RECRUITINGEarly Phase 1INTERVENTIONAL

Single Arm Romiplostim to Prevent CIT

Single Arm Evaluation of Romiplostim to Prevent Chemotherapy Induced Thrombocytopenia in Patients With Ewing Sarcoma

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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. - willing to sign a consent form: All patients and/or their parents or legally authorized representatives must sign a written willing to sign a consent form. Assent, when appropriate, will be obtained according to institutional guidelines. Who Should NOT Join This Trial: - 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language

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.

Treatments Being Tested

DRUG

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

Locations (2)

Phoenix Children's

Phoenix, Arizona, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States