Trial Outcomes & Findings for Phase II Eltrombopag in Chronic Lymphocytic Leukemia (CLL) (NCT NCT01168921)

Last Updated: 2023-03-23

Results Overview

Response is Complete Response (CR) + Major Response (MR). Complete response was defined as an increase in platelet count to ≥100K/µL for at least 4 weeks. Major response was defined as an increase in platelet count from \<20K/µL to ≥20K/μL and by at least 100% for at least 8 weeks; or for pts starting with \>20K/μL platelet count, absolute increase in platelet count of ≥30K/μL for at least 4 weeks.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

23 participants

Primary outcome timeframe

Up to 4 years

Results posted on

2023-03-23

Participant Flow

Participant milestones

Participant milestones
Measure	Eltrombopag Starting dose 75 mg by mouth (PO) daily for 28 day cycle. Eltrombopag: Starting dose 75 mg by mouth (PO) daily for 28 day cycle
Overall Study STARTED	23
Overall Study COMPLETED	23
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Phase II Eltrombopag in Chronic Lymphocytic Leukemia (CLL)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eltrombopag n=23 Participants Starting dose 75 mg by mouth (PO) daily for 28 day cycle. Eltrombopag: Starting dose 75 mg by mouth (PO) daily for 28 day cycle
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants
Age, Categorical >=65 years	10 Participants n=5 Participants
Age, Continuous	62 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	20 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	1 Participants n=5 Participants
Race (NIH/OMB) White	22 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	23 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 4 years

Outcome measures

Outcome measures
Measure	Eltrombopag n=23 Participants Starting dose 75 mg by mouth (PO) daily for 28 day cycle. Eltrombopag: Starting dose 75 mg by mouth (PO) daily for 28 day cycle
Number of Participants With a Response	12 Participants

SECONDARY outcome

Timeframe: Up to 4 years

The number of months from start of treatment to CLL progression, requiring leukemia treatment. Disease Progression (PD) is defined as a \>/= 50% increase in at least one of the following in responding participants: \>/= 2 lymph nodes (on 2 exams 2 weeks apart), liver or spleen (below costal margin) , or absolute number of circulating lymphocytes (\>/= 10L/ul).

Outcome measures

Outcome measures
Measure	Eltrombopag n=23 Participants Starting dose 75 mg by mouth (PO) daily for 28 day cycle. Eltrombopag: Starting dose 75 mg by mouth (PO) daily for 28 day cycle
Time to CLL Progression Requiring Leukemia Treatment	5.8 Months Interval 2.8 to 21.0

Adverse Events

Eltrombopag

Serious events: 6 serious events

Other events: 23 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Eltrombopag n=23 participants at risk Starting dose 75 mg by mouth (PO) daily for 28 day cycle. Eltrombopag: Starting dose 75 mg by mouth (PO) daily for 28 day cycle
General disorders Weakness	8.7% 2/23 • Number of events 2 • Up to 4 years
Infections and infestations Cellulitis	4.3% 1/23 • Number of events 1 • Up to 4 years
Vascular disorders Deep Vein Thrombosis	8.7% 2/23 • Number of events 2 • Up to 4 years
General disorders Flu	4.3% 1/23 • Number of events 1 • Up to 4 years
Psychiatric disorders Altered Mental Status	13.0% 3/23 • Number of events 3 • Up to 4 years
Metabolism and nutrition disorders Hyponatremia	4.3% 1/23 • Number of events 1 • Up to 4 years
Blood and lymphatic system disorders Anemia	4.3% 1/23 • Number of events 1 • Up to 4 years
Infections and infestations Pneumonia	8.7% 2/23 • Number of events 2 • Up to 4 years
Blood and lymphatic system disorders Neutropenia	4.3% 1/23 • Number of events 4 • Up to 4 years
Respiratory, thoracic and mediastinal disorders Shortness of Breath	4.3% 1/23 • Number of events 1 • Up to 4 years
General disorders Fever	4.3% 1/23 • Number of events 1 • Up to 4 years
Vascular disorders Hypotension	8.7% 2/23 • Number of events 2 • Up to 4 years
Infections and infestations Sepsis	4.3% 1/23 • Number of events 1 • Up to 4 years
Eye disorders Blurred Vision	4.3% 1/23 • Number of events 1 • Up to 4 years
Respiratory, thoracic and mediastinal disorders Pleural Effusion	4.3% 1/23 • Number of events 1 • Up to 4 years
Injury, poisoning and procedural complications Fracture	4.3% 1/23 • Number of events 1 • Up to 4 years
Surgical and medical procedures Splenectomy	4.3% 1/23 • Number of events 1 • Up to 4 years

Other adverse events

Other adverse events
Measure	Eltrombopag n=23 participants at risk Starting dose 75 mg by mouth (PO) daily for 28 day cycle. Eltrombopag: Starting dose 75 mg by mouth (PO) daily for 28 day cycle
Metabolism and nutrition disorders Hyperbilirubinemia	4.3% 1/23 • Number of events 1 • Up to 4 years
Metabolism and nutrition disorders Hyponatremia	4.3% 1/23 • Number of events 1 • Up to 4 years
General disorders Fever	8.7% 2/23 • Number of events 2 • Up to 4 years
Blood and lymphatic system disorders Neutropenia	100.0% 23/23 • Number of events 52 • Up to 4 years
Blood and lymphatic system disorders Anemia	21.7% 5/23 • Number of events 5 • Up to 4 years
Infections and infestations Bronchitis	4.3% 1/23 • Number of events 1 • Up to 4 years

Additional Information

William Wierda MD/Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-745-0428

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place