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RecruitingNCT04570423Updated Sep 1, 2026

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

A Phase 2 interventional study of Eflapegrastim and Chemotherapy in Solid Tumors and Lymphoma, sponsored by Spectrum Pharmaceuticals, Inc. Recruiting at 5 sites in United States. Open to participants aged 1 Month to 17 Years. Per ClinicalTrials.gov, last updated 2026-09-01.

Sponsored by Spectrum Pharmaceuticals, Inc · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
40
Allocation
Non-randomized
Ages
1 Month to 17 Years
Sex
All
01

Study summary

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.

Read the detailed 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.

02

Conditions studied

  • Solid Tumors
  • Lymphoma

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Keywords

  • Lymphomas
  • Solid Tumors
  • Chemotherapy
03

Who can participate

Ages eligible
1 Month to 17 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
40 participants (estimated)

Study arms

  • Experimental
    Cohort 1: ≥12 to <17 years

    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).

    Drug: Eflapegrastim · Drug: Chemotherapy

  • Experimental
    Cohort 2: ≥6 to <12 years

    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).

    Drug: Eflapegrastim · Drug: Chemotherapy

  • Experimental
    Cohort 3: ≥2 to <6 years

    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).

    Drug: Eflapegrastim · Drug: Chemotherapy

  • Experimental
    Cohort 4: ≥1 month to <2 years

    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).

    Drug: Eflapegrastim · Drug: Chemotherapy

Interventions

  • DrugEflapegrastim

    Eflapegrastim supplied in prefilled, single-use syringes for SC injection.

    Also known as: Rolontis®, SPI-2012

  • DrugChemotherapy

    Chemotherapy agents may include doxorubicin, ifosfamide, docetaxel, CHOP regimen, etoposide, cyclophosphamide and vincristine which will be administered as per standard of care per the Primary Care physician's treatment plan.

05

What researchers measure

Primary outcomes

  1. Number of Participants With Treatment Emergent Adverse Events (TEAEs)

    An AE is defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. A TEAE is any AE that occurs from the first dose of the study drug until 35 days after the last dose of study drug, or on the day a new/additional chemotherapy regimen, or on the day another granulocyte-colony stimulating factor (G-CSF) is administered.

    Time frame: From first dose of study drug to 35 days after the last dose of the study drug (Up to approximately 16 months)

Secondary outcomes

  1. Percentage of Participants With Severe Neutropenia in Cycle 1

    Severe neutropenia is defined as absolute neutrophil count (ANC) less than 0.5\*10\^9/liter (L).

    Time frame: Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected)

  2. Time to Absolute Neutrophil Count (ANC) Recovery of Severe Neutropenia in Cycle 1

    Time to ANC recovery of severe neutropenia is defined as the time from chemotherapy administration until the participants ANC increases to ≥1.0\*10\^9/L after the expected nadir.

    Time frame: Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected)

  3. Number of Participants With Febrile Neutropenia in Cycle 1

    Febrile neutropenia is defined as ANC less than 0.5\*10\^9/L with a single temperature of \>38.3 degree Celsius (°C) or a sustained temperature of ≥38°C for more than 1 hour.

    Time frame: Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected)

  4. Peak Concentration (Cmax) of Eflapegrastim in Cycle 1

    Time frame: Pre-dose and at multiple time points (up to Day 9 [Cohorts 1-3] and Day 6 [Cohort 4]) post-dose in Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected)

  5. Time to Reach Peak Concentration (Tmax) of Eflapegrastim in Cycle 1

    Time frame: Pre-dose and at multiple time points (up to Day 9 [Cohorts 1-3] and Day 6 [Cohort 4]) post-dose in Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected)

  6. Elimination Half-life (t½) of Eflapegrastim in Cycle 1

    Time frame: Pre-dose and at multiple time points (up to Day 9 [Cohorts 1-3] and Day 6 [Cohort 4]) post-dose in Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected)

06

Study locations

1 of 5 sites recruiting
  • New York Medical College
    Valhalla, New York 10595, United States
    Terminated
  • Carolinas Medical Center/ Levine Children's Hospital
    Charlotte, North Carolina 28203, United States
    Terminated
  • Levine Children's Health
    Charlotte, North Carolina 28203, United States
    Terminated
  • University Hospitals Cleveland Medical Center
    Cleveland, Ohio 44106, United States
    Recruiting
  • UT MD Anderson Cancer Center
    Houston, Texas 77030, United States
    Terminated
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT04570423
Lead sponsor
Spectrum Pharmaceuticals, Inc
Responsible party
Sponsor
First posted
Sep 30, 2020
Start date
May 20, 2021
Primary completion
Oct 2027 (estimated)
Completion
Oct 2027 (estimated)
Last update
Sep 1, 2026

Study contacts

Howard Franklin, MD
Contact
Hfranklin@assertiotx.com
224.419.7106

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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