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

NCT04610736 | Completed Phase 1 DMC

• 1 other identifier: ORP-TMZ-I- b
• Study Type: interventional
• Target: 49
• Locations: 5 countries
• Sites: 11

Brief Summary

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.

Conditions

Pediatric Cancer

Keywords

neuroblastoma; other pediatric cancers requiring temozolomide

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (3)

• Acceptability of the oral suspension of temozolomide: score

  At Day 1 and Day 5 of treatment cycle 1 (each cycle is 21 or 28 days)

• Incidence of treatment-emergent adverse events

  Through study completion, an average of 6 months including compassionate use period

• Activity of the oral suspension of temozolomide

  At the end of each 21- or 28-day treatment cycle of the compassionate use period

Study Arms (1)

Single arm

EXPERIMENTAL

Temozolomide 40 mg/ml, Oral suspension

Drug: Temozolomide Oral Suspension

Interventions

Temozolomide Oral Suspension DRUG

One prescribed oral dose (range 75 to 200 mg/m2) once daily for 5 days

Also known as: KIMOZO, Ped-TMZ

Single arm

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)

You may not qualify if:

• 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.
• Known contraindication or hypersensitivity to temozolomide or any chemically close substance

Sponsors & Collaborators

• Orphelia Pharma: lead
• ClinSearch: collaborator

Study Sites (11)

Centre Oscar Lambret

Lille, 59000, France

Location

Institut d'Hématologie et d'Oncologie Pédiatrique

Lyon, 69008, France

Location

CHU Timone Enfants

Marseille, 13005, France

Location

Institut Curie

Paris, 75005, France

Location

Gustave Roussy

Villejuif, 94800, France

Location

Charité University Medicine Berlin

Berlin, 13353, Germany

Location

Hopp Children's Cancer Center Heidelberg

Heidelberg, 69120, Germany

Location

Princess Maxima Center for Pediatric Oncology

Utrecht, 3584 CS, Netherlands

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

Location

Southampton General Hospital

Southampton, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Neoplasms; Neuroblastoma

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Samuel Abbou, MD

  Gustave Roussy, Cancer Campus, Grand Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Non-randomized, international, multi-centre, open-label, single arm study

Study Record Dates

• First Submitted: October 20, 2020
• First Posted: October 30, 2020
• Study Start: March 16, 2021
• Primary Completion: January 15, 2023
• Study Completion: July 3, 2023
• Last Updated: July 31, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

FR (5); DE (2); NL (1); GB (2); ES (1)