Trial Outcomes & Findings for Eltrombopag Olamine in Treating Thrombocytopenia in Patients With Chronic Myeloid Leukemia or Myelofibrosis Receiving Tyrosine Kinase Therapy (NCT NCT01428635)
NCT ID: NCT01428635
Last Updated: 2023-09-26
Results Overview
The primary endpoint is complete (platelet) response (yes/no). A complete (platelet) response will be defined as a sustained (3 months) platelet count of \> 50 x 109/L for patients with CML and \> 100 x 109/L for patients with MF and at least a 20% increase in platelet count from baseline.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
21 participants
Primary outcome timeframe
Up to 9 years
Results posted on
2023-09-26
Participant Flow
Recruitment Period: January 2012 to January 2022
Participant milestones
| Measure |
Supportive Care (Eltrombopag Olamine) CML Participants
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
Supportive Care (Eltrombopag Olamine) Myelofibrosis Participants
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
6
|
|
Overall Study
COMPLETED
|
15
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Eltrombopag Olamine in Treating Thrombocytopenia in Patients With Chronic Myeloid Leukemia or Myelofibrosis Receiving Tyrosine Kinase Therapy
Baseline characteristics by cohort
| Measure |
Supportive Care (Eltrombopag Olamine) CML Participants
n=15 Participants
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
Supportive Care (Eltrombopag Olamine) Myelofibrosis Participants
n=6 Participants
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
13 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Age, Continuous
|
46 years
n=5 Participants
|
73 years
n=7 Participants
|
57 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
13 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
6 participants
n=7 Participants
|
21 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 9 yearsThe primary endpoint is complete (platelet) response (yes/no). A complete (platelet) response will be defined as a sustained (3 months) platelet count of \> 50 x 109/L for patients with CML and \> 100 x 109/L for patients with MF and at least a 20% increase in platelet count from baseline.
Outcome measures
| Measure |
Supportive Care (Eltrombopag Olamine) CML Participants
n=15 Participants
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
Supportive Care (Eltrombopag Olamine) Myelofibrosis Participants
n=6 Participants
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
|---|---|---|
|
Number of Participants With a Platelet Response
|
12 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 9 yearsPopulation: Zero participants were analyzed in Myelofibrosis Participants as this outcome measure only analyzes those participants who had a platelet response . Zero participants in the Myelofibrosis group had a platelet response, therefore zero participants in this outcome were analyzed.
(CML) : Complete Hematologic Remission : Normalization for 4 weeks of the bone marrow (\< 5% blasts) and peripheral blood with WBC within normal institutional limits with no blasts, promyelocytes or myelocytes, and basophils \<5%,. Complete cytogenetic response: Ph positive 0%. Partial cytogenetic response: Ph positive 1-35%. Minor cytogenetic response: Ph positive 36-90%. No cytogenetic response: Ph positive 100%. Myelofibrosis : Complete Remission: absence of transfusion or growth factor support: complete resolution of disease-related symptoms/signs including palpable hepatosplenomegaly, hemoglobin \> 11 g/dL, platelet count ≥100 x 10\^9/L, absolute neutrophil count ≥ 1.0 x 10\^9/L. Normal leukocyte differential with disappearance of nucleated red blood cells and immature myeloid cells in peripheral smear, in the absence of splenectomy. Bone marrow histological remission:presence of age-adjusted normocellularity, \< 5% myeloblast , osteomyelofibrosis grade \</= 1.
Outcome measures
| Measure |
Supportive Care (Eltrombopag Olamine) CML Participants
n=12 Participants
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
Supportive Care (Eltrombopag Olamine) Myelofibrosis Participants
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
|---|---|---|
|
Number of Participants With a Response to TKI Therapy After Eltrombopag
|
9 Participants
|
0 Participants
|
Adverse Events
Supportive Care (Eltrombopag Olamine) CML Participants
Serious events: 10 serious events
Other events: 13 other events
Deaths: 1 deaths
Supportive Care (Eltrombopag Olamine) Myelofibrosis Participants
Serious events: 4 serious events
Other events: 4 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Supportive Care (Eltrombopag Olamine) CML Participants
n=15 participants at risk
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
Supportive Care (Eltrombopag Olamine) Myelofibrosis Participants
n=6 participants at risk
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Bullous dermatitis
|
0.00%
0/15 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Cardiac disorders
Cardiac Arrest
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Cardiac disorders
Chest Pain
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
General disorders
Edema Limbs
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
General disorders
Fatigue
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
16.7%
1/6 • Number of events 2 • Up to 10 years
|
|
General disorders
Flu Like Symptoms
|
0.00%
0/15 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Infections and infestations
Infection
|
13.3%
2/15 • Number of events 3 • Up to 10 years
|
33.3%
2/6 • Number of events 3 • Up to 10 years
|
|
Infections and infestations
Lung Infection
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Cardiac disorders
Myocardial Infarction
|
0.00%
0/15 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign
|
13.3%
2/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Gastrointestinal disorders
Pancreatitis
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
6.7%
1/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/15 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
Other adverse events
| Measure |
Supportive Care (Eltrombopag Olamine) CML Participants
n=15 participants at risk
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
Supportive Care (Eltrombopag Olamine) Myelofibrosis Participants
n=6 participants at risk
Patients receive eltrombopag olamine PO QD in the absence of disease progression or unacceptable toxicity.
Eltrombopag Olamine: Given PO
|
|---|---|---|
|
General disorders
Abdominal pain
|
13.3%
2/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Investigations
Alanine aminotransferase increased
|
53.3%
8/15 • Number of events 9 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Blood and lymphatic system disorders
Anemia
|
13.3%
2/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Investigations
Aspartate aminotransferase increased
|
46.7%
7/15 • Number of events 9 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Investigations
Blood bilirubin increased
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Eye disorders
Blurred vision
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
16.7%
1/6 • Number of events 2 • Up to 10 years
|
|
Injury, poisoning and procedural complications
Bruising
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Investigations
Cholesterol high
|
13.3%
2/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Eye disorders
Conjunctivitis
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Investigations
Creatinine increased
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Renal and urinary disorders
Cystitis noninfective
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Gastrointestinal disorders
Dehydration
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Psychiatric disorders
Depression
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Gastrointestinal disorders
Diarrhea
|
20.0%
3/15 • Number of events 5 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Gastrointestinal disorders
Dysphagia
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
General disorders
Edema
|
20.0%
3/15 • Number of events 3 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Nervous system disorders
Encephalopathy
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Eye disorders
Eye Disorders
|
13.3%
2/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Infections and infestations
Eye Infection
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/15 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
General disorders
Fatigue
|
40.0%
6/15 • Number of events 6 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
General disorders
Fever
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
General disorders
Flu Like Symptoms
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Gastrointestinal disorders
Gastric Hemorrhage
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Gastrointestinal disorders
Gastritis
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Gastrointestinal disorders
Gastroparesis
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Psychiatric disorders
Hallucinations
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Nervous system disorders
Headache
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Ear and labyrinth disorders
Hearing Impaired
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
16.7%
1/6 • Number of events 2 • Up to 10 years
|
|
Vascular disorders
Hypertension
|
13.3%
2/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Psychiatric disorders
Insomnia
|
20.0%
3/15 • Number of events 3 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Investigations
Thrombosis
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Infections and infestations
Lung Infection
|
6.7%
1/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Nervous system disorders
Memory Impairment
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Musculoskeletal and connective tissue disorders
Tendonitis
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Gastrointestinal disorders
Nausea
|
20.0%
3/15 • Number of events 3 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Investigations
Neutropenia
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
General disorders
Pain Extremity
|
13.3%
2/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Gastrointestinal disorders
Periodontal Disease
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Eye disorders
Periorbital edema
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Nervous system disorders
Peripheral Motor Neuropathy
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Nervous system disorders
Peripheral Sensory Neuropathy
|
0.00%
0/15 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Investigations
Thrombocytopenia
|
20.0%
3/15 • Number of events 3 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Skin and subcutaneous tissue disorders
Purpura
|
0.00%
0/15 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
13.3%
2/15 • Number of events 2 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Infections and infestations
Sepsis
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Cardiac disorders
Sinus tachycardia
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Nervous system disorders
Stroke
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Blood and lymphatic system disorders
Thrombotic thrombocytopenic purpura
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Infections and infestations
Upper respiratory infection
|
6.7%
1/15 • Number of events 1 • Up to 10 years
|
0.00%
0/6 • Up to 10 years
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/15 • Up to 10 years
|
16.7%
1/6 • Number of events 1 • Up to 10 years
|
Additional Information
Gautam Borthakur MD./Professor
The University of Texas MD Anderson Cancer Center
Phone: 713-563-1586
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place