Avatrombopag for Pediatric ITP

Not currently recruiting at 135 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called avatrombopag to determine its effectiveness for children and teens with immune thrombocytopenia (ITP), a condition where the immune system attacks and destroys platelets, causing easy bruising and bleeding. The trial aims to assess whether avatrombopag is safe and effective in increasing platelet counts. Participants will receive either avatrombopag or a placebo (a pill with no active medicine) for 12 weeks, with some continuing avatrombopag for up to two years. Children and teens who have had ITP for at least six months and have not responded to other treatments may be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially bringing a new treatment to market.

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.

Is there any evidence suggesting that avatrombopag is likely to be safe for pediatric patients with ITP?

Research has shown that avatrombopag is safe and well-tolerated for treating children with immune thrombocytopenia (ITP). Studies have found that this oral medication has few side effects and can be taken with any food, making it easy to incorporate into daily routines. Importantly, previous research with children reported no deaths, serious blood clots, or severe bleeding events. Additionally, the FDA has already approved avatrombopag for treating long-term ITP, supporting its safety. Overall, the evidence suggests that avatrombopag is a promising and safe option for children. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Avatrombopag is unique because it directly stimulates platelet production by activating the thrombopoietin receptor, which is different from how many current treatments for pediatric immune thrombocytopenia (ITP) work. Most existing treatments, like steroids or immunoglobulins, primarily aim to reduce the immune system's attack on platelets or increase platelet counts indirectly. Avatrombopag's targeted action offers the potential for fewer side effects and more sustained platelet production. Researchers are excited about this approach because it could provide a more effective and longer-lasting treatment option for children with ITP, potentially improving their quality of life.

What evidence suggests that Avatrombopag might be an effective treatment for pediatric ITP?

Research has shown that avatrombopag, which participants in this trial may receive, works well for children with immune thrombocytopenia (ITP) lasting six months or more. Studies have found that it raises platelet counts, reducing the risk of bleeding in these patients. Taken orally, avatrombopag has proven safe and effective for children. Data from several trials confirm that it quickly and consistently improves platelet counts, making it a promising option for managing ITP in children and teenagers. ^{3 4 6 7 8}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

Citations

1. ashpublications.org ashpublications.org/ashclinicalnews/news/8724/Phase-IIIb-Results-Show-Avatrombopag-to-Be-Safe

Phase IIIb Results Show Avatrombopag to Be Safe, Effective ... Results from the phase IIIb AVA-PED-301 trial indicate that avatrombopag is an effective oral treatment for children and adolescents with a ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40418942/

Avatrombopag for the treatment of children and ... Avatrombopag is an effective oral treatment for children and adolescents with ITP for at least 6 months and has a reassuring safety profile in the paediatric ...

3. thelancet.com thelancet.com/journals/lanhae/article/PIIS2352-3026(25)00107-3/abstract

Avatrombopag for the treatment of children and ... Avatrombopag for the treatment of children and adolescents with immune thrombocytopenia (AVA-PED-301): a multicentre, randomised, double-blind, placebo- ...

4. sobi.com sobi.com/en/press-releases/sobi-announces-positive-results-phase-3-study-dopteletr-treatment-children-and-adolescents-itp-2215547

Sobi announces positive results from phase 3 study of ... The primary endpoint was met, confirming the efficacy and safety of avatrombopag (Doptelet®) in treating children and adolescents with ITP ...

5. doptelethcp.com doptelethcp.com/pediatric-itp/efficacy

Clinical Efficacy of DOPTELET® for Pediatric ITP Doptelet and Doptelet Sprinkle delivered fast-acting, long-lasting platelet count improvements in pediatric patients with persistent or chronic ITP. 1. Doptelet ...

6. doptelethcp.com doptelethcp.com/pediatric-itp/safety-profile

Doptelet Safety Profile in Pediatric ITP Treatment Doptelet and Doptelet Sprinkle should not be administered to patients with persistent or chronic ITP in an attempt to normalize platelet counts. Monitor ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2025/219696s000lbl.pdf

Reference ID: 5631118 - accessdata.fda.gov The safety and effectiveness of DOPTELET tablet for the treatment of persistent or chronic ITP have been established in pediatric patients aged 6 years and ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT04516967

Study Details | NCT04516967 | Avatrombopag for the ... Study Overview. Brief Summary. A Phase 3b Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects ...