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

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ClinicalTrials.gov Identifier: NCT04610736
Recruitment Status : Recruiting
First Posted : October 30, 2020
Last Update Posted : May 11, 2022
Sponsor:
Collaborator:
ClinSearch
Information provided by (Responsible Party):
Orphelia Pharma

Tracking Information
First Submitted Date  ICMJE October 20, 2020
First Posted Date  ICMJE October 30, 2020
Last Update Posted Date May 11, 2022
Actual Study Start Date  ICMJE March 16, 2021
Estimated Primary Completion Date August 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 29, 2020)
  • Population Phamacokinetic parameter: AUC24 [ Time Frame: 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 ]
    Estimated by a population analysis performed with NONMEM (7.4)
  • Population Phamacokinetic parameter: Cmax [ Time Frame: 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 ]
    Estimated by a population analysis performed with NONMEM (7.4)
  • Population Phamacokinetic parameter: T1/2 [ Time Frame: 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 ]
    Estimated by a population analysis performed with NONMEM (7.4)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 29, 2020)
  • Acceptability of the oral suspension of temozolomide: score [ Time Frame: At Day 1 and Day 5 of treatment cycle 1 (each cycle is 21 or 28 days) ]
    Scoring with a standardized assessment tool: CAST - ClinSearch Acceptability Score Test®. This tool measures 9 observational drivers of drug acceptability.
  • Incidence of treatment-emergent adverse events [ Time Frame: Through study completion, an average of 6 months including compassionate use period ]
    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
  • Activity of the oral suspension of temozolomide [ Time Frame: At the end of each 21- or 28-day treatment cycle of the compassionate use period ]
    Activity assessment (complete or partial response, stable disease, disease progression)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE TEMOkids Study : A Phase I Pediatric Study for KIMOZO, Oral Suspension of Temozolomide
Official Title  ICMJE TEMOkids Study: A Population Pharmacokinetic, Acceptability and Safety Study for KIMOZO, a Paediatric Oral Suspension of Temozolomide
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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
Non-randomized, international, multi-centre, open-label, single arm study
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pediatric Cancer
Intervention  ICMJE Drug: Temozolomide Oral Suspension
One prescribed oral dose (range 75 to 200 mg/m2) once daily for 5 days
Other Name: KIMOZO, Ped-TMZ
Study Arms  ICMJE Experimental: Single arm
Temozolomide 40 mg/ml, Oral suspension
Intervention: Drug: Temozolomide Oral Suspension
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 29, 2020)
40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2022
Estimated Primary Completion Date August 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Caroline Lemarchand, PharmD +33142770818 caroline.lemarchand@orphelia-pharma.eu
Contact: Hugues Bienaymé, PhD +33142770818 hugues.bienayme@orphelia-pharma.eu
Listed Location Countries  ICMJE France,   Germany,   Netherlands,   Spain,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04610736
Other Study ID Numbers  ICMJE ORP-TMZ-I- b
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Orphelia Pharma
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Orphelia Pharma
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE ClinSearch
Investigators  ICMJE
Principal Investigator: Samuel Abbou, MD Gustave Roussy, Cancer Campus, Grand Paris
PRS Account Orphelia Pharma
Verification Date May 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP