Evaluating eflapegrastim for children with solid tumors or lymphomas undergoing chemotherapy

A Multicenter, Open-Label, Phase 2 Study to Evaluate the Safety and Pharmacokinetics of Eflapegrastim in Pediatric Patients With Solid Tumors or Lymphomas and Treated With Myelosuppressive Chemotherapy

This study is testing a new drug called eflapegrastim to see if it can help children with solid tumors or lymphomas feel better while they are getting chemotherapy.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 1 Month to 17 Years
• Sex: All
• Sponsor: Spectrum Pharmaceuticals, Inc (industry)
• Drugs / interventions: chemotherapy, radiation
• Locations: 5 sites (Valhalla, New York and 4 other locations)
• Trial ID: NCT04570423 on ClinicalTrials.gov

What this trial studies

This Phase 2, open-label, multicenter study aims to assess the safety and pharmacokinetics of eflapegrastim in pediatric patients aged 1 month to under 17 years who have solid tumors or lymphoma and are receiving myelosuppressive chemotherapy. Approximately 40 participants will be enrolled and divided into four age-based cohorts, with initial focus on dose-limiting toxicities in the first cohort before parallel enrollment in the others. All participants will receive standard chemotherapy followed by subcutaneous administration of eflapegrastim for up to four treatment cycles.

Who should consider this trial

Good fit: Ideal candidates include pediatric patients with newly diagnosed or relapsed solid tumors or lymphomas who are eligible for myelosuppressive chemotherapy.

Not a fit: Patients with bone marrow involvement or those not requiring myelosuppressive chemotherapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could improve the safety and effectiveness of chemotherapy in pediatric patients with solid tumors or lymphomas.

How similar studies have performed: Other studies have shown promise in using similar approaches for managing chemotherapy-related complications in pediatric oncology, suggesting potential for success.

Eligibility criteria

Inclusion Criteria:

1. Participant must have a pathologic/histologic confirmed newly diagnosed/relapsed/recurrent solid tumor or lymphoma without bone marrow involvement.
2. Participant must be a candidate to receive myelosuppressive chemotherapy, with a febrile neutropenia rate of at least 20% as outlined in the National Comprehensive Cancer Network (NCCN) guidelines.
3. Participant has adequate hematological, renal, and hepatic function.
4. Participant must have an echocardiogram (ECHO) or multigated acquisition (MUGA) within 14 days of Screening if receiving a cardiotoxic therapy and have a cardiac ejection fraction of \>50%.
5. Participant must have a lumbar puncture, if clinically indicated, to rule out central nervous system (CNS) involvement within 14 days of study entry.
6. Participant has a Karnofsky performance level ≥50% for patients ≥16 years of age or a Lansky performance level ≥50 for children \<16 years of age.

Exclusion Criteria:

1. Participant has an uncontrollable infection, has an underlying medical condition, and/or another serious illness that would impair the ability of the participant to receive protocol-specified treatment.
2. Participant has had previous exposure to filgrastim (within 7 days), pegfilgrastim (within 14 days), or other granulocyte colony stimulating factor (G-CSF) products in clinical development within 2 weeks prior to the administration of study drug (eflapegrastim)
3. Participant requires concurrent radiation therapy specifically in Cycle 1.
4. Participant has had prior bone marrow or hematopoietic stem cell transplant and/or has concurrent bone marrow involvement in their malignancy, including leukemia.
5. Participant has had spinal radiation therapy within 30 days prior to study enrollment.
6. Participant has used any investigational drugs, biologics or devices within 30 days prior to study treatment or plans to use any of these during the study.
7. Participant has a known sensitivity or previous reactions to any of the G-CSF products.
8. Participant with active CNS disease.
9. Participant has not recovered from previous treatment adverse events to ≤Grade 1.

Where this trial is running

Valhalla, New York and 4 other locations

• New York Medical College — Valhalla, New York, United States (TERMINATED)
• Carolinas Medical Center/ Levine Children's Hospital — Charlotte, North Carolina, United States (TERMINATED)
• Levine Children's Health — Charlotte, North Carolina, United States (TERMINATED)
• University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States (RECRUITING)
• UT MD Anderson Cancer Center — Houston, Texas, United States (TERMINATED)

Study contacts

• Study coordinator: Howard Franklin, MD

Conditions: Solid Tumors, Lymphoma, Lymphomas, Chemotherapy