Trial Outcomes & Findings for Donor Umbilical Cord Blood Natural Killer Cells, Aldesleukin and Umbilical Cord Blood Transplant in Patients With Refractory Hematologic Cancers. (NCT NCT00354172)

NCT ID: NCT00354172

Last Updated: 2017-12-28

Results Overview

Number of patients who were alive and free of disease (malignancy) at 6 months after transplant.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 16 participants
• Primary outcome timeframe: 6 Months Post Transplant
• Results posted on: 2017-12-28

Participant Flow

Participant milestones

Measure	Patients Treated for Refractory Hematologic Cancers All patients receiving at least partial study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Overall Study STARTED	16
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Patients Treated for Refractory Hematologic Cancers All patients receiving at least partial study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Overall Study Did not receive transplant	1

Baseline Characteristics

Donor Umbilical Cord Blood Natural Killer Cells, Aldesleukin and Umbilical Cord Blood Transplant in Patients With Refractory Hematologic Cancers.

Baseline characteristics by cohort

Measure	Patients Treated for Refractory Hematologic Cancers n=16 Participants All patients receiving at least partial study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Age, Categorical <=18 years	8 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	22 years n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Region of Enrollment United States	16 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 Months Post Transplant

Population: One patient did not receive umbilical cord transplant and was not included in this Evaluable patient group.

Number of patients who were alive and free of disease (malignancy) at 6 months after transplant.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) Who Were Disease-free and Alive at 6 Months	2 Participants

SECONDARY outcome

Timeframe: 12 Months Post transplant

Number of patients who were alive and free of disease (malignancy) at 12 months after transplant.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) Who Were Disease-free and Alive at 12 Months	1 Participants

SECONDARY outcome

Timeframe: 24 Months Post transplant

Number of patients who were alive and free of disease (malignancy) at 24 months after transplant.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Patients Who Were Disease-free and Alive at 24 Months	0 Participants

SECONDARY outcome

Timeframe: 6 Months Post Transplant

Patients who had transplant-related mortality (TRM). TRM = adverse event(s) that occur(s) after the patient has received a transplant, the principal investigator decides it is related to the procedure and the patient dies within 6 months.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) Who Died Due to Transplant.	4 Participants

SECONDARY outcome

Timeframe: Day 42 Post Transplant

Defined as absolute neutrophils (ANC) > 5 x 10^8/Liter for 3 consecutive days.

ANC is the real number of white blood cells (WBCs) that are neutrophils. The absolute neutrophil count is commonly called the ANC. The ANC is not measured directly. It is derived by multiplying the WBC count times the percent of neutrophils in the differential WBC count. The percent of neutrophils consists of the segmented (fully mature) neutrophils) + the bands (almost mature neutrophils). The normal range for the ANC = 1.5 to 8.0 (1,500 to 8,000/mm3).

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) Who Attained Neutrophil Engraftment	13 Participants

SECONDARY outcome

Timeframe: 1 Year Post Transplant

Platelet engraftment is defined as platelet counts > 50 x 10^9/Liter for 3 consecutive days.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) Who Attained Platelet Engraftment	5 Participants

SECONDARY outcome

Timeframe: Day 100 Post Transplant

Graft-versus-host disease (GVHD) is a common complication of transplantation in which functional immune cells in the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. The acute or fulminant form of the disease (aGVHD) is normally observed within the first 100 days post-transplant, and is a major challenge to transplants owing to associated morbidity and mortality. Acute GVHD is staged as follows: overall grade (skin-liver-gut) with each organ staged individually from a low of I to a high of IV. Patients with grade IV GVHD usually have a poor prognosis.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) With Acute Graft-versus-host Disease (GVHD) Grade II-IV	6 Participants

SECONDARY outcome

Timeframe: Day 100 post transplant

Graft-versus-host disease (GVHD) is a common complication of transplantation in which functional immune cells in the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. The acute or fulminant form of the disease (aGVHD) is normally observed within the first 100 days post-transplant, and is a major challenge to transplants owing to associated morbidity and mortality. Acute GVHD is staged as follows: overall grade (skin-liver-gut) with each organ staged individually from a low of I to a high of IV. Patients with grade IV GVHD usually have a poor prognosis.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) With Acute Graft-versus-Host Disease at Grade III-IV	1 Participants

SECONDARY outcome

Timeframe: Day 100 through 1 Year Post Transplant

The chronic form of graft-versus-host-disease (cGVHD) normally occurs after 100 days. The appearance of moderate to severe cases of cGVHD adversely influences long-term survival.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) With Chronic Graft-Versus-Host Disease	1 Participants

SECONDARY outcome

Timeframe: 1 year Post Transplant

Number of patients who died after receiving treatment within 12 months post transplant.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) Who Died by 12 Months	14 Participants

SECONDARY outcome

Timeframe: 2 years post-transplant

Number of patients who died after receiving treatment within 24 months post transplant.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) Who Died by 24 Months	15 Participants

SECONDARY outcome

Timeframe: 1 Year Post Transplant

Number of patients who experienced recurrence or progression of disease from the time of transplant.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) Who Experienced Relapse by 12 Months	10 Participants

SECONDARY outcome

Timeframe: 2 Years Post transplant

Number of patients who experienced recurrence or progression of disease from the time of transplant.

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) Who Experienced Relapse by 24 Months	11 Participants

SECONDARY outcome

Timeframe: 10-13 Days Post Infusion

Defined by an absolute circulating donor-derived natural killer cell count of >100 cells/microliter 10-13 days after infusion with <5% donor T and B cells in the mononuclear population

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Number of Participants (Patients) With Successful Natural Killer Cell Expansion	3 Participants

SECONDARY outcome

Timeframe: Day 21, Day 100, 6 Months

Population: 1 Year and 2 Year Post Transplant data was not applicable; no patients reached this timeframe to evaluate.

Calculation of Median (range) of percentage of donor cells engrafted (present) in the recipient (patient).

Outcome measures

Measure	Evaluable Patients n=15 Participants All patients receiving full study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Chimerism After Double Umbilical Cord Blood Transplant (UCBT) Day 100	100 Percentage of Engrafted Cells Interval 47.0 to 100.0
Chimerism After Double Umbilical Cord Blood Transplant (UCBT) Day 21	92 Percentage of Engrafted Cells Interval 14.0 to 100.0
Chimerism After Double Umbilical Cord Blood Transplant (UCBT) 6 Months	96.5 Percentage of Engrafted Cells Interval 93.0 to 100.0

Adverse Events

Patients Treated for Refractory Hematologic Cancers

Serious events: 16 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Patients Treated for Refractory Hematologic Cancers n=16 participants at risk All patients receiving at least partial study treatment with chemotherapy and radiation, along with natural killer cells, aldesleukin and umbilical cord blood transplant.
Cardiac disorders Cardiac failure	6.2% 1/16 • Number of events 1 • Serious adverse events were collected if deemed related to treatment from Day 1 through 1 year post transplant. It was expected that most treatment related adverse events would occur during this period. Only serious adverse events were captured for this study.
Blood and lymphatic system disorders Death	25.0% 4/16 • Number of events 4 • Serious adverse events were collected if deemed related to treatment from Day 1 through 1 year post transplant. It was expected that most treatment related adverse events would occur during this period. Only serious adverse events were captured for this study.
Blood and lymphatic system disorders Disease relapse	25.0% 4/16 • Number of events 5 • Serious adverse events were collected if deemed related to treatment from Day 1 through 1 year post transplant. It was expected that most treatment related adverse events would occur during this period. Only serious adverse events were captured for this study.
Respiratory, thoracic and mediastinal disorders Dyspnea	6.2% 1/16 • Number of events 2 • Serious adverse events were collected if deemed related to treatment from Day 1 through 1 year post transplant. It was expected that most treatment related adverse events would occur during this period. Only serious adverse events were captured for this study.
Respiratory, thoracic and mediastinal disorders Hemorrhage, lung	12.5% 2/16 • Number of events 2 • Serious adverse events were collected if deemed related to treatment from Day 1 through 1 year post transplant. It was expected that most treatment related adverse events would occur during this period. Only serious adverse events were captured for this study.
Hepatobiliary disorders Hepatic portal vein flow occluded	6.2% 1/16 • Number of events 2 • Serious adverse events were collected if deemed related to treatment from Day 1 through 1 year post transplant. It was expected that most treatment related adverse events would occur during this period. Only serious adverse events were captured for this study.
Respiratory, thoracic and mediastinal disorders Pneumonia	18.8% 3/16 • Number of events 3 • Serious adverse events were collected if deemed related to treatment from Day 1 through 1 year post transplant. It was expected that most treatment related adverse events would occur during this period. Only serious adverse events were captured for this study.

Other adverse events

Adverse event data not reported

Additional Information

Jeffrey Miller, M.D.

Masonic Cancer Center, University of Minnesota

Phone: 612-625-7409

Email: mille011@umn.edu

Results disclosure agreements

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