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Eltrombopag for Moderate Aplastic Anemia

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ClinicalTrials.gov Identifier: NCT01328587
Recruitment Status : Active, not recruiting
First Posted : April 4, 2011
Results First Posted : April 23, 2019
Last Update Posted : October 20, 2020
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Moderate Aplastic Anemia
Unilineage Bone Marrow Failure Disorders
Intervention Drug: Eltrombopag
Enrollment 34
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Eltrombopag
Hide Arm/Group Description Patients will receive Eltrombopag ( thrombopoietin receptor agonist) by mouth once a day.
Period Title: Overall Study
Started 34
Completed 34
Not Completed 0
Arm/Group Title Eltrombopag
Hide Arm/Group Description Patients will receive Eltrombopag ( thrombopoietin receptor agonist) by mouth once a day.
Overall Number of Baseline Participants 34
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants
<=18 years
4
  11.8%
Between 18 and 65 years
27
  79.4%
>=65 years
3
   8.8%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants
Female
17
  50.0%
Male
17
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants
Hispanic or Latino
3
   8.8%
Not Hispanic or Latino
31
  91.2%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants
American Indian or Alaska Native
0
   0.0%
Asian
4
  11.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
5
  14.7%
White
23
  67.6%
More than one race
0
   0.0%
Unknown or Not Reported
2
   5.9%
1.Primary Outcome
Title Proportion of Drug Responders
Hide Description The portion of drug responders as defined by changes in the platelet count and/or platelet transfusion requirements or hemoglobin and/or PRBC transfusion requirements and the toxicity profile as measured using the CTCAE criteria. Treatment response for the platelet lineage is defined as an absolute increase of ≥20x10^9/L above baseline at 16 or 20 weeks, measured on at least two serial measurements performed one week apart and sustained for 1 month or more without support of platelet transfusions, or for transfusion dependent patients stable platelet counts with transfusion independence for ≥ 8 weeks. For patients with anemia (untransfused hemoglobin ≤ 8.5 g/dL), a treatment response will be an increase in Hb by ≥1.5g/dL at four months, measured on at least 2 serial measurements 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 th
Time Frame 16-20 weeks from start of drug
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eltrombopag
Hide Arm/Group Description:

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.

Overall Number of Participants Analyzed 34
Measure Type: Count of Participants
Unit of Measure: Participants
17
  50.0%
Time Frame 42 months from enrollment
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Eltrombopag
Hide Arm/Group Description Patients will receive Eltrombopag ( thrombopoietin receptor agonist) by mouth once a day.
All-Cause Mortality
Eltrombopag
Affected / at Risk (%)
Total   1/34 (2.94%) 
Hide Serious Adverse Events
Eltrombopag
Affected / at Risk (%)
Total   1/34 (2.94%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Metastatic Ovarian CA   1/34 (2.94%) 
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Eltrombopag
Affected / at Risk (%)
Total   29/34 (85.29%) 
Blood and lymphatic system disorders   
blood and lymphatic system d/s other, specify   1/34 (2.94%) 
Gingival Bleeding   1/34 (2.94%) 
Hematuria   1/34 (2.94%) 
spleen disorder/splenic varices   1/34 (2.94%) 
spleen disorder/splenomegaly   1/34 (2.94%) 
Cardiac disorders   
abdominal edema   1/34 (2.94%) 
BLE edema   1/34 (2.94%) 
dyspnea on exertion   1/34 (2.94%) 
fatigue   2/34 (5.88%) 
fatigue/angina   1/34 (2.94%) 
Palpitations   1/34 (2.94%) 
scrotal edema   1/34 (2.94%) 
Ear and labyrinth disorders   
Ear Pain   1/34 (2.94%) 
External Ear pain/discomfort   1/34 (2.94%) 
otitis media/external ear inflammation   1/34 (2.94%) 
Eye disorders   
Burning Eyes   1/34 (2.94%) 
eye tingling/pain   1/34 (2.94%) 
Gastrointestinal disorders   
Abdominal Discomfort   1/34 (2.94%) 
Abdominal Pain   2/34 (5.88%) 
Blood in stool   1/34 (2.94%) 
collagenous colitis   1/34 (2.94%) 
Dehydration   1/34 (2.94%) 
diarrhea   6/34 (17.65%) 
diarrhea/stomach cramps   1/34 (2.94%) 
epigastric tenderness   1/34 (2.94%) 
food poisoning   1/34 (2.94%) 
foodpoisoning/gastroenteritis   1/34 (2.94%) 
Gastroenteritis   1/34 (2.94%) 
GI illness(nausea, vmitting, diarrhea)   1/34 (2.94%) 
Gi symptoms   1/34 (2.94%) 
Gum Bleeding   1/34 (2.94%) 
h pylori   1/34 (2.94%) 
intermittent lower pelvic pain   1/34 (2.94%) 
Loose Stool   1/34 (2.94%) 
Loose stools   2/34 (5.88%) 
Mild constipation   1/34 (2.94%) 
nausea   6/34 (17.65%) 
nausea/diarrhea   1/34 (2.94%) 
nausea/vomiting   2/34 (5.88%) 
nausea/vomiting/abd cramps   1/34 (2.94%) 
Pinworms   1/34 (2.94%) 
rectal bleeding   1/34 (2.94%) 
Viral Gastroenteritis   1/34 (2.94%) 
Vomiting   1/34 (2.94%) 
Worsening GERD   1/34 (2.94%) 
General disorders   
chills   1/34 (2.94%) 
facial edema   1/34 (2.94%) 
Fatigue   1/34 (2.94%) 
Fever   3/34 (8.82%) 
Flu like symptoms   1/34 (2.94%) 
Headache   4/34 (11.76%) 
Influenza   1/34 (2.94%) 
intermittent night sweats   1/34 (2.94%) 
LE edema   1/34 (2.94%) 
lightheaded   1/34 (2.94%) 
Hepatobiliary disorders   
cirrhosis   1/34 (2.94%) 
Elevated AST   1/34 (2.94%) 
Elevated Bilirubin   1/34 (2.94%) 
portal hypertension   1/34 (2.94%) 
Immune system disorders   
CTLA 4 deficiency   1/34 (2.94%) 
Infections and infestations   
Abscessed Tooth   2/34 (5.88%) 
C diff   1/34 (2.94%) 
strep throat   1/34 (2.94%) 
tooth #30 infection   1/34 (2.94%) 
UTI   1/34 (2.94%) 
UTI/Inflammed prostate   1/34 (2.94%) 
yeast infection   1/34 (2.94%) 
Injury, poisoning and procedural complications   
easy bruising   1/34 (2.94%) 
Foot pain from fall   1/34 (2.94%) 
shoulder pain from fall   1/34 (2.94%) 
Investigations   
ALT increase   1/34 (2.94%) 
AST increase   1/34 (2.94%) 
Elevated Liver Enzymes   1/34 (2.94%) 
yellowing skin/eye   1/34 (2.94%) 
Musculoskeletal and connective tissue disorders   
muscle cramps   1/34 (2.94%) 
R Breast Pain   1/34 (2.94%) 
R Calf Cramps   1/34 (2.94%) 
Rib pain   1/34 (2.94%) 
Nervous system disorders   
dizzy   1/34 (2.94%) 
fingers tingly&#39;-peripheral sensory neuropathy   1/34 (2.94%) 
headaches   1/34 (2.94%) 
Seizure   1/34 (2.94%) 
Renal and urinary disorders   
Dark urine   1/34 (2.94%) 
UTI   2/34 (5.88%) 
Respiratory, thoracic and mediastinal disorders   
Chest cold   1/34 (2.94%) 
Cold Sxs   2/34 (5.88%) 
cough   1/34 (2.94%) 
dry cough   1/34 (2.94%) 
Dyspnea on Exertion   1/34 (2.94%) 
Flu-like symptoms   1/34 (2.94%) 
hoarseness   1/34 (2.94%) 
Mild Flu-like symptoms   1/34 (2.94%) 
nasal congestion   1/34 (2.94%) 
shortness of breath   1/34 (2.94%) 
sinus pressure   1/34 (2.94%) 
sleep apnea   1/34 (2.94%) 
sore throat   1/34 (2.94%) 
URI   4/34 (11.76%) 
Viral URI   1/34 (2.94%) 
Skin and subcutaneous tissue disorders   
Dermatitis   1/34 (2.94%) 
facial rash   1/34 (2.94%) 
left post auricular skin   1/34 (2.94%) 
Rash on Arm   1/34 (2.94%) 
Shingles   1/34 (2.94%) 
skin rash   1/34 (2.94%) 
vitiligo   1/34 (2.94%) 
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dunbar, Cynthia
Organization: National Heart Lung and Blood Institute
Phone: +1 301 827 1164
EMail: dunbarc@nhlbi.nih.gov
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT01328587    
Other Study ID Numbers: 110134
11-H-0134
First Submitted: April 1, 2011
First Posted: April 4, 2011
Results First Submitted: March 29, 2019
Results First Posted: April 23, 2019
Last Update Posted: October 20, 2020