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

• ClinicalTrials.gov Identifier: NCT04570423
• Recruitment Status: Recruiting
• First Posted: September 30, 2020
• Last Update Posted: May 16, 2024
• Sponsor: Spectrum Pharmaceuticals, Inc
• Information provided by (Responsible Party): Spectrum Pharmaceuticals, Inc

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Solid Tumors; Lymphoma	Drug: Eflapegrastim; Drug: Chemotherapy	Phase 2

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 40 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: 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
• Actual Study Start Date: May 20, 2021
• Estimated Primary Completion Date: October 2027
• Estimated Study Completion Date: October 2027

Arms and Interventions

Arm	Intervention/treatment
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; Eflapegrastim supplied in prefilled, single-use syringes for SC injection.; Other Names: Rolontis®; SPI-2012; Drug: Chemotherapy; 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.
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; Eflapegrastim supplied in prefilled, single-use syringes for SC injection.; Other Names: Rolontis®; SPI-2012; Drug: Chemotherapy; 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.
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; Eflapegrastim supplied in prefilled, single-use syringes for SC injection.; Other Names: Rolontis®; SPI-2012; Drug: Chemotherapy; 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.
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; Eflapegrastim supplied in prefilled, single-use syringes for SC injection.; Other Names: Rolontis®; SPI-2012; Drug: Chemotherapy; 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.

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Treatment Emergent Adverse Events (TEAEs) [ Time Frame: From first dose of study drug to 35 days after the last dose of the study drug (Up to approximately 16 months) ]
   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.

Secondary Outcome Measures :

1. Percentage of Participants With Severe Neutropenia in Cycle 1 [ Time Frame: Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected) ]
   Severe neutropenia is defined as absolute neutrophil count (ANC) less than 0.5*10^9/liter (L).
2. Time to Absolute Neutrophil Count (ANC) Recovery of Severe Neutropenia in Cycle 1 [ Time Frame: Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected) ]
   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.
3. Number of Participants With Febrile Neutropenia in Cycle 1 [ Time Frame: Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected) ]
   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.
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) ]

Eligibility Criteria

• Ages Eligible for Study: 1 Month to 17 Years (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Contacts

Contact: Howard Franklin, MD; 224.419.7106; Hfranklin@assertiotx.com

Locations

United States, New York

New York Medical College; Recruiting

Valhalla, New York, United States, 10595

United States, North Carolina

Carolinas Medical Center/ Levine Children's Hospital; Recruiting

Charlotte, North Carolina, United States, 28203

Levine Children's Health; Recruiting

Charlotte, North Carolina, United States, 28203

United States, Texas

UT MD Anderson Cancer Center; Recruiting

Houston, Texas, United States, 77030

Sponsors and Collaborators

Spectrum Pharmaceuticals, Inc

More Information

• Responsible Party: Spectrum Pharmaceuticals, Inc
• ClinicalTrials.gov Identifier: NCT04570423
• Other Study ID Numbers: SPI-GCF-202
• First Posted: September 30, 2020
• Last Update Posted: May 16, 2024
• Last Verified: May 2024

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Spectrum Pharmaceuticals, Inc:

Lymphomas; Solid Tumors; Chemotherapy

Additional relevant MeSH terms:

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