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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 16-20 weeks from start of drug
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
I am at least 2 years old.
Select...
I have been diagnosed with moderate aplastic anemia or a similar bone marrow failure.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 16-20 weeks from start of drug
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~16-20 weeks from start of drug
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Proportion of Drug Responders
Side effects data
From 2014 Phase 3 trial • 92 Patients • NCT0152090917%
Nasopharyngitis
16%
Rhinitis
13%
Epistaxis
11%
Upper respiratory tract infection
11%
Cough
10%
Headache
10%
Abdominal pain
6%
Pyrexia
6%
Aspartate Aminotransferase increased
5%
Decreased appetite
5%
Alanine Aminotransferase increased
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%
Soft tissue injury
2%
Constipation
2%
Groin pain
2%
Furuncle
2%
Cellulitis
2%
Dyspepsia
2%
Osteoporosis
2%
Retinal vascular disorder
2%
Pneumonia fungal
2%
Ear pain
2%
Allergy to chemicals
2%
Paraesthesia
2%
Rash pruritic
2%
Gingivitis
2%
Excoriation
2%
Impetigo
2%
Anaemia
2%
Dermatitis allergic
2%
Influenza like illness
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
3,835 Previous Clinical Trials
47,310,636 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
Eligibility Criteria:
This trial includes the following eligibility criteria:- My bilirubin level is higher than 2.0 mg/dL.I have been diagnosed with Fanconi anemia.I am HIV positive.I am not pregnant, nursing, and willing to use birth control if I can have children.My recent tests show active blood cancer or abnormal bone marrow.I have not had horse or rabbit ATG or Campath treatment in the last 6 months.I am receiving treatment with cytokines like G-CSF or Erythropoietin.I am at least 2 years old.My infection is not getting better despite treatment.I can understand the study details and can give informed consent myself or have someone who can.My liver is severely damaged and my albumin levels are low.I have cancer and received chemotherapy or radiation within the last 6 months.I have been diagnosed with moderate aplastic anemia or a similar bone marrow failure.I cannot take the study medication.I do not have severe health issues that would stop me from tolerating the treatment.
Research Study Groups:
This trial has the following groups:- Group 1: Eltrombopag
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How many participants are there in this clinical experiment?
"This medical trial is no longer actively recruiting patients, having been first posted in April 2011 and last updated on December 2021. Those searching for other studies may be interested to know that there are currently 255 trials seeking participants with pancytopenia and 23 clinical investigations looking for people with Eltrombopag."
Answered by AI
Are there specific criteria which must be met to qualify for this research program?
"Requirements to join this medical research include pancytopenia and being between the ages of 2 and 100. Currently, up to 34 patients can be enrolled."
Answered by AI
Are there vacancies available for participants in this experiment?
"According to clinicaltrials.gov, the study is not actively searching for participants at this moment in time: it was first posted on April 1st 2011 and has been updated most recently on December 2nd 2021. Nonetheless, there are still 278 other trials which are currently open to new candidates."
Answered by AI
Has Eltrombopag been granted regulatory clearance by the FDA?
"The clinical trial data that is currently available suggests a moderate level of safety for Eltrombopag, so it was assigned a score of 2 on our scale. This Phase 2 study has yet to collect information regarding the drug's efficacy."
Answered by AI
Is this study open to individuals aged 35 and older?
"To be eligible for this trial, applicants must fall within the age range of 2 to 100. There are also 127 studies that target minors and 194 trials designed primarily for senior citizens."
Answered by AI
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