Trial Outcomes & Findings for Allogeneic Transplant in HIV Patients (BMT CTN 0903) (NCT NCT01410344)
NCT ID: NCT01410344
Last Updated: 2022-12-08
Results Overview
The events for non-relapse mortality are death due to any cause other than relapse of the underlying malignancy.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
Day 100, 1 Year, and 2 Years Post-transplant
Results posted on
2022-12-08
Participant Flow
Participant milestones
| Measure |
Allogeneic Transplant
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Participants with leukemia (AML or ALL)
Baseline characteristics by cohort
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|
|
Age, Continuous
|
47 years
n=17 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=17 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=17 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=17 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=17 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=17 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=17 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=17 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=17 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=17 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=17 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=17 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=17 Participants
|
|
Karnofsky Performance Score
100
|
4 Participants
n=17 Participants
|
|
Karnofsky Performance Score
90
|
9 Participants
n=17 Participants
|
|
Karnofsky Performance Score
80
|
3 Participants
n=17 Participants
|
|
Karnofsky Performance Score
70
|
1 Participants
n=17 Participants
|
|
Primary Malignancy
Acute Myeloid Leukemia (AML)
|
9 Participants
n=17 Participants
|
|
Primary Malignancy
Acute Lymphocytic Leukemia (ALL)
|
2 Participants
n=17 Participants
|
|
Primary Malignancy
Myelodysplastic Syndromes (MDS)
|
2 Participants
n=17 Participants
|
|
Primary Malignancy
Hodgkin's Lymphoma
|
1 Participants
n=17 Participants
|
|
Primary Malignancy
Non-Hodgkin's Lymphoma
|
3 Participants
n=17 Participants
|
|
Leukemia Stage
First Complete Remission
|
8 Participants
n=11 Participants • Participants with leukemia (AML or ALL)
|
|
Leukemia Stage
Second Complete Remission
|
3 Participants
n=11 Participants • Participants with leukemia (AML or ALL)
|
|
Lymphoma Stage
Complete Remission
|
3 Participants
n=4 Participants • Participants with lymphoma (Hodgkin's or non-Hodgkin's)
|
|
Lymphoma Stage
Partial Remission
|
1 Participants
n=4 Participants • Participants with lymphoma (Hodgkin's or non-Hodgkin's)
|
|
HIV Viral Load
Undetectable by Assay
|
15 Participants
n=17 Participants
|
|
HIV Viral Load
Detectable by Assay
|
2 Participants
n=17 Participants
|
|
Recipient Cytomegalovirus Status
Positive
|
12 Participants
n=17 Participants
|
|
Recipient Cytomegalovirus Status
Negative
|
5 Participants
n=17 Participants
|
|
Number of Regimens of Induction Chemotherapy
1
|
10 Participants
n=17 Participants
|
|
Number of Regimens of Induction Chemotherapy
2
|
6 Participants
n=17 Participants
|
|
Number of Regimens of Induction Chemotherapy
3
|
1 Participants
n=17 Participants
|
|
Number of Regimens of Salvage Chemotherapy
0
|
10 Participants
n=17 Participants
|
|
Number of Regimens of Salvage Chemotherapy
1
|
6 Participants
n=17 Participants
|
|
Number of Regimens of Salvage Chemotherapy
3
|
1 Participants
n=17 Participants
|
|
Donor Type
Matched Related
|
4 Participants
n=17 Participants
|
|
Donor Type
Matched Unrelated
|
9 Participants
n=17 Participants
|
|
Donor Type
Mismatched Related
|
3 Participants
n=17 Participants
|
|
Donor Type
Mismatched Unrelated
|
1 Participants
n=17 Participants
|
|
CD4 T-cell Count
|
224 cells/microliter
n=17 Participants
|
PRIMARY outcome
Timeframe: Day 100, 1 Year, and 2 Years Post-transplantThe events for non-relapse mortality are death due to any cause other than relapse of the underlying malignancy.
Outcome measures
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Percentage of Participants With Non-Relapse Mortality
Day 100
|
0.0 percentage of participants
Interval 0.0 to 0.0
|
—
|
|
Percentage of Participants With Non-Relapse Mortality
1 Year
|
11.8 percentage of participants
Interval 1.8 to 32.2
|
—
|
|
Percentage of Participants With Non-Relapse Mortality
2 Years
|
18.3 percentage of participants
Interval 4.1 to 40.7
|
—
|
SECONDARY outcome
Timeframe: Six months, 1 Year, and 2 Years Post-transplantOverall survival is defined as the time from transplant to death from any cause.
Outcome measures
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Percentage of Participants With Overall Survival
6 Months
|
82.4 percentage of participants
Interval 54.7 to 93.9
|
—
|
|
Percentage of Participants With Overall Survival
1 Year
|
58.8 percentage of participants
Interval 32.5 to 77.8
|
—
|
|
Percentage of Participants With Overall Survival
2 Years
|
50.2 percentage of participants
Interval 24.3 to 71.4
|
—
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantRelapse/Progression is defined as relapse or progression of the primary malignancy.
Outcome measures
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Percentage of Participants With Relapse/Progression
|
29.4 percentage of participants
Interval 10.2 to 51.9
|
—
|
SECONDARY outcome
Timeframe: Up to 2 Years Post-transplantOutcome measures
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Primary Cause of Death
Relapse/Progresion
|
5 Participants
|
—
|
|
Primary Cause of Death
Acute GVHD
|
1 Participants
|
—
|
|
Primary Cause of Death
Adult Respiratory Distress Syndrome
|
1 Participants
|
—
|
|
Primary Cause of Death
Liver Failure
|
1 Participants
|
—
|
|
Primary Cause of Death
Still Alive
|
9 Participants
|
—
|
SECONDARY outcome
Timeframe: Day 100 Post-transplantPatients will be assessed for disease status at Day 100 post-HCT, classified as complete remission, partial remission, stable disease, and relapse/progressive disease.
Outcome measures
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Disease Status
Complete remission
|
13 Participants
|
—
|
|
Disease Status
Relapse/progressive disease
|
4 Participants
|
—
|
SECONDARY outcome
Timeframe: Days 28 and 100 Post-transplantRecovery of hematologic function is described by the time to neutrophil and platelet recovery. Time to neutrophil recovery will be the first of three consecutive days of \> 500 neutrophils/μL following the expected nadir. Time to platelet engraftment will be described by the date when platelet count is \> 20,000/μL for the first of three consecutive labs with no platelet transfusions 7 days prior.
Outcome measures
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Percentage of Participants Recovering Hematologic Function
Day 28 Neutrophil Recovery
|
100.0 percentage of participants
|
—
|
|
Percentage of Participants Recovering Hematologic Function
Day 100 Platelet Recovery
|
94.1 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Week 4, Day 100, and 6 months Post-transplantDonor T-cell and myeloid chimerism will be described separately by conditioning regimen intensity (myeloablative or reduced intensity) according to proportions with mixed chimerism (5-95% donor cells out of all), full chimerism (\>95% donor cells), or graft rejection (\<5% donor cells).
Outcome measures
| Measure |
Allogeneic Transplant
n=8 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
n=9 Participants
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Chimerism
6 Months · Mixed Chimerism
|
2 Participants
|
2 Participants
|
|
Chimerism
6 Months · Graft Rejection
|
0 Participants
|
0 Participants
|
|
Chimerism
6 Months · Dead at Assessment
|
2 Participants
|
2 Participants
|
|
Chimerism
6 Months · No Assay Reported
|
0 Participants
|
0 Participants
|
|
Chimerism
Week 4 · Full Chimerism
|
4 Participants
|
5 Participants
|
|
Chimerism
Week 4 · Mixed Chimerism
|
2 Participants
|
4 Participants
|
|
Chimerism
Week 4 · Graft Rejection
|
1 Participants
|
0 Participants
|
|
Chimerism
Week 4 · Dead at Assessment
|
0 Participants
|
0 Participants
|
|
Chimerism
Week 4 · No Assay Reported
|
1 Participants
|
0 Participants
|
|
Chimerism
Day 100 · Full Chimerism
|
3 Participants
|
5 Participants
|
|
Chimerism
Day 100 · Mixed Chimerism
|
4 Participants
|
4 Participants
|
|
Chimerism
Day 100 · Graft Rejection
|
0 Participants
|
0 Participants
|
|
Chimerism
Day 100 · Dead at Assessment
|
1 Participants
|
0 Participants
|
|
Chimerism
Day 100 · No Assay Reported
|
0 Participants
|
0 Participants
|
|
Chimerism
6 Months · Full Chimerism
|
4 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Day 100 Post-transplantAcute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995: Skin stage: 0: No rash 1. Rash \<25% of body surface area 2. Rash on 25-50% of body surface area 3. Rash on \> 50% of body surface area 4. Generalized erythroderma with bullous formation Liver stage (based on bilirubin level)\*: 0: \<2 mg/dL 1.2-3 mg/dL 2.3.01-6 mg/dL 3.6.01-15.0 mg/dL 4.\>15 mg/dL GI stage\*: 0: No diarrhea or diarrhea \<500 mL/day 1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD 2. Diarrhea 1000-1499 mL/day 3. Diarrhea \>1500 mL/day 4. Severe abdominal pain with or without ileus \* If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1. GVHD grade: 0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4
Outcome measures
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Percentage of Participants With Acute Graft-Versus-Host Disease (GVHD)
Grade II-IV Acute GVHD
|
41.2 percentage of participants
Interval 17.8 to 63.4
|
—
|
|
Percentage of Participants With Acute Graft-Versus-Host Disease (GVHD)
Grade III-IV Acute GVHD
|
11.8 percentage of participants
Interval 1.8 to 31.9
|
—
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantChronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification.
Outcome measures
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Percentage of Participants With Chronic Graft-Versus-Host Disease (GVHD)
|
17.6 percentage of participants
Interval 4.0 to 39.2
|
—
|
SECONDARY outcome
Timeframe: 1 Year Post-transplantThe maximum grade of infections reported by participants are described, as defined in the BMT CTN Technical MOP.
Outcome measures
| Measure |
Allogeneic Transplant
n=17 Participants
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
Reduced Intensity Allogeneic Transplant
Reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|---|
|
Infection Severity
Grade 0-1
|
6 Participants
|
—
|
|
Infection Severity
Grade 2
|
3 Participants
|
—
|
|
Infection Severity
Grade 3
|
8 Participants
|
—
|
Adverse Events
Allogeneic Transplant
Serious events: 5 serious events
Other events: 0 other events
Deaths: 8 deaths
Serious adverse events
| Measure |
Allogeneic Transplant
n=20 participants at risk
One regimen from either reduced-intensity conditioning (RIC) (Fludarabine and Busulfan; or Fludarabine and Melphalan) or myeloablative conditioning (MAC) (Busulfan and Fludarabine; or Cyclophosphamide and Total Body Irradiation) will be administered prior to allogeneic hematopoietic cell transplantation (HCT).
|
|---|---|
|
Cardiac disorders
Supraventricular tachycardia
|
5.0%
1/20 • Number of events 1 • Up to 2 Years Post-transplant
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
5.0%
1/20 • Number of events 1 • Up to 2 Years Post-transplant
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
5.0%
1/20 • Number of events 3 • Up to 2 Years Post-transplant
|
|
Gastrointestinal disorders
Upper gastrointestinal haemorrhage
|
5.0%
1/20 • Number of events 1 • Up to 2 Years Post-transplant
|
|
Hepatobiliary disorders
Hepatic failure
|
5.0%
1/20 • Number of events 1 • Up to 2 Years Post-transplant
|
|
Infections and infestations
Sepsis
|
5.0%
1/20 • Number of events 1 • Up to 2 Years Post-transplant
|
|
Investigations
Hepatic enzyme increased
|
5.0%
1/20 • Number of events 1 • Up to 2 Years Post-transplant
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
5.0%
1/20 • Number of events 1 • Up to 2 Years Post-transplant
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
|
5.0%
1/20 • Number of events 1 • Up to 2 Years Post-transplant
|
|
Vascular disorders
Hypotension
|
5.0%
1/20 • Number of events 1 • Up to 2 Years Post-transplant
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place