Romiplostim for Chemotherapy-Induced Low Platelet Count

Recruiting at 128 trial locations

Overseen By Apostolos Kontzias

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Amgen

Must be taking: Carboplatin-based chemotherapy

Must not be taking: TPO receptor agonists

Disqualifiers: Leukemia, Myeloma, Heart failure, 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: 10-24 weeks

162 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests romiplostim (also known as Nplate or AMG 531), a treatment that may assist individuals with low platelet counts due to chemotherapy. It focuses on patients with non-small cell lung cancer, ovarian cancer, or breast cancer. The trial aims to determine if romiplostim enables these patients to continue their chemotherapy as planned without delays. Candidates may qualify if they have low platelet levels and are on a specific chemotherapy regimen delayed due to low platelets. As a Phase 3 trial, this study represents the final step before FDA approval, offering patients an opportunity to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, it does mention that certain investigational drugs or treatments cannot be taken concurrently, so it's best to discuss your specific medications with the trial team.

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

Research has shown that romiplostim is generally well-tolerated by people with low platelet counts due to chemotherapy. In previous studies, romiplostim increased platelet counts from 63,000 to 141,000 per microliter in about two weeks, a safe range for patients to continue chemotherapy.

Further studies indicate that 87% of patients experienced a significant increase in platelet counts, occurring about 67% of the time during treatment. This suggests that romiplostim not only boosts platelet counts but is also safe for long-term use in many patients.

Overall, romiplostim appears to be a safe option for managing low platelet counts caused by chemotherapy. It helps patients stay on track with their cancer treatments, which is important for their overall care plan. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for low platelet count?

Romiplostim is unique because it's designed to specifically target low platelet counts induced by chemotherapy, a common side effect that current treatments like platelet transfusions or thrombopoietin receptor agonists aim to manage but not prevent. Unlike these standard treatments, which often provide temporary relief, romiplostim works by stimulating the bone marrow to produce more platelets naturally. This mechanism could potentially reduce the need for frequent transfusions, offering a more sustainable and proactive management of platelet levels. Researchers are excited about romiplostim because of its potential to improve the quality of life for patients undergoing chemotherapy by minimizing treatment interruptions and associated complications.

What evidence suggests that romiplostim could be an effective treatment for chemotherapy-induced low platelet count?

Research has shown that romiplostim, which participants in this trial may receive, can help increase low platelet counts caused by chemotherapy. One study found that 93% of patients who took romiplostim reached their target platelet levels within three weeks. Another study reported that 71% of patients responded well to romiplostim, and 79% continued their chemotherapy without delays or dose reductions. Long-term use demonstrated that 87% of patients experienced a significant increase in platelet counts. These findings suggest that romiplostim can help keep chemotherapy on schedule by boosting platelet levels. ^{1 2 3 6 7}

Who Is on the Research Team?

MD

Principal Investigator

Amgen

Are You a Good Fit for This Trial?

Adults over 18 with advanced or recurrent NSCLC, breast, or ovarian cancer eligible for chemotherapy can join. They must have low platelet counts due to chemo and be able to undergo at least 3 more cycles. Exclusions include HIV, recent heart issues, other cancers within 5 years (with exceptions), active infections, certain blood disorders, and unwillingness to use contraception.

Inclusion Criteria

Your platelet count must be less than or equal to 85,000 on the first day of the study.

I finished my last chemotherapy cycle at least 21 or 28 days ago, depending on the cycle length.

I am on a 21- or 28-day cancer treatment cycle with specific drugs, or I am about to start one after a delay.

Exclusion Criteria

Currently receiving (or plan to receive) treatment in another investigational device or drug study, or less than 28 days since ending treatment on another investigational device or drug study(ies). Other investigational procedures while participating in this study are excluded.

I am undergoing a treatment plan that includes chemotherapy along with radiation therapy or surgery.

I have multiple myeloma.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Treatment

Participants receive romiplostim or placebo during chemotherapy cycles to assess efficacy in treating chemotherapy-induced thrombocytopenia

10-24 weeks

Administered in the clinic by a qualified healthcare provider

Follow-up

Participants are monitored for safety and effectiveness after treatment

48 days

Long-term Follow-up

Participants are monitored for secondary malignancies, adverse events, and other long-term outcomes

Up to 36 months

What Are the Treatments Tested in This Trial?

Interventions

• Romiplostim

Trial Overview The trial is testing Romiplostim's effectiveness in treating low platelet counts caused by chemotherapy in patients with NSCLC, ovarian cancer, or breast cancer. The goal is to see if it helps them receive full-dose chemo on schedule without delays.

How Is the Trial Designed?

Group I: Romiplostim Experimental Treatment 1 Intervention

The study in a 2:1 randomization ratio(108 subjects to romiplostim). Amgen investigational product (romiplostim or placebo) will be administered in the clinic by a qualified healthcare provider as a subcutaneous injection.

Group II: Placebo Placebo Group 1 Intervention

The study in a 2:1 randomization ratio (54 subjects to placebo) Amgen investigational product (romiplostim or placebo) will be administered in the clinic by a qualified healthcare provider as a subcutaneous injection.

Who Is Running the Clinical Trial?

Amgen

Lead Sponsor

Trials

1,508

Recruited

1,433,000+

Founded

1980

Headquarters

Thousand Oaks, USA

Known For

Human Therapeutics

Top Products

Enbrel, Prolia, Neulasta, Otezla

Robert A. Bradway

Amgen

Chief Executive Officer since 2012

MBA from Harvard Business School

Paul Burton

Amgen

Chief Medical Officer since 2023

MD from University of London, PhD in Molecular and Cellular Biology from Imperial College London

Published Research Related to This Trial

Romiplostim (Nplate) was approved by the US FDA in August 2008 as a treatment for thrombocytopaenia in patients with chronic immune thrombocytopaenic purpura who did not respond adequately to other treatments like corticosteroids, immunoglobulins, or splenectomy.

As a thrombopoietin receptor agonist, romiplostim works by stimulating the production of platelets, which can help improve blood clotting in patients with low platelet counts.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Romiplostim. Cines, DB., Yasothan, U., Kirkpatrick, P.[2016]

In two case studies, patients with immune thrombocytopenia experienced stable platelet counts only through continuous weekly dosing of romiplostim, even when their platelet counts exceeded the FDA-recommended threshold of 400k/µL.

This approach of persistent dosing, along with vigilant monitoring, may help prevent dangerous fluctuations in platelet levels, suggesting a potential alternative dosing strategy for romiplostim.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dynamic dosing of romiplostim in patients with immune thrombocytopenia purpura: Two case reports. Gilreath, JA., Wei, M., Paul, S., et al.[2019]

Romiplostim (AMG531) effectively increases megakaryocyte proliferation in a dose-dependent manner, as demonstrated in an in vitro study using human cord blood-derived progenitor cells.

The study suggests that the effects of AMG531 on megakaryocyte function are linked to changes in AKT phosphorylation, providing insights into the mechanisms of action for this treatment in immune thrombocytopenia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

High doses of romiplostim induce proliferation and reduce proplatelet formation by human megakaryocytes. Currao, M., Balduini, CL., Balduini, A.[2021]

In a study of 84 adult patients at NYU Langone Health, romiplostim was used off-label for conditions like chemotherapy-induced thrombocytopenia and post-hematopoietic stem cell transplantation, with a median initial dose of 3.8 mcg/kg, which is higher than the FDA-recommended starting dose of 1 mcg/kg.

Over half of the patients (51%) achieved a platelet count of ≥50 × 10^9/L by the end of the first week, suggesting that higher initial doses may be effective and safe for improving platelet responses in thrombocytopenic patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Antithrombotic and hemostatic stewardship: Evaluation of romiplostim for treatment of thrombocytopenia at a large academic medical center. Wong, A., Ahuja, T., Cirrone, F., et al.[2023]

In a study of 18 hospitalized patients with severe immune thrombocytopenia, those receiving higher initial doses of romiplostim (≥2 mcg/kg/week) achieved platelet levels of ≥10 × 10^9/L significantly faster (median of 2 days) compared to the standard dose of 1 mcg/kg/week (median of 4.5 days).

Patients on higher doses also had a shorter hospital stay (13.5 days vs. 20 days) and a lower incidence of clinically relevant nonmajor bleeding, suggesting that higher doses may improve treatment outcomes and safety for patients who are refractory to standard therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Initial romiplostim dosing and time to platelet response in patients with treatment refractory immune thrombocytopenia. DasGupta, RK., Levine, L., Wiczer, T., et al.[2019]

AMG 531 was generally well tolerated in a phase I study with 30 healthy Japanese men, showing safety profiles similar to placebo and only mild treatment-related adverse events reported.

The study demonstrated that AMG 531 effectively increased platelet counts, with significant increases observed in 4 out of 8 subjects at 1 microg/kg and 7 out of 8 at 2 microg/kg, indicating its potential efficacy for treating chronic immune thrombocytopenic purpura.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacodynamics and pharmacokinetics of AMG 531, a thrombopoiesis-stimulating peptibody, in healthy Japanese subjects: a randomized, placebo-controlled study. Kumagai, Y., Fujita, T., Ozaki, M., et al.[2016]

In a long-term study of 142 patients with chronic immune thrombocytopenic purpura (ITP), romiplostim significantly increased platelet counts in 87% of patients, maintaining effectiveness for up to 156 weeks without tachyphylaxis.

The treatment had an acceptable safety profile, with serious adverse events occurring in 9% of patients, including severe bleeding in 9% and thrombotic events in 5%, indicating that while effective, monitoring for side effects is important.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Bussel, JB., Kuter, DJ., Pullarkat, V., et al.[2022]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11319220/

Romiplostim in chemotherapy‐induced thrombocytopenia Another retrospective analysis of weekly romiplostim found that platelet counts improved in all patients, reaching ≥100 × 109/L in 19/20 ...

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

Romiplostim Treatment of Chemotherapy-Induced ... Fourteen of the 15 romiplostim patients (93%) achieved the primary end point of corrected platelet counts (≥ 100,000/μL) within 3 weeks. Only ...

3. 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 ...

4. 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 ... Platelet responses (platelet count ≥ 50 × 109/L and double baseline) were observed in 87% of all patients and occurred on average 67% of the time in responding ...

5. cdn.clinicaltrials.gov cdn.clinicaltrials.gov/large-docs/82/NCT02052882/Prot_SAP_000.pdf

MSK PROTOCOL COVER SHEET An Open Label Phase II ... If the pre-chemotherapy cycle platelet count is >200,000/mcL, the romiplostim dose will be reduced by 0.5 mcg/kg. 3. If the pre-chemotherapy cycle platelet ...

6. 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 ...

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

Romiplostim (Nplate) - Medical Clinical Policy Bulletins The authors concluded that romiplostim resulted in improvement in platelet counts, allowing resumption of chemotherapy without recurrence of dose-limiting CIT. ...