Avatrombopag for Pediatric ITP

Recruiting at 115 trial locations

Overseen By Sobi Inc

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Dova Pharmaceuticals

Disqualifiers: Secondary ITP, Thrombosis, MDS, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

75 Participants Needed

Trial Summary

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial team for guidance.

What makes the drug Avatrombopag unique for treating pediatric ITP?

Avatrombopag is unique because it is an oral medication that stimulates platelet production without the need for food restrictions and has a low risk of liver damage, making it a convenient and well-tolerated option compared to other treatments for immune thrombocytopenia (ITP). ^{1 2 3 4 5}

What is the purpose of this trial?

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.

Eligibility Criteria

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.

Inclusion Criteria

Your blood platelet counts have been consistently low during the screening period.

I am under 18, have had ITP for over 6 months, and treatments haven't worked well.

Exclusion Criteria

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

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

I have never been diagnosed with myelodysplastic syndrome.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive avatrombopag or placebo in a double-blind manner for 12 weeks

12 weeks

Weekly visits for platelet count monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of avatrombopag treatment for up to 2 years

2 years

Treatment Details

Interventions

• Avatrombopag
• Placebo

Trial Overview 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.

Participant Groups

Group I: Experimental: Avatrombopag Double Blind Experimental Treatment 1 Intervention

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

Group II: Avatrombopag Open Label Extension Experimental Treatment 1 Intervention

Investigational product administered orally for up to 2 years.

Group III: Placebo Comparator:Placebo Double Blind Placebo Group 1 Intervention

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

Avatrombopag is already approved in United States, European Union for the following indications:

Approved in United States as Doptelet for:

• Thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure
• Chronic immune thrombocytopenia

Approved in European Union as Doptelet for:

• Thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure
• Chronic immune thrombocytopenia

Who Is Running the Clinical Trial?

Dova Pharmaceuticals

Lead Sponsor

Trials

6

Recruited

310+

Sobi, Inc.

Lead Sponsor

Trials

14

Recruited

1,000+

Findings from Research

Avatrombopag is an effective oral treatment for immune thrombocytopenia (ITP) and periprocedural thrombocytopenia in chronic liver disease, providing a safer alternative to blood transfusions without the need for food restrictions.

It has a favorable safety profile, showing no significant hepatotoxicity or portal vein thrombosis, and is generally well-tolerated, with side effects similar to those of a placebo in clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Avatrombopag for the treatment of immune thrombocytopenia and thrombocytopenia of chronic liver disease. Cheloff, AZ., Al-Samkari, H.[2020]

Avatrombopag is an effective oral treatment for adults with chronic immune thrombocytopenia (ITP), showing significantly greater platelet responses compared to placebo in phase III studies.

The drug also reduces the need for platelet transfusions in patients with chronic liver disease undergoing invasive procedures, and it is generally well tolerated, making it a valuable second-line treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Avatrombopag: A Review in Thrombocytopenia. Markham, A.[2022]

Avatrombopag (Doptelet®) is an oral medication that acts as a thrombopoietin receptor agonist, specifically designed to treat low platelet counts (thrombocytopenia) in adults with chronic liver disease who are about to undergo a medical procedure.

It received its first global approval in May 2018 in the USA, and further clinical studies are being conducted to explore its effectiveness in treating other conditions related to low platelet counts, such as immune thrombocytopenic purpura and chemotherapy-induced thrombocytopenia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Avatrombopag: First Global Approval. Shirley, M.[2019]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Avatrombopag for the treatment of immune thrombocytopenia and thrombocytopenia of chronic liver disease. [2020]

2. England pubmed.ncbi.nlm.nih.gov

An evaluation of avatrombopag for the treatment of thrombocytopenia. [2021]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Avatrombopag: A Review in Thrombocytopenia. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Catastrophic antiphospholipid syndrome in an immune thrombocytopenia patient treated with avatrombopag. [2023]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Avatrombopag: First Global Approval. [2019]