Pediatric OncologyPhase 1NCT02085148
A Phase I Dose Finding Study in Children With Solid Tumors Recurrent or Refractory to Standard Therapy
A Phase 1 study testing Regorafenib (BAY73-4506), Vincristine (Cellcristin®) and Irinotecan (Irinotecan Cell pharm®) in people with Pediatric Oncology, run by Bayer at 12 sites in 4 countries.
Trial summary
Dose escalation phase of the study : To define the safety profile, maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of regorafenib administered orally as a single agent in a 3-weeks-on/1- week-off schedule in repeating cycles of 28 days in pediatric subjects with solid malignant tumors recurrent or… Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Pediatric Oncology
- What is being tested?
- Regorafenib (BAY73-4506), Vincristine (Cellcristin®) and Irinotecan (Irinotecan Cell pharm®)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 62 participants
- Where
- 12 sitesin 4 countries
- Study dates
- 11 Apr 2014to 13 Mar 2024
- 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 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Signed Informed Consent Form by subjects and/or subjects' parents/legal guardians and age appropriate Assent Form by the subjects obtained before any study specific procedure
- Age: from 6 months to less than 18 years old
- Diagnosis, Dose escalation phase of the study: subjects must have had histologic verification of solid malignancy at original diagnosis. Subjects with recurrent or refractory solid tumors are eligible, including primary central nervous system (CNS) tumors or subjects with known CNS metastases. Subject's current disease state must be one for which there is no known effective therapy or therapy proven to prolong survival with an acceptable quality of life. Effective therapy may include surgery, radiation therapy, chemotherapy or any combination of these modalities.
- Dose expansion phase of the study: subjects must have relapsed/refractory RMS or a solid malignant tumor (Ewing sarcoma, hepatoblastoma, neuroblastoma and Wilms tumor) in which treatment with vincristine/irinotecan is considered backbone chemotherapy at relapse and a scientific rationale to combine vincristine/irinotecan with regorafenib exists.
- Subjects must have at least one measurable or evaluable lesion according to Response Evaluation Criteria in Solid Tumors (RECIST), version 1.1. For the neuroblastoma subjects with osteomedullary disease, the SIOPEN (International Society of Pediatric Oncology Europe Neuroblastoma Group) score will be used. Bone scans (if clinically indicated) should be obtained ≤12 weeks prior to the start of treatment.
- Life expectancy of at least 12 weeks from the time of signing informed consent/assent.
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- Performance level: Karnofsky ≥ 70% for subjects > 12 years of age or Lansky ≥ 70% for subjects ≤ 12 years of age
- Adequate hematological function assessed by the following laboratory requirements conducted within 7 days before starting study treatment:
- Peripheral absolute neutrophil count (ANC): ≥ 1.0 x 109/L Platelet count : ≥ 100 x 109/L (transfusion independent) Hemoglobin: ≥ 8.0 g/dL
- -Adequate hepatic function defined as:
- Aspartate aminotransferase/alanine aminotransferase (AST/ALT) ≤ 3.0* ULN
- Bilirubin (sum of conjugated and unconjugated) ≤ 1.5 * ULN
Who cannot join
- Prior treatment with regorafenib. Subjects permanently withdrawn from study participation will not be allowed to re-enter the study.
- Dose expansion phase of the study only: Subjects with brain tumors or subjects with known CNS metastases are excluded.
- Subjects with uncontrolled baseline hypertension higher than Grade 1 NCICTCAE v. 4.0
- Subjects with evidence or history of disorders of coagulation or thrombosis
- Cardiac abnormalities and cardiac arrhythmias requiring anti-arrhythmic therapy (beta blockers or digoxin are permitted)
- History of organ allograft (including allogeneic bone marrow transplant)
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- Any other malignant disease treated prior to study entry
- Pregnancy or breast feeding
- Significant gastrointestinal disorders with diarrhea as a major symptom e.g., Crohn's disease or any malabsorption condition
- Close affiliation with the investigational site, e.g. a close relative of the investigator or a dependent person (e.g. employee or student of the investigational site)
- Unresolved toxicity higher than NCI-CTCAE v. 4.0 Grade 1 attributed to any prior therapy/procedure (excluding alopecia, chemotherapy-induced ototoxicity, Grade 2 chemotherapy-induced neuropathy and, as per above eligibility criteria, anemia with hemoglobin ≥ 8 mg/dL and ANC ≥ 1.0 x 10 9/L ).
- Any other malignant disease treated prior to the study
Trial locations and contacts
12 sites in 4 countries.
12 sites in 4 countries
France4 sites
Study site
Lyon, 69008
Study site
Marseille, 13005
Study site
Paris, 75248
Study site
Villejuif, 94805
United Kingdom4 sites
Study site
Birmingham, West Midlands, B4 6NH
Study site
Manchester, M13 9WL
Study site
Newcastle upon Tyne, Tyne and Wear, NE1 4LP
Study site
Sutton, Surrey, SM2 5PT
About this study
Official title: A Multi-center, Open-label, Non-randomized, Phase I Dose Escalation Study of Regorafenib (BAY 73-4506) in Pediatric Subjects With Solid Malignant Tumors That Are Recurrent or Refractory to Standard Therapy
Dose escalation phase of the study :
To define the safety profile, maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of regorafenib administered orally as a single agent in a 3-weeks-on/1- week-off schedule in repeating cycles of 28 days in pediatric subjects with solid malignant tumors recurrent or refractory to standard therapy. To characterize the pharmacokinetics (PK) of regorafenib The dose escalation phase of the study has been completed.
Read more
Expansion phase:
To define the safety profile, MTD and the RP2D of regorafenib administered orally in combination with backbone chemotherapy (vincristine and irinotecan) at relapse in pediatric subjects with rhabdomyosarcoma (RMS) and other solid malignant tumors recurrent or refractory to standard therapy.
Read the detailed description
Expansion Phase of the study:
Subjects must have relapsed/refractory RMS or a solid malignant tumor (Ewing sarcoma, hepatoblastoma, neuroblastoma and Wilms tumor).
- Conditions
- Pediatric Oncology
- Being tested
- Regorafenib (BAY73-4506), Vincristine (Cellcristin®), Irinotecan (Irinotecan Cell pharm®)
- Keywords
- Pediatric cancer, Rhabdomyosarcoma,, Ewing sarcoma, Hepatoblastoma, Neuroblastoma, Wilm's tumor
- Medical subject headings
- Neoplasms, Rhabdomyosarcoma, Sarcoma, Ewing, Hepatoblastoma, Neuroblastoma, Wilms Tumor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Regorafenib (BAY73-4506)Drug
Regorafenib will be given orally once a day, across cycles of 21 days each. During each cycle regorafenib is taken for 2 weeks followed by one week off the drug. Doses of the study drug used in this study are age-dependent and the children's dose will been adjusted based on the age and the body surface area and given either as tablets or granulate.
Vincristine (Cellcristin®)Drug
Vincristine will be given at a dose of 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg, maximum 2.0 mg) on Day 1 and Day 8 in 21-day cycles.
Irinotecan (Irinotecan Cell pharm®)Drug
Irinotecan will be administered at a starting dose of 50 mg/m2/day from Day 1 to Day 5, in 21 day cycles.
| Study group | Role | What they receive |
|---|---|---|
| Sequential dosing schedule | Experimental | Expansion phase: Schedule B - Sequential dosing schedule: Of a 21-day cycle, regorafenib will be dosed sequentially, following administration of VI: Vincristine：intravenous bolus, 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg), Day 1 and Day 8. Irinotecan: intravenously over 1 hour, 50mg/m2/day, Day 1 to Day 5. Regorafenib: orally, at a starting dose level of 72 mg/m2 (subjects 2 to less than 18 years old) or 60 mg/m2 (subjects 6 to less than 24 months old) once daily, Day 8 to Day 21. Receives: Drug: Regorafenib (BAY73-4506); Drug: Vincristine (Cellcristin®); Drug: Irinotecan (Irinotecan Cell pharm®) |
| Concomitant dosing schedule | Experimental | Expansion phase: Schedule A - Concomitant dosing schedule: Of a 21-day cycle, regorafenib will be concomitantly administered with vincristine and irinotecan (VI): Vincristine: intravenous bolus, 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg), Day 1 and Day 8. Irinotecan: intravenously over 1 hour, 50 mg/m2/day, Day 1 to Day 5. Regorafenib: orally, at a starting dose level of 72 mg/m2 (subjects 2 to less than 18 years old) or 60 mg/m2 (subjects 6 to less than 24 months old) once daily, Day 1 to Day 14. Receives: Drug: Regorafenib (BAY73-4506); Drug: Vincristine (Cellcristin®); Drug: Irinotecan (Irinotecan Cell pharm®) |
| Dose escalation | Experimental | Dose escalation phase: This phase of the study has been completed Receives: Drug: Regorafenib (BAY73-4506) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Maximum Tolerated Dose
MTD is defined as the dose level at which none or 1 of 6 participants experiences dose-limiting toxicity (DLT), when at least 2 of 3-6 participants experience a DLT at the next highest dose
Measured: approximately after 21 months
Safety: Recommended Phase II Dose
In order to establish a RP2D, the MTD cohort will be expanded to have at least 12 evaluable subjects to confirm the RP2D. It is expected that at least 15 subjects evaluable for DLTs will be necessary to establish the RP2D of the combination"
Measured: approximately after 21 months
Number of participants with Adverse Events
Individual listings of adverse events will be provided. The incidence of treatment-emergent adverse events and drug-related adverse events, respectively, will be summarized by worst NCI-CTCAE v 4.0 grade and by dose level
Measured: Dose escalation phase:approximately after 21 months; Expansion Phase: approximately after 21 months
AUC(0-24)md based on nominal dosing
Dose escalation phase has been completed
Measured: Dose escalation phase:Cycle 1 Day 1, Day 15 and Day 21
Secondary outcomes (10)
Overall survival
Measured: Dose escalation phase: approximately 21 months; Expansion phase: approximately 21 months
Time to progression
Measured: Dose escalation phase: approximately 21 months; Expansion phase: approximately 21 months
Tumor response: tumor assessment by RECIST v. 1.1
Measured: Dose escalation phase: approximately 21 months; Expansion phase: approximately 21 months
Taste and texture questionnaire of the regorafenib formulations
Expansion phase Dose escalation phase
Measured: Dose escalation phase: Cycle 1; Expansion phase:Concomitant: Cycle 1 Day 1;Sequential: Cycle 1 Day 8
AUC(0-24)md based on nominal dosing
Expansion phase
Measured: Expansion Phase:Cycle 1 Day1, Day 15 and Day 21
Cmax(0-24)md based on individual dosing
Expansion phase
Measured: Expansion Phase:Cycle 1 Day1, Day 15 and Day 21
Cav(0-24)md based on individual dosing
Expansion phase Dose escalation phase
Measured: Expansion phase:Cycle 1 Day1, Day 15 and Day 21; Dose escalation phase:Cycle 1 Day1, Day 15 and Day 21
t1/2eff,md based on individual dosing
Expansion phase Dose escalation phase
Measured: Expansion phase:Cycle 1 Day1, Day 15 and Day 21; Dose escalation phase:Cycle 1 Day1, Day 15 and Day 21
AUC(0-24)md based on individual dosing
Expansion phase
Measured: Expansion Phase:Cycle 1 Day1, Day 15 and Day 21
Clearance of irinotecan and SN-38
Expansion phase
Measured: Expansion Phase:Cycle 1 Day1, Day 15 and Day 21
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2014 (estimated)
- Study start11 Apr 2014
- Primary completion5 May 2019
- Study completion13 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Pediatric OncologyStudies of the same condition, open and under way.
- More trials run by BayerThe sponsor's other registered studies.
- 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
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 62 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bayer Study Director, Study director, Bayer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02085148
- Sponsor's protocol ID
- 15906
- Other IDs
- 2013-003579-36
- Status last verified
- Apr 2024
- First submitted
- 7 Mar 2014
- Last updated
- 22 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.
Publications
Casanova M, Bautista F, Campbell-Hewson Q, Makin G, Marshall LV, Verschuur AC, Canete Nieto A, Corradini N, Ploeger BA, Brennan BJ, Mueller U, Zebger-Gong H, Chung JW, Geoerger B. Regorafenib plus Vincristine and Irinotecan in Pediatric Patients with Recurrent/Refractory Solid Tumors: An Innovative Therapy for Children with Cancer Study. Clin Cancer Res. 2023 Nov 1;29(21):4341-4351. doi: 10.1158/1078-0432.CCR-23-0257.
PubMed 37606641Geoerger B, Morland B, Jimenez I, Frappaz D, Pearson ADJ, Vassal G, Maeda P, Kincaide J, Mueller U, Schlief S, Teufel M, Ploeger BA, Cleton A, Agostinho AC, Marshall LV. Phase 1 dose-escalation and pharmacokinetic study of regorafenib in paediatric patients with recurrent or refractory solid malignancies. Eur J Cancer. 2021 Aug;153:142-152. doi: 10.1016/j.ejca.2021.05.023. Epub 2021 Jun 20.
PubMed 34157616
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open-label, Non-randomized, Phase I Dose Escalation Study of Regorafenib (BAY 73-4506) in Pediatric Subjects With Solid Malignant Tumors That Are Recurrent or Refractory to Standard Therapy
- ClinicalTrials.gov ID
- NCT02085148
- Acronym / sponsor ID
- 15906
- Conditions
- Pediatric Oncology
- Keywords
- Pediatric cancer, Rhabdomyosarcoma,, Ewing sarcoma, Hepatoblastoma, Neuroblastoma, Wilm's tumor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 11 Apr 2014 (actual)
- Primary completion
- 5 May 2019 (actual)
- Study completion
- 13 Mar 2024 (actual)
- First posted
- 12 Mar 2014 (estimated)
- 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.
Dose escalation phase of the study :
To define the safety profile, maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of regorafenib administered orally as a single agent in a 3-weeks-on/1- week-off schedule in repeating cycles of 28 days in pediatric subjects with solid malignant tumors recurrent or refractory to standard therapy. To characterize the pharmacokinetics (PK) of regorafenib The dose escalation phase of the study has been completed.
Expansion phase:
To define the safety profile, MTD and the RP2D of regorafenib administered orally in combination with backbone chemotherapy (vincristine and irinotecan) at relapse in pediatric subjects with rhabdomyosarcoma (RMS) and other solid malignant tumors recurrent or refractory to standard therapy.
Detailed description
Expansion Phase of the study:
Subjects must have relapsed/refractory RMS or a solid malignant tumor (Ewing sarcoma, hepatoblastoma, neuroblastoma and Wilms tumor).
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Sequential dosing schedule | Experimental | Drug: Regorafenib (BAY73-4506), Drug: Vincristine (Cellcristin®), Drug: Irinotecan (Irinotecan Cell pharm®)Expansion phase: Schedule B - Sequential dosing schedule: Of a 21-day cycle, regorafenib will be dosed sequentially, following administration of VI: Vincristine：intravenous bolus, 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg), Day 1 and Day 8. Irinotecan: intravenously over 1 hour, 50mg/m2/day, Day 1 to Day 5. Regorafenib: orally, at a starting dose level of 72 mg/m2 (subjects 2 to less than 18 years old) or 60 mg/m2 (subjects 6 to less than 24 months old) once daily, Day 8 to Day 21. |
| Concomitant dosing schedule | Experimental | Drug: Regorafenib (BAY73-4506), Drug: Vincristine (Cellcristin®), Drug: Irinotecan (Irinotecan Cell pharm®)Expansion phase: Schedule A - Concomitant dosing schedule: Of a 21-day cycle, regorafenib will be concomitantly administered with vincristine and irinotecan (VI): Vincristine: intravenous bolus, 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg), Day 1 and Day 8. Irinotecan: intravenously over 1 hour, 50 mg/m2/day, Day 1 to Day 5. Regorafenib: orally, at a starting dose level of 72 mg/m2 (subjects 2 to less than 18 years old) or 60 mg/m2 (subjects 6 to less than 24 months old) once daily, Day 1 to Day 14. |
| Dose escalation | Experimental | Drug: Regorafenib (BAY73-4506)Dose escalation phase: This phase of the study has been completed |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- Signed Informed Consent Form by subjects and/or subjects' parents/legal guardians and age appropriate Assent Form by the subjects obtained before any study specific procedure
- Age: from 6 months to less than 18 years old
- Diagnosis, Dose escalation phase of the study: subjects must have had histologic verification of solid malignancy at original diagnosis. Subjects with recurrent or refractory solid tumors are eligible, including primary central nervous system (CNS) tumors or subjects with known CNS metastases. Subject's current disease state must be one for which there is no known effective therapy or therapy proven to prolong survival with an acceptable quality of life. Effective therapy may include surgery, radiation therapy, chemotherapy or any combination of these modalities.
- Dose expansion phase of the study: subjects must have relapsed/refractory RMS or a solid malignant tumor (Ewing sarcoma, hepatoblastoma, neuroblastoma and Wilms tumor) in which treatment with vincristine/irinotecan is considered backbone chemotherapy at relapse and a scientific rationale to combine vincristine/irinotecan with regorafenib exists.
- Subjects must have at least one measurable or evaluable lesion according to Response Evaluation Criteria in Solid Tumors (RECIST), version 1.1. For the neuroblastoma subjects with osteomedullary disease, the SIOPEN (International Society of Pediatric Oncology Europe Neuroblastoma Group) score will be used. Bone scans (if clinically indicated) should be obtained ≤12 weeks prior to the start of treatment.
Key exclusion criteria (12 listed)
- Prior treatment with regorafenib. Subjects permanently withdrawn from study participation will not be allowed to re-enter the study.
- Dose expansion phase of the study only: Subjects with brain tumors or subjects with known CNS metastases are excluded.
- Subjects with uncontrolled baseline hypertension higher than Grade 1 NCICTCAE v. 4.0
- Subjects with evidence or history of disorders of coagulation or thrombosis
- Cardiac abnormalities and cardiac arrhythmias requiring anti-arrhythmic therapy (beta blockers or digoxin are permitted)
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 |
|---|---|---|
| Regorafenib (BAY73-4506)Regorafenib will be given orally once a day, across cycles of 21 days each. During each cycle regorafenib is taken for 2 weeks followed by one week off the drug. Doses of the study drug used in this study are age-dependent and the children's dose will been adjusted based on the age and the body surface area and given either as tablets or granulate. | Drug | Concomitant dosing schedule, Dose escalation, Sequential dosing schedule |
| Vincristine (Cellcristin®)Vincristine will be given at a dose of 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg, maximum 2.0 mg) on Day 1 and Day 8 in 21-day cycles. | Drug | Concomitant dosing schedule, Sequential dosing schedule |
| Irinotecan (Irinotecan Cell pharm®)Irinotecan will be administered at a starting dose of 50 mg/m2/day from Day 1 to Day 5, in 21 day cycles. | Drug | Concomitant dosing schedule, Sequential dosing schedule |
Primary outcome
Safety: Maximum Tolerated Dose
Time frame: approximately after 21 months
MTD is defined as the dose level at which none or 1 of 6 participants experiences dose-limiting toxicity (DLT), when at least 2 of 3-6 participants experience a DLT at the next highest dose
Safety: Recommended Phase II Dose
Time frame: approximately after 21 months
In order to establish a RP2D, the MTD cohort will be expanded to have at least 12 evaluable subjects to confirm the RP2D. It is expected that at least 15 subjects evaluable for DLTs will be necessary to establish the RP2D of the combination"
Number of participants with Adverse Events
Time frame: Dose escalation phase:approximately after 21 months; Expansion Phase: approximately after 21 months
Individual listings of adverse events will be provided. The incidence of treatment-emergent adverse events and drug-related adverse events, respectively, will be summarized by worst NCI-CTCAE v 4.0 grade and by dose level
AUC(0-24)md based on nominal dosing
Time frame: Dose escalation phase:Cycle 1 Day 1, Day 15 and Day 21
Dose escalation phase has been completed
10 secondary outcomes
Overall survival
Time frame: Dose escalation phase: approximately 21 months; Expansion phase: approximately 21 months
Time to progression
Time frame: Dose escalation phase: approximately 21 months; Expansion phase: approximately 21 months
Tumor response: tumor assessment by RECIST v. 1.1
Time frame: Dose escalation phase: approximately 21 months; Expansion phase: approximately 21 months
Taste and texture questionnaire of the regorafenib formulations
Time frame: Dose escalation phase: Cycle 1; Expansion phase:Concomitant: Cycle 1 Day 1;Sequential: Cycle 1 Day 8
AUC(0-24)md based on nominal dosing
Time frame: Expansion Phase:Cycle 1 Day1, Day 15 and Day 21
Cmax(0-24)md based on individual dosing
Time frame: Expansion Phase:Cycle 1 Day1, Day 15 and Day 21
Cav(0-24)md based on individual dosing
Time frame: Expansion phase:Cycle 1 Day1, Day 15 and Day 21; Dose escalation phase:Cycle 1 Day1, Day 15 and Day 21
t1/2eff,md based on individual dosing
Time frame: Expansion phase:Cycle 1 Day1, Day 15 and Day 21; Dose escalation phase:Cycle 1 Day1, Day 15 and Day 21
AUC(0-24)md based on individual dosing
Time frame: Expansion Phase:Cycle 1 Day1, Day 15 and Day 21
Clearance of irinotecan and SN-38
Time frame: Expansion Phase:Cycle 1 Day1, Day 15 and Day 21
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 22 Apr 2024.
- Derived from this study: PubMed 37606641Casanova M, Bautista F, Campbell-Hewson Q, Makin G, Marshall LV, Verschuur AC, Canete Nieto A, Corradini N, Ploeger BA, Brennan BJ, Mueller U, Zebger-Gong H, Chung JW, Geoerger B. Regorafenib plus Vincristine and Irinotecan in Pediatric Patients with Recurrent/Refractory Solid Tumors: An Innovative Therapy for Children with Cancer Study. Clin Cancer Res. 2023 Nov 1;29(21):4341-4351. doi: 10.1158/1078-0432.CCR-23-0257.
- Derived from this study: PubMed 34157616Geoerger B, Morland B, Jimenez I, Frappaz D, Pearson ADJ, Vassal G, Maeda P, Kincaide J, Mueller U, Schlief S, Teufel M, Ploeger BA, Cleton A, Agostinho AC, Marshall LV. Phase 1 dose-escalation and pharmacokinetic study of regorafenib in paediatric patients with recurrent or refractory solid malignancies. Eur J Cancer. 2021 Aug;153:142-152. doi: 10.1016/j.ejca.2021.05.023. Epub 2021 Jun 20.
- Individual participant dataSharing planned: No. Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.
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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.
Bayer Study Director study director
Bayer
12 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Regorafenib (BAY73-4506), Vincristine (Cellcristin®) and Irinotecan (Irinotecan Cell pharm®) in people with Pediatric Oncology, run by Bayer at 12 sites in 4 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 12 inclusion and 12 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 22 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in France, United Kingdom, Italy, 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?
Safety: Maximum Tolerated Dose (time frame: approximately after 21 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02085148, which also lists 2 linked publications.
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 22 Apr 2024.