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: 2025-04-30

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.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 47 participants
• Primary outcome timeframe: 1 Year after transplant
• Results posted on: 2025-04-30

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=5 Participants
Age, Categorical Between 18 and 65 years	11 Participants n=5 Participants
Age, Categorical >=65 years	35 Participants n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	30 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	40 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	37 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Region of Enrollment United States	46 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 Year after transplant

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.

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 transplant

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
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 events: 0 serious events

Other events: 32 other events

Deaths: 16 deaths

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

Phone: 215-955-4367

Email: Neal.Flomenberg@jefferson.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place