Trial Outcomes & Findings for Chemotherapy, Total-Body Irradiation, Donor Natural Killer Cell Infusion, Aldesleukin, and UCB Transplant in Treating Patients With Relapsed or Refractory AML (NCT NCT00871689)

NCT ID: NCT00871689

Last Updated: 2017-12-28

Results Overview

Number of patient with absolute neutrophils \>500\*10\^8/kg by 42 days post transplant.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

Day 42

Results posted on

2017-12-28

Participant Flow

Study entry was open to patients aged 0 to 45 years regardless of gender or ethnic background at the University of Minnesota, Masonic Cancer Center.

Participant milestones

Participant milestones
Measure	Patients Receiving Double Umbilical Cord Blood Transplant Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Overall Study STARTED	2
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Chemotherapy, Total-Body Irradiation, Donor Natural Killer Cell Infusion, Aldesleukin, and UCB Transplant in Treating Patients With Relapsed or Refractory AML

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 Participants Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Age, Categorical <=18 years	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	10.5 years STANDARD_DEVIATION 2.1 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 42

Number of patient with absolute neutrophils \>500\*10\^8/kg by 42 days post transplant.

Outcome measures

Outcome measures
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 Participants Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Number of Patients With Neutrophil Engraftment	1 Participants

PRIMARY outcome

Timeframe: Day 100 Post Transplant

Number of patients with Grade III-IV GVHD. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Acute GVHD usually happens within the first 3 months after transplant.

Outcome measures

Outcome measures
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 Participants Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Number of Patients With Grade III-IV Acute Graft-Versus-Host (GVHD) Disease	0 participants

SECONDARY outcome

Timeframe: Day 42

Incidence of graft failure defined as an absolute neutrophil count of less than 500/uL and a bone marrow that is less than 5% cellular (marrow aplasia) on day 42.

Outcome measures

Outcome measures
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 Participants Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Incidence of Primary Graft Failure	1 Participants

SECONDARY outcome

Timeframe: Day 100 Post Transplant

Number of patients with any grade of GVHD. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Acute GVHD usually happens within the first 3 months after transplant.

Outcome measures

Outcome measures
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 Participants Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Number of Patients With Acute Graft-Versus-Host (GVHD) Disease	1 participants

SECONDARY outcome

Timeframe: 1 Year Post Transplant

Number of patients whose death is related to study treatment received. TRD is defined as the number of patients that die without prior relapse.

Outcome measures

Outcome measures
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 Participants Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Number of Patients With Transplant-Related Death (TRD)	1 Participants

SECONDARY outcome

Timeframe: Day 100

Disease response will be measured by rate of leukemic clearance (clearance of blasts in blood at timepoint 0) and complete remission (less than 5% blasts and recovery of hematopoiesis).

Outcome measures

Outcome measures
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 Participants Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Number of Patients With Complete Remission of Disease	1 Participants

SECONDARY outcome

Timeframe: Month 6

Average number of days the patients were alive after receiving UCB transplantation.

Outcome measures

Outcome measures
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 Participants Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Median Overall Survival	98.5 Days Interval 98.0 to 99.0

SECONDARY outcome

Timeframe: Day 72 Post Transplant

Successful in vivo donor NK cell expansion will be defined as an absolute circulating donor-derived NK cell count of \>100 cells/μl.

Outcome measures

Outcome measures
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 Participants Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Number of Patients With Successful Natural Killer Expansion	1 participants

Adverse Events

Patients Receiving Double Umbilical Cord Blood Transplant

Serious events: 2 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 participants at risk Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
General disorders Death	100.0% 2/2 • Number of events 2 • Patients were followed from Day 1 up to death ( within 6 months).
Blood and lymphatic system disorders Leukoencephalopathy	50.0% 1/2 • Number of events 2 • Patients were followed from Day 1 up to death ( within 6 months).
Nervous system disorders CNS Demyelinating Disease	50.0% 1/2 • Number of events 2 • Patients were followed from Day 1 up to death ( within 6 months).
General disorders Progressive Disease	50.0% 1/2 • Number of events 1 • Patients were followed from Day 1 up to death ( within 6 months).

Other adverse events

Other adverse events
Measure	Patients Receiving Double Umbilical Cord Blood Transplant n=2 participants at risk Patients that receive myeloablative preparative regimen (allopurinol 300 mg by mouth Day -8 through +14; fludarabine 25 mg/m\^2 intravenously on days -7 through -5; cyclophosphamide 60 mg/kg intravenously on days -7 and -6; total body irradiation 165 cGy\*2 on days -4 through -2), 2 units T cell depleted umbilical cord blood transplant on day 0, followed by IL-2 every other day beginning day +3 and day +60 for a total of 6 doses.
Respiratory, thoracic and mediastinal disorders Dyspnea	100.0% 2/2 • Number of events 4 • Patients were followed from Day 1 up to death ( within 6 months).
Respiratory, thoracic and mediastinal disorders Hypoxia	50.0% 1/2 • Number of events 1 • Patients were followed from Day 1 up to death ( within 6 months).
General disorders Fever	100.0% 2/2 • Number of events 7 • Patients were followed from Day 1 up to death ( within 6 months).
Nervous system disorders Chills	100.0% 2/2 • Number of events 6 • Patients were followed from Day 1 up to death ( within 6 months).
Cardiac disorders Hypertension	50.0% 1/2 • Number of events 3 • Patients were followed from Day 1 up to death ( within 6 months).
Cardiac disorders Hypotension	50.0% 1/2 • Number of events 1 • Patients were followed from Day 1 up to death ( within 6 months).
General disorders Fatigue	100.0% 2/2 • Number of events 6 • Patients were followed from Day 1 up to death ( within 6 months).
Endocrine disorders Edema	100.0% 2/2 • Number of events 4 • Patients were followed from Day 1 up to death ( within 6 months).
Respiratory, thoracic and mediastinal disorders Pneumonitis	50.0% 1/2 • Number of events 2 • Patients were followed from Day 1 up to death ( within 6 months).
Skin and subcutaneous tissue disorders Injection site reaction	100.0% 2/2 • Number of events 3 • Patients were followed from Day 1 up to death ( within 6 months).
Skin and subcutaneous tissue disorders Rash	100.0% 2/2 • Number of events 3 • Patients were followed from Day 1 up to death ( within 6 months).
Gastrointestinal disorders Nausea	50.0% 1/2 • Number of events 2 • Patients were followed from Day 1 up to death ( within 6 months).
Respiratory, thoracic and mediastinal disorders Cough	50.0% 1/2 • Number of events 1 • Patients were followed from Day 1 up to death ( within 6 months).
Respiratory, thoracic and mediastinal disorders Lung disorder	50.0% 1/2 • Number of events 1 • Patients were followed from Day 1 up to death ( within 6 months).
Musculoskeletal and connective tissue disorders Myalgia	50.0% 1/2 • Number of events 3 • Patients were followed from Day 1 up to death ( within 6 months).
Skin and subcutaneous tissue disorders Skin disorder	50.0% 1/2 • Number of events 2 • Patients were followed from Day 1 up to death ( within 6 months).
Nervous system disorders Sweats	50.0% 1/2 • Number of events 3 • Patients were followed from Day 1 up to death ( within 6 months).
Gastrointestinal disorders Vomiting	50.0% 1/2 • Number of events 2 • Patients were followed from Day 1 up to death ( within 6 months).

Additional Information

Michael Verneris, M.D.

Masonic Cancer Center, University of Minnesota

Phone: 612-626-2961

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place