Romiplostim to prevent chemotherapy-related low platelets in Ewing sarcoma
Single Arm Evaluation of Romiplostim to Prevent Chemotherapy Induced Thrombocytopenia in Patients With Ewing Sarcoma
This trial will test whether giving romiplostim during interval-compressed chemotherapy can reduce episodes of low platelets in people with newly diagnosed Ewing sarcoma.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 26 (estimated) |
| Ages | 1 Year and up |
| Sex | All |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Drugs / interventions | rituximab, chemotherapy, radiation, cyclophosphamide, doxorubicin |
| Locations | 2 sites (Phoenix, Arizona and 1 other locations) |
| Trial ID | NCT07048249 on ClinicalTrials.gov |
What this trial studies
This is a single-arm, multi-center trial giving romiplostim (Nplate) to patients with newly diagnosed Ewing sarcoma who are receiving interval-compressed chemotherapy. Participants start romiplostim as early as cycle 1 day 1 or no later than two weeks after the start of cycle 5, with dosing based on weight and weekly escalation if platelet counts remain below 200,000/µL. The main outcome is the rate of chemotherapy-induced thrombocytopenia (failure to achieve platelet recovery by the planned cycle start) during the continuation phase, compared to published historical controls, and safety of the drug will also be monitored. Treatment is delivered at participating pediatric oncology centers with regular platelet monitoring and dose adjustments as needed.
Who should consider this trial
Good fit: Ideal candidates are people older than 1 year with newly diagnosed Ewing sarcoma receiving interval-compressed chemotherapy per AEWS protocols who do not have bone marrow metastasis and can receive care at a participating site.
Not a fit: Patients with bone marrow metastatic disease, those receiving large-field or high-dose marrow radiation, or those undergoing early pneumonectomy are excluded and therefore unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, romiplostim could reduce low-platelet events and thereby decrease platelet transfusions or chemotherapy delays for patients on intensive Ewing sarcoma regimens.
How similar studies have performed: Romiplostim is proven to raise platelets in immune thrombocytopenia and has shown promise for chemotherapy-related low platelets in other cancers, but its preventive use specifically in Ewing sarcoma is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Phoenix, Arizona and 1 other locations
- Phoenix Children's — Phoenix, Arizona, United States (Recruiting)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
Study contacts
- Principal investigator: Brian Turpin, DO — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Brian Turpin, DO
- Email: cancer@cchmc.org
- Phone: 513-636-2799
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.