Romiplostim for Bone Cancer

Overseen By Brian Turpin, DO

Age: Any Age

Sex: Any

Trial Phase: Phase < 1

Sponsor: Children's Hospital Medical Center, Cincinnati

Must not be taking: Romiplostim, Eltrombopag, Steroids, others

Disqualifiers: Metastatic marrow disease, Bone marrow failure, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

26 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests romiplostim, a supportive treatment, for individuals newly diagnosed with Ewing sarcoma, a type of bone cancer, who are undergoing intensive chemotherapy. The primary goal is to determine if romiplostim can manage low platelet counts caused by chemotherapy, which can lead to easy bruising or bleeding. The trial also assesses the safety and practicality of including romiplostim in the treatment plan. Suitable candidates are those recently diagnosed with Ewing sarcoma who are not receiving certain other cancer treatments, such as extensive radiation or therapies affecting bone marrow. As an Early Phase 1 trial, this research focuses on understanding how romiplostim works in people, offering participants a chance to contribute to groundbreaking treatment development.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but certain treatments like previous use of romiplostim, eltrombopag, or other platelet-producing agents, and erythropoietin-stimulating agents are not allowed. If you are on these or similar treatments, you may need to stop them to participate.

Is there any evidence suggesting that romiplostim is likely to be safe for humans?

Research has shown that romiplostim has been tested for safety in various situations. In some studies, about 9% of patients experienced serious side effects from the treatment. A small number of patients had changes in their bone marrow, where blood cells are made, but this was uncommon.

Romiplostim has also been used in patients with solid tumors. In these cases, 71% of patients responded well to the treatment, and 79% avoided issues like delayed chemotherapy or needing lower doses. This suggests that many people generally tolerate the treatment well.

Romiplostim is already approved for some other uses, which can be reassuring about its safety. However, discussing any concerns with a healthcare provider before joining a trial is always advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Romiplostim is unique because it targets platelet production, which is not a typical focus in bone cancer treatment. Most existing therapies for bone cancer, like chemotherapy and radiation, primarily aim to kill cancer cells directly. Romiplostim, however, acts as a thrombopoietin receptor agonist, stimulating the body's production of platelets. This feature can be particularly beneficial for patients undergoing chemotherapy, as it can help manage and prevent low platelet counts, a common side effect. Researchers are excited about Romiplostim because it offers a novel way to support patients' blood health during intensive cancer treatments.

What evidence suggests that romiplostim might be an effective treatment for Ewing sarcoma?

Research has shown that romiplostim can help cancer patients manage low platelet counts caused by chemotherapy. In one study, 71% of patients with solid tumors responded well to romiplostim. Another study found that 93% of patients had their platelet counts return to normal within three weeks. Romiplostim also helped 79% of patients avoid delays or reductions in their chemotherapy doses. In this trial, participants will receive romiplostim alongside chemotherapy cycles, which might help patients with Ewing sarcoma maintain healthier platelet levels during treatment. ^{6 7 8 9 10}

Who Is on the Research Team?

Brian Turpin, DO

Principal Investigator

Children's Hospital Medical Center, Cincinnati

Are You a Good Fit for This Trial?

This trial is for patients newly diagnosed with Ewing sarcoma, a type of bone cancer, who are about to start chemotherapy and are at risk of developing low platelet counts (thrombocytopenia). Specific eligibility criteria were not provided.

Inclusion Criteria

Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines

I am older than 1 year.

I have been newly diagnosed with Ewing sarcoma and am treated with specific chemotherapy protocols.

Exclusion Criteria

Concomitant therapy, non-cancer directed: Patients requiring hematopoietic stem cell rescue are not eligible. Previous use of romiplostim, eltrombopag or any other platelet-producing agent is not allowed. Previous therapy for immune thrombocytopenia and related conditions, including rituximab, mycophenolic acid, protracted systemic steroids, and/or IVIG, is prohibited. Treatment with erythropoietin-stimulating agents is prohibited. Patients receiving another investigational drug are not eligible. Patients who are receiving prophylactic dosing of heparin (i.e. enoxaparin) or oral anticoagulants (i.e. rivaroxaban) for thrombosis prevention may be considered for enrollment but will be excluded from secondary aim 'a' analysis (efficacy measured as the median platelet count and transfusion dependency) given shift in transfusion thresholds

Patients who in the opinion of the investigator may not be able to comply with the study (including safety monitoring requirements of the study) are not eligible

My Ewing sarcoma has not spread to my bone marrow.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive romiplostim as a supportive care measure alongside interval-compressed chemotherapy for Ewing sarcoma

52 weeks

Weekly visits for romiplostim administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Romiplostim

Trial Overview The study tests romiplostim's effectiveness in preventing low platelet counts caused by chemotherapy in Ewing sarcoma patients. It aims to see if this drug can help maintain safe platelet levels during treatment.

How Is the Trial Designed?

Group I: Romiplostim administration with chemotherapy cycles Experimental Treatment 1 Intervention

Enrolled patients may start romiplostim, as early as cycle 1 day 1; all patients MUST initiate romiplostim no later than 2 weeks from the start of the 5th cycle of chemotherapy. If plt count \< 200,000/mm3, patients will start romiplostim based on their weight.

Who Is Running the Clinical Trial?

Children's Hospital Medical Center, Cincinnati

Lead Sponsor

Trials

844

Recruited

6,566,000+

Citations

1. journals.lww.com journals.lww.com/md-journal/fulltext/2025/08080/successful_treatment_of_refractory_cancer.125.aspx

Successful treatment of refractory cancer therapy-induced... Romiplostim N01 effectively managed refractory CTIT in this case, showing rapid and sustained platelet recovery. This case demonstrates the ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8018116/

A multicenter study of romiplostim for chemotherapy-induced ... Romiplostim was effective in solid tumor patients: 71% of patients achieved a romiplostim response, 79% avoided chemotherapy dose reductions/treatment delays, ...

3. library.ehaweb.org library.ehaweb.org/eha/2025/eha2025-congress/4162746/ji.ma.real-world.effectiveness.of.romiplostim.n01.in.cancer.treatment-induced.html?f=listing%3D3%2Abrowseby%3D8%2Asortby%3D1%2Amedia%3D1

real-world effectiveness of romiplostim n01 in cancer ... The overall treatment efficacy within 3 weeks was 80.8%. Anti-platelet antibody testing in 24 patients showed 50% positivity, with lower ...

4. ashpublications.org ashpublications.org/blood/article/134/Supplement_1/389/426153/A-Multicenter-Study-of-Romiplostim-to-Treat

A Multicenter Study of Romiplostim to Treat Chemotherapy ... Both weekly and intracycle dosing were effective and equally safe, but weekly dosing maintained higher Plt overall and higher Plt nadirs with ...

5. ascopubs.org ascopubs.org/doi/10.1200/JCO.18.01931

Romiplostim Treatment of Chemotherapy-Induced ... In the randomization phase, 14 of 15 romiplostim-treated patients (93%) experienced correction of their platelet count within 3 weeks, compared ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4888762/

Long-term safety and efficacy of romiplostim for treatment ... This review focuses on the efficacy data and safety analysis of the pooled data from the clinical trials evaluating romiplostim for treatment of adults with ...

7. ashpublications.org ashpublications.org/blood/article/113/10/2161/24354/Safety-and-efficacy-of-long-term-treatment-with

Safety and efficacy of long-term treatment with romiplostim in ... Treatment-related serious adverse events were reported in 13 patients (9%). Bone marrow reticulin was observed in 8 patients; marrows were not routinely ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT07048249

NCT07048249 | Single Arm Romiplostim to Prevent CIT The goal of this clinical trial is to to assess the efficacy of romiplostim as a supportive care measure in patients with a new diagnosis of Ewing sarcoma ...

9. haematologica.org haematologica.org/article/view/9776

A multicenter study of romiplostim for chemotherapy ... Romiplostim was effective in solid tumor patients: 71% of patients achieved a romiplostim response, 79% avoided chemotherapy dose reductions/treatment delays ...

10. aetna.com aetna.com/cpb/medical/data/700_799/0768.html

Romiplostim (Nplate) - Medical Clinical Policy Bulletins The major safety concerns consisted of risks for bone marrow reticulin formation and worsened thrombocytopenia (compared to baseline) following romiplostim ...