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

Pediatric Cancer Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04610736
• Other study ID #: ORP-TMZ-I- b
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: March 16, 2021
• Est. completion date: July 2023

Study information

• Verified date: February 2023
• Source: Orphelia Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 49
• Est. completion date: July 2023
• Est. primary completion date: January 15, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Pediatric Cancer

Intervention

Drug:
• Temozolomide Oral Suspension
One prescribed oral dose (range 75 to 200 mg/m2) once daily for 5 days

Locations

Country	Name	City	State
France	Centre Oscar Lambret	Lille
France	Institut d'Hématologie et d'Oncologie Pédiatrique	Lyon
France	CHU Timone Enfants	Marseille
France	Institut Curie	Paris
France	Gustave Roussy	Villejuif
Germany	Charité University Medicine Berlin	Berlin
Germany	Hopp Children's Cancer Center Heidelberg	Heidelberg
Netherlands	Princess Maxima Center for Pediatric Oncology	Utrecht
Spain	Hospital Universitari Vall d'Hebron	Barcelona
United Kingdom	Great Ormond Street Hospital for Children	London
United Kingdom	Southampton General Hospital	Southampton

Sponsors (2)

Lead Sponsor	Collaborator
Orphelia Pharma	ClinSearch

Countries where clinical trial is conducted

France,  Germany,  Netherlands,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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	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)
Secondary	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
Secondary	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