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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 : March 3, 2021
Sponsor:
Collaborator:
ClinSearch
Information provided by (Responsible Party):
Orphelia Pharma

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.

Condition or disease Intervention/treatment Phase
Pediatric Cancer Drug: Temozolomide Oral Suspension Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
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
Official Title: TEMOkids Study: A Population Pharmacokinetic, Acceptability and Safety Study for KIMOZO, a Paediatric Oral Suspension of Temozolomide
Estimated Study Start Date : March 2021
Estimated Primary Completion Date : February 2022
Estimated Study Completion Date : August 2022

Resource links provided by the National Library of Medicine


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 Name: KIMOZO, Ped-TMZ




Primary Outcome Measures :
  1. 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)

  2. 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)

  3. 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)


Secondary Outcome Measures :
  1. 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.

  2. 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

  3. 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)



Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04610736


Contacts
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Contact: Caroline Lemarchand, PharmD +33142770818 caroline.lemarchand@orphelia-pharma.eu
Contact: Hugues Bienaymé, PhD +33142770818 hugues.bienayme@orphelia-pharma.eu

Locations
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France
Centre Oscar Lambret Not yet recruiting
Lille, France, 59000
Contact: Anne-Sophie Defachelles, MD         
Institut d'Hématologie et d'Oncologie Pédiatrique Not yet recruiting
Lyon, France, 69008
Contact: Pierre Leblond, MD         
CHU Timone Enfants Not yet recruiting
Marseille, France, 13005
Contact: Nicolas André, MD         
Institut Curie Not yet recruiting
Paris, France, 75005
Contact: Isabelle Aerts, MD         
Gustave Roussy Recruiting
Villejuif, France, 94800
Contact: Samuel Abbou, MD         
Germany
Charité University Medicine Berlin Not yet recruiting
Berlin, Germany, 13353
Contact: Anne Thorwarth, MD         
Hopp Children's Cancer Center Heidelberg Not yet recruiting
Heidelberg, Germany, 69120
Contact: Olaf Witt, MD         
Netherlands
Princess Maxima Center for Pediatric Oncology Not yet recruiting
Utrecht, Netherlands, 3584 CS
Contact: Natasha Van Eijkelenburg, MD         
Spain
Hospital Universitari Vall d'Hebron Not yet recruiting
Barcelona, Spain, 08035
Contact: Lucas Moreno, MD         
United Kingdom
Southampton General Hospital Not yet recruiting
Southampton, United Kingdom, SO16 6YD
Contact: Juliet Gray, MD         
Sponsors and Collaborators
Orphelia Pharma
ClinSearch
Investigators
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Principal Investigator: Samuel Abbou, MD Gustave Roussy, Cancer Campus, Grand Paris
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Responsible Party: Orphelia Pharma
ClinicalTrials.gov Identifier: NCT04610736    
Other Study ID Numbers: ORP-TMZ-I- b
First Posted: October 30, 2020    Key Record Dates
Last Update Posted: March 3, 2021
Last Verified: March 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Orphelia Pharma:
neuroblastoma
other pediatric cancers requiring temozolomide
Additional relevant MeSH terms:
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Temozolomide
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents