← Back to Search

Thrombopoietin Receptor Agonist

Eltrombopag for Aplastic Anemia

Phase 2
Waitlist Available
Led By Cynthia E Dunbar, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age greater than or equal to 2 years old
Current diagnosis of moderate aplastic anemia or unilineage bone marrow failure disorders
Must not have
Bilirubin > 2.0 mg/dL, including congenital abnormalities in the bilirubin count
Known diagnosis of Fanconi anemia
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 16-20 weeks from start of drug
Awards & highlights

Summary

This trial is evaluating the safety and effectiveness of eltrombopag, a drug given by mouth, in people with moderate aplastic anemia or bone marrow failure and unilineage cytopenia who need treatment for significantly low blood cell counts.

Who is the study for?
This trial is for people over 2 years old with moderate aplastic anemia or certain bone marrow failure disorders, who have low blood cell counts and weigh more than 12 kg. They shouldn't have severe aplastic anemia, uncontrolled infections, HIV, significant liver/kidney disease, known sensitivity to eltrombopag, be pregnant/nursing without contraception use if applicable, or unable to consent.
What is being tested?
The trial tests the safety and effectiveness of a drug called Eltrombopag in patients with moderate aplastic anemia. Participants will take this medication orally once daily and undergo regular blood tests to adjust dosage based on response and side effects.
What are the potential side effects?
Eltrombopag may cause liver issues (elevated enzymes), digestive problems (nausea or diarrhea), headaches, muscle pain or cramps. It can also increase the risk of developing cataracts which would be monitored through eye exams.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am at least 2 years old.
Select...
I have been diagnosed with moderate aplastic anemia or a similar bone marrow failure.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
My bilirubin level is higher than 2.0 mg/dL.
Select...
I have been diagnosed with Fanconi anemia.
Select...
I am HIV positive.
Select...
I am not pregnant, nursing, and willing to use birth control if I can have children.
Select...
I have not had horse or rabbit ATG or Campath treatment in the last 6 months.
Select...
I am receiving treatment with cytokines like G-CSF or Erythropoietin.
Select...
My infection is not getting better despite treatment.
Select...
I can understand the study details and can give informed consent myself or have someone who can.
Select...
My liver is severely damaged and my albumin levels are low.
Select...
I cannot take the study medication.
Select...
I do not have severe health issues that would stop me from tolerating the treatment.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~16-20 weeks from start of drug
This trial's timeline: 3 weeks for screening, Varies for treatment, and 16-20 weeks from start of drug for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Proportion of Drug Responders

Side effects data

From 2014 Phase 3 trial • 92 Patients • NCT01520909
17%
Nasopharyngitis
16%
Rhinitis
13%
Epistaxis
11%
Upper respiratory tract infection
11%
Cough
10%
Headache
10%
Abdominal pain
6%
Pyrexia
6%
Aspartate Aminotransferase increased
5%
Alanine Aminotransferase increased
5%
Decreased appetite
5%
Vitamin D deficiency
5%
Abdominal pain upper
5%
Oropharyngeal pain
5%
Rash
5%
Toothache
5%
Diarrhoea
3%
Activated partial thromboplastin time prolonged
3%
Blood alkaline Phosphatase increased
3%
Blood creatinine increased
3%
Bronchitis
3%
Contusion
3%
Gingival bleeding
3%
Mouth haemorrhage
3%
Nausea
3%
Rhinorrhoea
3%
Vomiting
2%
Anaemia
2%
Constipation
2%
Impetigo
2%
Retinal vascular disorder
2%
Cellulitis
2%
Allergy to chemicals
2%
Furuncle
2%
Dermatitis allergic
2%
Dyspepsia
2%
Ear pain
2%
Excoriation
2%
Gingivitis
2%
Groin pain
2%
Osteoporosis
2%
Influenza like illness
2%
Paraesthesia
2%
Rash pruritic
2%
Soft tissue injury
2%
Pneumonia fungal
2%
Lip haemorrhage
2%
Menorrhagia
2%
Viral pharyngitis
2%
Pneumonia
2%
Influenza
2%
Joint injury
2%
Lice infestation
2%
Motion sickness
2%
Pharyngitis
2%
Platelet count increased
2%
Somnolence
2%
Subcutaneous abscess
2%
Tongue haemorrhage
2%
Tonsillar hypertrophy
2%
Meningitis aseptic
2%
Alanine aminotransferase abnormal
2%
Aspartate aminotransferase abnormal
2%
Gastritis
2%
Asthenia
2%
Back pain
2%
Bronchospasm
2%
Bulimia nervosa
2%
Non-cardiac chest pain
2%
Pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part 1: Placebo
Part 1: Eltrombopag
Part 2: Eltrombopag

Trial Design

1Treatment groups
Experimental Treatment
Group I: EltrombopagExperimental Treatment1 Intervention
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Eltrombopag
2013
Completed Phase 4
~970

Find a Location

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,889 Previous Clinical Trials
47,834,173 Total Patients Enrolled
11 Trials studying Aplastic Anemia
922 Patients Enrolled for Aplastic Anemia
Cynthia E Dunbar, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
2 Previous Clinical Trials
83 Total Patients Enrolled

Media Library

Eltrombopag (Thrombopoietin Receptor Agonist) Clinical Trial Eligibility Overview. Trial Name: NCT01328587 — Phase 2
Aplastic Anemia Research Study Groups: Eltrombopag
Aplastic Anemia Clinical Trial 2023: Eltrombopag Highlights & Side Effects. Trial Name: NCT01328587 — Phase 2
Eltrombopag (Thrombopoietin Receptor Agonist) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01328587 — Phase 2
~2 spots leftby Sep 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top