Trial Outcomes & Findings for Donor Stem Cell Transplant in Treating Young Patients With Acute Myeloid Leukemia With Monosomy 7, -5/5q-, High FLT3-ITD AR, or Refractory or Relapsed Acute Myelogenous Leukemia (NCT NCT00553202)
NCT ID: NCT00553202
Last Updated: 2020-04-16
Results Overview
OS - Time from HSCT until death
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
158 participants
Primary outcome timeframe
At 5 years from HSCT date
Results posted on
2020-04-16
Participant Flow
Participant milestones
| Measure |
Treatment (Chemotherapy and Allogeneic SCT)
All patients
|
|---|---|
|
Overall Study
STARTED
|
158
|
|
Overall Study
COMPLETED
|
90
|
|
Overall Study
NOT COMPLETED
|
68
|
Reasons for withdrawal
| Measure |
Treatment (Chemotherapy and Allogeneic SCT)
All patients
|
|---|---|
|
Overall Study
Death
|
16
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Physician Decision
|
28
|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
No donor identified
|
16
|
|
Overall Study
Fails to meet organ function
|
6
|
Baseline Characteristics
Donor Stem Cell Transplant in Treating Young Patients With Acute Myeloid Leukemia With Monosomy 7, -5/5q-, High FLT3-ITD AR, or Refractory or Relapsed Acute Myelogenous Leukemia
Baseline characteristics by cohort
| Measure |
Treatment (Chemotherapy and Allogeneic SCT)
n=158 Participants
All Patients
|
|---|---|
|
Age, Categorical
<=18 years
|
149 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
3215.34 Days
STANDARD_DEVIATION 2240.48 • n=5 Participants
|
|
Sex: Female, Male
Female
|
75 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
83 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 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
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
128 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
15 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
11 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
147 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At 5 years from HSCT datePopulation: Patients without completion of planned therapy (n=68) are excluded from analyses of OS
OS - Time from HSCT until death
Outcome measures
| Measure |
Treatment (Chemotherapy and Allogeneic SCT)
n=90 Participants
All Patients
|
|---|---|
|
Overall Survival (OS)
|
45.9 Percentage of participants
Interval 33.7 to 57.2
|
PRIMARY outcome
Timeframe: At 5 years from HSCT datePopulation: Patients without completion of planned therapy (n=68) or without NK cell status (n=38) are excluded from analyses of TExp
Cumulative incidence of successful reconstitution to donor level is calculated.
Outcome measures
| Measure |
Treatment (Chemotherapy and Allogeneic SCT)
n=52 Participants
All Patients
|
|---|---|
|
Cumulative Incidence of NK Cell Reconstitution
|
48.1 Percentage of participants
Interval 33.8 to 61.0
|
OTHER_PRE_SPECIFIED outcome
Timeframe: From the date of SCT to the date of relapse, the date of death, or the date of last follow-up, whichever occurs firstThe cumulative incidence of relapse or death after SCT will be calculated by considering relapse and death due to other causes as competing events.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 5 yearsAcute and chronic GVHD will be summarized.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 42 days after SCTThe presence of donor cells is demonstrated by the detection of informative variable-number tandem-repeat polymorphisms or by fluorescent in situ hybridization with a Y-chromosome-specific probe in cases of sex-mismatched transplants. Independent variables that will be examined include donor-recipient KIR mismatch, taking into consideration the interactions with donor-recipient human leukocyte antigen (HLA) compatibility, and the numbers of CD34+ cells and CD3+ cells in the graft.
Outcome measures
Outcome data not reported
Adverse Events
Group 1
Serious events: 7 serious events
Other events: 32 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Group 1
n=90 participants at risk
All patients
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Adult respiratory distress syndrome
|
1.1%
1/90
|
|
Investigations
Alanine aminotransferase increased
|
1.1%
1/90
|
|
Gastrointestinal disorders
Ascites
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
1.1%
1/90
|
|
Investigations
Blood bilirubin increased
|
1.1%
1/90
|
|
Investigations
Creatinine increased
|
1.1%
1/90
|
|
General disorders
Death NOS
|
2.2%
2/90
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other, specify
|
1.1%
1/90
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
1.1%
1/90
|
|
Immune system disorders
Immune system disorders - Other, specify
|
1.1%
1/90
|
|
Infections and infestations
Infections and infestations - Other, specify
|
2.2%
2/90
|
|
Gastrointestinal disorders
Mucositis oral
|
1.1%
1/90
|
|
General disorders
Multi-organ failure
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
1.1%
1/90
|
|
Hepatobiliary disorders
Portal hypertension
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
1.1%
1/90
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, specify
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
3.3%
3/90
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
1.1%
1/90
|
|
Infections and infestations
Sepsis
|
2.2%
2/90
|
Other adverse events
| Measure |
Group 1
n=90 participants at risk
All patients
|
|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
1.1%
1/90
|
|
Gastrointestinal disorders
Abdominal pain
|
1.1%
1/90
|
|
Renal and urinary disorders
Acute kidney injury
|
5.6%
5/90
|
|
Respiratory, thoracic and mediastinal disorders
Adult respiratory distress syndrome
|
1.1%
1/90
|
|
Immune system disorders
Allergic reaction
|
1.1%
1/90
|
|
Gastrointestinal disorders
Ascites
|
8.9%
8/90
|
|
Investigations
Aspartate aminotransferase increased
|
2.2%
2/90
|
|
Renal and urinary disorders
Bladder spasm
|
1.1%
1/90
|
|
Investigations
Blood bilirubin increased
|
12.2%
11/90
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
|
1.1%
1/90
|
|
Cardiac disorders
Cardiac disorders - Other, specify
|
2.2%
2/90
|
|
Investigations
Creatinine increased
|
1.1%
1/90
|
|
Renal and urinary disorders
Cystitis noninfective
|
3.3%
3/90
|
|
Immune system disorders
Cytokine release syndrome
|
1.1%
1/90
|
|
General disorders
Death NOS
|
1.1%
1/90
|
|
Gastrointestinal disorders
Diarrhea
|
3.3%
3/90
|
|
Investigations
Electrocardiogram QT corrected interval prolonged
|
5.6%
5/90
|
|
Infections and infestations
Encephalitis infection
|
1.1%
1/90
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
1.1%
1/90
|
|
Gastrointestinal disorders
Gastric hemorrhage
|
1.1%
1/90
|
|
Cardiac disorders
Heart failure
|
1.1%
1/90
|
|
Renal and urinary disorders
Hematuria
|
2.2%
2/90
|
|
Hepatobiliary disorders
Hepatic pain
|
4.4%
4/90
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other, specify
|
2.2%
2/90
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
3.3%
3/90
|
|
Metabolism and nutrition disorders
Hypernatremia
|
2.2%
2/90
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
2.2%
2/90
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
2.2%
2/90
|
|
Metabolism and nutrition disorders
Hypokalemia
|
4.4%
4/90
|
|
Metabolism and nutrition disorders
Hyponatremia
|
1.1%
1/90
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
1.1%
1/90
|
|
Vascular disorders
Hypotension
|
2.2%
2/90
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
3.3%
3/90
|
|
Gastrointestinal disorders
Ileus
|
1.1%
1/90
|
|
Infections and infestations
Infections and infestations - Other, specify
|
3.3%
3/90
|
|
Cardiac disorders
Left ventricular systolic dysfunction
|
4.4%
4/90
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
1.1%
1/90
|
|
Infections and infestations
Lung infection
|
1.1%
1/90
|
|
Gastrointestinal disorders
Mucositis oral
|
2.2%
2/90
|
|
General disorders
Multi-organ failure
|
2.2%
2/90
|
|
General disorders
Pain
|
1.1%
1/90
|
|
Cardiac disorders
Pericardial effusion
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal hemorrhage
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
1.1%
1/90
|
|
Hepatobiliary disorders
Portal hypertension
|
3.3%
3/90
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
2.2%
2/90
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
1.1%
1/90
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
5.6%
5/90
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
2.2%
2/90
|
|
Nervous system disorders
Seizure
|
1.1%
1/90
|
|
Infections and infestations
Sepsis
|
2.2%
2/90
|
|
Investigations
Serum amylase increased
|
1.1%
1/90
|
|
Cardiac disorders
Sinus tachycardia
|
1.1%
1/90
|
|
Infections and infestations
Sinusitis
|
1.1%
1/90
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
1.1%
1/90
|
|
Skin and subcutaneous tissue disorders
Stevens-Johnson syndrome
|
1.1%
1/90
|
|
Metabolism and nutrition disorders
Tumor lysis syndrome
|
1.1%
1/90
|
|
Gastrointestinal disorders
Upper gastrointestinal hemorrhage
|
1.1%
1/90
|
|
Infections and infestations
Urinary tract infection
|
1.1%
1/90
|
|
Investigations
Urine output decreased
|
2.2%
2/90
|
|
Reproductive system and breast disorders
Vaginal hemorrhage
|
1.1%
1/90
|
|
Vascular disorders
Vascular disorders - Other, specify
|
2.2%
2/90
|
|
Investigations
Weight gain
|
8.9%
8/90
|
|
Investigations
White blood cell decreased
|
1.1%
1/90
|
Additional Information
Results Reporting Coordinator
Children's Oncology Group
Phone: 626-447-0064
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Must obtain prior Sponsor approval.
- Publication restrictions are in place
Restriction type: OTHER