Trial Outcomes & Findings for Haploidentical Donor Bone Marrow Transplant in Treating Patients With High-Risk Hematologic Cancer (NCT NCT00049504)

NCT ID: NCT00049504

Last Updated: 2017-05-17

Results Overview

Defined by the detection of at least 50% donor derived T-cells (CD3+), as a proportion of the total T-cell population

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 53 participants
• Primary outcome timeframe: At day +84 after transplantation
• Results posted on: 2017-05-17

Participant Flow

Participant milestones

Measure	Treatment (Nonmyeloablative HSCT) NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 1 hour on days -6 and -5. Patients undergo total body irradiation on day -1. TRANSPLANTATION: Patients undergo BMT, from an HLA-haploidentical donor, on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive cyclophosphamide IV over 1 hour on day 3. GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV over 1-2 hours and then tacrolimus PO, once tolerated, on days 4-180, with taper on day 86 in the absence of graft-versus-host disease. Patients also receive mycophenolate mofetil PO three times daily on days 4-35.
Overall Study STARTED	55
Overall Study COMPLETED	55
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Haploidentical Donor Bone Marrow Transplant in Treating Patients With High-Risk Hematologic Cancer

Baseline characteristics by cohort

Measure	Treatment (Nonmyeloablative HSCT) n=55 Participants NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 1 hour on days -6 and -5. Patients undergo total body irradiation on day -1. TRANSPLANTATION: Patients undergo BMT, from an HLA-haploidentical donor, on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive cyclophosphamide IV over 1 hour on day 3. GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV over 1-2 hours and then tacrolimus PO, once tolerated, on days 4-180, with taper on day 86 in the absence of graft-versus-host disease. Patients also receive mycophenolate mofetil PO three times daily on days 4-35.
Age, Continuous	40 years n=5 Participants
Sex: Female, Male Female	21 Participants n=5 Participants
Sex: Female, Male Male	34 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	49 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 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	5 Participants n=5 Participants
Race (NIH/OMB) White	41 Participants n=5 Participants
Race (NIH/OMB) More than one race	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Region of Enrollment United States	55 participants n=5 Participants

PRIMARY outcome

Timeframe: At day +84 after transplantation

Population: Patients receiving haploidentical transplant who had T cell chimerism tested at day +84

Defined by the detection of at least 50% donor derived T-cells (CD3+), as a proportion of the total T-cell population

Outcome measures

Measure

Treatment (Nonmyeloablative HSCT) n=36 Participants NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 1 hour on days -6 and -5. Patients undergo total body irradiation on day -1. TRANSPLANTATION: Patients undergo BMT, from an HLA-haploidentical donor, on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive cyclophosphamide IV over 1 hour on day 3. GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV over 1-2 hours and then tacrolimus PO, once tolerated, on days 4-180, with taper on day 86 in the absence of graft-versus-host disease. Patients also receive mycophenolate mofetil PO three times daily on days 4-35.

Donor Engraftment (Chimerism)

34 Participants

PRIMARY outcome

Timeframe: At any time within 200 days after transplantation

Grade III GVHD represents moderate severity. Grade IV GVHD represents extreme severity

Outcome measures

Measure

Treatment (Nonmyeloablative HSCT) n=55 Participants NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 1 hour on days -6 and -5. Patients undergo total body irradiation on day -1. TRANSPLANTATION: Patients undergo BMT, from an HLA-haploidentical donor, on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive cyclophosphamide IV over 1 hour on day 3. GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV over 1-2 hours and then tacrolimus PO, once tolerated, on days 4-180, with taper on day 86 in the absence of graft-versus-host disease. Patients also receive mycophenolate mofetil PO three times daily on days 4-35.

Incidence of Grades III-IV Acute GVHD

4 Participants

PRIMARY outcome

Timeframe: Up to 200 days after transplantation

Number of deaths without progression or recurrence of malignant disease

Outcome measures

Measure

Treatment (Nonmyeloablative HSCT) n=55 Participants NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 1 hour on days -6 and -5. Patients undergo total body irradiation on day -1. TRANSPLANTATION: Patients undergo BMT, from an HLA-haploidentical donor, on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive cyclophosphamide IV over 1 hour on day 3. GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV over 1-2 hours and then tacrolimus PO, once tolerated, on days 4-180, with taper on day 86 in the absence of graft-versus-host disease. Patients also receive mycophenolate mofetil PO three times daily on days 4-35.

Non-relapse-related Mortality

12 Participants

Adverse Events

Treatment (Nonmyeloablative HSCT)

Serious events: 11 serious events

Other events: 55 other events

Deaths: 30 deaths

Serious adverse events

Measure	Treatment (Nonmyeloablative HSCT) n=55 participants at risk NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 1 hour on days -6 and -5. Patients undergo total body irradiation on day -1. TRANSPLANTATION: Patients undergo BMT, from an HLA-haploidentical donor, on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive cyclophosphamide IV over 1 hour on day 3. GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV over 1-2 hours and then tacrolimus PO, once tolerated, on days 4-180, with taper on day 86 in the absence of graft-versus-host disease. Patients also receive mycophenolate mofetil PO three times daily on days 4-35.
Infections and infestations Bacterial infection	12.7% 7/55
Immune system disorders Graft-versus-host disease	3.6% 2/55
Blood and lymphatic system disorders Secondary myeloid malignancy	1.8% 1/55
Respiratory, thoracic and mediastinal disorders Diffuse alveolar hemorrhage	1.8% 1/55

Other adverse events

Measure	Treatment (Nonmyeloablative HSCT) n=55 participants at risk NONMYELOABLATIVE CONDITIONING: Patients receive fludarabine phosphate IV over 1 hour on days -6 to -2 and cyclophosphamide IV over 1 hour on days -6 and -5. Patients undergo total body irradiation on day -1. TRANSPLANTATION: Patients undergo BMT, from an HLA-haploidentical donor, on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive cyclophosphamide IV over 1 hour on day 3. GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS: Patients receive tacrolimus IV over 1-2 hours and then tacrolimus PO, once tolerated, on days 4-180, with taper on day 86 in the absence of graft-versus-host disease. Patients also receive mycophenolate mofetil PO three times daily on days 4-35.
Infections and infestations Invasive Fungal Infection	10.9% 6/55
Infections and infestations CMV reactivation	79.4% 27/34
Blood and lymphatic system disorders Recurrent or progressive malignancy	40.0% 22/55

Additional Information

Rachel Salit

Fred Hutchinson Cancer Research Center

Phone: 206-667-1317

Email: rsalit@fredhutch.gov

Results disclosure agreements

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