Trial Outcomes & Findings for Retreatment Protocol for BL22 Immunotherapy in Relapsed or Refractory Hairy Cell Leukemia (NCT NCT00924040)
NCT ID: NCT00924040
Last Updated: 2015-10-29
Results Overview
Response is defined by the Response Evaluation Criteria in the protocol, namely the earliest point where all relevant tests (i.e. lab tests, physical exam, radiology results) are consistent with complete response (CR) or partial response (PR). CR or PR must be confirmed for at least 4 weeks. Complete response: No evidence of leukemic cells by routine H/E stains of the peripheral blood and bone marrow. Partial response:neutrophils \>/= 1,500/micrograms/L or 50% improvement over baseline without growth factors for at least 4 weeks.
TERMINATED
PHASE2
1 participants
2/14/2009 till 6/24/2010
2015-10-29
Participant Flow
The study was designed to enroll 21 patients with an expected accrual of 2-3 patients every 2-3 months. Accrual ceiling was 25 patients.
Participant milestones
| Measure |
BL22 Immunotherapy
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Retreatment Protocol for BL22 Immunotherapy in Relapsed or Refractory Hairy Cell Leukemia
Baseline characteristics by cohort
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
65.2 years
STANDARD_DEVIATION 0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 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
|
0 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
|
1 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
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2/14/2009 till 6/24/2010Response is defined by the Response Evaluation Criteria in the protocol, namely the earliest point where all relevant tests (i.e. lab tests, physical exam, radiology results) are consistent with complete response (CR) or partial response (PR). CR or PR must be confirmed for at least 4 weeks. Complete response: No evidence of leukemic cells by routine H/E stains of the peripheral blood and bone marrow. Partial response:neutrophils \>/= 1,500/micrograms/L or 50% improvement over baseline without growth factors for at least 4 weeks.
Outcome measures
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Number of Months to Response to Treatment
|
2 Months
|
SECONDARY outcome
Timeframe: 2 years & 6 monthsHere are the number of participants with adverse events. For the detailed list of adverse events see the adverse event module.
Outcome measures
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Number of Participants With Adverse Events
|
1 Participants
|
SECONDARY outcome
Timeframe: Bone marrow biopsy and MRI 4 weeks after patients meeting blood criteria for CR, and if CR is present, repeat bone marrow biopsy and MRI every 12 months. Bone marrow biopsy and MRI is not done in patients with PR as best response.Patients are assessed by MRI to determine which ones resolve their marrow abnormality. A non-parametric Wilcoxon test was to be used to determine whether CR correlated with resolution of MRI abnormality
Outcome measures
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Number of Participants With Complete Response (CR) Who Resolve the Bone Marrow Abnormality by Magnetic Resonance Imaging (MRI)
|
0 Participants
|
SECONDARY outcome
Timeframe: 24 weeksFresh malignant cells are isolated from blood, bone marrow, lymph nodes or other tissue and incubated with recombinant immunotoxins to determine sensitivity to BL22 and other agents to estimate the amount of cancer cells in the body by measuring proteins which fall off cancer cells and go into the blood.
Outcome measures
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Number of Patients Who Developed Neutralizing Antibodies After One or More Cycles of BL22
|
0 Participants
|
SECONDARY outcome
Timeframe: Time to CR can be between 2 months and 1 yearAlthough some hairy cell leukemia (HCL) cells from some patients may have ex vivo sensitivity, they might not respond clinically. Number of participants with pretreatment ex vivo sensitivity (\<10 ng/ml IC50) who go on to achieve CR as best response. CR required abscence of HCL in the bone marrow and resolution of cytopenias.
Outcome measures
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Number of Patients With ex Vivo Sensitivity Who Respond Clinically
|
0 Participants
|
SECONDARY outcome
Timeframe: patients may undergo lymphapheresis before the first and/or later cycles up to 12 months after achieving CR or PRCD25 (sCD25)and CD22 (sCD22) quantify hairy cell leukemia (HCL) tumor burden. Patients with either PR or CR are evaluated for soluble forms of CD25 (sCD25) and soluble CD22 (sCD22). The number of patients with PR or CR who have normalization of sCD25 and sCD22 will be recorded. Normalization is considered \<3 ng/ml for sCD25, and \<2 ng/ml for sCD22. Patients will be assessed for normalization of sCD25 and sCD22 for at least 12 months after achieving PR or CR.
Outcome measures
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Percentage of Patients Who Respond Clinically, Who Also Have Normalization in sCD22 or sCD25
|
100 Percentage of participants
|
SECONDARY outcome
Timeframe: Within 2 months of end of treatment ( measure antibodies before each cycle)Percent of patients neutralizing \>75% of 1000 ng/ml of BL22 in a biologic assay by end of treatment, with respect to the number of prior cycles of BL22 prior to entry on this protocol
Outcome measures
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Correlation Between Number of Prior Cycles of BL22 With Immunogenicity on This Protocol
|
0 correlation coefficient
|
SECONDARY outcome
Timeframe: 24 weeksDetermination of antibodies against BL22 is determined by the Clinical Laboratory Improvement Amendments (CLIA) certified blood tests in our contract lab. NCI-Frederick in the laboratory of Dr. David Waters (Science Applications International Corporation (SAIC). He is CLIA certified.
Outcome measures
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Percentage of Patients Who Make Antibodies
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: 24 weeksDetermination of dose limiting toxicity (DLT) is by the standard toxicity assessment Common Terminology Criteria for Adverse Events version 3.0 (CTCAEv3.0) done every cycle. For detailed information about the CTCAEv3.0 see the protocol Link module.
Outcome measures
| Measure |
BL22 Immunotherapy
n=1 Participants
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Percentage of Patients Who Have Dose Limiting Toxicity (DLT)
|
0 Percentage of participants
|
Adverse Events
BL22 Immunotherapy
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
BL22 Immunotherapy
n=1 participants at risk
30 micrograms/kg intravenous over 30 minutes every other day (QOD) on days 1, 3, 5, of a 4 week cycle (at least 26 days) for a maximum of 16 cycles or until they become ineligible.
|
|---|---|
|
Investigations
ALT, SGPT (serum glutamic pyruvic transaminase)
|
100.0%
1/1 • Number of events 4
|
|
Investigations
AST, SGOT(serum glutamic oxaloacetic transaminase)
|
100.0%
1/1 • Number of events 2
|
|
Investigations
Albumin, serum-low (hypoalbuminemia)
|
100.0%
1/1 • Number of events 3
|
|
Gastrointestinal disorders
Constipation
|
100.0%
1/1 • Number of events 2
|
|
Nervous system disorders
Constitutional Symptoms - Other (Specify, __)
|
100.0%
1/1 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin - Other (Specify, __)
|
100.0%
1/1 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhea
|
100.0%
1/1 • Number of events 2
|
|
General disorders
Edema: limb
|
100.0%
1/1 • Number of events 2
|
|
General disorders
Fatigue (asthenia, lethargy, malaise)
|
100.0%
1/1 • Number of events 2
|
|
General disorders
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)
|
100.0%
1/1 • Number of events 4
|
|
Gastrointestinal disorders
Heartburn/dyspepsia
|
100.0%
1/1 • Number of events 1
|
|
Investigations
Hemoglobin
|
100.0%
1/1 • Number of events 3
|
|
Gastrointestinal disorders
Hemorrhage, GI::Rectum
|
100.0%
1/1 • Number of events 1
|
|
Vascular disorders
Hypotension
|
100.0%
1/1 • Number of events 1
|
|
General disorders
Infection with normal ANC or Grade 1 or 2 neutrophils::Bladder (urinary)
|
100.0%
1/1 • Number of events 1
|
|
Psychiatric disorders
Insomnia
|
100.0%
1/1 • Number of events 1
|
|
Investigations
Leukocytes (total WBC)
|
100.0%
1/1 • Number of events 14
|
|
Investigations
Lipase
|
100.0%
1/1 • Number of events 2
|
|
Investigations
Lymphopenia
|
100.0%
1/1 • Number of events 11
|
|
Metabolism and nutrition disorders
Magnesium, serum-high (hypermagnesemia)
|
100.0%
1/1 • Number of events 4
|
|
Metabolism and nutrition disorders
Metabolic/Laboratory - Other (Specify, __)
|
100.0%
1/1 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Pain::Back
|
100.0%
1/1 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Pain::Bone
|
100.0%
1/1 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Pain::Muscle
|
100.0%
1/1 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Pain::Throat/pharynx/larynx
|
100.0%
1/1 • Number of events 1
|
|
Cardiac disorders
Palpitations
|
100.0%
1/1 • Number of events 1
|
|
Investigations
Platelets
|
100.0%
1/1 • Number of events 1
|
|
Metabolism and nutrition disorders
Sodium, serum-low (hyponatremia)
|
100.0%
1/1 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Sweating (diaphoresis)
|
100.0%
1/1 • Number of events 1
|
|
Nervous system disorders
Syncope (fainting)
|
100.0%
1/1 • Number of events 1
|
|
Eye disorders
Vision-blurred vision
|
100.0%
1/1 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
100.0%
1/1 • Number of events 1
|
|
Nervous system disorders
Dizziness
|
100.0%
1/1 • Number of events 1
|
Additional Information
Robert J. Kreitman, M.D.
National Cancer Institute, National Institutes of Health
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place