Eltrombopag for Aplastic Anemia

Recruiting at 36 trial locations

Overseen By Novartis Pharmaceuticals

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Novartis Pharmaceuticals

Must be taking: Eltrombopag, Immunosuppressive therapy

Must not be taking: Thrombopoietin receptor agonists

Disqualifiers: Fanconi anemia, Myelodysplastic syndrome, Malignancy, HIV, Hepatitis B or C, others

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

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

Compensation: Varies

Number of Visits: 1

Duration: 26 weeks

51 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to study how eltrombopag (also known as Promacta or Revolade) works with other treatments for children with severe aplastic anemia, a condition where the bone marrow doesn't produce enough blood cells. The trial will test different combinations of eltrombopag with other medications to determine the most effective approach. It is open to children with aplastic anemia that hasn't been treated before or has recurred after treatment. This trial might suit children struggling with severe aplastic anemia, especially if options like a stem cell transplant aren't possible. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants, offering a promising opportunity for children to benefit from innovative therapies.

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

A previous study found eltrombopag to be generally safe for treating severe aplastic anemia (SAA) in adults. Some patients experienced changes in skin color and unusual liver test results, but these did not significantly impact overall safety. The FDA has approved eltrombopag for patients over 2 years old with SAA, providing some reassurance about its safety.

For treatment involving hATG, there is a risk of serious allergic reactions, including anaphylaxis, a severe and life-threatening reaction. Therefore, careful monitoring is crucial when using hATG.

Cyclosporine (CsA) is generally considered safe as an immune-suppressing treatment for severe aplastic anemia. Research has shown its safety, with no major unexpected side effects reported.

Each treatment has its own safety considerations, but they have been studied enough to provide a reasonable understanding of their risks. Participants should discuss these with their healthcare provider to determine what best fits their health needs. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Eltrombopag is unique because it offers a novel approach to treating aplastic anemia by stimulating the production of blood cells through a new mechanism. Unlike the standard treatments that primarily focus on suppressing the immune system with drugs like cyclosporine (CsA) and antithymocyte globulin (hATG), eltrombopag works as a thrombopoietin receptor agonist, directly encouraging the bone marrow to produce more platelets, red blood cells, and white blood cells. Researchers are excited about eltrombopag because it has the potential to enhance blood cell counts more effectively and safely, offering hope for patients who may not respond adequately to conventional therapies.

What evidence suggests that this trial's treatments could be effective for aplastic anemia?

Research has shown that eltrombopag, when combined with immunosuppressive therapy, may help treat severe aplastic anemia. In studies, about 55% of patients improved after 26 weeks, indicating that more than half experienced positive results. For some patients with hard-to-treat severe aplastic anemia, improvement rates were even higher, around 40–50%. In this trial, participants in Cohort A (Option 2) will receive eltrombopag combined with CsA, while those in Cohort A (Option 1) and Cohort B will receive eltrombopag, CsA, and horse ATG (hATG) therapy. Eltrombopag alone increased platelet counts in about 41% of patients. Additionally, hATG therapy has demonstrated good results, with high survival rates and effective blood cell responses. These findings suggest that eltrombopag, especially when combined with other treatments, can be effective for this condition. ^{6 7 8 9 10}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for pediatric patients aged 1 to <18 with severe aplastic anemia (SAA) or recurrent aplastic anemia who haven't had a transplant option. They must not have certain bone marrow failure syndromes, prior immunosuppressive therapy, or active infections. Participants will receive eltrombopag alongside standard immunosuppressive treatments.

Inclusion Criteria

I agree to be treated with hATG, CsA, and eltrombopag together.

I have never been treated with IST.

I was previously diagnosed with severe aplastic anemia.

Exclusion Criteria

My tests show genetic abnormalities in my cells.

I have a known genetic condition affecting my bone marrow.

I have been diagnosed with Fanconi anemia.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1 week

1 visit (in-person)

Treatment

Participants receive eltrombopag in combination with immunosuppressive therapy for 26 weeks

26 weeks

Regular visits for dose assessment and modification

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

Periodic visits for monitoring

Long-term Follow-up

Participants are monitored annually for up to 3 years for long-term safety and effectiveness

3 years

Annual visits

What Are the Treatments Tested in This Trial?

Interventions

• CsA
• Eltrombopag
• hATG

Trial Overview The study tests how the body processes the drug eltrombopag when given with hATG and CsA in children with SAA or recurrent AA. It's an open-label, multi-center trial where doses of eltrombopag are increased within each patient to understand its pharmacokinetics.

How Is the Trial Designed?

Group I: Cohort B Experimental Treatment 3 Interventions

previously untreated SAA, hATG (ATGAM®), CsA and eltrombopag begin on Day 1 and all patients will be treated with the same regimen

Group II: Cohort A (option 2) Experimental Treatment 2 Interventions

CsA and eltrombopag begin on Day 1.

Group III: Cohort A (Option 1) Experimental Treatment 3 Interventions

Regimen 1: hATG (ATGAM®), CsA and eltrombopag begin on Day 1.

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8718619/

Long-term outcomes in patients with severe aplastic ... The majority of treated patients achieved an overall hematologic response rate (ORR) at 6 months, and most importantly, EPAG addition resulted in a more robust ...

2. ashpublications.org ashpublications.org/bloodadvances/article/9/15/3728/537097/Eltrombopag-in-combination-with-immunosuppressive

Eltrombopag in combination with immunosuppressive therapy ... The overall response rate (ORR; per North American Pediatric Aplastic Anemia Consortium criteria) at 26 weeks was 54.9% in both cohorts combined and 71.4% and ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7940771/

Current evidence and the emerging role of eltrombopag in ... First studies with eltrombopag as a single agent at 150 mg/day showed an overall response rate of 40–50% in patients with refractory severe AA (rSAA).

4. tandfonline.com tandfonline.com/doi/full/10.1080/16078454.2024.2335419

Safety and efficacy of eltrombopag in patients with aplastic ... Eltrombopag combined with immunotherapy showed significant improvements in 6-month CR (OR: 2.20; 95% CI;1.54–3.12; P < 0.0001) and 6-month OR ( ...

5. onclive.com onclive.com/view/generic-eltrombopag-is-now-available-for-severe-aplastic-anemia-and-immune-thrombocytopenia

Generic Eltrombopag Is Now Available for Severe Aplastic ... The same patient population was evaluated in PETIT2, and those treated with eltrombopag (n = 63) achieved a platelet count response rate of 41% ...

6. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/5636/504533/Real-Life-Data-of-Cyclosporine-CsA-Tapering-in

Real-Life Data of Cyclosporine (CsA) Tapering in Patients with ... Real-life data of cyclosporine (CsA) tapering in patients with very/severe aplastic anemia (v/sAA) treated with horse anti-thymocyte globulin (hATG/CsA) ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6089711/

Cyclosporine monotherapy for severe aplastic anemia Our results support findings that cyclosporine monotherapy is an effective and safe immunosuppressive therapy for SAA, and that it could be a reasonable IST ...

8. nature.com nature.com/articles/s41408-025-01328-3

Efficacy and safety of immunosuppressive therapy versus ... The combination of CsA + AVA achieved comparable efficacy with superior safety compared to the combination of ATG + CsA + AVA in older adults ...

9. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497123122361

Real-Life Data of Cyclosporine (CsA) Tapering in Patients ... We analyzed data from our registries including CsA plasma levels of 20 s/vsAA pts. with and without relapse during CsA tapering after treatment with horse ATG/ ...

10. clinicaltrials.gov clinicaltrials.gov/study/NCT01193283

Cyclophosphamide Plus Cyclosporine in Treatment-Naive ... Moderate-dose cyclophosphamide for severe aplastic anemia has significant toxicity and does not prevent relapse and clonal evolution. Blood. 2014 Oct 30;124 ...