Trial Outcomes & Findings for Phase II Trial of LMB-2, Fludarabine and Cyclophosphamide for Adult T-Cell Leukemia (NCT NCT00924170)

NCT ID: NCT00924170

Last Updated: 2023-11-14

Results Overview

Response is based on the International Workshop to Standardize Response Criteria for Non-Hodgkin's Lymphoma and must last \>8 weeks to meet the primary endpoint of the study. Complete remission (CR) is complete disappearance of all detectable clinical and radiographic evidence of disease and disappearance of all disease related symptoms if present before therapy and normalization of those biochemical abnormalities definitely assignable to the lymphoma. Partial response is reduction by ≥50% of leukemia cell count or ≥50% reduction in the size of all measurable lesions, and no increase in size of any measurable or evaluable lesion or appearance of new lesion. Stable disease is neither a response nor progressive disease. Progressive disease is appearance of new lesions, or an increase of 50% or greater in the sum of the product of the perpendicular diameters of the measurable lesions or persistent (at least two determinations) doubling of the peripheral blood leukemic cell count.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 18 participants
• Primary outcome timeframe: 8 weeks
• Results posted on: 2023-11-14

Participant Flow

Participant milestones

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).
Overall Study STARTED	10	8
Overall Study COMPLETED	3	0
Overall Study NOT COMPLETED	7	8

Reasons for withdrawal

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).
Overall Study Refused further treatment	0	1
Overall Study Physician Decision	0	1
Overall Study Progressive disease	3	6
Overall Study Stopped due to neutralizing antibodies	3	0
Overall Study Death	1	0

Baseline Characteristics

One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.

Baseline characteristics by cohort

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Total n=18 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=10 Participants	0 Participants n=8 Participants	0 Participants n=18 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=10 Participants	8 Participants n=8 Participants	17 Participants n=18 Participants
Age, Categorical >=65 years	1 Participants n=10 Participants	0 Participants n=8 Participants	1 Participants n=18 Participants
Age, Continuous	42 years STANDARD_DEVIATION 14.25 • n=10 Participants	39.8 years STANDARD_DEVIATION 10.77 • n=8 Participants	40.9 years STANDARD_DEVIATION 12.51 • n=18 Participants
Sex: Female, Male Female	7 Participants n=10 Participants	5 Participants n=8 Participants	12 Participants n=18 Participants
Sex: Female, Male Male	3 Participants n=10 Participants	3 Participants n=8 Participants	6 Participants n=18 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=10 Participants	0 Participants n=8 Participants	1 Participants n=18 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=10 Participants	7 Participants n=8 Participants	16 Participants n=18 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=10 Participants	1 Participants n=8 Participants	1 Participants n=18 Participants
Race/Ethnicity, Customized African American	2 Participants n=10 Participants	2 Participants n=8 Participants	4 Participants n=18 Participants
Race/Ethnicity, Customized Tabago, Black	1 Participants n=10 Participants	0 Participants n=8 Participants	1 Participants n=18 Participants
Race/Ethnicity, Customized Peruvian, Hispanic	1 Participants n=10 Participants	0 Participants n=8 Participants	1 Participants n=18 Participants
Race/Ethnicity, Customized American from Jamaica, Black	1 Participants n=10 Participants	0 Participants n=8 Participants	1 Participants n=18 Participants
Race/Ethnicity, Customized Jamaican, Black	5 Participants n=10 Participants	2 Participants n=8 Participants	7 Participants n=18 Participants
Race/Ethnicity, Customized Ethiopian, Black	0 Participants n=10 Participants	1 Participants n=8 Participants	1 Participants n=18 Participants
Race/Ethnicity, Customized Jamaican	0 Participants n=10 Participants	0 Participants n=8 Participants	0 Participants n=18 Participants
Race/Ethnicity, Customized Barbados, Black	0 Participants n=10 Participants	2 Participants n=8 Participants	2 Participants n=18 Participants
Race/Ethnicity, Customized American, from Ghana, Black	0 Participants n=10 Participants	1 Participants n=8 Participants	1 Participants n=18 Participants
Region of Enrollment United States	10 Participants n=10 Participants	8 Participants n=8 Participants	18 Participants n=18 Participants
Prior Treatment CHOP	2 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	3 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	5 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Prior Treatment EPOCH-C	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Prior Treatment C, ESHAP, PTX, Gem	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Prior Treatment Medi507, Ontak, HAT	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Prior Treatment CHOP, C	2 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	2 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Prior Treatment EPOCH-RS	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	2 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 17 cm tumor	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Prior Treatment None	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Prior Treatment Hydroxyurea	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	2 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Prior Treatment CHOP, Hydroxyurea	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Prior Treatment LSG-15, radiation	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Type of Leukemia Acute	10 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	3 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	13 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Type of Leukemia Lymphoma	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	4 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	4 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Bone, lung, skin	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Thigh (skin)	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Pelvis	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Muscle	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Skin	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Lymph node (LN)	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	2 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	3 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Spleen, LN	2 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	3 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Subcutaneous	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	2 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites None	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Bone, subcutaneous	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Extravascular sites Spleen	2 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	2 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Participants with High Lactate Hydrogenase (LDH) Yes	10 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	7 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	17 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Participants with High Lactate Hydrogenase (LDH) No	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Participants with High Calcium++ Yes	5 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	2 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	7 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Participants with High Calcium++ No	5 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	5 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	10 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 0 cells	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	3 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	3 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 1 cell	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 80 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 206 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 246 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 408 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 450 cells	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 687 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 5,604 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 7,737 cells	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=8 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=18 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 8,216 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 13,153 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 24,112 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 210,000 cells	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Adult T-cell Leukemia (ATL) cells/mm^3 in the Peripheral Blood 213,000 cells	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size <1 cm tumor	4 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	5 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 1.9 cm tumor	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 2 cm tumor	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 17.5 cm tumor	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 2.2 cm tumor	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 5.6 cm tumor	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 5.8 cm tumor	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 6 cm tumor	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 9 cm tumor	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 15, 2.5 cm tumor	0 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 18.5, 1.7 cm tumor	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.
Maximum tumor size 20, 1.8 cm tumor	1 Participants n=10 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	0 Participants n=7 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.	1 Participants n=17 Participants • One participant was not evaluable. Participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.

PRIMARY outcome

Timeframe: 8 weeks

Population: All 10 patients receiving LMB-2 and at least 25+250 mg/m\^2 Fludarabine/Cyclophosphamide (FC) were evaluable for response. Of the 8 other patients, only 5 received LMB-2 and were therefore evaluable for response.

Response is based on the International Workshop to Standardize Response Criteria for Non-Hodgkin's Lymphoma and must last >8 weeks to meet the primary endpoint of the study. Complete remission (CR) is complete disappearance of all detectable clinical and radiographic evidence of disease and disappearance of all disease related symptoms if present before therapy and normalization of those biochemical abnormalities definitely assignable to the lymphoma. Partial response is reduction by ≥50% of leukemia cell count or ≥50% reduction in the size of all measurable lesions, and no increase in size of any measurable or evaluable lesion or appearance of new lesion. Stable disease is neither a response nor progressive disease. Progressive disease is appearance of new lesions, or an increase of 50% or greater in the sum of the product of the perpendicular diameters of the measurable lesions or persistent (at least two determinations) doubling of the peripheral blood leukemic cell count.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Percentage of Participants With a Minimally Durable Clinical Response Rate Overall Response	80 percentage of participants Interval 44.4 to 97.5	0 percentage of participants Interval 0.0 to 0.0	—
Percentage of Participants With a Minimally Durable Clinical Response Rate Complete Response	60 percentage of participants Interval 26.0 to 88.0	0 percentage of participants Interval 0.0 to 0.0	—
Percentage of Participants With a Minimally Durable Clinical Response Rate Partial Response	20 percentage of participants Interval 2.5 to 55.6	0 percentage of participants Interval 0.0 to 0.0	—
Percentage of Participants With a Minimally Durable Clinical Response Rate Stable Disease	20 percentage of participants Interval 2.5 to 55.6	25 percentage of participants Interval 3.2 to 65.1	—
Percentage of Participants With a Minimally Durable Clinical Response Rate Progressive Disease	0 percentage of participants Interval 0.0 to 0.0	37.5 percentage of participants Interval 8.5 to 75.5	—
Percentage of Participants With a Minimally Durable Clinical Response Rate Not Evaluable	0 percentage of participants Interval 0.0 to 0.0	37.5 percentage of participants Interval 8.5 to 75.5	—

SECONDARY outcome

Timeframe: First 24 hours after the dose given on Cycle 2, day 1

The maximum analyte concentration in serum was reported using a cytotoxicity assay measuring the level of LMB-2 in the plasma and using purified LMB-2 as a standard curve.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=5 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Peak Level of LMB-2 in Adult T-Cell Lymphoma	602 ng/mL Interval 189.0 to 1072.0	484 ng/mL Interval 205.0 to 748.0	—

SECONDARY outcome

Timeframe: 70 months

PFS was determined by the Kaplan Meier method beginning at the on study date and continuing until progression or last follow-up without progression. Progressive disease is assessed by the International Workshop to Standardize Response Criteria for Non-Hodgkin's Lymphoma, and is the appearance of new lesions, or an increase of 50% or greater in the sum of the product of the perpendicular diameters of the measurable lesions or persistent (at least two determinations) doubling of the peripheral blood leukemic cell count.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Progression Free Survival (PFS)	11.6 Months Interval 1.5 to 25.2	1.05 Months Interval 0.23 to 1.54	—

SECONDARY outcome

Timeframe: 70 months

Population: One participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2.

OS is the time between the first day of treatment to the day of disease progression. Progressive disease is assessed by the International Workshop to Standardize Response Criteria for Non-Hodgkin's Lymphoma, and is the appearance of new lesions, or an increase of 50% or greater in the sum of the product of the perpendicular diameters of the measurable lesions or persistent (at least two determinations) doubling of the peripheral blood leukemic cell count.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=7 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Overall Survival (OS)	18.6 Months Interval 2.3 to 53.0	3.75 Months Interval 1.3 to 16.0	—

SECONDARY outcome

Timeframe: 7 years and 12 days

Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Number of Participants With Serious and Non-serious Adverse Events	10 Participants	8 Participants	—

SECONDARY outcome

Timeframe: 30 days after last dose of LMB2

DLT is a grade II-IV LMB-2 or fludarabine and cyclophosphamide (FC)-related toxicity, except vascular leak syndrome, alopecia, grade II-III allergic reaction with asymptomatic bronchospasm or urticarial is considered DLT. Grade III aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase, and fever are not considered DLT. Grade IV creatine phosphokinase associated with any other DLT or not resolving to <grade II within 2 weeks is considered DLT. Hematologic toxicity is not considered DLT unless it fails to resolve to <grade 2 or baseline by day 18 after cycle 1 or after day 25 after cycles 2-7. DLT from hepatotoxicity, creatine phosphokinase, and vascular leak syndrome is assumed from LMB-2, and hematologic toxicity from fludarabine and cyclophosphamide. Grade III proteinuria lasting <2 weeks after the last dose of LMB-2 is not considered DLT, and needs to resolve to grade 0-2 prior to retreatment.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Number of Participants With Dose Limiting Toxicity (DLT)	0 Participants	0 Participants	—

SECONDARY outcome

Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Population: The number 8 represents the number of responders in the Arm/Group and the number 2 represents the number of non-responders in the Arm/Group.

Tumor tissue, including lymph node or skin biopsies was examined and analysis performed by flow cytometry to determine the amount of cancer cells in the body by measuring proteins which fall off cancer cells and go into the blood.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Soluble Cluster of Differentiation 25 (sCD25) Between Responders and Nonresponders Soluble CD25, lowest post-treatment (nadir) value	1094 pg/ml Interval 0.0 to 113000.0	34591 pg/ml Interval 5198.0 to 319000.0	—
Soluble Cluster of Differentiation 25 (sCD25) Between Responders and Nonresponders Median sCD25 Nadir for responders	1082 pg/ml Interval 0.0 to 5999.0	—	—
Soluble Cluster of Differentiation 25 (sCD25) Between Responders and Nonresponders Median sCD25 Nadir for non-responders	70713 pg/ml Interval 28425.0 to 113000.0	34591 pg/ml Interval 5198.0 to 319000.0	—
Soluble Cluster of Differentiation 25 (sCD25) Between Responders and Nonresponders Median sCD25 PRE-treatment for responders	31893 pg/ml Interval 3163.0 to 190000.0	—	—
Soluble Cluster of Differentiation 25 (sCD25) Between Responders and Nonresponders Median sCD25 PRE-treatment for non-responders	66419 pg/ml Interval 5838.0 to 127000.0	17079 pg/ml Interval 3668.0 to 122000.0	—

SECONDARY outcome

Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Population: Three participants were non-evaluable. One participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2. One participant had progressive disease before first cycle of LMB2, and one participant had progressive disease after first cycle of LMB2

AUC is a measure of the plasma concentration of drug over time. It is used to characterize drug absorption. Plasma levels were analyzed by cytotoxicity assay.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=5 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Area Under the Plasma Concentration (AUC) - LMB2	161 µg/-min/mL Interval 53.5 to 244.0	144 µg/-min/mL Interval 49.8 to 205.0	—

SECONDARY outcome

Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Population: One patient did not have flow cytometry measuring it.

Peripheral blood was obtained and analyzed by flow cytometry.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Post Treatment Effects of LMB-2 + Fludarabine and Cyclophosphamide (FC) on Normal B and T Cell Subsets by Flow Cytometry Normal T-cells	28 Cells/µL Interval 6.0 to 87.0	23.5 Cells/µL Interval 1.0 to 156.0	—
Post Treatment Effects of LMB-2 + Fludarabine and Cyclophosphamide (FC) on Normal B and T Cell Subsets by Flow Cytometry Normal CD4+ cells	99.5 Cells/µL Interval 4.0 to 427.0	186 Cells/µL Interval 1.0 to 3818.0	—
Post Treatment Effects of LMB-2 + Fludarabine and Cyclophosphamide (FC) on Normal B and T Cell Subsets by Flow Cytometry Normal CD8+ cells	28 Cells/µL Interval 6.0 to 87.0	23.5 Cells/µL Interval 1.0 to 156.0	—
Post Treatment Effects of LMB-2 + Fludarabine and Cyclophosphamide (FC) on Normal B and T Cell Subsets by Flow Cytometry Normal B-cells	8 Cells/µL Interval 0.0 to 119.0	0.5 Cells/µL Interval 0.0 to 11.0	—

SECONDARY outcome

Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Population: Three participants were non-evaluable. One participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2. One participant had progressive disease before first cycle of LMB2, and one participant had progressive disease after first cycle of LMB2

Blood was drawn prior to each cycle of LMB-2 to determine if the level, >75% neutralization of 1000ng/ml of LMB-2 of neutralizing antibodies is too high to give additional LMB-2. Analysis was performed by cytotoxicity assay.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=4 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Percentage of Patients Who Developed Neutralizing Antibodies After One or More Cycles of LMB-2	40 percentage of participants Interval 3.1 to 77.0	0 percentage of participants Interval 0.0 to 0.0	—

SECONDARY outcome

Timeframe: 69 months

Duration of response is defined as a response lasting for at least 4 weeks but >8 weeks and is assessed by the International Workshop to Standardize Response Criteria for Non-Hodgkin's Lymphoma. Complete remission (CR) is complete disappearance of all detectable clinical and radiographic evidence of disease and disappearance of all disease related symptoms if present before therapy and normalization of those biochemical abnormalities definitely assignable to the lymphoma. Partial response is reduction by ≥50% of leukemia cell count or ≥50% reduction in the size of all measurable lesions, and no increase in size of any measurable or evaluable lesion or appearance of new lesion.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Duration of Response (Complete Response + Partial Response)	69.6 Weeks Interval 8.0 to 263.0	0 Weeks Interval 0.0 to 0.0	—

SECONDARY outcome

Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Population: Three participants were non-evaluable. One participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2. One participant had progressive disease before first cycle of LMB2, and one participant had progressive disease after first cycle of LMB2

Dilutions of patient plasma was tested by cytotoxicity assays to determine plasma clearance of LMB-2.The CL is a quantitative measure of the rate at which a drug substance is removed from the body.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=5 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Plasma Clearance (CL) of LMB-2	109 mL/min Interval 10.9 to 134.0	101 mL/min Interval 13.5 to 191.0	—

SECONDARY outcome

Timeframe: 24 hours

Population: Three participants were non-evaluable. One participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2. One participant had progressive disease before first cycle of LMB2, and one participant had progressive disease after first cycle of LMB2

Dilutions of patient plasma were tested by cytotoxicity assays to determine volume of distribution of LMB-2. Volume distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. This was measured during the first 24 hours after administration of the dose on cycle 2 day 1.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=5 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Volume of Distribution of LMB-2	49.6 Liters Interval 3.3 to 313.0	26.6 Liters Interval 4.89 to 133.0	—

SECONDARY outcome

Timeframe: First 24 hours after the dose given on Cycle 2, day 1

Population: Three participants were non-evaluable. One participant withdrew due to inability to receive second cycle, which was the first cycle containing LMB-2. One participant had progressive disease before first cycle of LMB2, and one participant had progressive disease after first cycle of LMB2

Dilutions of patient plasma was tested by cytotoxicity assays to determine half life. Plasma decay half-life is the time measured for the plasma concentration of the drug to decrease by one half.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=5 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 Patients CF08 and CF09, both received LMB-2 40 µg/kg
Half Life (t1/2) of LMB-2	360 min Interval 105.0 to 542.0	251 min Interval 91.1 to 484.0	—

SECONDARY outcome

Timeframe: 7 years and 12 days

Population: Data for this outcome measure is reported as in the publication noted in the References module.

Adverse events is assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. Grade 3 (mild), Grade 4 (life threatening) and Grade 5 (death).

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=3 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=12 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 n=2 Participants Patients CF08 and CF09, both received LMB-2 40 µg/kg
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Mucositis	0 Participants	3 Participants	1 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Neutropenia	2 Participants	10 Participants	2 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Nausea/vomiting/anorexia	2 Participants	10 Participants	2 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Fever/chills	1 Participants	10 Participants	1 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Leukopenia/lymphopenia	1 Participants	9 Participants	2 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Transaminases	3 Participants	8 Participants	1 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Hypotension/tachycardia	1 Participants	8 Participants	2 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Thrombocytopenia	1 Participants	8 Participants	2 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Anemia	0 Participants	9 Participants	1 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Myalgia/headache	2 Participants	7 Participants	2 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Hypoalbuminemia	2 Participants	6 Participants	1 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Hematuria	1 Participants	5 Participants	1 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Proteinuria	0 Participants	7 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Fatigue/dizziness	1 Participants	4 Participants	2 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Edema	0 Participants	6 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Abdominal pain	0 Participants	5 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Diarrhea	1 Participants	3 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Creatine phosphokinase (CPK)	0 Participants	4 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Hypomagnesemia	0 Participants	3 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Bilirubin	1 Participants	2 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Alkaline phosphatase/gamma-glutamyl transferase	0 Participants	3 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Pneumonitis	0 Participants	3 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Low cluster of differentiation 4 (CD4) count	1 Participants	1 Participants	1 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Lipase	0 Participants	3 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Bladder infection	0 Participants	2 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Dyspnea	0 Participants	2 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Prothrombin time	1 Participants	1 Participants	0 Participants
Count of Participants With Grades 3-5 Adverse Events of > 1 Adult T-Cell Leukemia (ATL) Patients Treated With 20+200, 25+250, and 30+300 mg/m^2 LMB-2/Fludarabine and Cyclophosphamide Pruritis	1 Participants	0 Participants	1 Participants

SECONDARY outcome

Timeframe: 7 years and 12 days

Population: \*Grade 5 (death) event. Data for this outcome measure is reported as in the publication noted in the References module.

Adverse events is assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=3 Participants Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=12 Participants Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).	Fludarabine and Cyclophosphamide: 30 + 300mg/m^2 n=2 Participants Patients CF08 and CF09, both received LMB-2 40 µg/kg
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Neutropenia	1 Participants	9 Participants	2 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Leukopenia/lymphopenia	1 Participants	9 Participants	2 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Anemia	0 Participants	8 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Transaminases	1 Participants	4 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Thrombocytopenia	0 Participants	5 Participants	1 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Fever/chills	0 Participants	3 Participants	1 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Pneumonitis	0 Participants	3 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Low cluster of differentiation 4 (CD4) count	1 Participants	1 Participants	1 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Hypotension/tachycardia	0 Participants	2 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Rectal hemorrhage	0 Participants	1 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Bilirubin	0 Participants	1 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Dysuria	0 Participants	1 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Bladder infection	0 Participants	1 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Hypoxia	0 Participants	1 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Proteinuria	0 Participants	1 Participants	0 Participants
Count of Participants With Adverse Events Attributed At Least Possibly to Patients Treated With Fludarabine and Cyclophosphamide Dose Levels 20+200, 25+250, and 30+300 mg/m^2 Sepsis*	0 Participants	1 Participants	0 Participants

Adverse Events

Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC

Serious events: 7 serious events

Other events: 10 other events

Deaths: 8 deaths

All Other Patients

Serious events: 6 serious events

Other events: 8 other events

Deaths: 6 deaths

Serious adverse events

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 participants at risk Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 participants at risk Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).
Metabolism and nutrition disorders Acidosis	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Renal and urinary disorders Acute kidney injury	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Cardiac disorders Cardiac arrest	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Metabolism and nutrition disorders Dehydration	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Nervous system disorders Depressed level of consciousness	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Nervous system disorders Dizziness	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Dyspnea	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
General disorders Edema limbs	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Injury, poisoning and procedural complications Fall	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Metabolism and nutrition disorders Hypercalcemia	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Hypoxia	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Lung infection	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Nervous system disorders Presyncope	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Sepsis	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
General disorders Death related to disease	70.0% 7/10 • Number of events 7 • 7 years and 12 days	75.0% 6/8 • Number of events 6 • 7 years and 12 days

Other adverse events

Measure	Leukemic Patients Receiving LMB-2 & At Least 25+250 mg/m^2 FC n=10 participants at risk Leukemic patients receiving LMB-2 and at least 25+250 mg/m\^2 fludarabine and cyclophosphamide; CF03-CF05, CF07-CF10, and CF12-CF14.	All Other Patients n=8 participants at risk Patients CF01-CF02, CF06, CF11, and CF15-CF18. This is a mixture of patients who did not receive LMB-2, who had lymphoma-type adult T-cell leukemia, and who received 20+250 mg/m\^2 of fludarabine cyclophosphamide (FC).
Gastrointestinal disorders Abdominal pain	50.0% 5/10 • Number of events 7 • 7 years and 12 days	37.5% 3/8 • Number of events 4 • 7 years and 12 days
Investigations Activated partial thromboplastin time prolonged	10.0% 1/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Psychiatric disorders Agitation	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Alanine aminotransferase increased	50.0% 5/10 • Number of events 20 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Investigations Alkaline phosphatase increased	70.0% 7/10 • Number of events 9 • 7 years and 12 days	37.5% 3/8 • Number of events 4 • 7 years and 12 days
Immune system disorders Allergic reaction	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Allergic rhinitis	20.0% 2/10 • Number of events 2 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Blood and lymphatic system disorders Anemia	80.0% 8/10 • Number of events 108 • 7 years and 12 days	62.5% 5/8 • Number of events 16 • 7 years and 12 days
Metabolism and nutrition disorders Anorexia	50.0% 5/10 • Number of events 6 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Psychiatric disorders Anxiety	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Arthralgia	10.0% 1/10 • Number of events 1 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Investigations Aspartate aminotransferase increased	70.0% 7/10 • Number of events 44 • 7 years and 12 days	62.5% 5/8 • Number of events 7 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Back pain	40.0% 4/10 • Number of events 5 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Gastrointestinal disorders Bloating	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Investigations Blood bilirubin increased	50.0% 5/10 • Number of events 10 • 7 years and 12 days	37.5% 3/8 • Number of events 3 • 7 years and 12 days
Blood and lymphatic system disorders Blood and lymphatic system disorders - Other,Prothrombin time prolonged	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Eye disorders Blurred vision	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Bone pain	30.0% 3/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Buttock pain	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations CD4 lymphocytes decreased	20.0% 2/10 • Number of events 6 • 7 years and 12 days	12.5% 1/8 • Number of events 2 • 7 years and 12 days
Investigations CPK increased	50.0% 5/10 • Number of events 6 • 7 years and 12 days	37.5% 3/8 • Number of events 4 • 7 years and 12 days
Vascular disorders Capillary leak syndrome	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Carbon monoxide diffusing capacity decreased	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Cardiac troponin I increased	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Catheter related infection	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Chest wall pain	40.0% 4/10 • Number of events 5 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
General disorders Chills	50.0% 5/10 • Number of events 10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Psychiatric disorders Confusion	20.0% 2/10 • Number of events 3 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Eye disorders Conjunctivitis	20.0% 2/10 • Number of events 3 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Gastrointestinal disorders Constipation	40.0% 4/10 • Number of events 6 • 7 years and 12 days	25.0% 2/8 • Number of events 3 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Cough	30.0% 3/10 • Number of events 3 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Investigations Creatinine increased	0.00% 0/10 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Metabolism and nutrition disorders Dehydration	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Psychiatric disorders Depression	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Gastrointestinal disorders Diarrhea	50.0% 5/10 • Number of events 10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Nervous system disorders Dizziness	50.0% 5/10 • Number of events 7 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Eye disorders Dry eye	20.0% 2/10 • Number of events 2 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Skin and subcutaneous tissue disorders Dry skin	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Gastrointestinal disorders Dyspepsia	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Gastrointestinal disorders Dysphagia	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Nervous system disorders Dysphasia	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Dyspnea	40.0% 4/10 • Number of events 7 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Ear and labyrinth disorders Ear pain	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
General disorders Edema face	40.0% 4/10 • Number of events 8 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
General disorders Edema limbs	70.0% 7/10 • Number of events 12 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Cardiac disorders Electrocardiogram QT corrected interval prolonged	20.0% 2/10 • Number of events 3 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Nervous system disorders Encephalopathy	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Epistaxis	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Ear and labyrinth disorders External ear inflammation	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Eye disorders Eye pain	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
General disorders Fatigue	50.0% 5/10 • Number of events 7 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Blood and lymphatic system disorders Febrile neutropenia	30.0% 3/10 • Number of events 6 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
General disorders Fever	100.0% 10/10 • Number of events 23 • 7 years and 12 days	87.5% 7/8 • Number of events 7 • 7 years and 12 days
Investigations Fibrinogen decreased	20.0% 2/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Eye disorders Floaters	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations GGT increased	40.0% 4/10 • Number of events 5 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
General disorders Gait disturbance	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Haptoglobin decreased	20.0% 2/10 • Number of events 2 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Nervous system disorders Headache	70.0% 7/10 • Number of events 16 • 7 years and 12 days	37.5% 3/8 • Number of events 5 • 7 years and 12 days
Renal and urinary disorders Hematuria	50.0% 5/10 • Number of events 10 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Investigations Hemoglobin increased	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Vascular disorders Hot flashes	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Metabolism and nutrition disorders Hypercalcemia	30.0% 3/10 • Number of events 9 • 7 years and 12 days	37.5% 3/8 • Number of events 4 • 7 years and 12 days
Metabolism and nutrition disorders Hyperglycemia	20.0% 2/10 • Number of events 6 • 7 years and 12 days	37.5% 3/8 • Number of events 4 • 7 years and 12 days
Metabolism and nutrition disorders Hyperkalemia	30.0% 3/10 • Number of events 4 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Metabolism and nutrition disorders Hypermagnesemia	30.0% 3/10 • Number of events 5 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Metabolism and nutrition disorders Hypernatremia	30.0% 3/10 • Number of events 9 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Vascular disorders Hypertension	10.0% 1/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Metabolism and nutrition disorders Hypertriglyceridemia	30.0% 3/10 • Number of events 4 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Metabolism and nutrition disorders Hyperuricemia	20.0% 2/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Metabolism and nutrition disorders Hypoalbuminemia	70.0% 7/10 • Number of events 36 • 7 years and 12 days	62.5% 5/8 • Number of events 6 • 7 years and 12 days
Metabolism and nutrition disorders Hypocalcemia	50.0% 5/10 • Number of events 39 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Metabolism and nutrition disorders Hypoglycemia	20.0% 2/10 • Number of events 2 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Metabolism and nutrition disorders Hypokalemia	40.0% 4/10 • Number of events 52 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Metabolism and nutrition disorders Hypomagnesemia	60.0% 6/10 • Number of events 22 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Metabolism and nutrition disorders Hyponatremia	80.0% 8/10 • Number of events 33 • 7 years and 12 days	50.0% 4/8 • Number of events 4 • 7 years and 12 days
Metabolism and nutrition disorders Hypophosphatemia	60.0% 6/10 • Number of events 24 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Vascular disorders Hypotension	90.0% 9/10 • Number of events 15 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Endocrine disorders Hypothyroidism	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Hypoxia	20.0% 2/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations INR increased	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Infections and infestations - Other, CMV reaction	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Psychiatric disorders Insomnia	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Investigations - Other, Bicarbonate, low; Bicarbonate, serum-low	10.0% 1/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Lipase increased	20.0% 2/10 • Number of events 2 • 7 years and 12 days	37.5% 3/8 • Number of events 3 • 7 years and 12 days
Infections and infestations Lung infection	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Lymphocyte count decreased	90.0% 9/10 • Number of events 136 • 7 years and 12 days	62.5% 5/8 • Number of events 22 • 7 years and 12 days
Nervous system disorders Memory impairment	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Metabolism and nutrition disorders Metabolism and nutrition disorders - Other, Bicarbonate, Low	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Mucosal infection	40.0% 4/10 • Number of events 4 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Gastrointestinal disorders Mucositis oral	30.0% 3/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Myalgia	80.0% 8/10 • Number of events 16 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Myositis	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Gastrointestinal disorders Nausea	80.0% 8/10 • Number of events 23 • 7 years and 12 days	75.0% 6/8 • Number of events 10 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Neck pain	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Neutrophil count decreased	90.0% 9/10 • Number of events 83 • 7 years and 12 days	62.5% 5/8 • Number of events 23 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Non-cardiac chest pain	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Gastrointestinal disorders Oral pain	10.0% 1/10 • Number of events 1 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
General disorders Pain	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 3 • 7 years and 12 days
Musculoskeletal and connective tissue disorders Pain in extremity	40.0% 4/10 • Number of events 12 • 7 years and 12 days	37.5% 3/8 • Number of events 4 • 7 years and 12 days
Eye disorders Papilledema	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Nervous system disorders Paresthesia	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Pharyngolaryngeal pain	30.0% 3/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Skin and subcutaneous tissue disorders Photosensitivity	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Platelet count decreased	100.0% 10/10 • Number of events 100 • 7 years and 12 days	50.0% 4/8 • Number of events 6 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Pleural effusion	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Nervous system disorders Presyncope	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Renal and urinary disorders Proteinuria	60.0% 6/10 • Number of events 7 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Skin and subcutaneous tissue disorders Pruritus	60.0% 6/10 • Number of events 8 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Skin and subcutaneous tissue disorders Rash maculo-papular	50.0% 5/10 • Number of events 9 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Gastrointestinal disorders Rectal hemorrhage	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Rhinitis infective	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Nervous system disorders Seizure	10.0% 1/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Sepsis	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations Serum amylase increased	40.0% 4/10 • Number of events 5 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Nervous system disorders Sinus pain	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Cardiac disorders Sinus tachycardia	40.0% 4/10 • Number of events 6 • 7 years and 12 days	25.0% 2/8 • Number of events 2 • 7 years and 12 days
Infections and infestations Sinusitis	30.0% 3/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other,Dermatology/Skin: Rash	10.0% 1/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Skin infection	10.0% 1/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Skin and subcutaneous tissue disorders Skin ulceration	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Nervous system disorders Somnolence	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Nervous system disorders Syncope	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Tooth infection	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Nervous system disorders Tremor	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Renal and urinary disorders Urinary incontinence	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Infections and infestations Urinary tract infection	30.0% 3/10 • Number of events 7 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Renal and urinary disorders Urinary tract pain	30.0% 3/10 • Number of events 3 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Eye disorders Uveitis	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Reproductive system and breast disorders Vaginal inflammation	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Injury, poisoning and procedural complications Vascular access complication	10.0% 1/10 • Number of events 1 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Gastrointestinal disorders Vomiting	60.0% 6/10 • Number of events 17 • 7 years and 12 days	37.5% 3/8 • Number of events 6 • 7 years and 12 days
Investigations Weight gain	20.0% 2/10 • Number of events 2 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Respiratory, thoracic and mediastinal disorders Wheezing	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Investigations White blood cell decreased	80.0% 8/10 • Number of events 155 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days
Injury, poisoning and procedural complications Wound dehiscence	10.0% 1/10 • Number of events 1 • 7 years and 12 days	62.5% 5/8 • Number of events 22 • 7 years and 12 days
Infections and infestations Infections and Infestations - Other, Bladder	0.00% 0/10 • 7 years and 12 days	12.5% 1/8 • Number of events 1 • 7 years and 12 days
Infections and infestations Infections and infestations - Other, tooth infection	10.0% 1/10 • Number of events 1 • 7 years and 12 days	0.00% 0/8 • 7 years and 12 days

Additional Information

Dr. Robert Kreitman

National Cancer Institute

Phone: 301-496-6947

Email: Robert_Kreitman@nih.gov

Results disclosure agreements

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