Trial Outcomes & Findings for Phase II Study of the Efficacy and Toxicity of Ontak(Registered Trademark) (Denileukin Diftitox) in the Therapy of Adult T-Cell Leukemia (NCT NCT00117845)

Last Updated: 2019-11-19

Results Overview

Response rate is based on the number of patients who achieve either a complete response (CR) or partial response (PR) to therapy. Complete response 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 (for example LDH) definitely assignable to the lymphoma. Partial response is reduction by \>=50% of leukemia cell count or \>=50% reduction is the size of all measurable lesions, and no increase in size of any measurable or evaluable lesion or appearance of new lesion.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

up to 12 months

Results posted on

2019-11-19

Participant Flow

Participant milestones

Participant milestones
Measure	Denileukin Diftitox in ATL Denileukin Diftitox in adult T-cell leukemia (ATL) Patients will be treated with Denileukin Diftitox 9 mcg/kg/d intravenously for 5 days every 2 weeks.
Overall Study STARTED	17
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Denileukin Diftitox in ATL Denileukin Diftitox in adult T-cell leukemia (ATL) Patients will be treated with Denileukin Diftitox 9 mcg/kg/d intravenously for 5 days every 2 weeks.
Overall Study stopped treatment/drug supplyinterrupted	1

Baseline Characteristics

Phase II Study of the Efficacy and Toxicity of Ontak(Registered Trademark) (Denileukin Diftitox) in the Therapy of Adult T-Cell Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Denileukin Diftitox in ATL n=17 Participants Denileukin Diftitox in adult T-cell leukemia (ATL) Patients will be treated with Denileukin Diftitox 9 mcg/kg/d intravenously for 5 days every 2 weeks.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	15 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	49.46 years STANDARD_DEVIATION 11.64 • n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	16 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	17 Participants n=5 Participants

PRIMARY outcome

Timeframe: up to 12 months

Outcome measures

Outcome measures
Measure	Denileukin Diftitox in ATL n=17 Participants Denileukin Diftitox in adult T-cell leukemia (ATL) Patients will be treated with Denileukin Diftitox 9 mcg/kg/d intravenously for 5 days every 2 weeks.
Response Rate Complete Response	0 Participants
Response Rate Partial Response	0 Participants

SECONDARY outcome

Timeframe: Date treatment consent signed to date off study, approximately 72 months

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v3.0). 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

Outcome measures
Measure	Denileukin Diftitox in ATL n=17 Participants Denileukin Diftitox in adult T-cell leukemia (ATL) Patients will be treated with Denileukin Diftitox 9 mcg/kg/d intravenously for 5 days every 2 weeks.
Number of Participants With Serious and Non-Serious Adverse Events	17 Participants

Adverse Events

Denileukin Diftitox in ATL

Serious events: 5 serious events

Other events: 17 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Denileukin Diftitox in ATL n=17 participants at risk Denileukin Diftitox in adult T-cell leukemia (ATL) Patients will be treated with Denileukin Diftitox 9 mcg/kg/d intravenously for 5 days every 2 weeks.
Metabolism and nutrition disorders ALT, SGPT (serum glutamic pyruvic transaminase)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders AST, SGOT(serum glutamic oxaloacetic transaminase)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Alkaline phosphatase	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Dehydration	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Cardiac disorders Hypotension	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Infections and infestations Infection (documented clinically or microbiologically)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Respiratory, thoracic and mediastinal disorders Obstruction/stenosis of airway::Larynx	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Perforation, GI::Duodenum	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Rigors/chills	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Vascular disorders Thrombosis/thrombus/embolism	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.

Other adverse events

Other adverse events
Measure	Denileukin Diftitox in ATL n=17 participants at risk Denileukin Diftitox in adult T-cell leukemia (ATL) Patients will be treated with Denileukin Diftitox 9 mcg/kg/d intravenously for 5 days every 2 weeks.
Metabolism and nutrition disorders ALT, SGPT (serum glutamic pyruvic transaminase)	58.8% 10/17 • Number of events 39 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders AST, SGOT(serum glutamic oxaloacetic transaminase)	70.6% 12/17 • Number of events 48 • Date treatment consent signed to date off study, approximately 72 months.
Vascular disorders Acute vascular leak syndrome	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Albumin, serum-low (hypoalbuminemia)	70.6% 12/17 • Number of events 36 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Alkaline phosphatase	52.9% 9/17 • Number of events 20 • Date treatment consent signed to date off study, approximately 72 months.
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Immune system disorders Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Amylase	11.8% 2/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Anorexia	23.5% 4/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Bicarbonate, serum-low	17.6% 3/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Bilirubin (hyperbilirubinemia)	23.5% 4/17 • Number of events 16 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders CPK (creatine phosphokinase)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Calcium, serum-high (hypercalcemia)	47.1% 8/17 • Number of events 9 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Calcium, serum-low (hypocalcemia)	23.5% 4/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 72 months.
Cardiac disorders Cardiac troponin I (cTnT)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Colitis	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Nervous system disorders Confusion	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Respiratory, thoracic and mediastinal disorders Cough	17.6% 3/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Creatinine	23.5% 4/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Dehydration	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Diarrhea	5.9% 1/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
Nervous system disorders Dizziness	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
Respiratory, thoracic and mediastinal disorders Dyspnea	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Blood and lymphatic system disorders Edema: limb	35.3% 6/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Fatigue (asthenia, lethargy, malaise)	29.4% 5/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 72 months.
Infections and infestations Febrile neutropenia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)	52.9% 9/17 • Number of events 27 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders GGT (gamma-Glutamyl transpeptidase)	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Glucose, serum-low (hyperglycemia)	29.4% 5/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Glucose, serum-low (hypoglycemia)	11.8% 2/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Heartburn/dyspepsia	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Blood and lymphatic system disorders Hemoglobin	82.4% 14/17 • Number of events 44 • Date treatment consent signed to date off study, approximately 72 months.
Cardiac disorders Hypertension	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Cardiac disorders Hypotension	23.5% 4/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 72 months.
Respiratory, thoracic and mediastinal disorders Hypoxia	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Infections and infestations Infection	5.9% 1/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
Infections and infestations Infection - Other (Specify, infection with neutropenia)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils::Blood	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils::Colon	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils::Eye NOS	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils::Skin (cellulitis)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils::Urinary tract NOS	11.8% 2/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Insomnia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Blood and lymphatic system disorders Leukocytes (total WBC)	47.1% 8/17 • Number of events 19 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Lipase	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Blood and lymphatic system disorders Lymphopenia	82.4% 14/17 • Number of events 56 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Magnesium, serum-high (hypermagnesemia)	23.5% 4/17 • Number of events 5 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Magnesium, serum-low (hypomagnesemia)	35.3% 6/17 • Number of events 10 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Metabolic/Laboratory - Other (Specify, hypokalemia)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Nervous system disorders Mood alteration::Anxiety	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Mucositis/stomatitis (clinical exam)::Oral cavity	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Respiratory, thoracic and mediastinal disorders Nasal cavity/paranasal sinus reactions	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Nausea	35.3% 6/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 72 months.
Nervous system disorders Neuropathy: cranial::CN I Smell	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Nervous system disorders Neuropathy: sensory	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC)	52.9% 9/17 • Number of events 17 • Date treatment consent signed to date off study, approximately 72 months.
Blood and lymphatic system disorders PTT (Partial Thromboplastin Time)	41.2% 7/17 • Number of events 9 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Pain - Other (Specify, Rt lumbar radiculopathy)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Pain::Abdomen NOS	5.9% 1/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
Musculoskeletal and connective tissue disorders Pain::Back	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Respiratory, thoracic and mediastinal disorders Pain::Chest/thorax NOS	5.9% 1/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
Musculoskeletal and connective tissue disorders Pain::Extremity-limb	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Nervous system disorders Pain::Head/headache	41.2% 7/17 • Number of events 9 • Date treatment consent signed to date off study, approximately 72 months.
Musculoskeletal and connective tissue disorders Pain::Joint	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
Respiratory, thoracic and mediastinal disorders Pain::Larynx	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Musculoskeletal and connective tissue disorders Pain::Neck	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Pain::Tumor pain	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Cardiac disorders Palpitations	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Phosphate, serum-low (hypophosphatemia)	29.4% 5/17 • Number of events 11 • Date treatment consent signed to date off study, approximately 72 months.
Blood and lymphatic system disorders Platelets	52.9% 9/17 • Number of events 13 • Date treatment consent signed to date off study, approximately 72 months.
Respiratory, thoracic and mediastinal disorders Pneumonitis/pulmonary infiltrates	5.9% 1/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Potassium, serum-high (hyperkalemia)	17.6% 3/17 • Number of events 3 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Potassium, serum-low (hypokalemia)	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Proteinuria	23.5% 4/17 • Number of events 6 • Date treatment consent signed to date off study, approximately 72 months.
Blood and lymphatic system disorders Prothrombin time (PT)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Skin and subcutaneous tissue disorders Pruritus/itching	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Respiratory, thoracic and mediastinal disorders Pulmonary/Upper Respiratory - Other (Specify,)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Skin and subcutaneous tissue disorders Rash/desquamation	23.5% 4/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Rigors/chills	41.2% 7/17 • Number of events 10 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Sodium, serum-high (hypernatremia)	17.6% 3/17 • Number of events 4 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Sodium, serum-low (hyponatremia)	47.1% 8/17 • Number of events 14 • Date treatment consent signed to date off study, approximately 72 months.
Cardiac disorders Supraventricular and nodal arrhythmia::Sinus bradycardia	5.9% 1/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
Cardiac disorders Supraventricular and nodal arrhythmia::Sinus tachycardia	41.2% 7/17 • Number of events 11 • Date treatment consent signed to date off study, approximately 72 months.
Cardiac disorders Supraventricular and nodal arrhythmia::Supraventricular tachycardia	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Skin and subcutaneous tissue disorders Sweating (diaphoresis)	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Vascular disorders Thrombosis/thrombus/embolism	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Nervous system disorders Tremor	5.9% 1/17 • Number of events 1 • Date treatment consent signed to date off study, approximately 72 months.
Metabolism and nutrition disorders Uric acid, serum-high (hyperuricemia)	23.5% 4/17 • Number of events 8 • Date treatment consent signed to date off study, approximately 72 months.
Gastrointestinal disorders Vomiting	41.2% 7/17 • Number of events 9 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Weight gain	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.
General disorders Weight loss	11.8% 2/17 • Number of events 2 • Date treatment consent signed to date off study, approximately 72 months.

Additional Information

Thomas Waldmann, M.D.

National Cancer Institute, National Institutes of Health

Phone: 301-496-6656

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place