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

NCT04570423 | Recruiting Phase 2 FDA Drug

• 1 other identifier: SPI-GCF-202
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 5

Brief 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.

Conditions

Solid Tumors; Lymphoma

Keywords

Lymphomas; Solid Tumors; Chemotherapy

Outcome Measures

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.

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

Secondary Outcomes (6)

• Percentage of Participants With Severe Neutropenia in Cycle 1

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

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

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

• Number of Participants With Febrile Neutropenia in Cycle 1

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

• Peak Concentration (Cmax) of Eflapegrastim in Cycle 1

  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)

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

  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)

Study Arms (4)

Cohort 1: ≥12 to <17 years

EXPERIMENTAL

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

Cohort 2: ≥6 to <12 years

EXPERIMENTAL

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

Cohort 3: ≥2 to <6 years

EXPERIMENTAL

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

Cohort 4: ≥1 month to <2 years

EXPERIMENTAL

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

Eflapegrastim DRUG

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

Also known as: Rolontis®, SPI-2012

Cohort 1: ≥12 to <17 years; Cohort 2: ≥6 to <12 years; Cohort 3: ≥2 to <6 years; Cohort 4: ≥1 month to <2 years

Chemotherapy DRUG

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.

Cohort 1: ≥12 to <17 years; Cohort 2: ≥6 to <12 years; Cohort 3: ≥2 to <6 years; Cohort 4: ≥1 month to <2 years

Eligibility Criteria

• Age: 1 Month - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant must have a pathologic/histologic confirmed newly diagnosed/relapsed/recurrent solid tumor or lymphoma without bone marrow involvement.
• 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.
• Participant has adequate hematological, renal, and hepatic function.
• 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%.
• Participant must have a lumbar puncture, if clinically indicated, to rule out central nervous system (CNS) involvement within 14 days of study entry.
• 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.

You may not qualify if:

• 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.
• 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)
• Participant requires concurrent radiation therapy specifically in Cycle 1.
• Participant has had prior bone marrow or hematopoietic stem cell transplant and/or has concurrent bone marrow involvement in their malignancy, including leukemia.
• Participant has had spinal radiation therapy within 30 days prior to study enrollment.
• 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.
• Participant has a known sensitivity or previous reactions to any of the G-CSF products.
• Participant with active CNS disease.
• Participant has not recovered from previous treatment adverse events to ≤Grade 1.

Sponsors & Collaborators

• Spectrum Pharmaceuticals, Inc: lead

Study Sites (5)

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

MeSH Terms

Conditions

Lymphoma

Interventions

eflapegrastim; Drug Therapy

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Therapeutics

Central Study Contacts

Howard Franklin, MD

CONTACT

224.419.7106; Hfranklin@assertiotx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 15, 2020
• First Posted: September 30, 2020
• Study Start: May 20, 2021
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): October 1, 2027
• Last Updated: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)