Trial Outcomes & Findings for Reduced Intensity Flu/Mel/TBI Conditioning for HAPLO HCT Patients With Hematologic Malignancies (NCT NCT04191187)
NCT ID: NCT04191187
Last Updated: 2025-12-11
Results Overview
Disease Free Survival (DFS) is defined as the time from the date of Peripheral Blood Stem Cell Transplant (PBSCT) to first documentation of relapse or death due to any cause, whichever comes first.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
34 participants
Primary outcome timeframe
Up to 18 months post-transplant
Results posted on
2025-12-11
Participant Flow
Participant milestones
| Measure |
Conditioning Regimen + Transplant
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Overall Study
STARTED
|
34
|
|
Overall Study
COMPLETED
|
34
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Reduced Intensity Flu/Mel/TBI Conditioning for HAPLO HCT Patients With Hematologic Malignancies
Baseline characteristics by cohort
| Measure |
Conditioning Regimen + Transplant
n=34 Participants
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=237 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
19 Participants
n=237 Participants
|
|
Age, Categorical
>=65 years
|
15 Participants
n=237 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=237 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=237 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=237 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
29 Participants
n=237 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=237 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=237 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=237 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=237 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=237 Participants
|
|
Race (NIH/OMB)
White
|
26 Participants
n=237 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=237 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=237 Participants
|
|
Region of Enrollment
United States
|
34 participants
n=237 Participants
|
PRIMARY outcome
Timeframe: Up to 18 months post-transplantPopulation: Evaluable Participants
Disease Free Survival (DFS) is defined as the time from the date of Peripheral Blood Stem Cell Transplant (PBSCT) to first documentation of relapse or death due to any cause, whichever comes first.
Outcome measures
| Measure |
Conditioning Regimen + Transplant
n=34 Participants
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Disease Free Survival
|
70.6 percentage of participants
Interval 59.2 to 100.0
|
SECONDARY outcome
Timeframe: At 180 days post-transplantPopulation: Evaluable Participants
GVHD-free survival is defined as the time from the date of PBSCT to date of events which include grade III-IV acute GVHD and systemic therapy-requiring chronic GVHD.
Outcome measures
| Measure |
Conditioning Regimen + Transplant
n=34 Participants
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Percentage of Participants With Graft vs Host Disease (GVHD) Free Survival
|
61.8 percentage of participants
Interval 43.4 to 75.7
|
SECONDARY outcome
Timeframe: Up to 18 monthsPopulation: Evaluable Participants
OS is defined as the time from the date of PBSCT to the date of death due to any cause.
Outcome measures
| Measure |
Conditioning Regimen + Transplant
n=34 Participants
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Percentage of Participants Overall Survival (OS)
|
73.3 percentage of participants
Interval 54.9 to 85.1
|
SECONDARY outcome
Timeframe: at 6 months post-transplantPopulation: Evaluable Participants
TRM is defined as death not directly due to disease
Outcome measures
| Measure |
Conditioning Regimen + Transplant
n=34 Participants
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Percentage of Participants With Treatment Related Mortality (TRM) at 6 Months
|
17.6 percentage of participants
Interval 7.0 to 32.2
|
SECONDARY outcome
Timeframe: at 18 months post-transplantPopulation: Evaluable Participants
TRM is defined as death not directly due to disease
Outcome measures
| Measure |
Conditioning Regimen + Transplant
n=34 Participants
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Percentage of Participants With Treatment Related Mortality (TRM) at 18 Months
|
17.6 percentage of participants
Interval 7.0 to 32.2
|
SECONDARY outcome
Timeframe: Up to 18 months post-transplantPopulation: Evaluable Participants
RFS is defined as the time from the date of PBSCT to relapse or death.
Outcome measures
| Measure |
Conditioning Regimen + Transplant
n=34 Participants
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Percentage of Participants With Relapse Free Survival (RFS)
|
70.6 percentage of participants
Interval 59.2 to 79.3
|
Adverse Events
Conditioning Regimen + Transplant
Serious events: 21 serious events
Other events: 16 other events
Deaths: 9 deaths
Serious adverse events
| Measure |
Conditioning Regimen + Transplant
n=34 participants at risk
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Immune system disorders
GVHD
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Cytomegalovirus infection reactivation
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Sepsis
|
26.5%
9/34 • Number of events 10 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
5.9%
2/34 • Number of events 3 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
COVID-19+
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
General disorders
Multi-organ failure
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, specify
|
8.8%
3/34 • Number of events 3 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Metabolism and nutrition disorders
Anorexia
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Skin and subcutaneous tissue disorders
Stevens-Johnson syndrome
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Vascular disorders
Thromboembolic event
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
General disorders
Fever
|
14.7%
5/34 • Number of events 5 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Bacteremia
|
2.9%
1/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Viremia
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Nervous system disorders
Encephalopathy
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Gastrointestinal disorders
Gastric hemorrhage
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other, specify
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Lung Infection
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Infections and infestations - Other, specify
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Pharyngitis
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Immune system disorders
Anaphylaxis
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Renal and urinary disorders
Dysuria
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Renal and urinary disorders
Urinary retention
|
5.9%
2/34 • Number of events 3 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Metabolism and nutrition disorders
Acidosis
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Renal and urinary disorders
Acute kidney injury
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Encephalitis infection
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications - Other, specify
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
Other adverse events
| Measure |
Conditioning Regimen + Transplant
n=34 participants at risk
All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)
Fludarabine: Fludarabine 30mg/m\^2/day will be administered over 30-60 minutes intravenous infusion on Days -6 through -2 for a total dose of 150 mg/m\^2
Melphalan: Melphalan 70 mg/m\^2 over 45 minutes will be administered Day -6. Melphalan dose will be calculated based on Actual Body Weight.
Total Body Irradiation: Total Body Irradiation (TBI) will be delivered at a dose of 200 centigray units (cGy)
|
|---|---|
|
Investigations
Aspartate aminotransferase increased
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Nervous system disorders
Encephalopathy
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Renal and urinary disorders
Acute kidney injury
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Sepsis
|
14.7%
5/34 • Number of events 6 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Cytomegalovirus infection reactivation
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Infections and infestations - Other, specify
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Lung Infection
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Skin Infection
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Infections and infestations
Viremia
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
General disorders
Fever
|
11.8%
4/34 • Number of events 4 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
General disorders
Multi-organ failure
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
General disorders
Disease Progression
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
5.9%
2/34 • Number of events 3 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
2.9%
1/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Metabolism and nutrition disorders
Acidosis
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Metabolism and nutrition disorders
Anorexia
|
8.8%
3/34 • Number of events 3 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, specify
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Immune system disorders
Immune system disorders - Other, specify
|
5.9%
2/34 • Number of events 2 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Investigations
Alanine aminotransferase increased
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Skin and subcutaneous tissue disorders
Stevens-Johnson syndrome
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
|
Vascular disorders
Thromboembolic event
|
2.9%
1/34 • Number of events 1 • All events beginning with start of study intervention until 30 days after the last day of study intervention (up to 36 months) will be reported.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place