Trial Outcomes & Findings for Eltrombopag for Moderate Aplastic Anemia (NCT NCT01328587)
NCT ID: NCT01328587
Last Updated: 2024-12-03
Results Overview
Defined by changes in platelet count and/or platelet transfusion requirements or hemoglobin (Hb) and/or PRBC transfusion requirements and the toxicity profile as measured by CTCAE. Response for platelet lineage is defined as absolute increase of ≥20x10\^9/L above baseline at 16 or 20 weeks, with at least 2 serial measurements performed 1 week apart and sustained for 1 month or more without platelet transfusions, or for transfusion dependent patients stable platelet counts with transfusion independence for ≥ 8 weeks. Patients with anemia (untransfused hemoglobin ≤ 8.5 g/dL), a response will be an increase in Hb by ≥1.5g/dL at 4 months, with at least 2 serial measurments and sustained for 1 month or more without transfusion support OR for transfusion dependent patients, reduction of units of RCC transfused by 50%/8 weeks compared with the pretreatment transfusion number in the pretreatment transfusion number in the previous 8 weeks or transfusion independence (no transfusions ≥ 8 weeks).
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
34 participants
Primary outcome timeframe
16-20 weeks from start of drug
Results posted on
2024-12-03
Participant Flow
Participant milestones
| Measure |
Eltrombopag
Patients will receive Eltrombopag ( thrombopoietin receptor agonist) by mouth once a day.
|
|---|---|
|
Overall Study
STARTED
|
34
|
|
Overall Study
COMPLETED
|
34
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Eltrombopag for Moderate Aplastic Anemia
Baseline characteristics by cohort
| Measure |
Eltrombopag
n=34 Participants
Patients will receive Eltrombopag ( thrombopoietin receptor agonist) by mouth once a day.
|
|---|---|
|
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
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
31 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
|
4 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
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
23 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
|
PRIMARY outcome
Timeframe: 16-20 weeks from start of drugDefined by changes in platelet count and/or platelet transfusion requirements or hemoglobin (Hb) and/or PRBC transfusion requirements and the toxicity profile as measured by CTCAE. Response for platelet lineage is defined as absolute increase of ≥20x10\^9/L above baseline at 16 or 20 weeks, with at least 2 serial measurements performed 1 week apart and sustained for 1 month or more without platelet transfusions, or for transfusion dependent patients stable platelet counts with transfusion independence for ≥ 8 weeks. Patients with anemia (untransfused hemoglobin ≤ 8.5 g/dL), a response will be an increase in Hb by ≥1.5g/dL at 4 months, with at least 2 serial measurments and sustained for 1 month or more without transfusion support OR for transfusion dependent patients, reduction of units of RCC transfused by 50%/8 weeks compared with the pretreatment transfusion number in the pretreatment transfusion number in the previous 8 weeks or transfusion independence (no transfusions ≥ 8 weeks).
Outcome measures
| Measure |
Eltrombopag
n=34 Participants
Eltrombopag will be administered for 16 to 20 weeks at a starting dose of 50mg/day (East Asian ancestry 25mg/day). The dose will decreased and increased (maximum dose 300mg/day) based on safety and response.
Eltrombopag: Eltrombopag will be administered for 16 to 20 weeks at a starting dose of 150mg/day (East Asian ancestry 75mg /day). The dose will decreased and increased (maximum dose 300mg/day) based on safety and response.
|
|---|---|
|
Proportion of Drug Responders
|
17 Participants
|
Adverse Events
Eltrombopag
Serious events: 1 serious events
Other events: 29 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Eltrombopag
n=34 participants at risk
Patients will receive Eltrombopag ( thrombopoietin receptor agonist) by mouth once a day.
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastatic Ovarian CA
|
2.9%
1/34 • 42 months from enrollment
|
Other adverse events
| Measure |
Eltrombopag
n=34 participants at risk
Patients will receive Eltrombopag ( thrombopoietin receptor agonist) by mouth once a day.
|
|---|---|
|
Blood and lymphatic system disorders
blood and lymphatic system d/s other, specify
|
2.9%
1/34 • 42 months from enrollment
|
|
Blood and lymphatic system disorders
Gingival Bleeding
|
2.9%
1/34 • 42 months from enrollment
|
|
Blood and lymphatic system disorders
Hematuria
|
2.9%
1/34 • 42 months from enrollment
|
|
Blood and lymphatic system disorders
spleen disorder/splenic varices
|
2.9%
1/34 • 42 months from enrollment
|
|
Blood and lymphatic system disorders
spleen disorder/splenomegaly
|
2.9%
1/34 • 42 months from enrollment
|
|
Cardiac disorders
abdominal edema
|
2.9%
1/34 • 42 months from enrollment
|
|
Cardiac disorders
BLE edema
|
2.9%
1/34 • 42 months from enrollment
|
|
Cardiac disorders
dyspnea on exertion
|
2.9%
1/34 • 42 months from enrollment
|
|
Cardiac disorders
fatigue
|
5.9%
2/34 • 42 months from enrollment
|
|
Cardiac disorders
fatigue/angina
|
2.9%
1/34 • 42 months from enrollment
|
|
Cardiac disorders
Palpitations
|
2.9%
1/34 • 42 months from enrollment
|
|
Cardiac disorders
scrotal edema
|
2.9%
1/34 • 42 months from enrollment
|
|
Ear and labyrinth disorders
Ear Pain
|
2.9%
1/34 • 42 months from enrollment
|
|
Ear and labyrinth disorders
External Ear pain/discomfort
|
2.9%
1/34 • 42 months from enrollment
|
|
Ear and labyrinth disorders
otitis media/external ear inflammation
|
2.9%
1/34 • 42 months from enrollment
|
|
Eye disorders
Burning Eyes
|
2.9%
1/34 • 42 months from enrollment
|
|
Eye disorders
eye tingling/pain
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Abdominal Discomfort
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Abdominal Pain
|
5.9%
2/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Blood in stool
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
collagenous colitis
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Dehydration
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
diarrhea
|
17.6%
6/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
diarrhea/stomach cramps
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
epigastric tenderness
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
food poisoning
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
foodpoisoning/gastroenteritis
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Gastroenteritis
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
GI illness(nausea, vmitting, diarrhea)
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Gi symptoms
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Gum Bleeding
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
h pylori
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
intermittent lower pelvic pain
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Loose Stool
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Loose stools
|
5.9%
2/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Mild constipation
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
nausea
|
17.6%
6/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
nausea/diarrhea
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
nausea/vomiting
|
5.9%
2/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
nausea/vomiting/abd cramps
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Pinworms
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
rectal bleeding
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Viral Gastroenteritis
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Vomiting
|
2.9%
1/34 • 42 months from enrollment
|
|
Gastrointestinal disorders
Worsening GERD
|
2.9%
1/34 • 42 months from enrollment
|
|
General disorders
chills
|
2.9%
1/34 • 42 months from enrollment
|
|
General disorders
facial edema
|
2.9%
1/34 • 42 months from enrollment
|
|
General disorders
Fatigue
|
2.9%
1/34 • 42 months from enrollment
|
|
General disorders
Fever
|
8.8%
3/34 • 42 months from enrollment
|
|
General disorders
Flu like symptoms
|
2.9%
1/34 • 42 months from enrollment
|
|
General disorders
Headache
|
11.8%
4/34 • 42 months from enrollment
|
|
General disorders
Influenza
|
2.9%
1/34 • 42 months from enrollment
|
|
General disorders
intermittent night sweats
|
2.9%
1/34 • 42 months from enrollment
|
|
General disorders
LE edema
|
2.9%
1/34 • 42 months from enrollment
|
|
General disorders
lightheaded
|
2.9%
1/34 • 42 months from enrollment
|
|
Hepatobiliary disorders
cirrhosis
|
2.9%
1/34 • 42 months from enrollment
|
|
Hepatobiliary disorders
Elevated AST
|
2.9%
1/34 • 42 months from enrollment
|
|
Hepatobiliary disorders
Elevated Bilirubin
|
2.9%
1/34 • 42 months from enrollment
|
|
Hepatobiliary disorders
portal hypertension
|
2.9%
1/34 • 42 months from enrollment
|
|
Immune system disorders
CTLA 4 deficiency
|
2.9%
1/34 • 42 months from enrollment
|
|
Infections and infestations
Abscessed Tooth
|
5.9%
2/34 • 42 months from enrollment
|
|
Infections and infestations
C diff
|
2.9%
1/34 • 42 months from enrollment
|
|
Infections and infestations
strep throat
|
2.9%
1/34 • 42 months from enrollment
|
|
Infections and infestations
tooth #30 infection
|
2.9%
1/34 • 42 months from enrollment
|
|
Infections and infestations
UTI
|
2.9%
1/34 • 42 months from enrollment
|
|
Infections and infestations
UTI/Inflammed prostate
|
2.9%
1/34 • 42 months from enrollment
|
|
Infections and infestations
yeast infection
|
2.9%
1/34 • 42 months from enrollment
|
|
Injury, poisoning and procedural complications
easy bruising
|
2.9%
1/34 • 42 months from enrollment
|
|
Injury, poisoning and procedural complications
Foot pain from fall
|
2.9%
1/34 • 42 months from enrollment
|
|
Injury, poisoning and procedural complications
shoulder pain from fall
|
2.9%
1/34 • 42 months from enrollment
|
|
Investigations
ALT increase
|
2.9%
1/34 • 42 months from enrollment
|
|
Investigations
AST increase
|
2.9%
1/34 • 42 months from enrollment
|
|
Investigations
Elevated Liver Enzymes
|
2.9%
1/34 • 42 months from enrollment
|
|
Investigations
yellowing skin/eye
|
2.9%
1/34 • 42 months from enrollment
|
|
Musculoskeletal and connective tissue disorders
muscle cramps
|
2.9%
1/34 • 42 months from enrollment
|
|
Musculoskeletal and connective tissue disorders
R Breast Pain
|
2.9%
1/34 • 42 months from enrollment
|
|
Musculoskeletal and connective tissue disorders
R Calf Cramps
|
2.9%
1/34 • 42 months from enrollment
|
|
Musculoskeletal and connective tissue disorders
Rib pain
|
2.9%
1/34 • 42 months from enrollment
|
|
Nervous system disorders
dizzy
|
2.9%
1/34 • 42 months from enrollment
|
|
Nervous system disorders
fingers tingly'-peripheral sensory neuropathy
|
2.9%
1/34 • 42 months from enrollment
|
|
Nervous system disorders
headaches
|
2.9%
1/34 • 42 months from enrollment
|
|
Nervous system disorders
Seizure
|
2.9%
1/34 • 42 months from enrollment
|
|
Renal and urinary disorders
Dark urine
|
2.9%
1/34 • 42 months from enrollment
|
|
Renal and urinary disorders
UTI
|
5.9%
2/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
Chest cold
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
Cold Sxs
|
5.9%
2/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
dry cough
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea on Exertion
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
Flu-like symptoms
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
hoarseness
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
Mild Flu-like symptoms
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
nasal congestion
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
shortness of breath
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
sinus pressure
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
sleep apnea
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
sore throat
|
2.9%
1/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
URI
|
11.8%
4/34 • 42 months from enrollment
|
|
Respiratory, thoracic and mediastinal disorders
Viral URI
|
2.9%
1/34 • 42 months from enrollment
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
2.9%
1/34 • 42 months from enrollment
|
|
Skin and subcutaneous tissue disorders
facial rash
|
2.9%
1/34 • 42 months from enrollment
|
|
Skin and subcutaneous tissue disorders
left post auricular skin
|
2.9%
1/34 • 42 months from enrollment
|
|
Skin and subcutaneous tissue disorders
Rash on Arm
|
2.9%
1/34 • 42 months from enrollment
|
|
Skin and subcutaneous tissue disorders
Shingles
|
2.9%
1/34 • 42 months from enrollment
|
|
Skin and subcutaneous tissue disorders
skin rash
|
2.9%
1/34 • 42 months from enrollment
|
|
Skin and subcutaneous tissue disorders
vitiligo
|
2.9%
1/34 • 42 months from enrollment
|
Additional Information
Dunbar, Cynthia
National Heart Lung and Blood Institute
Phone: +1 301 827 1164
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place