TEMOkids study (ORP-TMZ-I- b): A Population pharmacokinetic, acceptability and safety study for KIMOZO, a paediatric oral suspension of temozolomide

Phase 2

Completed

• Conditions: pediatric cancers such as malignant glioma and also relapsed or refractory neuroblastoma, rhabdomyosarcoma, medulloblastoma, and Ewing sarcoma; Paediatric cancer

• Registration Number: NL-OMON50808
• Lead Sponsor: ORPHELIA Pharma

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2023-02-23
• Last Update Posted: 9 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 1

Inclusion Criteria

Paediatric patients already receiving commercially available temozolomide-based
treatment or naïve paediatric patients requiring temozolomide-based treatment
as per investigator*s decision (all indications with 5-day treatment per 21- or
28-day cycle). Indications include those described in the Temodal SmPC (i.e.
malignant glioma such as glioblastoma and anaplastic astrocytoma). For patients
having no therapeutic alternatives, the IMP may be used in off-label
indications in accordance with current treatment protocols recommended by
European and International Medical Associations (e.g. SIOPEN, EPSSG, COG,
European ITCC, SIOP,*). Such indications include but are not limited to
primarily neuroblastoma, medulloblastoma and also rhabdomyosarcoma, or Ewing
sarcoma
• Male and female patients aged 1 to less than 18 years
• Patients who have signed the informed consent or for which one, both parents
or legal guardian (depending on local legislation) have signed the informed
consent.
• Patients having records of coverage by a health insurance
• Life expectancy >= 3 months
• Adequate haematological function:
o haemoglobin >= 80 g/L (transfusion support authorized)
o neutrophil count >= 1.0 x 109 cells/L
o platelet count >= 100 x 109 cells/L (without transfusion support)
o in case of bone marrow involvement: neutrophils >= 0.5 x 109 cells/L and
platelets >=75 x 109 cells/L
• Adequate renal function:
o Creatine clearance >= 60 mL/min.1.73m² according to the Schwartz formula [1]
or its modified form
[2]
• Adequate hepatic function:
o bilirubin <=1.5 x ULN
o AST and ALT <= 2.5 x ULN (AST, ALT 5xULN in case of liver metastases)
• Lansky Score >= 70%

Exclusion Criteria

• Patient treated with sodium valproate within two weeks prior to receiving
Kimozo or patients who are coadministrated on day one of Kimozo administration
with sodium valproate as it decreases the clearance of temozolomide.
• Patients with (naso)gastric tube administration of Kimozo
• Patients already enrolled in studies investigating temozolomide or other
investigational new drugs.
• A post-menarche female with a positive blood/urine pregnancy test at
inclusion.
• Known contraindication or hypersensitivity to temozolomide or any chemically
close substance.
• Persons who are living in a facility by order of a court or an administrative
order.
• Patients infected by a SARS-CoV-2 variant.

Study & Design

• Study Type: Interventional
• Study Design: Not specified