TEMOkids Study : A Phase I Pediatric Study for KIMOZO, Oral Suspension of Temozolomide

TEMOkids Study: A Population Pharmacokinetic, Acceptability and Safety Study for KIMOZO, a Paediatric Oral Suspension of Temozolomide

Non-randomized, international, multi-centre, open-label, single arm study to determine the pharmacokinetic (PK) parameters of a single dose of an oral suspension of temozolomide (KIMOZO) in the pediatric population aged 1 year and over.

Study Overview

• Status: Completed
• Conditions: Pediatric Cancer
• Intervention / Treatment: Drug: Temozolomide Oral Suspension

Detailed Description

The TEMOkids study is an international open-label, non-randomized, prospective, single-arm phase 1 study to determine the pharmacokinetic (PK) parameters of a single dose of an oral suspension of temozolomide (KIMOZO) in the pediatric population aged 1 year and over.

Patients will be subject to at least one 21 or 28-day treatment cycle involving administration of an oral suspension of temozolomide (KIMOZO) for five consecutive days followed by 16 or 23-days resting period, with determination of the PK parameters on the first treatment day.

Five (5) additional cycles will be allowed under compassionate use regimen. The compassionate use period could be extended at the discretion of the investigator and in agreement with the sponsor. Safety and activity data will be collected during the compassionate follow-up period.

The study will be held in multiple sites spread across Europe.

• Study Type: Interventional
• Enrollment (Actual): 49
• Phase: Phase 1

Contacts and Locations

Study Locations

• France
  • Lille, France, 59000
    • Centre Oscar Lambret
  • Lyon, France, 69008
    • Institut d'Hématologie et d'Oncologie Pédiatrique
  • Marseille, France, 13005
    • CHU Timone Enfants
  • Paris, France, 75005
    • Institut Curie
  • Villejuif, France, 94800
    • Gustave Roussy
• Germany
  • Berlin, Germany, 13353
    • Charite University Medicine Berlin
  • Heidelberg, Germany, 69120
    • Hopp Children's Cancer Center Heidelberg
• Netherlands
  • Utrecht, Netherlands, 3584 CS
    • Princess Maxima Center for Pediatric Oncology
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d'Hebron
• United Kingdom
  • London, United Kingdom, WC1N 3JH
    • Great Ormond Street Hospital for Children
  • Southampton, United Kingdom, SO16 6YD
    • Southampton General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Pediatric patients in need of temozolomide (all indications with 5-day treatment per 21- or 28-day cycle).
• 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:

  • haemoglobin ≥ 80 g/L (transfusion support authorized)
  • neutrophil count ≥ 1.0 x 10e9 cells/L
  • platelet count ≥ 100 x 10e9 cells/L (without transfusion support)
  • in case of bone marrow involvement: neutrophils ≥ 0.5 x 10e9 cells/L and platelets ≥75 x 10e9 cells/L

• Adequate renal function:

  • Creatine clearance ≥ 60 mL/min.1.73m² according to the Schwartz formula [1] or its modified form [2]

• Adequate hepatic function:

  • bilirubin ≤1.5 x ULN
  • AST and ALT ≤ 2.5 x ULN (AST, ALT 5xULN in case of liver metastases)
• Lansky Score ≥ 70%

Exclusion Criteria:

• Patients who are co-administrated at day one with sodium valproate as it decreases the clearance of temozolomide
• Patients with (naso)gastric tube administration of temozolomide during first cycle of treatment.
• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Single arm; Temozolomide 40 mg/ml, Oral suspension	Drug: Temozolomide Oral Suspension; One prescribed oral dose (range 75 to 200 mg/m2) once daily for 5 days; Other Names: KIMOZO, Ped-TMZ

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Population Phamacokinetic parameter: AUC24	Estimated by a population analysis performed with NONMEM (7.4)	At Day 1 of treatment cycle 1 (each cycle is 21 or 28 days) with post-dose samples collected at about 0.15, 0.5, 1, 2.5, and 7 hours post dose
Population Phamacokinetic parameter: Cmax	Estimated by a population analysis performed with NONMEM (7.4)	At Day 1 of treatment cycle 1 (each cycle is 21 or 28 days) with post-dose samples collected at about 0.15, 0.5, 1, 2.5, and 7 hours post dose
Population Phamacokinetic parameter: T1/2	Estimated by a population analysis performed with NONMEM (7.4)	At Day 1 of treatment cycle 1 (each cycle is 21 or 28 days) with post-dose samples collected at about 0.15, 0.5, 1, 2.5, and 7 hours post dose

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Acceptability of the oral suspension of temozolomide: score	Scoring with a standardized assessment tool: CAST - ClinSearch Acceptability Score Test®. This tool measures 9 observational drivers of drug acceptability.	At Day 1 and Day 5 of treatment cycle 1 (each cycle is 21 or 28 days)
Incidence of treatment-emergent adverse events	Adverse events collected directly by investigators when patient is hospitalized and through patient diary completed by caregivers and medically controlled by investigators when patient is at home	Through study completion, an average of 6 months including compassionate use period
Activity of the oral suspension of temozolomide	Activity assessment (complete or partial response, stable disease, disease progression)	At the end of each 21- or 28-day treatment cycle of the compassionate use period

Collaborators and Investigators

• Sponsor: Orphelia Pharma
• Collaborators: ClinSearch
• Investigators: Principal Investigator: Samuel Abbou, MD, Gustave Roussy, Cancer Campus, Grand Paris

Study record dates

Study Major Dates

• Study Start (Actual): March 16, 2021
• Primary Completion (Actual): January 15, 2023
• Study Completion (Actual): July 3, 2023

Study Registration Dates

• First Submitted: October 20, 2020
• First Submitted That Met QC Criteria: October 29, 2020
• First Posted (Actual): October 30, 2020

Study Record Updates

• Last Update Posted (Actual): July 31, 2025
• Last Update Submitted That Met QC Criteria: July 28, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Keywords: neuroblastoma; other pediatric cancers requiring temozolomide
• Additional Relevant MeSH Terms: Antineoplastic Agents; Molecular Mechanisms of Pharmacological Action; Antineoplastic Agents, Alkylating; Alkylating Agents; Temozolomide
• Other Study ID Numbers: ORP-TMZ-I- b

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No