Trial Outcomes & Findings for Rituximab Plus Sargramostim (GM-CSF) In Patients With Chronic Lymphocytic Leukemia (NCT NCT00940342)

Last Updated: 2018-09-19

Results Overview

Overall Response Rate - Complete Response (CR) + Partial Response (PR). CR is the absence of Lymphocyte infiltrates on biopsy, \<30% Lymphocytes on Bone Marrow Aspirate, Lymphocytes \< 4,000ul , Hemoglobin \>11.0 g/dl , Platelets \>1000,000/ul, Polymorphonuclear neutrophils (PMN) \>1,500/ul, Liver/Spleen not palpable, Nodes none. PR is \<30% Lymphocytes with residual disease on biopsy for nodular PR, Lymphocytes \>50% decrease, Hemoglobin (un-transfused) \> 11.0 g/dl or \>50% improvement from baseline, Platelets \> 100,000/ul or 50% improvement from baseline, PMN \>1,500 ul or 50% improvement from baseline, Liver/Spleen \>/= 50% decrease, Nodes \>/= 50%.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

130 participants

Primary outcome timeframe

Blood tests once a week during 8 weeks of treatment.

Results posted on

2018-09-19

Participant Flow

Recruitment Period: October 2004 through January 2007. Of the 130 participants recruited, 119 participants were recruited at The University of Texas (UT) MD Anderson Cancer Center, 6 at The University of California San Diego, and 5 participants were recruited at Dana Farber Cancer Institute.

Participant milestones

Participant milestones
Measure	Treated and Relapsed - Group 1 Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	Untreated and High-Risk for Progression - Group 2 Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	70 Years of Age and Refused Chemo - Group 3 Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks
Overall Study STARTED	50	40	40
Overall Study COMPLETED	50	38	39
Overall Study NOT COMPLETED	0	2	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Treated and Relapsed - Group 1 Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	Untreated and High-Risk for Progression - Group 2 Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	70 Years of Age and Refused Chemo - Group 3 Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks
Overall Study Adverse Event	0	2	1

Baseline Characteristics

Rituximab Plus Sargramostim (GM-CSF) In Patients With Chronic Lymphocytic Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treated and Relapsed - Group 1 n=50 Participants Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	Untreated and High-Risk for Progression - Group 2 n=40 Participants Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	70 Years of Age and Refused Chemo - Group 3 n=40 Participants Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	Total n=130 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants	0 Participants n=31 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=39 Participants	36 Participants n=41 Participants	0 Participants n=35 Participants	56 Participants n=31 Participants
Age, Categorical >=65 years	30 Participants n=39 Participants	4 Participants n=41 Participants	40 Participants n=35 Participants	74 Participants n=31 Participants
Age, Continuous	68 years n=39 Participants	56.5 years n=41 Participants	73 years n=35 Participants	69 years n=31 Participants
Sex: Female, Male Female	16 Participants n=39 Participants	19 Participants n=41 Participants	14 Participants n=35 Participants	49 Participants n=31 Participants
Sex: Female, Male Male	34 Participants n=39 Participants	21 Participants n=41 Participants	26 Participants n=35 Participants	81 Participants n=31 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants	0 Participants n=31 Participants
Race (NIH/OMB) Asian	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants	0 Participants n=31 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants	0 Participants n=31 Participants
Race (NIH/OMB) Black or African American	1 Participants n=39 Participants	2 Participants n=41 Participants	4 Participants n=35 Participants	7 Participants n=31 Participants
Race (NIH/OMB) White	49 Participants n=39 Participants	38 Participants n=41 Participants	36 Participants n=35 Participants	123 Participants n=31 Participants
Race (NIH/OMB) More than one race	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants	0 Participants n=31 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants	0 Participants n=31 Participants
Region of Enrollment United States	50 participants n=39 Participants	40 participants n=41 Participants	40 participants n=35 Participants	130 participants n=31 Participants

PRIMARY outcome

Timeframe: Blood tests once a week during 8 weeks of treatment.

Outcome measures

Outcome measures
Measure	Treated and Relapsed - Group 1 n=50 Participants Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	Untreated and High-Risk for Progression - Group 2 n=38 Participants Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	70 Years of Age and Refused Chemo - Group 3 n=39 Participants Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks
Overall Response Rate	23 Participants	31 Participants	23 Participants

Adverse Events

Treated and Relapsed - Group 1

Serious events: 5 serious events

Other events: 21 other events

Deaths: 3 deaths

Untreated and High-Risk for Progression - Group 2

Serious events: 3 serious events

Other events: 16 other events

Deaths: 0 deaths

70 Years of Age and Refused Chemo - Group 3

Serious events: 4 serious events

Other events: 15 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Treated and Relapsed - Group 1 n=50 participants at risk Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	Untreated and High-Risk for Progression - Group 2 n=40 participants at risk Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	70 Years of Age and Refused Chemo - Group 3 n=40 participants at risk Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks
General disorders Fever	4.0% 2/50 • Number of events 2 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Infections and infestations Neutropenic Fever	4.0% 2/50 • Number of events 3 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Nervous system disorders Confusion	2.0% 1/50 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Musculoskeletal and connective tissue disorders Muscle Weakness	2.0% 1/50 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Infections and infestations Infection	2.0% 1/50 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
General disorders Pain	0.00% 0/50 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	5.0% 2/40 • Number of events 2 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Blood and lymphatic system disorders Hemolysis	0.00% 0/50 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Metabolism and nutrition disorders Hyponatremia	0.00% 0/50 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Respiratory, thoracic and mediastinal disorders Dyspnea	0.00% 0/50 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Vascular disorders Ischemia	0.00% 0/50 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
General disorders Fatigue	0.00% 0/50 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks

Other adverse events

Other adverse events
Measure	Treated and Relapsed - Group 1 n=50 participants at risk Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	Untreated and High-Risk for Progression - Group 2 n=40 participants at risk Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks	70 Years of Age and Refused Chemo - Group 3 n=40 participants at risk Participants receive one (1) course of therapy. One course of therapy consists of four (4) doses of Rituximab 375 mg/m2, administered once weekly by vein for 4 weeks along with GM-CSF 250 mcg subcutaneously three times weekly for 8 weeks. GM-CSF (Sargramostim): 250 mcg injection under the skin, three times a week for eight weeks. Rituximab: 375 mg/m\^2 administered intravenously once weekly for four weeks
Skin and subcutaneous tissue disorders Injection Site Reaction	12.0% 6/50 • Number of events 6 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	25.0% 10/40 • Number of events 10 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	25.0% 10/40 • Number of events 10 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
General disorders Pain	8.0% 4/50 • Number of events 4 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	10.0% 4/40 • Number of events 4 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
General disorders Fever	6.0% 3/50 • Number of events 3 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
General disorders Chills	6.0% 3/50 • Number of events 3 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	2.5% 1/40 • Number of events 1 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Infections and infestations Neutropenia	22.0% 11/50 • Number of events 11 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	10.0% 4/40 • Number of events 4 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	15.0% 6/40 • Number of events 6 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Blood and lymphatic system disorders Thrombocytopenia	6.0% 3/50 • Number of events 3 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	5.0% 2/40 • Number of events 2 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks
Respiratory, thoracic and mediastinal disorders Shortness of Breath	0.00% 0/50 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	0.00% 0/40 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks	5.0% 2/40 • Number of events 2 • Adverse events captured from the time of participant consent and will be followed to resolution or stabilization. Adverse events reported during each treatment course, up to 8 weeks

Additional Information

Alessandra Ferrajoli, MD/Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-792-7734

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place