Trial Outcomes & Findings for Stem Cell Transplantation for Hematological Malignancies (NCT NCT00176839)

Last Updated: 2017-12-05

Results Overview

Number of participants with long-term disease free survival after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

11 participants

Primary outcome timeframe

1 year

Results posted on

2017-12-05

Participant Flow

The study was offered to patients at the time different treatment options were being discussed in the clinic or in the hospital.

Participant milestones

Participant milestones
Measure	Treatment Arm Patients treated with therapy plan ((Busulfan, Cyclophosphamide, Melphalan, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation.
Overall Study STARTED	11
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplantation for Hematological Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment Arm n=11 Participants Patients treated with therapy plan ((Busulfan, Cyclophosphamide, Melphalan, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation.
Age, Categorical <=18 years	9 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	10.3 years STANDARD_DEVIATION 8.4 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 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	1 Participants n=5 Participants
Race (NIH/OMB) White	8 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	11 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Number of participants with long-term disease free survival after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.

Outcome measures

Outcome measures
Measure	Treatment Arm n=11 Participants Patients treated with therapy plan ((Busulfan, Cyclophosphamide, Melphalan, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation.
Probability of Long-term Disease-free Survival (DFS)	3 participants

SECONDARY outcome

Timeframe: 1 year

Number of participants with engraftment after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies..

Outcome measures

Outcome measures
Measure	Treatment Arm n=11 Participants Patients treated with therapy plan ((Busulfan, Cyclophosphamide, Melphalan, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation.
Probability of Engraftment	10 participants

SECONDARY outcome

Timeframe: 100 days post-transplant

Number of participants with acute GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.

Outcome measures

Outcome measures
Measure	Treatment Arm n=11 Participants Patients treated with therapy plan ((Busulfan, Cyclophosphamide, Melphalan, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation.
Incidence of Acute Graft-versus-host Disease (GVHD)	7 participants

SECONDARY outcome

Timeframe: 1 year

Number of participants with chronic GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.

Outcome measures

Outcome measures
Measure	Treatment Arm n=11 Participants Patients treated with therapy plan ((Busulfan, Cyclophosphamide, Melphalan, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation.
Incidence Chronic Graft-versus-host Disease (GVHD)	0 participants

SECONDARY outcome

Timeframe: 100 days post-transplant

Number of participants with regimen-related toxicity 100 days post transplant after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.

Outcome measures

Outcome measures
Measure	Treatment Arm n=11 Participants Patients treated with therapy plan ((Busulfan, Cyclophosphamide, Melphalan, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation.
Incidence of Regimen-related Toxicity 100 Days Post Transplant	3 participants

SECONDARY outcome

Timeframe: 1 year

Number of patients with relapse after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.

Outcome measures

Outcome measures
Measure	Treatment Arm n=11 Participants Patients treated with therapy plan ((Busulfan, Cyclophosphamide, Melphalan, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation.
Incidence of Relapse	2 participants

Adverse Events

Treatment Arm

Serious events: 10 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment Arm n=11 participants at risk Patients treated with therapy plan ((Busulfan, Cyclophosphamide, Melphalan, antithymocyte globulin (ATG), G-CSF (granulocyte colony-stimulating factor) and stem cell transplantation.
Infections and infestations Fungal Infection	9.1% 1/11 • Number of events 1 • 1 year post transplant.
Respiratory, thoracic and mediastinal disorders Pulmonary Failure	9.1% 1/11 • Number of events 1 • 1 year post transplant.
Immune system disorders Acute Graft Versus Host Disease	63.6% 7/11 • Number of events 7 • 1 year post transplant.
Immune system disorders Graft Failure	9.1% 1/11 • Number of events 1 • 1 year post transplant.

Other adverse events

Adverse event data not reported

Additional Information

Margaret MacMillan, M.D.

University of Minnesota Masonic Cancer Center

Phone: 612-626-2778

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place