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Thrombopoietin Receptor Agonist

Eltrombopag for Diamond-Blackfan Anemia

Phase 1 & 2

Waitlist Available

Led By David J Young, 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

Weight greater than or equal to 12 kilograms

Clinically-significant anemia as defined as either: hemoglobin less than 9.0 g/dL red cell transfusion of at least 2 units PRBC for adults or 30 cc/kg for children (whichever is less) in the eight weeks prior to study enrollment

Must not have

Stage 4 or greater kidney disease as defined by creatinine > 2.5 mg/ dL or GFR < 30 mL/min/1.73 m(2)

Known active or uncontrolled infections not adequately responding to appropriate therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the safety and efficacy of eltrombopag in people with Diamond-Blackfan anemia (DBA) who have not responded to steroids or could not take them.

Who is the study for?

This trial is for people aged 2 and older with Diamond-Blackfan anemia who haven't responded to steroids or can't take them. They must weigh at least 12 kg, live in the US or be able to stay there during the trial, and not have certain severe health conditions or be pregnant. People taking certain drugs or with a history of blood clots (other than catheter-related) are excluded.

What is being tested?

The study tests Eltrombopag's ability to increase red blood cells in patients with refractory Diamond-Blackfan anemia. Participants will take daily pills for 24 weeks, have regular blood tests, and undergo various assessments including MRI scans and bone marrow biopsies. If successful, treatment may continue up to three years.

What are the potential side effects?

Eltrombopag might cause liver issues, heart problems if predisposed (QT prolongation), potential bleeding risks due to increased platelets count especially if it exceeds normal levels (>400k/microliter), as well as possible interactions leading to altered drug metabolism.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I weigh at least 12 kilograms.

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I have severe anemia or needed a blood transfusion recently.

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I am at least 2 years old.

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My condition worsened after or didn't respond to steroids.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My kidney function is severely reduced.

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I have an infection that isn't getting better with treatment.

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

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

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My condition meets the WHO criteria for MDS or AML.

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I am not on medications that affect eltrombopag's action.

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I do not have significant heart problems or recent serious heart issues.

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I have had blood clots not related to catheter use.

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I cannot take the study medication.

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I am at risk of my cancer returning within the next year.

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I am not pregnant or nursing.

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My bilirubin level is higher than 2.0 mg/dL.

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I can understand the study details and can give informed consent, or I have someone who can do it on my behalf.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of Adverse Events

Participants That Responded to Eltrombopag

Time (Weeks) to Response

Secondary study objectives

Health-related Quality-of-life

Iron Overload

Long-term Safety of Drug

+4 more

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: Refractory Diamond-Blackfan Anemia in EltrombopagExperimental Treatment1 Intervention

Participants with Refractory Diamond-Blackfan Anemia will be administered Eltrombopag. Participants 12 years of age an above will receive the adult dose of 150 mg by mouth daily. Participants between ages of 6 and 11 years old will start at 75 mg by mouth daily, and children 2 and 5 years of age will start at 2.5 mg/kg, not to exceed 75 mg by mouth daily. To adjust for the higher expected exposure in participants of East Asian and South East Asian ancestry, the starting dose for East Asian and South East Asian participants 12 years of age and above will be 75 mg by mouth once daily. For East Asian and South East Asian participants between 6 and 11 years of age, the starting dose will be 37.5 mg once daily by mouth, and for children between 2 and 5, the starting dose will be 1.25 mg/kg by mouth.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Eltrombopag

2013

Completed Phase 4

~970

0 / 17Eligibility criteria met