Recruiting
Early Phase 1

Romiplostim

Sponsor:

Children's Hospital Medical Center, Cincinnati

Code:

NCT07048249

Conditions

Ewings Sarcoma

Chemotherapy Induced Thrombocytopenia

Eligibility Criteria

Sex: All

Age: 1+

Healthy Volunteers: Not accepted

Interventions

Romiplostim (AMG-531)

Study Details

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. 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. To determine the safety of incorporation of romiplostim supportive care when given concurrently with Ewing sarcoma therapy.
3. To determine the feasibility of incorporation of romiplostim supportive care into upfront Ewing sarcoma regimens.

Conditions

Ewings Sarcoma

Chemotherapy Induced Thrombocytopenia

Study ID

NCT07048249

Start date

Jan 26, 2024

Status verified date

Feb, 2026

Completion date

Jan 1, 2028

Anticipated

Primary completion date

Jan 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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, cancer directed: Patients receiving whole lung radiation, >50% of pelvic irradiation, other substantial bone marrow radiation (i.e. ≥ 50% of vertebral marrow space), or patients undergoing pneumonectomy as a component of local control before cycle 14, are not eligible. These therapies are not an exclusion if instituted during or after cycle 14.
  • 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.

Study Design

Enrollment

26 participants

Anticipated

Intervention Model

Single group

Primary purpose

Supportive Care

Interventions and Outcome Measures

Arms

experimental: Romiplostim administration with chemotherapy cycles

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.

Interventions

Romiplostim (AMG-531)

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

Primary outcome measure

  • 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. [ Time Frame: 52 weeks ]
  • Measure adverse events with the addition of romiplostim when given with chemotherapy. [ Time Frame: 52 weeks ]
  • Number of patients able to receive the majority of planned romiplostim doses. [ Time Frame: 52 weeks ]

Central Contacts and Locations

Central contacts

Brian Turpin, DO

513-636-2799cancer@cchmc.org

Locations

Phoenix Children's

Recruiting

Phoenix, Arizona, United States, 85016

Contacts

Felicia Frank

602-933-5004dg_EDDT@phoenixchildrens.com

Chris Oless, RN

602-933-0188coless@phoenixchildrens.com

Principal Investigator:

Nawal Merjaneh, MD, MS

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Brian Turpin, DO

513-636-2799cancer@cchmc.org

Site Public Contact

513-636-2799cancer@cchmc.org

More Information

Sponsor

Children's Hospital Medical Center, Cincinnati

Last update posted

Mar 18, 2026

Last verified

Feb, 2026

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Children's Hospital Medical Center, Cincinnati on 2026-03-18.