A Study to Evaluate the Safety and Pharmacokinetics of Eflapegrastim in Pediatric Participants With Solid Tumors or Lymphomas and Treated With Myelosuppressive Chemotherapy

Spectrum Pharmaceuticals

Status and phase: Enrolling; Phase 2

Conditions: Lymphoma; Solid Tumors

Treatments: Drug: Chemotherapy; Drug: Eflapegrastim

Study type: Interventional

Funder types: Industry

Identifiers

NCT04570423

SPI-GCF-202

Details and patient eligibility

About

The purpose of this study is to evaluate the safety and pharmacokinetics of eflapegrastim in pediatric participants with solid tumors or lymphoma and treated with myelosuppressive chemotherapy.

Full description

This is a Phase 2, open label, multicenter study of eflapegrastim in pediatric participants (≥1 month to <17 years) with solid tumors or lymphoma.

Approximately 40 participants will be enrolled and assigned to one of 4 age-based cohorts. Participants enrolled in Cohort 1 will be followed for dose-limiting toxicities (DLTs) prior to initiating parallel enrollment into Cohorts 2 through 4.

All participants will receive chemotherapy as Standard of Care after which a subcutaneous (SC) dose of eflapegrastim will be administered up to 4 treatment cycles.

Enrollment: 40 estimated patients

Sex: All

Ages: 1 month to 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

40 participants in 4 patient groups

Cohort 1: ≥12 to <17 years

Experimental group

Description:

Participants will receive a SC injection of eflapegrastim after completion of each cycle of chemotherapy up to four cycles of treatment (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy regimen selected).

Treatment:

Drug: Eflapegrastim

Drug: Chemotherapy

Cohort 2: ≥6 to <12 years

Experimental group

Description:

Participants will receive a SC injection eflapegrastim after completion of each cycle of chemotherapy up to four cycles of treatment (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy regimen selected).

Treatment:

Drug: Eflapegrastim

Drug: Chemotherapy

Cohort 3: ≥2 to <6 years

Experimental group

Description:

Participants will receive a SC injection eflapegrastim after completion of each cycle of chemotherapy up to four cycles of treatment (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy regimen selected).

Treatment:

Drug: Eflapegrastim

Drug: Chemotherapy

Cohort 4: ≥1 month to <2 years

Experimental group

Description:

Participants will receive a SC injection eflapegrastim after completion of each cycle of chemotherapy up to four cycles of treatment (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy regimen selected).

Treatment:

Drug: Eflapegrastim

Drug: Chemotherapy