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Thrombopoietin Receptor Agonist

Eltrombopag vs Standard Treatment for ITP in Children

Phase 3
Waitlist Available
Led By Jenny Despotovic, DO
Research Sponsored by Baylor College of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Upfront treatment: Patient within 10 days of ITP diagnosis who has not received previous treatment OR Treatment failure: Patients who have failed standard management (observation or treatment with one or more first-line agents)
Treatment options include one of three standard therapies, (IVIg, steroids, or Anti-D). For example, if patient has previously shown no response to IVIg or steroids and is Rh-negative, patient would not be eligible for study.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Study Summary

This trial is testing a new treatment for ITP in children aged 1-18.

Who is the study for?
This trial is for children aged 1 to less than 18 with newly diagnosed ITP, a condition causing low platelet count. They must not have received prior treatment or have failed standard treatments like IVIG, steroids, or Anti-D. Participants need pharmacologic treatment and should be able to return for lab studies.Check my eligibility
What is being tested?
The study compares Eltrombopag against three standard therapies (IVIG, Rho(D) Immune Globulin, Steroids) in treating ITP in children. It's an open label phase 3 trial where participants are randomly assigned to receive either the new intervention or one of the standard options.See study design
What are the potential side effects?
Eltrombopag may cause liver issues, infections that don't respond well to drugs, heart problems if there's pre-existing cardiac disease, bleeding risks especially if severe bleeding has occurred before and potential cataract development.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I was diagnosed with ITP within the last 10 days and haven't been treated, or my initial ITP treatments didn't work.
Select...
I have not responded to IVIg, steroids, or Anti-D and am Rh-negative.
Select...
I am younger than 18 years old.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Proportion of patients with a platelet response
Secondary outcome measures
Bleeding Score
Blood Iron values
Hockenberry Fatigue Scale-Parent
+7 more
Other outcome measures
Change in Hockenberry fatigue
Extreme thrombocytosis
Global Change Scale scores
+7 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
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%
Constipation
2%
Groin pain
2%
Furuncle
2%
Cellulitis
2%
Allergy to chemicals
2%
Retinal vascular disorder
2%
Dyspepsia
2%
Osteoporosis
2%
Pneumonia fungal
2%
Ear pain
2%
Gingivitis
2%
Rash pruritic
2%
Paraesthesia
2%
Soft tissue injury
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

2Treatment groups
Experimental Treatment
Active Control
Group I: EltrombopagExperimental Treatment1 Intervention
Patients randomized to eltrombopag will be treated for 12 weeks, with the possibility to continue therapy for up to 1 year depending on response.
Group II: Standard first-line therapyActive Control3 Interventions
Subjects randomized to the standard therapy arm will receive one of three treatments at the discretion of the treating physician. Patients who previously failed standard management prior to study entry must be treated with a different agent than their original failed agent. e.g. Patient who failed steroids could receive either IVIg or anti-D if randomized to the standard treatment arm. Standard therapy will be administered as commercially available drug. Investigator may choose amongst the following: IVIg: IVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy) Steroids: Prednisone/Prednisolone 4 mg/kg/day (Max 120 mg/day) x 4 days Rho(D) Immune Globulin: Anti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Eltrombopag
FDA approved

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Thrombocytopenic Purpura (ITP) include immune modulation and platelet production enhancement. Corticosteroids, such as prednisone, work by suppressing the immune system to reduce the destruction of platelets. Intravenous immunoglobulin (IVIG) provides a temporary increase in platelet count by interfering with the immune system's ability to destroy platelets. Thrombopoietin receptor agonists like eltrombopag and romiplostim stimulate the bone marrow to produce more platelets. These treatments are crucial for ITP patients as they help manage bleeding risks and improve platelet counts, thereby enhancing the patient's quality of life and reducing the likelihood of severe bleeding complications.

Find a Location

Who is running the clinical trial?

Baylor College of MedicineLead Sponsor
1,003 Previous Clinical Trials
6,002,948 Total Patients Enrolled
Boston Children's HospitalOTHER
767 Previous Clinical Trials
5,580,387 Total Patients Enrolled
University of California, San FranciscoOTHER
2,518 Previous Clinical Trials
15,241,607 Total Patients Enrolled

Media Library

Thrombocytopenic Purpura Research Study Groups: Standard first-line therapy, Eltrombopag
Thrombocytopenic Purpura Clinical Trial 2023: Eltrombopag Highlights & Side Effects. Trial Name: NCT03939637 — Phase 3
~16 spots leftby Mar 2025
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