Phase 2
N=21
Study to Assess Safety and Preliminary Activity of Eribulin Mesylate in Pediatric Participants With Relapsed/Refractory Rhabdomyosarcoma (RMS), Non-rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS) and Ewing Sarcoma (EWS)
Relapsed/Refractory Rhabdomyosarcoma · Non-rhabdomyosarcoma Soft Tissue Sarcoma · Ewing Sarcoma
Bottom Line
View on ClinicalTrials.gov: NCT03441360 ↗Enrolled (actual)
21
Serious AEs
52.4%
Results posted
Jan 2022
Primary outcome: Primary: Percentage of Participants With Objective Response — 0; 0; 0 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Eribulin mesylate (Drug)
- Age
- Pediatric, Adult · 0+ yrs
- Sex
- All
- Sponsor
- Eisai Inc.
- Primary completion
- Jan 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Objective Response |
0; 0; 0 | — |
| SECONDARY Progression-free Survival (PFS) |
1.74; 1.30; 0.76 | — |
| SECONDARY Number of Participants With Any Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
8; 8; 5; 6; 2; 3 | — |
| SECONDARY Number of Participants With a Shift From Baseline to Worst Post-Baseline Common Terminology Criteria for Adverse Events (CTCAE) Grade in Laboratory Value |
0; 0; 0; 3; 4; 3 | — |
| SECONDARY Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Values |
0; 0; 0 | — |
| SECONDARY Number of Participants With Clinically Significant Change From Baseline in Vital Signs Values |
0; 0; 0 | — |
| SECONDARY Number of Participants With Shift From Baseline to Worst Post-baseline for Lansky Play-performance Scale |
1; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With Shift From Baseline to Worst Post-baseline for Karnofsky Performance Status Scores |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Duration of Response (DOR) |
— | — |
| SECONDARY Overall Survival (OS) |
5.08; 6.95; 7.89 | — |
Summary
This study will be conducted as an assessment of the safety and preliminary activity of eribulin mesylate in pediatric participants with relapsed/refractory rhabdomyosarcoma (RMS), non-rhabdomyosarcoma soft tissue sarcoma (NRSTS), or Ewing sarcoma (EWS) to determine whether each cohort warrants further investigation.
Eligibility Criteria
Inclusion Criteria
- Age: ≥12 months to 16 years of age) or Lansky (for participants ≤16 years of age). Participants who are unable to walk because of paralysis and/or previous surgeries, but who are in a wheelchair, will be considered ambulatory for the purpose of assessing performance score.
- Participants must have fully recovered from the acute toxic effects of all prior anticancer therapy and must meet the following minimum duration from prior anticancer directed therapy prior to study drug administration. If, after the required time frame, the numerical eligibility criteria are met, eg, blood count criteria, the participant is considered to have recovered adequately:
- Cytotoxic chemotherapy or other chemotherapy known to be myelosuppressive: ≥21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea).
- Anticancer agents not known to be myelosuppressive (eg, not associated with reduced platelet or absolute neutrophil count [ANC] counts): ≥7 days after the last dose of agent.
- Monoclonal antibodies ≥ 3 half-lives must have elapsed from infusion of last dose of antibody (including checkpoint inhibitors), and toxicity related to prior antibody therapy must be recovered to Grade ≤1.
- Hematopoietic growth factors: ≥14 days after the last dose of a long-acting growth factor (eg, Neulasta) or 7 days for a short-acting growth factor. For agents that have known adverse events (AEs) occurring beyond 7 days after administration, this period must be extended beyond the time during which AEs are known to occur. The duration of this interval must be discussed with the sponsor.
- Interleukins, interferons, and cytokines (other than hematopoietic growth factors): ≥21 days after the completion of interleukins, interferons, or cytokines (other than hematopoietic growth factors)
- Stem cell infusions (with or without total body irradiation [TBI]): ≥84 days
- Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion or boost infusion: ≥84 days after infusion and no evidence of graft versus host disease (GVHD)
- Autologous stem cell infusion including boost infusion: ≥42 days
- Cellular therapy: ≥42 days after the completion of any type of cellular therapy (eg, modified T-cells, natural killer cells, dendritic cells, etc)
- Radiation therapy (XRT)/External Beam Irradiation including Protons: ≥14 days after local XRT; ≥150 days after TBI, craniospinal XRT or if radiation to ≥50% of the pelvis; ≥42 days if other substantial BM radiation
- Radiopharmaceutical therapy (eg, radiolabeled antibody, 131I-metaiodobenzylguanidine): ≥42 days after systemically administered radiopharmaceutical therapy.
- Adequate bone marrow function, defined as:
- ANC ≥1.0 × 10^9/Liter (L)
- Platelet count ≥100 × 10^9/L (transfusion independent, defined as not receiving platelet transfusions within a 7-day period prior to study drug administration)
- Hemoglobin at least 8.0 grams per deciliter (g/dL) at Baseline (blood transfusions are allowed during the screening period to correct hemoglobin values less than 8.0 g/dL) Note: As blood transfusions are permitted to meet the hemoglobin criteria, participants requiring transfusion must not be known to be refractory to red blood cell or platelet transfusions.
- Adequate renal function, defined as:
- A serum creatinine based on age/gender, derived from the Schwartz formula for estimating glomerular filtration rate (GFR)
- Or creatinine clearance or GFR ≥50 milliliters per minute (mL/min)/1.73 meters squared (m^2) based on a 12 or 24 hour urine creatinine collection
- Adequate liver function, defined as:
- Bilirubin (sum of conjugated + unconjugated) ≤1.5 × upper limit of normal (ULN) for age
- Alanine aminotransferase (ALT) ≤110 units per Liter (U/L). For the purpose of this study, the ULN for ALT is 45 U/L
- Serum albumin ≥2 g/dL
- Informed consent: All participants and/or their parents or legally auth
Data sourced from ClinicalTrials.gov (NCT03441360). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.