Trial Outcomes & Findings for Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia (NCT NCT01703169)
NCT ID: NCT01703169
Last Updated: 2017-10-19
Results Overview
Defined as a stable platelet count of 50,000/μl or more during any 4 week period within the possible 12 weeks while on study,and including maximal platelet counts achieved in patients with moderate to very severe aplastic anemia.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
13 participants
Primary outcome timeframe
up to 12 weeks
Results posted on
2017-10-19
Participant Flow
Participant milestones
| Measure |
Eltrombopag
Single arm study. Dose Escalation.
Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
13
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia
Baseline characteristics by cohort
| Measure |
Eltrombopag
n=13 Participants
Single arm study. Dose Escalation.
Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
13 Count of Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: up to 12 weeksDefined as a stable platelet count of 50,000/μl or more during any 4 week period within the possible 12 weeks while on study,and including maximal platelet counts achieved in patients with moderate to very severe aplastic anemia.
Outcome measures
| Measure |
Eltrombopag
n=13 Participants
Single arm study. Dose Escalation.
Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count
|
|---|---|
|
Proportion of Participants With Platelet Response
|
0.23 proportion of participants
Interval 0.05 to 0.54
|
SECONDARY outcome
Timeframe: Between weeks 1-12.Population: Data was not collected for this outcome variable.
Proportion of subjects who achieve platelet counts at least twice their baseline value at any point while on study medication, in patients with moderate to very severe aplastic anemia.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeksPopulation: Data was not collected for this outcome variable.
Association between eltrombopag use and response in hemoglobin, hematocrit, total white blood cell count, and absolute neutrophil count to be evaluate by maximal hemoglobin, hematocrit, total white blood cell count, and absolute neutrophil counts achieved in patients with moderate to very severe aplastic anemia
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeksPopulation: Data was not collected for this outcome variable.
Evaluated weekly, up to 12 weeks. Association between eltrombopag use, dose, and tolerability in patients with moderate to very severe aplastic anemia
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Weeks 2, 6 and 12Population: Data was not collected for this outcome variable.
Samples will for PK analysis will collected as a trough level weeks 2, 6 and 12, prior to dose of eltrombopag. Additional PK level drawn at 2, 4 and 6 hours post-dose at the scheduled week 2 visit.
Outcome measures
Outcome data not reported
Adverse Events
Eltrombopag
Serious events: 3 serious events
Other events: 13 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Eltrombopag
n=13 participants at risk
Single arm study. Dose Escalation.
Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count
|
|---|---|
|
Cardiac disorders
congestive heart failure
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Hepatobiliary disorders
cholecystitis
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Nervous system disorders
syncope
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
Other adverse events
| Measure |
Eltrombopag
n=13 participants at risk
Single arm study. Dose Escalation.
Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count
|
|---|---|
|
Gastrointestinal disorders
abdominal pain
|
7.7%
1/13 • Number of events 2 • Approximately 3 years.
|
|
General disorders
aches/pains
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Blood and lymphatic system disorders
anemia
|
23.1%
3/13 • Number of events 13 • Approximately 3 years.
|
|
Psychiatric disorders
anxiety
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Cardiac disorders
arrhythmia
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Musculoskeletal and connective tissue disorders
back pain
|
15.4%
2/13 • Number of events 3 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
purpura
|
38.5%
5/13 • Number of events 8 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
bronzing of the skin
|
15.4%
2/13 • Number of events 3 • Approximately 3 years.
|
|
Injury, poisoning and procedural complications
bruising
|
23.1%
3/13 • Number of events 5 • Approximately 3 years.
|
|
Cardiac disorders
chest pain
|
7.7%
1/13 • Number of events 2 • Approximately 3 years.
|
|
General disorders
chills
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Psychiatric disorders
confusion
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Gastrointestinal disorders
constipation
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Musculoskeletal and connective tissue disorders
cramps
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Investigations
decreased lymphocyte count
|
23.1%
3/13 • Number of events 22 • Approximately 3 years.
|
|
Investigations
decreased neutrophil count
|
23.1%
3/13 • Number of events 17 • Approximately 3 years.
|
|
Investigations
thrombocytopenia
|
46.2%
6/13 • Number of events 24 • Approximately 3 years.
|
|
Metabolism and nutrition disorders
dehydration
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Gastrointestinal disorders
diarrhea
|
15.4%
2/13 • Number of events 4 • Approximately 3 years.
|
|
Nervous system disorders
dizziness
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Gastrointestinal disorders
dry mouth
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
dry skin
|
7.7%
1/13 • Number of events 3 • Approximately 3 years.
|
|
Nervous system disorders
dysgeusia
|
15.4%
2/13 • Number of events 3 • Approximately 3 years.
|
|
Renal and urinary disorders
dysuria
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
ecchymosis
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
General disorders
edema
|
30.8%
4/13 • Number of events 10 • Approximately 3 years.
|
|
Investigations
Alkaline phosphatase increased
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Investigations
elevated ALT
|
23.1%
3/13 • Number of events 10 • Approximately 3 years.
|
|
Investigations
elevated AST
|
23.1%
3/13 • Number of events 5 • Approximately 3 years.
|
|
Investigations
elevated bilirubin
|
30.8%
4/13 • Number of events 7 • Approximately 3 years.
|
|
Investigations
elevated creatinine
|
23.1%
3/13 • Number of events 8 • Approximately 3 years.
|
|
Metabolism and nutrition disorders
hyperglycemia
|
7.7%
1/13 • Number of events 2 • Approximately 3 years.
|
|
Respiratory, thoracic and mediastinal disorders
epistaxis
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
erythema
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
General disorders
fatigue
|
53.8%
7/13 • Number of events 8 • Approximately 3 years.
|
|
Infections and infestations
gum hypertrophy
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Infections and infestations
gum lesions
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
facial hair growth
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Nervous system disorders
headache
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Vascular disorders
hematoma
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Vascular disorders
hot flashes
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Vascular disorders
hypertension
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Investigations
hypoalbunemia
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Metabolism and nutrition disorders
hypocalcemia
|
15.4%
2/13 • Number of events 5 • Approximately 3 years.
|
|
Metabolism and nutrition disorders
hyponatremia
|
15.4%
2/13 • Number of events 5 • Approximately 3 years.
|
|
Respiratory, thoracic and mediastinal disorders
hypoxia
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Psychiatric disorders
insomnia
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
jaundice
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Reproductive system and breast disorders
menorrhagia
|
15.4%
2/13 • Number of events 3 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
mouth sores
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Respiratory, thoracic and mediastinal disorders
nasal congestion
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Infections and infestations
otitis externa
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Gastrointestinal disorders
nausea
|
15.4%
2/13 • Number of events 3 • Approximately 3 years.
|
|
General disorders
night sweats
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Eye disorders
opacity
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
General disorders
pain
|
38.5%
5/13 • Number of events 7 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
pallor
|
23.1%
3/13 • Number of events 3 • Approximately 3 years.
|
|
Blood and lymphatic system disorders
petechiae
|
84.6%
11/13 • Number of events 18 • Approximately 3 years.
|
|
General disorders
port leak
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Renal and urinary disorders
pyelonephritis
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
rash
|
23.1%
3/13 • Number of events 4 • Approximately 3 years.
|
|
Gastrointestinal disorders
rectal hemorrhage
|
7.7%
1/13 • Number of events 2 • Approximately 3 years.
|
|
Renal and urinary disorders
renal insufficiency
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Respiratory, thoracic and mediastinal disorders
respiratory infection
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Nervous system disorders
sciatica
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Eye disorders
scleral icterus
|
15.4%
2/13 • Number of events 2 • Approximately 3 years.
|
|
Respiratory, thoracic and mediastinal disorders
shortness of breath
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Hepatobiliary disorders
spelnomegaly
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Skin and subcutaneous tissue disorders
thrush
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Blood and lymphatic system disorders
transfusion reaction
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Nervous system disorders
tremor
|
7.7%
1/13 • Number of events 1 • Approximately 3 years.
|
|
Infections and infestations
urinary tract infection
|
15.4%
2/13 • Number of events 3 • Approximately 3 years.
|
|
Investigations
weight gain
|
30.8%
4/13 • Number of events 4 • Approximately 3 years.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place