Romiplostim for Aplastic Anemia

Overseen By Anjali Sharathkumar, MD

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Anjali Sharathkumar

Must not be taking: Eltrombopag, Androgens, others

Disqualifiers: PNH, Thrombosis, Cancer predisposition, MDS, 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: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the effectiveness of a drug called romiplostim for individuals with severe aplastic anemia (SAA), a condition where the bone marrow produces insufficient blood cells. Participants are divided into two groups: one group, consisting of those untreated previously, will receive romiplostim with immunosuppressive therapy; the other group, comprising individuals whose previous treatments failed, will receive romiplostim alone. This trial may suit children or young adults with severe aplastic anemia, whether they are new to treatment or have tried other treatments without success. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications like eltrombopag, avatrombopag, lusutrombopag, and androgens before participating. If you've stopped androgen therapy for more than 2 weeks, you may be eligible to join the study.

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

Research has shown that romiplostim has been tested for safety in people with aplastic anemia (AA). One study found that romiplostim, used alone, was safe starting at doses of 20 micrograms per kilogram each week. It worked quickly in those whose aplastic anemia did not respond to other treatments, with most patients tolerating the treatment well and experiencing no serious side effects.

When combined with immunosuppressive therapy (IST), romiplostim also appeared safe. Although long-term safety data is limited, current information suggests that romiplostim, whether used alone or with IST, is generally well-tolerated in both children and adults with severe aplastic anemia. ^{1 2 3 4 5}

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

Romiplostim is unique because it boosts the production of platelets by mimicking the natural hormone thrombopoietin, which is a different approach compared to traditional treatments for aplastic anemia that often involve immunosuppressive therapy or bone marrow transplants. Researchers are excited because Romiplostim offers a potential new way to help patients who are either new to treatment or have not responded to previous therapies. By directly stimulating platelet production, Romiplostim could reduce the need for transfusions and improve blood counts more effectively than existing options.

What evidence suggests that romiplostim might be an effective treatment for severe aplastic anemia?

In this trial, participants with severe aplastic anemia (SAA) will receive treatments tailored to their condition. Research has shown that romiplostim, administered alone to participants in Cohort B, can effectively treat severe aplastic anemia, particularly in patients unresponsive to other treatments. Approximately 70% of patients have experienced improvements in blood counts with this treatment. Participants in Cohort A will receive romiplostim combined with immunosuppressive therapy (IST). Studies have shown that this combination yields promising results, with response rates between 70% and 95%. This suggests that romiplostim can enhance blood cell production, crucial for managing aplastic anemia. However, researchers continue to study the long-term effectiveness and safety beyond one year. ^{1 6 7 8 9}

Who Is on the Research Team?

Anjali Sharathkumar, MD

Principal Investigator

University of Iowa

Are You a Good Fit for This Trial?

This trial is for children and young adults with severe aplastic anemia (SAA), who haven't been treated yet or those whose condition didn't improve after previous treatments. Specific eligibility details are not provided, but typically participants must meet certain health standards.

Inclusion Criteria

Parent, guardian, or patient (if >18 years of old) must be able to provide written and voluntary informed consent and assent (if >12 years of age)

I have been diagnosed with severe aplastic anemia or a related condition.

My organs are functioning well enough for me to join the study.

Exclusion Criteria

Treatment with an investigational drug within specified timeframe

Patients infected with Human Immunodeficiency Virus (HIV)

Known hypersensitivity reactions or allergies to specific substances

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive romiplostim, with or without immunosuppressive therapy, for severe aplastic anemia

24 weeks

Regular visits as per protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year

Periodic visits for safety assessment

What Are the Treatments Tested in This Trial?

Interventions

• Romiplostim

Trial Overview The study tests the effectiveness of adding romiplostim to standard immunosuppressive therapy (IST) in inducing blood cell production across all three lineages in patients with SAA. It's a Phase II trial, meaning it focuses on efficacy and side effects.

How Is the Trial Designed?

Group I: Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST) Experimental Treatment 2 Interventions

Treatment naïve SAA (Cohort A) will be treated with romiplostim and immunosuppressive therapy (IST).

Group II: Relapsed or refractory SAA (Cohort B): Romiplostim Experimental Treatment 1 Intervention

Relapsed or refractory SAA (Cohort B) will be treated with romiplostim alone.

Who Is Running the Clinical Trial?

Anjali Sharathkumar

Lead Sponsor

Trials

1

Recruited

20+

Department of Health and Human Services

Collaborator

Trials

240

Recruited

944,000+

Food and Drug Administration (FDA)

Collaborator

Trials

184

Recruited

1,553,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12180318/

Effective treatment of refractory aplastic anemia with ... High-dose ROM with an initial dosage of 20 μg/kg per week is effective and safe for patients with refractory AA who experienced treatment failure.

2. ashpublications.org ashpublications.org/bloodadvances/article/8/6/1415/506709/Long-term-efficacy-and-safety-of-romiplostim-in

Long-term efficacy & safety of romiplostim in aplastic anemia However, long-term efficacy and safety data of romiplostim for refractory AA beyond 53 weeks remain scarce. To clarify this, we prospectively ...

3. haematologica.org haematologica.org/article/view/12263/78781

Romiplostim for aplastic anemia: beyond an eltrombopag ... Table 1 summarizes four studies that reported similar response rates of approximately 70%, suggesting that ROMI at 20 μg/kg/week may exert a ...

4. link.springer.com link.springer.com/article/10.1007/s00277-025-06337-7

Predictive Factors of Romiplostim Response in Anemia At 27 weeks, no patients achieved a complete response, 57.4% (35/61) achieved a partial response, 49.0% (24/49) became red blood cell ...

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40027000/

Romiplostim in Aplastic Anemia: Single-Center Study The primary outcome was to evaluate the increase in platelet count/percentage and the safety of romiplostim in these patients. The secondary ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10950812/

Long-term efficacy and safety of romiplostim in refractory ... However, long-term efficacy and safety data of romiplostim for refractory AA beyond 53 weeks remain scarce. To clarify this, we prospectively evaluated clinical ...

7. haematologica.org haematologica.org/article/view/12189/78655

Safety and efficacy of romiplostim in children with acquired ... High-dose romiplostim accelerates hematologic recovery in patients with aplastic anemia refractory to eltrombopag. Leukemia. 2021;35(3):906-909.

8. tandfonline.com tandfonline.com/doi/full/10.1080/07853890.2025.2514791

Full article: Effective treatment of refractory aplastic anemia ... Romiplostim was safe and effective at an initial dosage of 20 μg/kg per week, with rapid responses in patients with refractory aplastic anemia who experienced ...

9. aamds.org aamds.org/research-article/romiplostim-treatment-children-and-young-adults-severe-aplastic-anemia-and

Romiplostim for Treatment of Children and Young Adults ... This proof-of-concept study provides data about short-term safety, tolerability, and preliminary efficacy of romiplostim (±IST) for treatment of pediatric SAA/ ...