Eltrombopag vs Standard Treatment for ITP in Children

No longer recruiting at 27 trial locations

Overseen By Rohith Jesudas, MD

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Baylor College of Medicine

Must be taking: IVIg, Steroids, Anti-D

Must not be taking: Anticoagulants, Anti-platelets

Disqualifiers: Severe bleeding, HIV, Hepatitis C, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 5 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 explores the effectiveness of eltrombopag, a medication, compared to standard treatments for children with Immune Thrombocytopenia (ITP). ITP causes low platelet counts, leading to easy bruising and bleeding. Participants will receive either eltrombopag or a typical treatment such as steroids or IVIg. The trial targets children diagnosed with ITP within the last three months who need treatment to manage their condition. Participants should either not have responded well to standard treatments previously or be newly diagnosed and untreated. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

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

The trial does not specify if you need to stop taking your current medications, but it mentions that patients should not require additional therapy at the time of enrollment. It's best to discuss your current medications with the trial investigator to see if they might affect your eligibility.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that eltrombopag is generally safe for treating immune thrombocytopenia (ITP) in both adults and children. In everyday use, about 32% of patients report experiencing one or more side effects while taking eltrombopag, but only a few of these are serious.

Long-term studies indicate that eltrombopag effectively maintains healthy platelet levels, reducing the risk of bleeding. This suggests the treatment is usually well-tolerated. Eltrombopag is also approved for long-term use in ITP, which adds to its safety assurance.

Overall, while some side effects may occur, evidence supports eltrombopag as a safe option for managing ITP. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ITP?

Eltrombopag is unique because it works by stimulating the production of platelets in the bone marrow, offering a different mechanism of action compared to standard treatments like steroids, IVIg, or anti-D globulin, which primarily focus on suppressing the immune system. Researchers are excited about eltrombopag because it targets the underlying problem of low platelet production in children with ITP (Immune Thrombocytopenia). Additionally, eltrombopag provides an oral treatment option, potentially making it easier for children and their families to manage compared to intravenous options. This novel approach could improve patient outcomes and quality of life by addressing the root cause rather than just managing symptoms.

What evidence suggests that Eltrombopag could be an effective treatment for ITP in children?

Studies have shown that eltrombopag, one of the treatments tested in this trial, effectively treats immune thrombocytopenia (ITP) in children. In one study, children who took eltrombopag had higher platelet counts, which help prevent bleeding. Most of these children maintained their platelet levels and experienced fewer bleeding episodes. Long-term use of eltrombopag has proven safe and effective, keeping platelet counts steady over time. Additionally, real-world evidence suggests eltrombopag leads to better results compared to other treatments like romiplostim, rituximab, and splenectomy (removal of the spleen). These findings support eltrombopag as a reliable option for managing ITP in children. Participants in this trial will be randomized to receive either eltrombopag or standard first-line therapy, which includes options like IVIg, steroids, or Rho(D) Immune Globulin. ^{2 6 7 8 9}

Who Is on the Research Team?

Amanda Grimes, MD

Principal Investigator

Baylor College of Medicine - Texas Children's Hospital

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I am seeking treatment for the first time or after a previous treatment didn't work.

I was diagnosed with ITP within the last 10 days and haven't been treated, or my initial ITP treatments didn't work.

My doctor says I need medication for my condition.

Exclusion Criteria

My heart does not function properly.

You have a history of using drugs or alcohol in a harmful way.

You have had a bad reaction to eltrombopag or any of its ingredients in the past.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either eltrombopag or standard therapy for 12 weeks

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

40 weeks

Open-label extension

Participants may continue eltrombopag treatment for up to 1 year based on response

1 year

What Are the Treatments Tested in This Trial?

Interventions

• Eltrombopag

Trial Overview 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.

How Is the Trial Designed?

Group I: Eltrombopag Experimental Treatment 1 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 therapy Active Control 3 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)

Eltrombopag is already approved in United States, European Union, Canada, Japan, China for the following indications:

Approved in United States as Promacta for:

• Severe aplastic anemia
• Chronic immune thrombocytopenia
• Thrombocytopenia in patients with chronic hepatitis C

Approved in European Union as Revolade for:

• Severe aplastic anemia
• Chronic immune thrombocytopenia

Approved in Canada as Promacta for:

• Severe aplastic anemia
• Chronic immune thrombocytopenia

Approved in Japan as Revolade for:

• Severe aplastic anemia
• Chronic immune thrombocytopenia

Approved in China as Promacta for:

• Severe aplastic anemia
• Chronic immune thrombocytopenia

Who Is Running the Clinical Trial?

Baylor College of Medicine

Lead Sponsor

Trials

1,044

Recruited

6,031,000+

Boston Children's Hospital

Collaborator

Trials

801

Recruited

5,584,000+

University of California, San Francisco

Collaborator

Trials

2,636

Recruited

19,080,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5971401/

Eltrombopag in immune thrombocytopenia: efficacy review ... This review summarizes the experience reported using eltrombopag in ITP, paying attention to efficacy and safety. Results from clinical trials will be discussed ...

2. revolade.com revolade.com/efficacy/immune-thrombocytopenia/

Efficacy: ITP Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015;386(10004):1649-1658.

3. novartis.com novartis.com/us-en/news/media-releases/real-world-data-show-novartis-drug-promacta-improves-outcomes-itp-patients-compared-other-second-line-therapies

Real-world data show Novartis drug Promacta® improves ... Promacta (eltrombopag) showed lower rate of bleeding-related episodes and similar rate of thrombotic events vs. romiplostim, rituximab and splenectomy, ...

4. ashpublications.org ashpublications.org/blood/article/130/23/2527/36754/Safety-and-efficacy-of-long-term-treatment-of

Safety and efficacy of long-term treatment of chronic/persistent ... Long-term use of eltrombopag was effective in maintaining platelet counts of 50 × 10 9 /L or more and reducing bleeding in most patients with ITP of more than ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8060759/

Eltrombopag Effectiveness and Tolerability in Chronic Immune ... Eltrombopag treatment in patients with chronic immune-mediated thrombocytopenia can prolong survival.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7675127/

Real-world experience of eltrombopag in immune ... Seventeen patients (17/53, 32%) reported one or more adverse events while on eltrombopag therapy. A total of 49 adverse events (n=4 with grade ≥3 CTCAEv4) were ...

7. revolade.com revolade.com/safety/immune-thrombocytopenia/

Revolade™ (eltrombopag olamine) | Safety: ITP REVOLADE® (eltrombopag) has a proven, well-established safety profile in adult and paediatric patients with chronic immune thrombocytopenia (ITP), ...

8. novartis.com novartis.com/us-en/news/media-releases/novartis-drug-promacta-shows-long-term-disease-control-chronicpersistent-immune-thrombocytopenia-itp

Novartis drug Promacta® shows long-term disease control ... Nearly 70% of patients maintained platelet counts of ≥30×10[9]/L without rescue therapy for prolonged periods, reducing the overall risk of bleeding- More ...

9. clinicaltrials.gov clinicaltrials.gov/study/NCT03771378

Efficacy and Safety of rhTPO and Eltrombopag in Patients ... This trial will compare the efficacy and safety of intravenous rhTPO or oral use of eltrombopag after 14 days of treatment in Chinese ITP patients.