Trial Outcomes & Findings for A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients (NCT NCT00922883)
NCT ID: NCT00922883
Last Updated: 2020-10-20
Results Overview
Defined as unilineage or multilineage recovery by 1 or more of the following: 1) platelet response (increase to 20 × 103/μL above baseline or stable platelet counts with transfusion independence for a minimum of 8 weeks in those who were transfusion dependent on entry into the protocol); (2) erythroid response (when pretreatment hemoglobin was \<9 g/dL, defined as an increase in hemoglobin by 1.5 g/dL or, in transfused patients, a reduction in the units of packed red blood cell transfusions by an absolute number of at least 4 transfusions for 8 consecutive weeks, compared with the pretreatment transfusion number in the previous 8 weeks); and (3) neutrophil response (when pretreatment absolute neutrophil count \[ANC\] of \<0.5 × 103/μL as at least a 100% increase in ANC, or an ANC increase \>0.5 × 103/μL, and the toxicity profile as measured using Common Terminology Criteria for Adverse Events).
COMPLETED
PHASE2
43 participants
12-16 weeks
2020-10-20
Participant Flow
44 patients were consented, but 1 patient was removed before treatment with the investigational agent was initiated because an additional review of baseline bone marrow revealed that the patient was not eligible, because the patient had changes in the bone marrow inconsistent with a diagnosis of aplastic anemia.
Participant milestones
| Measure |
Eltrombopag
Eltrombopag (Promacta): Subjects commenced eltrombopag at a dose of 50 mg, which was increased by 25 mg every 2 weeks if the platelet count had not increased by 20 × 103/µL, to a maximum dose of 150 mg.
|
|---|---|
|
Overall Study
STARTED
|
43
|
|
Overall Study
COMPLETED
|
43
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients
Baseline characteristics by cohort
| Measure |
Eltrombopag
n=44 Participants
Eltrombopag (Promacta): Subjects commenced eltrombopag at a dose of 50 mg, which was increased by 25 mg every 2 weeks if the platelet count had not increased by 20 × 103/µL, to a maximum dose of 150 mg.
|
|---|---|
|
Age, Categorical
<=18 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
27 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
25 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 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
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
21 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
8 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12-16 weeksPopulation: All subjects who received Eltrombopag were analyzed.
Defined as unilineage or multilineage recovery by 1 or more of the following: 1) platelet response (increase to 20 × 103/μL above baseline or stable platelet counts with transfusion independence for a minimum of 8 weeks in those who were transfusion dependent on entry into the protocol); (2) erythroid response (when pretreatment hemoglobin was \<9 g/dL, defined as an increase in hemoglobin by 1.5 g/dL or, in transfused patients, a reduction in the units of packed red blood cell transfusions by an absolute number of at least 4 transfusions for 8 consecutive weeks, compared with the pretreatment transfusion number in the previous 8 weeks); and (3) neutrophil response (when pretreatment absolute neutrophil count \[ANC\] of \<0.5 × 103/μL as at least a 100% increase in ANC, or an ANC increase \>0.5 × 103/μL, and the toxicity profile as measured using Common Terminology Criteria for Adverse Events).
Outcome measures
| Measure |
Eltrombopag
n=43 Participants
Eltrombopag (Promacta): Subjects commenced eltrombopag at a dose of 50 mg, which was increased by 25 mg every 2 weeks if the platelet count had not increased by 20 × 103/µL, to a maximum dose of 150 mg.
|
|---|---|
|
The Portion of Drug Responders as Defined by Hematologic Improvements
|
17 Participants
|
Adverse Events
Eltrombopag
Serious adverse events
| Measure |
Eltrombopag
n=43 participants at risk
Eltrombopag (Promacta): Subjects commenced eltrombopag at a dose of 50 mg, which was increased by 25 mg every 2 weeks if the platelet count had not increased by 20 × 103/µL, to a maximum dose of 150 mg.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
2.3%
1/43
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
16.3%
7/43
|
|
Blood and lymphatic system disorders
Neutropenia
|
2.3%
1/43
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
2.3%
1/43
|
|
Eye disorders
Conjunctival haemorrhage
|
4.7%
2/43
|
|
Gastrointestinal disorders
Abdominal pain
|
2.3%
1/43
|
|
Gastrointestinal disorders
Clostridium difficile colitis
|
2.3%
1/43
|
|
Gastrointestinal disorders
Gingivitis ulcerative
|
2.3%
1/43
|
|
General disorders
Drug eruption
|
2.3%
1/43
|
|
General disorders
Pyrexia
|
2.3%
1/43
|
|
Hepatobiliary disorders
Hepatic neoplasm
|
2.3%
1/43
|
|
Infections and infestations
Bacteraemia
|
2.3%
1/43
|
|
Infections and infestations
Influenza
|
2.3%
1/43
|
|
Infections and infestations
Sepsis
|
7.0%
3/43
|
|
Infections and infestations
Urinary tract infection
|
2.3%
1/43
|
|
Investigations
Aspartate aminotransferase increased
|
2.3%
1/43
|
|
Vascular disorders
Deep vein thrombosis
|
2.3%
1/43
|
|
Vascular disorders
Hypotension
|
2.3%
1/43
|
Other adverse events
| Measure |
Eltrombopag
n=43 participants at risk
Eltrombopag (Promacta): Subjects commenced eltrombopag at a dose of 50 mg, which was increased by 25 mg every 2 weeks if the platelet count had not increased by 20 × 103/µL, to a maximum dose of 150 mg.
|
|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
7.0%
3/43
|
|
Blood and lymphatic system disorders
Hyperbilirubinaemia
|
2.3%
1/43
|
|
Blood and lymphatic system disorders
Jaundice
|
2.3%
1/43
|
|
Blood and lymphatic system disorders
Petechiae
|
7.0%
3/43
|
|
Blood and lymphatic system disorders
Purpura
|
2.3%
1/43
|
|
Cardiac disorders
Chest pain
|
4.7%
2/43
|
|
Cardiac disorders
Dizziness
|
9.3%
4/43
|
|
Cardiac disorders
Dyspnoea
|
2.3%
1/43
|
|
Cardiac disorders
Dyspnoea exertional
|
4.7%
2/43
|
|
Eye disorders
Cataract
|
2.3%
1/43
|
|
Eye disorders
Conjunctival haemorrhage
|
4.7%
2/43
|
|
Eye disorders
Dry eye
|
4.7%
2/43
|
|
Eye disorders
Eye contusion
|
2.3%
1/43
|
|
Eye disorders
Eye irritation
|
2.3%
1/43
|
|
Eye disorders
Myopia
|
2.3%
1/43
|
|
Eye disorders
Ocular hyperaemia
|
2.3%
1/43
|
|
Eye disorders
Vision blurred
|
2.3%
1/43
|
|
Eye disorders
Vitreous floaters
|
2.3%
1/43
|
|
Gastrointestinal disorders
Abdominal discomfort
|
4.7%
2/43
|
|
Gastrointestinal disorders
Abdominal injury
|
7.0%
3/43
|
|
Gastrointestinal disorders
Abdominal pain
|
9.3%
4/43
|
|
Gastrointestinal disorders
Anal fissure
|
2.3%
1/43
|
|
Gastrointestinal disorders
Anorectal discomfort
|
2.3%
1/43
|
|
Gastrointestinal disorders
Constipation
|
2.3%
1/43
|
|
Gastrointestinal disorders
Diarrhoea
|
9.3%
4/43
|
|
Gastrointestinal disorders
Dyspepsia
|
2.3%
1/43
|
|
Gastrointestinal disorders
Gastrointestinal disorder
|
2.3%
1/43
|
|
Gastrointestinal disorders
Gingival bleeding
|
4.7%
2/43
|
|
Gastrointestinal disorders
Gingival disorder
|
2.3%
1/43
|
|
Gastrointestinal disorders
Gingival swelling
|
2.3%
1/43
|
|
Gastrointestinal disorders
Lip ulceration
|
2.3%
1/43
|
|
Gastrointestinal disorders
Nausea
|
25.6%
11/43
|
|
Gastrointestinal disorders
Oral candidiasis
|
2.3%
1/43
|
|
Gastrointestinal disorders
Oropharyngeal pain
|
9.3%
4/43
|
|
Gastrointestinal disorders
Pelvic organ injury
|
2.3%
1/43
|
|
Gastrointestinal disorders
Pericoronitis
|
2.3%
1/43
|
|
Gastrointestinal disorders
Periodontal disease
|
2.3%
1/43
|
|
Gastrointestinal disorders
Tooth abscess
|
2.3%
1/43
|
|
Gastrointestinal disorders
Tooth injury
|
2.3%
1/43
|
|
Gastrointestinal disorders
Toothache
|
2.3%
1/43
|
|
Gastrointestinal disorders
Vomiting
|
7.0%
3/43
|
|
General disorders
Asthenia
|
2.3%
1/43
|
|
General disorders
Axillary pain
|
2.3%
1/43
|
|
General disorders
Catheter site pain
|
2.3%
1/43
|
|
General disorders
Fatigue
|
25.6%
11/43
|
|
General disorders
Induration
|
2.3%
1/43
|
|
General disorders
Injection site infection
|
2.3%
1/43
|
|
General disorders
Night sweats
|
2.3%
1/43
|
|
General disorders
Pain
|
4.7%
2/43
|
|
General disorders
Pyrexia
|
9.3%
4/43
|
|
Hepatobiliary disorders
Cholecystitis
|
2.3%
1/43
|
|
Hepatobiliary disorders
Hepatitis viral
|
2.3%
1/43
|
|
Immune system disorders
Hypersensitivity
|
2.3%
1/43
|
|
Immune system disorders
Rhinitis allergic
|
4.7%
2/43
|
|
Immune system disorders
Urticaria
|
4.7%
2/43
|
|
Infections and infestations
Cellulitis
|
2.3%
1/43
|
|
Infections and infestations
Herpes zoster
|
2.3%
1/43
|
|
Infections and infestations
Influenza
|
2.3%
1/43
|
|
Infections and infestations
Kidney infection
|
2.3%
1/43
|
|
Infections and infestations
Upper respiratory tract infection
|
18.6%
8/43
|
|
Infections and infestations
Viral infection
|
2.3%
1/43
|
|
Investigations
Alanine aminotransferase increased
|
7.0%
3/43
|
|
Investigations
Aspartate aminotransferase increased
|
4.7%
2/43
|
|
Investigations
Blood creatine phosphokinase increased
|
2.3%
1/43
|
|
Investigations
Neutrophil count decreased
|
2.3%
1/43
|
|
Investigations
Weight decreased
|
2.3%
1/43
|
|
Metabolism and nutrition disorders
Gout
|
2.3%
1/43
|
|
Metabolism and nutrition disorders
Osteoporosis
|
2.3%
1/43
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.7%
2/43
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
4.7%
2/43
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
2.3%
1/43
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
11.6%
5/43
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal disorder
|
2.3%
1/43
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
4.7%
2/43
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
4.7%
2/43
|
|
Musculoskeletal and connective tissue disorders
Piriformis syndrome
|
2.3%
1/43
|
|
Musculoskeletal and connective tissue disorders
Synovial cyst
|
2.3%
1/43
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Ovarian cyst
|
2.3%
1/43
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Seborrhoeic keratosis
|
2.3%
1/43
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin cancer
|
2.3%
1/43
|
|
Nervous system disorders
Headache
|
16.3%
7/43
|
|
Nervous system disorders
Insomnia
|
7.0%
3/43
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
2.3%
1/43
|
|
Psychiatric disorders
Anxiety
|
2.3%
1/43
|
|
Psychiatric disorders
Depressed mood
|
2.3%
1/43
|
|
Psychiatric disorders
Depression
|
2.3%
1/43
|
|
Psychiatric disorders
Libido decreased
|
2.3%
1/43
|
|
Psychiatric disorders
Mood altered
|
2.3%
1/43
|
|
Psychiatric disorders
Nightmare
|
2.3%
1/43
|
|
Psychiatric disorders
Personality disorder
|
2.3%
1/43
|
|
Renal and urinary disorders
Chromaturia
|
2.3%
1/43
|
|
Renal and urinary disorders
Dysuria
|
2.3%
1/43
|
|
Renal and urinary disorders
Pollakiuria
|
2.3%
1/43
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.0%
3/43
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
4.7%
2/43
|
|
Respiratory, thoracic and mediastinal disorders
Nasal disorder
|
2.3%
1/43
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary haemorrhage
|
2.3%
1/43
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
2.3%
1/43
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
2.3%
1/43
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
4.7%
2/43
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.0%
3/43
|
|
Skin and subcutaneous tissue disorders
Skin discolouration
|
2.3%
1/43
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
2.3%
1/43
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
2.3%
1/43
|
|
Surgical and medical procedures
Platelet transfusion
|
2.3%
1/43
|
|
Surgical and medical procedures
Tooth extraction
|
2.3%
1/43
|
|
Vascular disorders
Haematoma
|
2.3%
1/43
|
|
Vascular disorders
Hypotension
|
2.3%
1/43
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place