
Advancing Pediatric Clinical Trials: Overcoming Challenges in the Clinical Trial of Pegfilgrastim for Rhabdomyosarcoma and Wilms' Tumor
09 Oct 2023
Overcoming Challenges in the Clinical Trial of Pegfilgrastim for Rhabdomyosarcoma and Wilms' Tumor
Study Overview:
Pegfilgrastim, a drug with proven efficacy in adults, was investigated in pediatric patients. Although studies in adults demonstrated similar efficacy to filgrastim, limited data existed for the pediatric population, specifically in rhabdomyosarcoma and Wilms' tumor patients.
Study Design:
A randomized active-controlled multi-center open-label two-arm study was designed to assess the safety, efficacy, pharmacodynamics, and pharmacokinetics of Pegfilgrastim PFS in comparison to the Reference Product in pediatric participants under 6 years old with rhabdomyosarcoma or Wilms' tumor.
Challenges and Solutions:
Challenges | Solutions |
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Recruitment of Pediatric Patients: | |
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Dosing and IMP Management: | |
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Handling of PK & PD Samples: | |
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Achievements:
- Successfully randomized 12 pediatric patients within an impressive 7-month period, showcasing effective patient enrollment strategies.
- Executed the trial efficiently within a timeline of 12 months, from the first site initiation visit (SIV) to the last patient's last visit, meeting the study's objectives and timelines for EMEA submission compliance.
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