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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

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

No Placebo-Only Group

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

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I am at least 2 years old.

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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:

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My bilirubin level is higher than 2.0 mg/dL.

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I have been diagnosed with Fanconi anemia.

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I am HIV positive.

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I am not pregnant, nursing, and willing to use birth control if I can have children.

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I have not had horse or rabbit ATG or Campath treatment in the last 6 months.

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I am receiving treatment with cytokines like G-CSF or Erythropoietin.

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My infection is not getting better despite treatment.

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I can understand the study details and can give informed consent myself or have someone who can.

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My liver is severely damaged and my albumin levels are low.

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I cannot take the study medication.

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I do not have severe health issues that would stop me from tolerating the treatment.

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

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

Pyrexia

Aspartate Aminotransferase increased

Alanine Aminotransferase increased

Decreased appetite

Vitamin D deficiency

Abdominal pain upper

Oropharyngeal pain

Rash

Toothache

Diarrhoea

Activated partial thromboplastin time prolonged

Blood alkaline Phosphatase increased

Blood creatinine increased

Bronchitis

Contusion

Gingival bleeding

Mouth haemorrhage

Nausea

Rhinorrhoea

Vomiting

Pneumonia fungal

Impetigo

Furuncle

Dyspepsia

Retinal vascular disorder

Constipation

Excoriation

Paraesthesia

Soft tissue injury

Cellulitis

Anaemia

Allergy to chemicals

Ear pain

Rash pruritic

Dermatitis allergic

Gingivitis

Groin pain

Osteoporosis

Influenza like illness

Lip haemorrhage

Menorrhagia

Viral pharyngitis

Pneumonia

Influenza

Joint injury

Lice infestation

Motion sickness

Pharyngitis

Platelet count increased

Somnolence

Subcutaneous abscess

Tongue haemorrhage

Tonsillar hypertrophy

Meningitis aseptic

Alanine aminotransferase abnormal

Aspartate aminotransferase abnormal

Gastritis

Asthenia

Back pain

Bronchospasm

Bulimia nervosa

Non-cardiac chest pain

Pain

100%

80%

60%

40%

20%

Study treatment Arm

Part 1: Placebo

Part 1: Eltrombopag

Part 2: Eltrombopag

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

~960

0 / 13Eligibility criteria met