Trial Outcomes & Findings for A Study of Bone Marrow Transplantation Using Fully-Matched Relatives as Donors for Patients With Hematological Malignancies (NCT NCT01315132)
NCT ID: NCT01315132
Last Updated: 2026-03-19
Results Overview
The primary objective of this prospective, phase II trial was to obtain an OS rate of \>60% at 1 year in patients undergoing a 2 step HSCT from an HLA compatible family donor. The \>60% threshold was selected as a composite efficacy measure as patients with any hematologic diagnosis, stage of disease, or age as old as 65 years were eligible for this treatment protocol.
COMPLETED
PHASE2
47 participants
1 Year after transplant
2026-03-19
Participant Flow
Participant milestones
| Measure |
Allogeneic Transplantation
Matched Sibling Allogeneic Transplantation
Matched Sibling Allogeneic Transplantation: Patients undergoing myeloablative hematopoietic stem cell transplant from HLA identical related donors using cyclophosphamide tolerization
|
|---|---|
|
Overall Study
STARTED
|
47
|
|
Overall Study
COMPLETED
|
46
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Allogeneic Transplantation
Matched Sibling Allogeneic Transplantation
Matched Sibling Allogeneic Transplantation: Patients undergoing myeloablative hematopoietic stem cell transplant from HLA identical related donors using cyclophosphamide tolerization
|
|---|---|
|
Overall Study
not treated due to disease progression
|
1
|
Baseline Characteristics
A Study of Bone Marrow Transplantation Using Fully-Matched Relatives as Donors for Patients With Hematological Malignancies
Baseline characteristics by cohort
| Measure |
Allogeneic Transplantation
n=46 Participants
Matched Sibling Allogeneic Transplantation
Matched Sibling Allogeneic Transplantation: Patients undergoing myeloablative hematopoietic stem cell transplant from HLA identical related donors using cyclophosphamide tolerization
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=110 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=110 Participants
|
|
Age, Categorical
>=65 years
|
35 Participants
n=110 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=110 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=110 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=110 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
40 Participants
n=110 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=110 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=110 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=110 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=110 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=110 Participants
|
|
Race (NIH/OMB)
White
|
37 Participants
n=110 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=110 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=110 Participants
|
|
Region of Enrollment
United States
|
46 participants
n=110 Participants
|
PRIMARY outcome
Timeframe: 1 Year after transplantThe primary objective of this prospective, phase II trial was to obtain an OS rate of \>60% at 1 year in patients undergoing a 2 step HSCT from an HLA compatible family donor. The \>60% threshold was selected as a composite efficacy measure as patients with any hematologic diagnosis, stage of disease, or age as old as 65 years were eligible for this treatment protocol.
Outcome measures
| Measure |
Allogeneic Transplantation
n=46 Participants
Matched Sibling Allogeneic Transplantation
Matched Sibling Allogeneic Transplantation: Patients undergoing myeloablative hematopoietic stem cell transplant from HLA identical related donors using cyclophosphamide tolerization
|
|---|---|
|
Number of Patients With Overall Survival
|
41 Participants
|
SECONDARY outcome
Timeframe: 1 Year after transplantOutcome measures
| Measure |
Allogeneic Transplantation
n=46 Participants
Matched Sibling Allogeneic Transplantation
Matched Sibling Allogeneic Transplantation: Patients undergoing myeloablative hematopoietic stem cell transplant from HLA identical related donors using cyclophosphamide tolerization
|
|---|---|
|
Graft Versus Host Disease (GVHD)
Acute GVHD
|
6 Participants
|
|
Graft Versus Host Disease (GVHD)
Chronic GVHD
|
4 Participants
|
|
Graft Versus Host Disease (GVHD)
No GVHD
|
36 Participants
|
Adverse Events
Allogeneic Transplantation
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Allogeneic Transplantation
n=46 participants at risk
Matched Sibling Allogeneic Transplantation
Matched Sibling Allogeneic Transplantation: Patients undergoing myeloablative hematopoietic stem cell transplant from HLA identical related donors using cyclophosphamide tolerization
|
|---|---|
|
Gastrointestinal disorders
Mucositis
|
69.6%
32/46 • Number of events 32 • from baseline through one year after transplant
|
|
Gastrointestinal disorders
Diarrhea
|
65.2%
30/46 • Number of events 35 • from baseline through one year after transplant
|
Additional Information
Neal Flomenberg, MD
Sidney Kimmel Cancer Center at Thomas Jefferson University
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place