Trial Outcomes & Findings for LMB-2 to Treat Hairy Cell Leukemia (NCT NCT00321555)
NCT ID: NCT00321555
Last Updated: 2023-11-14
Results Overview
Partial response requires all of the following for a period of at least 4 weeks: ≥50% decrease in hairy cell count from pretreatment baseline value by flow cytometry. ≥50% reduction in abnormal hepatosplenomegaly by computed tomography or physical exam. No growth in lymphadenopathy. Neutrophils ≥1,500/µL or 50% improvement over baseline without growth factors for at least 4 weeks. Platelets ≥100,000/µL or 50% improvement over baseline. Complete remission requires all of the following: no evidence of leukemic cells by routine hematoxylin and eosin stains of the peripheral blood and bone marrow. No hepatomegaly, splenomegaly, or lymphadenopathy (not \>2 cm in short axis) by physical examination and appropriate radiographic techniques. Normal complete blood count as exhibited by Neutrophils ≥1,500/µL, Platelets ≥100,000/µL, and Hemoglobin ≥11.0g/dL without transfusions or growth factors for at least 4 weeks.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
6 months
Results posted on
2023-11-14
Participant Flow
Participant milestones
| Measure |
LMB-2 to Treat Hairy Cell Leukemia
LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin: LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium Chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
LMB-2 to Treat Hairy Cell Leukemia
LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin: LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium Chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
|
|---|---|
|
Overall Study
Developed antibodies
|
1
|
|
Overall Study
Switched to alternative treatment
|
3
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Disease progression on study
|
1
|
Baseline Characteristics
LMB-2 to Treat Hairy Cell Leukemia
Baseline characteristics by cohort
| Measure |
LMB-2 to Treat Hairy Cell Leukemia
n=15 Participants
LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin: LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium Chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
7 Participants
n=5 Participants
|
|
Age, Continuous
|
63.49 years
STANDARD_DEVIATION 9.29 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
14 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
|
15 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPartial response requires all of the following for a period of at least 4 weeks: ≥50% decrease in hairy cell count from pretreatment baseline value by flow cytometry. ≥50% reduction in abnormal hepatosplenomegaly by computed tomography or physical exam. No growth in lymphadenopathy. Neutrophils ≥1,500/µL or 50% improvement over baseline without growth factors for at least 4 weeks. Platelets ≥100,000/µL or 50% improvement over baseline. Complete remission requires all of the following: no evidence of leukemic cells by routine hematoxylin and eosin stains of the peripheral blood and bone marrow. No hepatomegaly, splenomegaly, or lymphadenopathy (not \>2 cm in short axis) by physical examination and appropriate radiographic techniques. Normal complete blood count as exhibited by Neutrophils ≥1,500/µL, Platelets ≥100,000/µL, and Hemoglobin ≥11.0g/dL without transfusions or growth factors for at least 4 weeks.
Outcome measures
| Measure |
LMB-2 to Treat Hairy Cell Leukemia
n=15 Participants
LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin: LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium Chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
|
|---|---|
|
Number of Patients Who Obtain Partial Response/Complete Remission
Complete Remission
|
1 Participants
|
|
Number of Patients Who Obtain Partial Response/Complete Remission
Partial Response
|
5 Participants
|
SECONDARY outcome
Timeframe: Participants were assessed at 5.0658, 12.0066, 20.1645, 26.9079, 35.0987, and 45.1316 monthsDuration of response is the duration that the patients qualifies for at least a Partial Response (PR). Partial response requires all of the following for a period of at least 4 weeks: ≥50% decrease in hairy cell count from pretreatment baseline value by flow cytometry. ≥50% reduction in abnormal hepatosplenomegaly by computed tomography or physical exam. No growth in lymphadenopathy. Neutrophils ≥1,500/µL or 50% improvement over baseline without growth factors for at least 4 weeks. Platelets ≥100,000/µL or 50% improvement over baseline. Hemoglobin ≥11.0 g/dL or 50% improvement over baseline without transfusions or growth factors for at least 4 weeks.
Outcome measures
| Measure |
LMB-2 to Treat Hairy Cell Leukemia
n=6 Participants
LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin: LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium Chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
|
|---|---|
|
Duration of Response
|
23.5 months
Interval 5.1 to 45.1
|
SECONDARY outcome
Timeframe: Date treatment consent signed to date off study, approximately 83 months and 15 days.Here is the count 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 |
LMB-2 to Treat Hairy Cell Leukemia
n=15 Participants
LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin: LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium Chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
|
|---|---|
|
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)
|
13 Participants
|
Adverse Events
LMB-2 to Treat Hairy Cell Leukemia
Serious events: 4 serious events
Other events: 13 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
LMB-2 to Treat Hairy Cell Leukemia
n=15 participants at risk
LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin: LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium Chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
|
|---|---|
|
Immune system disorders
Allergic reaction
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Immune system disorders
Anaphylaxis
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
General disorders
Death NOS
|
13.3%
2/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Nervous system disorders
Vasovagal reaction
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
White blood cell decreased
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
Other adverse events
| Measure |
LMB-2 to Treat Hairy Cell Leukemia
n=15 participants at risk
LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
Anti-Tac(Fv)-PE38 (LMB-2) Immunotoxin: LMB-2 Infusion: 40 micro-g/Kg will be infused in 50 ml of 0.9% Sodium Chloride (NaCl) and 0.2% albumin via over 30 minutes every other day for 3 doses. Patients may receive up to six treatment cycles every 4 weeks.
|
|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
13.3%
2/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Alanine aminotransferase increased
|
26.7%
4/15 • Number of events 9 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Immune system disorders
Allergic reaction
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
6.7%
1/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Blood and lymphatic system disorders
Anemia
|
33.3%
5/15 • Number of events 12 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Metabolism and nutrition disorders
Anorexia
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Aspartate aminotransferase increased
|
33.3%
5/15 • Number of events 11 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Cardiac disorders
Atrial fibrillation
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
13.3%
2/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Blood bilirubin increased
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
CD4 lymphocytes decreased
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Vascular disorders
Capillary leak syndrome
|
6.7%
1/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
General disorders
Chills
|
6.7%
1/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Psychiatric disorders
Confusion
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Gastrointestinal disorders
Constipation
|
6.7%
1/15 • Number of events 3 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.3%
2/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Creatinine increased
|
6.7%
1/15 • Number of events 3 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Gastrointestinal disorders
Diarrhea
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Nervous system disorders
Dizziness
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Gastrointestinal disorders
Dyspepsia
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
20.0%
3/15 • Number of events 3 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
General disorders
Edema face
|
13.3%
2/15 • Number of events 4 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
General disorders
Edema limbs
|
46.7%
7/15 • Number of events 9 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
General disorders
Fatigue
|
20.0%
3/15 • Number of events 3 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
General disorders
Fever
|
20.0%
3/15 • Number of events 6 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Haptoglobin decreased
|
13.3%
2/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Nervous system disorders
Headache
|
26.7%
4/15 • Number of events 4 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Renal and urinary disorders
Hematuria
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Renal and urinary disorders
Hemoglobinuria
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
13.3%
2/15 • Number of events 6 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
13.3%
2/15 • Number of events 4 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
6.7%
1/15 • Number of events 3 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Vascular disorders
Hypertension
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
60.0%
9/15 • Number of events 26 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
6.7%
1/15 • Number of events 3 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
6.7%
1/15 • Number of events 5 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
33.3%
5/15 • Number of events 9 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Vascular disorders
Hypotension
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Psychiatric disorders
Insomnia
|
6.7%
1/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Investigations - Other, Bicarbonate serum low
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Cardiac disorders
Left ventricular systolic dysfunction
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Lymphocyte count decreased
|
33.3%
5/15 • Number of events 12 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
26.7%
4/15 • Number of events 4 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Gastrointestinal disorders
Nausea
|
33.3%
5/15 • Number of events 10 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Neutrophil count decreased
|
20.0%
3/15 • Number of events 4 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Musculoskeletal and connective tissue disorders
Non-cardiac chest pain
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
General disorders
Pain
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
13.3%
2/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Cardiac disorders
Palpitations
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Cardiac disorders
Paroxysmal atrial tachycardia
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Cardiac disorders
Pericardial effusion
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Platelet count decreased
|
26.7%
4/15 • Number of events 10 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Renal and urinary disorders
Proteinuria
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
13.3%
2/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Nervous system disorders
Syncope
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Infections and infestations
Upper respiratory infection
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Vascular disorders
Vascular disorders - Other, Vascular leak syndrome
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Nervous system disorders
Vasovagal reaction
|
6.7%
1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Gastrointestinal disorders
Vomiting
|
13.3%
2/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
Weight gain
|
26.7%
4/15 • Number of events 5 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
|
Investigations
White blood cell decreased
|
13.3%
2/15 • Number of events 2 • Date treatment consent signed to date off study, approximately 83 months and 15 days.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place