Telpegfilgrastim versus filgrastim to prevent low white blood counts after chemotherapy in children and young adults

A Multicenter, Randomized, Controlled Study of Telpegfilgrastim Versus Filgrastim for Secondary Prevention of Chemotherapy-Induced Neutropenia in Children and Adolescents With Solid Tumors Receiving High-Intensity Chemotherapy Regimens

This study tries to see if a single long‑acting injection of telpegfilgrastim can reduce febrile neutropenia compared with daily filgrastim in 6–24‑year‑olds receiving high‑intensity chemotherapy for solid tumors.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 132 (estimated)
• Ages: 6 Years to 24 Years
• Sex: All
• Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Beijing)
• Trial ID: NCT06926751 on ClinicalTrials.gov

What this trial studies

This is a Phase 2, randomized, open‑label, adaptive trial comparing a long‑acting PEGylated G‑CSF (telpegfilgrastim) with daily filgrastim for secondary prevention of chemotherapy‑induced neutropenia in patients aged 6–24 with malignant solid tumors. An initial PK/safety lead‑in (20 patients) informs dose and safety before expanding to a 3:1 randomized cohort (99 vs 33) stratified by age groups. Telpegfilgrastim is given as a single subcutaneous dose after chemotherapy while filgrastim is given daily until neutrophil recovery; primary endpoints include febrile neutropenia in the first post‑randomization cycle plus duration/severity of neutropenia and treatment disruptions. The trial excludes other myelostimulatory agents and monitors ANC closely to compare efficacy and safety outcomes.

Who should consider this trial

Good fit: Children and young adults aged 6–24 with malignant solid tumors who experienced febrile or dose‑limiting neutropenia in the prior chemotherapy cycle and who have adequate performance status and blood counts are ideal candidates.

Not a fit: Patients with bone marrow tumor involvement, uncontrolled infections, known hypersensitivity to the study drugs, inadequate blood counts, or those receiving other myelostimulatory agents are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If successful, the long‑acting drug could mean fewer injections, lower rates of febrile neutropenia, and fewer chemotherapy delays or hospitalizations for young patients.

How similar studies have performed: Long‑acting pegylated G‑CSF agents (for example pegfilgrastim) have reduced neutropenia in adults and have some pediatric experience, but telpegfilgrastim's specific Y‑branched PEG formulation is newer and less extensively tested in children.

Eligibility criteria

Inclusion Criteria:

1. Histologically or cytologically confirmed malignant solid tumor requiring high-intensity chemotherapy, with ≥2 remaining chemotherapy cycles, and either:

   * Previous febrile neutropenia(FN) or dose-limiting neutropenia in the prior chemotherapy cycle without prophylactic granulocyte colony-stimulating factor (G-CSF);
   * Previous FN or dose-limiting neutropenia in the prior chemotherapy cycle despite prophylactic G-CSF.
2. Age ≥6 to ≤24 years.
3. Eastern Cooperative Oncology Group Performance Status ≤1.
4. Normal bone marrow hematopoietic function: hemoglobin ≥75 g/L, white blood cell count ≥3.0×10\^9/L, platelets ≥80×10\^9/L, and neutrophils ≥1.5×10\^9/L.
5. Anticipated survival ≥8 months.
6. Willing to participate, with written informed consent signed by the patient or legal guardian.

Exclusion Criteria:

1. Bone marrow involvement at screening.
2. Uncontrolled localized or systemic infection.
3. Known hypersensitivity to Telpegfilgrastim, recombinant human granulocyte-colony stimulating factor(rhG-CSF), or other pegylated recombinant human granulocyte colony stimulating factor(PEG-rhG-CSF) agents.
4. Concurrent participation in any other investigational drug or device trial.
5. Severe organ dysfunction: total bilirubin, alanine aminotransferase(ALT), or aspartate transaminase(AST) \>2.5 × upper limit of normal(ULN) (or \>5 × ULN in patients with liver metastases), serum creatinine \>5 × ULN.
6. Severe psychiatric disorders affecting informed consent provision or adverse event assessment.
7. Any condition deemed by the investigator to compromise patient safety or interfere with study outcomes, including risks from the investigational product or confounding adverse event evaluation.

Where this trial is running

Beijing

• Cancer Hospital, Chinese Academy of Medical Sciences — Beijing, China (Recruiting)

Study contacts

• Principal investigator: Sidan Li — Cancer Institute and Hospital, Chinese Academy of Medical Sciences
• Study coordinator: Sidan Li

Conditions Solid Tumors; Children; Adolescent; Chemotherapy Induced Neutropenia