Trial Outcomes & Findings for NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders (NCT NCT00053989)
NCT ID: NCT00053989
Last Updated: 2020-02-10
Results Overview
treatment related mortality within 100 days from hematopoietic stem cell (HSC) infusion on day 0
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
41 participants
Primary outcome timeframe
from start or conditioning (day -6 or -5) through day +100 after HSC infusion
Results posted on
2020-02-10
Participant Flow
Participant milestones
| Measure |
All Patients
All patients enrolled on study Patients received cyclophosphamide 50 mg/kg on days -5 and -4, and fludarabine 25 mg/m2 on days -5, -4, -3, -2, -1.
Hematopoietic cells from peripheral blood or marrow were infused on day 0. Patients who received hematopoietic cells from cord blood additionally received ATG 30 mg/kg on days -3, -2, -1.
Fanconi anemia patients received cyclophosphamide 7.5 mg/kg on days -6, -5, -4, -3, fludarabine 25 mg/m2 on days -6, -5, -4, -3, -2 and ATG 30 mg/kg on days -3, -2, -1 and hematopoietic cells infused on day 0.
|
|---|---|
|
Overall Study
STARTED
|
41
|
|
Overall Study
COMPLETED
|
41
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders
Baseline characteristics by cohort
| Measure |
All Patients
n=41 Participants
All patients enrolled on study Patients received cyclophosphamide 50 mg/kg on days -5 and -4, and fludarabine 25 mg/m2 on days -5, -4, -3, -2, -1.
Hematopoietic cells from peripheral blood or marrow were infused on day 0. Patients who received hematopoietic cells from cord blood additionally received ATG 30 mg/kg on days -3, -2, -1.
Fanconi anemia patients received cyclophosphamide 7.5 mg/kg on days -6, -5, -4, -3, fludarabine 25 mg/m2 on days -6, -5, -4, -3, -2 and ATG 30 mg/kg on days -3, -2, -1 and hematopoietic cells infused on day 0.
|
|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
37 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=99 Participants
|
|
Age, Continuous
|
52 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
40 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
41 participants
n=99 Participants
|
|
Diagnosis
Acute lymphoblastic or myeloid leukemia
|
12 Participants
n=99 Participants
|
|
Diagnosis
Hodgkin/non-Hodgkin lymphoma/prolymphocytic leukem
|
21 Participants
n=99 Participants
|
|
Diagnosis
Multiple Myeloma
|
2 Participants
n=99 Participants
|
|
Diagnosis
Myelodysplastic syndrome
|
5 Participants
n=99 Participants
|
|
Diagnosis
Fanconi anemia
|
1 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: from start or conditioning (day -6 or -5) through day +100 after HSC infusiontreatment related mortality within 100 days from hematopoietic stem cell (HSC) infusion on day 0
Outcome measures
| Measure |
All Patients
n=41 Participants
All patients enrolled on study
|
|---|---|
|
Day 100 TRM
|
4 Participants
|
PRIMARY outcome
Timeframe: from start of conditioning on day -6 or -5 through day +100 after HSC infusionBest disease response measured within 100 days from hematopoietic stem cell (HSC) infusion on day 0 using disease specific response criteria defined in the protocol
Outcome measures
| Measure |
All Patients
n=41 Participants
All patients enrolled on study
|
|---|---|
|
Day 100 Best Response
Complete Response
|
22 Participants
|
|
Day 100 Best Response
Partial Response
|
5 Participants
|
|
Day 100 Best Response
Early Death/NE
|
4 Participants
|
|
Day 100 Best Response
No Response/Stable Disease
|
2 Participants
|
|
Day 100 Best Response
Progressed
|
8 Participants
|
SECONDARY outcome
Timeframe: 1 yearProgression free survival defined as time from HSC infusion (day 0) until progression of disease or death due to any cause. Patients are censored if alive without disease progression through 1 year after HSC infusion
Outcome measures
| Measure |
All Patients
n=41 Participants
All patients enrolled on study
|
|---|---|
|
PFS
|
27 percentage of participants
Interval 13.0 to 40.0
|
SECONDARY outcome
Timeframe: 1 yearOverall survival with events defined as death due to any cause and censored patients are alive as of 1 year post HSC infusion
Outcome measures
| Measure |
All Patients
n=41 Participants
All patients enrolled on study
|
|---|---|
|
OS
|
44 percentage of participants
Interval 29.0 to 59.0
|
SECONDARY outcome
Timeframe: Day +100Population: 2 participants died before they were eligible for this outcome
overall grade II-IV acute GvHD
Outcome measures
| Measure |
All Patients
n=39 Participants
All patients enrolled on study
|
|---|---|
|
Acute GvHD
|
16 Participants
|
Adverse Events
All Patients
Serious events: 3 serious events
Other events: 6 other events
Deaths: 8 deaths
Serious adverse events
| Measure |
All Patients
n=41 participants at risk
All patients enrolled on study
|
|---|---|
|
Blood and lymphatic system disorders
DBE/FTE
|
7.3%
3/41 • Number of events 3 • 100 days
SAE=died before absolute neutrophil count recovery (DBE) or survived to day +28 without absolute neutrophil count recovery (FTE) AE=overall grade III-IV acute GVHD
|
Other adverse events
| Measure |
All Patients
n=41 participants at risk
All patients enrolled on study
|
|---|---|
|
Immune system disorders
acute GVHD grade III-IV
|
14.6%
6/41 • Number of events 6 • 100 days
SAE=died before absolute neutrophil count recovery (DBE) or survived to day +28 without absolute neutrophil count recovery (FTE) AE=overall grade III-IV acute GVHD
|
Additional Information
Theresa Hahn, PhD
Roswell Park Comprehensive Cancer Center
Phone: 716084505819
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place