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A Phase I Dose Finding Study in Children With Solid Tumors Recurrent or Refractory to Standard Therapy | Clinical Trials | Clareo Health

COMPLETEDPHASE1

A Phase I Dose Finding Study in Children With Solid Tumors Recurrent or Refractory to Standard Therapy

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

NCT02085148•Bayer

View on ClinicalTrials.gov

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 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).

Eligibility

Age

0 - 18 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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.
•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 10\*9/L Platelet count : ≥ 100 x 10\*9/L (transfusion independent) Hemoglobin: ≥ 8.0 g/dL
•Adequate hepatic function defined as:
+2 more criteria

Exclusion Criteria

•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)
•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)
+2 more criteria

Locations (12)

Lyon, France

Marseille, France

Paris, France

Villejuif, France

Genoa, Liguria, Italy

Milan, Lombardy, Italy

Madrid, Spain

Valencia, Spain

Sutton, Surrey, United Kingdom

Newcastle upon Tyne, Tyne and Wear, United Kingdom

Key Dates

Start Date

April 11, 2014

Primary Completion Date

May 5, 2019

Completion Date

March 13, 2024

Last Updated

April 22, 2024

Enrollment

ACTUAL participants

Conditions

Pediatric Oncology

Interventions

Regorafenib (BAY73-4506)

DRUG

Vincristine (Cellcristin®)

DRUG

Irinotecan (Irinotecan Cell pharm®)

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: April 22, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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