Single Arm Romiplostim to Prevent CIT

NCT07048249 | Recruiting Early Phase 1 DMC FDA Drug

• 1 other identifier: Romiplostim-BT-2201
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 2

Brief Summary

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 receiving interval-compressed chemotherapy. The main questions it aims to answer are:

1. 1.To demonstrate the efficacy of romiplostim in patients with newly diagnosed Ewing sarcoma, measured specifically as the rate of CIT, defined as a failure to achieve platelet recovery (≥ 75,000/µL post nadir, without transfusion, or a platelet count sufficient to resume chemotherapy per provider and institutional standard) within 7 days of planned chemotherapy cycle start, measured during the continuation phase (cycle 7 to end of cycle 13 or 16, per AEWS0031/AEWS1221, or AEWS1031 respectively) of interval-compressed chemotherapy (every 2 week vincristine/cyclophosphamide +/- doxorubicin and ifosfamide/etoposide chemotherapy) as compared to published institutional historical control rate.
2. 2.To determine the safety of incorporation of romiplostim supportive care when given concurrently with Ewing sarcoma therapy.
3. 3.To determine the feasibility of incorporation of romiplostim supportive care into upfront Ewing sarcoma regimens.

Conditions

Ewings Sarcoma; Chemotherapy Induced Thrombocytopenia

Keywords

romiplostim; ewing sarcoma; chemotherapy induced thrombocytopenia; CIT; NPLATE

Outcome Measures

Primary Outcomes (3)

• Number of evaluable participants (11 or fewer of 26) that develop CIT during the continuation phase of compressed-interval chemotherapy compared to institutional historical control rate.

  CIT is defined as failure to achieve platelet recovery ((≥ 75,000/µL post nadir, without transfusion, or a platelet count sufficient to resume chemotherapy per provider and institutional standard) within 7 days of planned chemotherapy cycle start, measured during the continuation phase (beyond cycle 6)

  52 weeks

• Measure adverse events with the addition of romiplostim when given with chemotherapy.

  To determine the safety of incorporation of romiplostim supportive care when given concurrently with Ewing sarcoma therapy.

  52 weeks

• Number of patients able to receive the majority of planned romiplostim doses.

  Feasibility will be met if fewer than 9 of 26 enrolled patients fail to receive at least 60% of planned romiplostim doses (excluding doses held for thrombocytosis or post-operatively) from initiation through at least the end of the 13th cycle of chemotherapy or through at least the end of the 16th cycle of chemotherapy.

  52 weeks

Study Arms (1)

Romiplostim administration with chemotherapy cycles

EXPERIMENTAL

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.

Drug: Romiplostim (AMG-531)

Interventions

Romiplostim (AMG-531) DRUG

Romiplostim may be started as supportive care, 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 (see exception below for patients with platelet count of 200,000 or greater). If plt count \< 200,000/mm3, patients will start romiplostim based on their weight

Also known as: NPLATE

Romiplostim administration with chemotherapy cycles

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age: Patients must be \>1 year old at the time of study consent.
• Diagnosis: Patients with a new diagnosis of Ewing sarcoma treated with interval-compressed chemotherapy as per AEWS0031, AEWS1221, or AEWS1031.
• 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.

You may not qualify if:

• Marrow disease: Patients with metastatic Ewing sarcoma to the bone marrow are not eligible. Marrow staging is not required for this study but should be performed if clinically indicated.
• 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.
• Concurrent Illnesses: Patients with a history of or current diagnosis of bone marrow failure, hematologic malignancy, pro-thrombotic condition, or platelet disorder (including immune or heparin induced thrombocytopenia) are not eligible.
• 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.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (2)

Phoenix Children's

Phoenix, Arizona, 85016, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Related Publications (28)

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MeSH Terms

Conditions

Sarcoma, Ewing

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Osteosarcoma; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Sarcoma

Study Officials

• Brian Turpin, DO

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Brian Turpin, DO

CONTACT

513-636-2799; cancer@cchmc.org

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 24, 2025
• First Posted: July 2, 2025
• Study Start: January 26, 2024
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2028
• Last Updated: March 18, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)