What is the mechanism of action of this treatment?

Thrombopoietin Receptor Agonists (TRAs) such as Avatrombopag, Romiplostim, and Eltrombopag work by mimicking the action of thrombopoietin, a natural hormone that regulates platelet production. These drugs bind to and activate the thrombopoietin receptor on megakaryocytes, the bone marrow cells responsible for producing platelets, thereby increasing platelet production. This mechanism is crucial for patients with Thrombocytopenic Purpura, a condition characterized by abnormally low platelet counts, as it helps to elevate platelet levels, reduce bleeding risks, and improve overall quality of life. By targeting the underlying issue of insufficient platelet production, TRAs offer a targeted and effective treatment option for managing this condition.

What side effects are associated with this type of intervention?

Thrombopoietin receptor agonists (TRAs) such as avatrombopag, eltrombopag, and romiplostim are commonly used to treat thrombocytopenic purpura. Known side effects of these treatments include an increased risk of thromboembolic events, such as deep vein thrombosis and portal vein thrombosis, particularly in patients with underlying conditions like advanced liver disease. Other potential side effects include bone marrow changes, such as reticulin formation, and the development of neutralizing antibodies, although this is less common with non-peptide mimetics. Overall, these treatments are generally well-tolerated, but monitoring for thrombotic complications is essential.

What prior FDA approvals exist?

The FDA has approved several thrombopoietin receptor agonists for the treatment of thrombocytopenic purpura. Eltrombopag and romiplostim are notable examples. Eltrombopag has been approved for increasing platelet counts in patients with chronic immune thrombocytopenia (ITP) and for use in patients with chronic hepatitis C to allow antiviral therapy. Romiplostim is approved for chronic ITP to increase platelet counts and reduce the risk of bleeding. Both drugs work by stimulating the thrombopoietin receptor, similar to avatrombopag, which is currently being studied for its efficacy and safety in pediatric patients with immune thrombocytopenia.

What other types of research exist?

Promising alternatives to Avatrombopag for thrombocytopenic purpura patients include Romiplostim and Eltrombopag. Both are thrombopoietin receptor agonists with established efficacy and safety profiles. Romiplostim has shown effectiveness in increasing platelet counts and reducing bleeding events, while Eltrombopag has been effective in both adult and pediatric populations. Additionally, ongoing research and clinical trials may introduce new TPO-RAs or other novel therapies by 2024.

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Thrombopoietin Receptor Agonist

Avatrombopag for Pediatric ITP

Phase 3

Waitlist Available

Research Sponsored by Dova Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Must not have

Participants must not have any history of arterial or venous thrombosis, including partial or complete thrombosis

Participants must not have a known history of congenital heart abnormalities or arrhythmias.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up throughout study

Awards & highlights

Pivotal Trial

Summary

This trial is testing a medication called avatrombopag, which helps increase blood platelets. It targets children who have had low platelet counts for an extended period due to their immune system attacking these cells. The medication works by encouraging the body to make more platelets, which are important for stopping bleeding. Avatrombopag has been shown to increase platelet counts in patients with low platelet levels, including those with liver disease.

Who is the study for?

This trial is for kids and teens (1-17 years old) with a condition called immune thrombocytopenia (ITP) that's lasted at least 6 months. They should have low platelet counts despite previous treatments but can't join if they've had blood clots, bone marrow disorders, certain heart issues, inherited platelet problems, or ITP caused by another disease.

What is being tested?

The study tests Avatrombopag against a placebo to see if it's effective and safe in treating low platelet counts due to ITP in children. It's a phase 3b trial where participants are randomly chosen to receive either the actual drug or a dummy pill without any active ingredients.

What are the potential side effects?

Avatrombopag may cause headaches, tiredness, stomach pain, nausea or vomiting. There might also be risks of liver problems and formation of blood clots. The exact side effects in children are being studied.

Eligibility Criteria

Exclusion Criteria

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

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I have never had a blood clot in my arteries or veins.

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I do not have a history of heart defects or irregular heartbeats.

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I have never been diagnosed with myelodysplastic syndrome.

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I do not have a genetic condition that causes low platelet counts.

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I do not have a history of secondary immune thrombocytopenia.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ last 8 weeks of 12 week treatment regimen

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~last 8 weeks of 12 week treatment regimen

This trial's timeline: 3 weeks for screening, Varies for treatment, and last 8 weeks of 12 week treatment regimen for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Durable platelet response

Secondary study objectives

Bleeding Symptoms

Platelet Counts

Platelet percentage

+2 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: Experimental: AvatrombopagActive Control1 Intervention

Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks

Group II: Placebo Comparator:PlaceboPlacebo Group1 Intervention

Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks

0 / 5Eligibility criteria met

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

Thrombopoietin Receptor Agonists (TRAs) such as Avatrombopag, Romiplostim, and Eltrombopag work by mimicking the action of thrombopoietin, a natural hormone that regulates platelet production. These drugs bind to and activate the thrombopoietin receptor on megakaryocytes, the bone marrow cells responsible for producing platelets, thereby increasing platelet production. This mechanism is crucial for patients with Thrombocytopenic Purpura, a condition characterized by abnormally low platelet counts, as it helps to elevate platelet levels, reduce bleeding risks, and improve overall quality of life. By targeting the underlying issue of insufficient platelet production, TRAs offer a targeted and effective treatment option for managing this condition.