Single Arm Romiplostim to Prevent CIT
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age1+
SponsorChildren's Hospital Medical Center, Cincinnati
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Patients requiring hematopoietic stem cell rescue are not eligible.
Previous use of romiplostim, eltrombopag or any other platelet-producing agent is not allowed.
Trial design
Design model
Single group
Treatments tested in this trial
Romiplostim (AMG-531)
DrugRomiplostim 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
Treatment groups
26 Participants
are divided into 1 treatment groupGroup A: Romiplostim administration with chemotherapy cyclesExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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)
Time frame
52 weeks2
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.
Time frame
52 weeks3
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.
Time frame
52 weeksSponsors and contacts
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Source ClinicalTrials.gov