Trial Outcomes & Findings for Reduced Intensity Conditioning Transplant Using Haploidentical Donors (NCT NCT02581007)
NCT ID: NCT02581007
Last Updated: 2023-04-27
Results Overview
Measurement of donor cells vs. recipient cells
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
100 days
Results posted on
2023-04-27
Participant Flow
Participant milestones
| Measure |
Reduced-Intensity Mismatched Transplant
Fludarabine, Melphalan \& Post-transplant cyclophosphamide
Fludarabine: fludarabine (30mg/m2) given every day starting on Day -6 through Day -2;
Melphalan
Cyclophosphamide: cyclophosphamide (50mg/kg) given every day starting on Day 3 through Day 4.
peripheral blood stem cell transplant
|
|---|---|
|
Overall Study
STARTED
|
25
|
|
Overall Study
COMPLETED
|
25
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Reduced-Intensity Mismatched Transplant
n=25 Participants
Fludarabine, Melphalan \& Post-transplant cyclophosphamide
Fludarabine: fludarabine (30mg/m2) given every day starting on Day -6 through Day -2;
Melphalan
Cyclophosphamide: cyclophosphamide (50mg/kg) given every day starting on Day 3 through Day 4.
peripheral blood stem cell transplant
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=25 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
22 Participants
n=25 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=25 Participants
|
|
Age, Continuous
|
57 years
n=25 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=25 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=25 Participants
|
|
Region of Enrollment
United States
|
25 participants
n=25 Participants
|
PRIMARY outcome
Timeframe: 100 daysMeasurement of donor cells vs. recipient cells
Outcome measures
| Measure |
Reduced-Intensity Mismatched Transplant
n=25 Participants
Fludarabine, Melphalan \& Post-transplant cyclophosphamide
Fludarabine: fludarabine (30mg/m2) given every day starting on Day -6 through Day -2;
Melphalan
Cyclophosphamide: cyclophosphamide (50mg/kg) given every day starting on Day 3 through Day 4.
peripheral blood stem cell transplant
|
|---|---|
|
Graft Rejection
|
3 Participants
|
SECONDARY outcome
Timeframe: 2 yearsNumber of participants still alive 2 years after transplant
Outcome measures
| Measure |
Reduced-Intensity Mismatched Transplant
n=25 Participants
Fludarabine, Melphalan \& Post-transplant cyclophosphamide
Fludarabine: fludarabine (30mg/m2) given every day starting on Day -6 through Day -2;
Melphalan
Cyclophosphamide: cyclophosphamide (50mg/kg) given every day starting on Day 3 through Day 4.
peripheral blood stem cell transplant
|
|---|---|
|
Overall Survival
1 yr post-transplant
|
68 percentage of patients
Interval 46.0 to 82.0
|
|
Overall Survival
2 yr post-transplant
|
56 percentage of patients
Interval 33.0 to 74.0
|
SECONDARY outcome
Timeframe: 2 yearsNumber of patients with disease reoccurrence at 1 and 2 years post-transplant
Outcome measures
| Measure |
Reduced-Intensity Mismatched Transplant
n=25 Participants
Fludarabine, Melphalan \& Post-transplant cyclophosphamide
Fludarabine: fludarabine (30mg/m2) given every day starting on Day -6 through Day -2;
Melphalan
Cyclophosphamide: cyclophosphamide (50mg/kg) given every day starting on Day 3 through Day 4.
peripheral blood stem cell transplant
|
|---|---|
|
Relapse Incidence
1 yr post-transplant
|
24 percentage of patients
Interval 10.0 to 42.0
|
|
Relapse Incidence
2 yr post-transplant
|
36 percentage of patients
Interval 17.0 to 55.0
|
SECONDARY outcome
Timeframe: 100 daysThe number of participants that developed graft-versus-host-disease before or at 100 days after transplant
Outcome measures
| Measure |
Reduced-Intensity Mismatched Transplant
n=25 Participants
Fludarabine, Melphalan \& Post-transplant cyclophosphamide
Fludarabine: fludarabine (30mg/m2) given every day starting on Day -6 through Day -2;
Melphalan
Cyclophosphamide: cyclophosphamide (50mg/kg) given every day starting on Day 3 through Day 4.
peripheral blood stem cell transplant
|
|---|---|
|
GVHD Incidence
Grades II to IV acute GVHD
|
20 percentage of patients
Interval 8.0 to 37.0
|
|
GVHD Incidence
Grades III to IV acute GVHD
|
8 percentage of patients
Interval 2.0 to 22.0
|
|
GVHD Incidence
Moderate chronic GVHD
|
16 percentage of patients
Interval 5.0 to 33.0
|
|
GVHD Incidence
Severe chronic GVHD
|
12 percentage of patients
Interval 3.0 to 27.0
|
Adverse Events
Reduced-Intensity Mismatched Transplant
Serious events: 23 serious events
Other events: 25 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Reduced-Intensity Mismatched Transplant
n=25 participants at risk
Fludarabine, Melphalan \& Post-transplant cyclophosphamide
Fludarabine: fludarabine (30mg/m2) given every day starting on Day -6 through Day -2;
Melphalan
Cyclophosphamide: cyclophosphamide (50mg/kg) given every day starting on Day 3 through Day 4.
peripheral blood stem cell transplant
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
respiratory failure
|
8.0%
2/25 • 100 days for adverse events, overall survival over 2 years
|
|
Nervous system disorders
confusion
|
8.0%
2/25 • 100 days for adverse events, overall survival over 2 years
|
|
Infections and infestations
sepsis
|
16.0%
4/25 • 100 days for adverse events, overall survival over 2 years
|
|
Renal and urinary disorders
renal failure
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Respiratory, thoracic and mediastinal disorders
hypoxia
|
28.0%
7/25 • 100 days for adverse events, overall survival over 2 years
|
|
Cardiac disorders
pericardial effusion
|
12.0%
3/25 • 100 days for adverse events, overall survival over 2 years
|
|
Respiratory, thoracic and mediastinal disorders
pulmonary edema
|
12.0%
3/25 • 100 days for adverse events, overall survival over 2 years
|
|
Infections and infestations
sinusitis
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Gastrointestinal disorders
esophagitis
|
8.0%
2/25 • 100 days for adverse events, overall survival over 2 years
|
|
General disorders
chest pain
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Gastrointestinal disorders
mucositis
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Cardiac disorders
tachycardia
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Metabolism and nutrition disorders
elevated liver enzymes
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
General disorders
fever
|
60.0%
15/25 • 100 days for adverse events, overall survival over 2 years
|
|
Psychiatric disorders
hallucinations
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Cardiac disorders
cardiac arrest
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Gastrointestinal disorders
anorexia
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Immune system disorders
graft-versus-host-disease
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Respiratory, thoracic and mediastinal disorders
pneumonia
|
8.0%
2/25 • 100 days for adverse events, overall survival over 2 years
|
|
Infections and infestations
bacteremia infection
|
24.0%
6/25 • 100 days for adverse events, overall survival over 2 years
|
|
Gastrointestinal disorders
nausea
|
16.0%
4/25 • 100 days for adverse events, overall survival over 2 years
|
|
Gastrointestinal disorders
vomiting
|
16.0%
4/25 • 100 days for adverse events, overall survival over 2 years
|
|
Gastrointestinal disorders
diarrhea
|
16.0%
4/25 • 100 days for adverse events, overall survival over 2 years
|
|
Cardiac disorders
superficial venous thrombosis
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Cardiac disorders
hypotension
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Cardiac disorders
hypertension
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Investigations
thrombocytopenia
|
12.0%
3/25 • 100 days for adverse events, overall survival over 2 years
|
|
Cardiac disorders
deep venous thrombosis
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Metabolism and nutrition disorders
tumor lysis syndrome
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Immune system disorders
cytokine release syndrome
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
Other adverse events
| Measure |
Reduced-Intensity Mismatched Transplant
n=25 participants at risk
Fludarabine, Melphalan \& Post-transplant cyclophosphamide
Fludarabine: fludarabine (30mg/m2) given every day starting on Day -6 through Day -2;
Melphalan
Cyclophosphamide: cyclophosphamide (50mg/kg) given every day starting on Day 3 through Day 4.
peripheral blood stem cell transplant
|
|---|---|
|
Metabolism and nutrition disorders
hyperglycemia
|
12.0%
3/25 • 100 days for adverse events, overall survival over 2 years
|
|
Metabolism and nutrition disorders
hypomagnesemia
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Metabolism and nutrition disorders
hypermagnesemia
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Investigations
leukopenia
|
100.0%
25/25 • 100 days for adverse events, overall survival over 2 years
|
|
Investigations
neutropenia
|
100.0%
25/25 • 100 days for adverse events, overall survival over 2 years
|
|
Renal and urinary disorders
BK virus
|
8.0%
2/25 • 100 days for adverse events, overall survival over 2 years
|
|
Investigations
anemia
|
40.0%
10/25 • 100 days for adverse events, overall survival over 2 years
|
|
Skin and subcutaneous tissue disorders
rash
|
16.0%
4/25 • 100 days for adverse events, overall survival over 2 years
|
|
Metabolism and nutrition disorders
hypokalemia
|
12.0%
3/25 • 100 days for adverse events, overall survival over 2 years
|
|
Metabolism and nutrition disorders
hyponatremia
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Gastrointestinal disorders
weight loss
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Nervous system disorders
syncope
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
|
Metabolism and nutrition disorders
hypocalcemia
|
8.0%
2/25 • 100 days for adverse events, overall survival over 2 years
|
|
Metabolism and nutrition disorders
elevated liver enzymes
|
32.0%
8/25 • 100 days for adverse events, overall survival over 2 years
|
|
Cardiac disorders
hypertension
|
12.0%
3/25 • 100 days for adverse events, overall survival over 2 years
|
|
Investigations
thrombocytopenia
|
100.0%
25/25 • 100 days for adverse events, overall survival over 2 years
|
|
Renal and urinary disorders
urinary tract infection
|
4.0%
1/25 • 100 days for adverse events, overall survival over 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place