Pediatric CancerPhase 1NCT04610736
TEMOkids Study : A Phase I Pediatric Study for KIMOZO, Oral Suspension of Temozolomide
A Phase 1 study testing Temozolomide Oral Suspension in people with Pediatric Cancer, run by Orphelia Pharma at 11 sites in 5 countries.
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. Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Pediatric Cancer
- What is being tested?
- Temozolomide Oral Suspension
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 11 sitesin 5 countries
- Study dates
- 16 Mar 2021to 3 Jul 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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:
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- 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%
Who cannot join
- 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
Trial locations and contacts
11 sites in 5 countries.
11 sites in 5 countries
France5 sites
Centre Oscar Lambret
Lille, 59000
Institut d'Hématologie et d'Oncologie Pédiatrique
Lyon, 69008
CHU Timone Enfants
Marseille, 13005
Institut Curie
Paris, 75005
Gustave Roussy
Villejuif, 94800
Germany2 sites
Charité University Medicine Berlin
Berlin, 13353
Hopp Children's Cancer Center Heidelberg
Heidelberg, 69120
United Kingdom2 sites
Great Ormond Street Hospital for Children
London, WC1N 3JH
Southampton General Hospital
Southampton, SO16 6YD
Netherlands1 site
Princess Maxima Center for Pediatric Oncology
Utrecht, 3584 CS
About this study
Official title: 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.
Read the 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.
- Conditions
- Pediatric Cancer
- Being tested
- Temozolomide Oral Suspension
- Keywords
- neuroblastoma, other pediatric cancers requiring temozolomide
- Medical subject headings
- Neoplasms, Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Temozolomide Oral SuspensionDrug
Also called KIMOZO, Ped-TMZ
One prescribed oral dose (range 75 to 200 mg/m2) once daily for 5 days
| Study group | Role | What they receive |
|---|---|---|
| Single arm | Experimental | Temozolomide 40 mg/ml, Oral suspension Receives: Drug: Temozolomide Oral Suspension |
What is being measured
The primary outcome is the main question the study is designed to answer.
Population Phamacokinetic parameter: AUC24
Estimated by a population analysis performed with NONMEM (7.4)
Measured: 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)
Measured: 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)
Measured: 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
Scoring with a standardized assessment tool: CAST - ClinSearch Acceptability Score Test®. This tool measures 9 observational drivers of drug acceptability.
Measured: At Day 1 and Day 5 of treatment cycle 1 (each cycle is 21 or 28 days)
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
Measured: Through study completion, an average of 6 months including compassionate use period
Activity of the oral suspension of temozolomide
Activity assessment (complete or partial response, stable disease, disease progression)
Measured: At the end of each 21- or 28-day treatment cycle of the compassionate use period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2020
- Study start16 Mar 2021
- Primary completion15 Jan 2023
- Study completion3 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Non-randomized, international, multi-centre, open-label, single arm study
- Masking
- None (open label)
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- Orphelia Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ClinSearch
- Responsible party
- Sponsor
- Investigators
- Samuel Abbou, MD, Principal investigator, Gustave Roussy, Cancer Campus, Grand Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04610736
- Sponsor's protocol ID
- ORP-TMZ-I- b
- Status last verified
- Jul 2025
- First submitted
- 20 Oct 2020
- Last updated
- 31 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- TEMOkids Study: A Population Pharmacokinetic, Acceptability and Safety Study for KIMOZO, a Paediatric Oral Suspension of Temozolomide
- ClinicalTrials.gov ID
- NCT04610736
- Acronym / sponsor ID
- ORP-TMZ-I- b
- Conditions
- Pediatric Cancer
- Keywords
- neuroblastoma, other pediatric cancers requiring temozolomide
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Orphelia PharmaIndustry (pharma, biotech, devices)
- Collaborators
- ClinSearch
- Study start
- 16 Mar 2021 (actual)
- Primary completion
- 15 Jan 2023 (actual)
- Study completion
- 3 Jul 2023 (actual)
- First posted
- 30 Oct 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Non-randomized, international, multi-centre, open-label, single arm study
| Arm | Type | Receives |
|---|---|---|
| Single arm | Experimental | Drug: Temozolomide Oral SuspensionTemozolomide 40 mg/ml, Oral suspension |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (16 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Temozolomide Oral SuspensionAlso: KIMOZO, Ped-TMZOne prescribed oral dose (range 75 to 200 mg/m2) once daily for 5 days | Drug | Single arm |
Primary outcome
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)
3 secondary outcomes
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)
Incidence of treatment-emergent adverse events
Time frame: Through study completion, an average of 6 months including compassionate use period
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Samuel Abbou, MD principal investigator
Gustave Roussy, Cancer Campus, Grand Paris
11 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Temozolomide Oral Suspension in people with Pediatric Cancer, run by Orphelia Pharma at 11 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in France, Germany, United Kingdom, Netherlands, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jul 2025.