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.

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.

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