Paediatric Patients Under 6 years of Age with Rhabdomyosarcoma or Wilms’ Tumour on Myelosuppressive Chemotherapy (CmT) Regimen MedDRA version: 20.0 Level: HLGT Classification code 10047954 Term: White blood cell disorders System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Product Name: Pegfilgrastim Pre-filled syringe for Injection 1.5mgper0.15 mL,2.5 mg per 0.25 mL and4.0 mg per0.4ml
Pharmaceutical Form: Solution for injection in pre-filled syringe
INN or Proposed INN
Sponsors
Intas Pharmaceuticals Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female infants and children from under 6 years of age 2.Children with a pathologically confirmed diagnosis of rhabdomyosarcoma or high-risk Wilms’ tumour, planned for treatment with 1 of the following CmT regimens: • Rhabdomyosarcoma: - Ifosfamide plus vincristine plus actinomycin D (IVA) - Ifosfamide plus vincristine plus actinomycin D plus doxorubicin (IVADo) - Vincristine plus actinomycin D plus cyclophosphamide (VAC) • High-risk Wilms’ tumour: - Cyclophosphamide with doxorubicin and /or etoposide with carboplatin 3. Written informed consent provided by parent(s)/legal representative(s) of the paediatric participant and participant’s assent if able to understand and/or follow study instructions alone or with parental assistance 4. Parents / legally acceptable representative should have signed consent for a CmT regimen that is known to be myelotoxic, with counts expected to drop below an absolute neutrophil count (ANC) of 0.5 × 109/L for at least 3 days. 5. ANC and platelet count: Participants must have an ANC >1 × 109/L and a pl atelet count >100 × 109/L to be eligible for therapy at the start of CmT 6.Normal cardiac, renal, and hepatic function 7.All participants must have a life expectancy of >4 months in the opinion of the investigator 8.On-treatment Eastern Cooperative Oncology Group (ECOG) performance status =2 9.Participants with baseline laboratory values acceptable for them to receive chemotherapy Are the trial subjects under 18? yes Number of subjects for this age range: 12 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: Any potential participant who meets any of the following criteria will be excluded from participating in the study: 1.Known hypersensitivity to any component of this product. 2.Previous treatment with long-acting G-CSF 3.History of congenital neutropenia or cyclic neutropenia 4.Any illness or condition that in the opinion of the investigator may affect the safety of the participant or the evaluation of any study endpoint 5.Bone marrow involvement 6.Prior bone marrow or stem cell transplant, or prior radiation to =25% of bone marrow (e.g., whole pelvic radiation) for any reason, or any therapeutic radiation within the 4 weeks prior to the first dose 7.Ongoing active infection or history of infectious disease within 2 weeks prior to the screening visit 8.A positive polymerase chain reaction test for COVID-19. 9.Treatment with lithium at screening or planned during the study 10.Participation in an interventional clinical study within 30 days or 5 half-lives of the investigational product before enrollment, whichever is longer 11.Participants with autoimmune diseases 12.Participants with severe liver, kidney, heart, or lung dysfunction precluding the expected delivery of the intended chemotherapy regimen
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Assess the efficacy of a subcutaneous (SC) dose administration of pegfilgrastim per chemotherapy cycle compared to daily SC dose administrations of filgrastim in children receiving CmT;Secondary Objective: Assess the pharmacodynamics, pharmacokinetics, safety, and tolerability including local (injection site) tolerability of a single SC dose administration of pegfilgrastim per chemotherapy cycle compared to daily SC dose administrations of filgrastim in children receiving CmT;Primary end point(s): To assess the efficacy of a single subcutaneous (SC) dose administration of pegfilgrastim per chemotherapy cycle compared to daily SC dose administrations of filgrastim in children receiving CmT;Timepoint(s) of evaluation of this end point: • Incidence and duration of severe neutropenia (ANC 38.3°C or 2 consecutive readings higher than 37.8°C measured at the axilla or external ear at least 2 hours apart; and ANC <0.5 × 10^9 /L per chemotherapy cycle and across all chemotherapy cycles. • Area under the curve (AUC) of absolute neutrophil count (AUCANC) in a chemotherapy cycle. ANC nadir (measured in 10^9/L), which is the lowest ANC recorded across all cycles. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): To assess the pharmacodynamics, pharmacokinetics, safety, and tolerability including local (injection site) tolerability of a single SC dose administration of pegfilgrastim per chemotherapy cycle compared to daily SC dose administrations of filgrastim in children receiving CmT;Timepoint(s) of evaluation of this end point: • Total time (days) in Intensive Care Unit (ICU) across all cycles • Percentage of scheduled chemotherapy dose that was delivered across all cycles • Proportion with chemotherapy doses reduced, omitted, or delayed across all cycles • Time in days in hospital and time in the ICU due to FN or associated infections across all cycles • Number of days of delay of chemotherapy across all cycles • Occurrence and/or resolution of chemotherapy-induced mucositis across all cycles | — |
Countries
India
Contacts
Public ContactDr. Pravin Ghadge
Intas Pharmaceuticals Ltd
Outcome results
None listed