric acid
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Cardiac disorders
acute systolic heart failure
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Cardiac disorders
cardiogenic shock
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Respiratory, thoracic and mediastinal disorders
hypoxemic respiratory failure
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Vascular disorders
refractory hypotension
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
General disorders
refractory shock
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Cardiac disorders
systolic dysfunction
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Cardiac disorders
cardiomyopathy
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
General disorders
multiorgan failure
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Infections and infestations
Lung infection - pulmonary aspergillosis
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Infections and infestations
skin infection - R leg cellulitis w/ abscess
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Infections and infestations
Lung infection - Stenotrophomonas bacteremia
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
|
Infections and infestations
primary stenotrophomonas secondary AML
|
7.1%
1/14 • Number of events 1 • 2 years
Other \[Not Including Serious\] Adverse Events were not monitored/assessed.
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place