Overview

This trial is active, not recruiting.

Condition pediatric oncology
Treatment regorafenib (bay73-4506)
Phase phase 1
Sponsor Bayer
Start date April 2014
End date November 2015
Trial size 41 participants
Trial identifier NCT02085148, 15906, 2013-003579-36

Summary

The aim of this study is to determine at which dose regorafenib is safe for children and adolescents and also to investigate the pharmacokinetics (explores what the body does to the drug) of regorafenib (study drug) in children and adolescents. Additional objectives of the study are:

- To assess the tolerability of regorafenib in children and adolescents

- To assess whether there is any tumor response to regorafenib

- To assess the palatability and acceptability of regorafenib tablets in children

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Arm
(Experimental)
regorafenib (bay73-4506)
Regorafenib will be given orally once a day, across cycles of 28 days each. During each cycle regorafenib is taken for 3 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.

Primary Outcomes

Measure
Safety: Maximum Tolerated Dose
time frame: approximately after 21 months
Safety: Recommended Phase II Dose
time frame: approximately after 21 months
Safety: Individual listings of Adverse Events
time frame: approximately after 21 months
Pharmacokinetics: Area under the concentration curve based on nominal dosing
time frame: approximately after 21 months
Pharmacokinetics: Maximum plasma concentration
time frame: approximately after 21 months
Pharmacokinetics: Average plasma concentration
time frame: approximately after 21 months
Pharmacokinetics: Effective half-life
time frame: approximately after 21 months
Pharmacokinetics: Area under the concentration curve based on individual dosing
time frame: approximately after 21 months

Secondary Outcomes

Measure
Overall survival
time frame: approximately after 21 months
Time to progression
time frame: approximately after 21 months
Tumor response: tumor assessment by RECIST v. 1.1
time frame: approximately after 21 months
Acceptability and palatability of the formulations - tablets and granulates: Taste and texture questionnaire
time frame: approximately after 21 months

Eligibility Criteria

Male or female participants from 6 months up to 18 years old.

Inclusion Criteria: - Age: from 6 months to less than 18 years old - Diagnosis: subjects must have had histologic verification of solid malignancy at original diagnosis. Subjects with recurrent or refractory solid tumors are eligible, including primary 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. - Subjects must have at least one measurable or evaluable lesion according to Response Evaluation Criteria in Solid Tumors (RECIST), version 1.1. For 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 and Lansky ≥ 70% for subjects ≤ 12 years of age. Note the performance level should not be considered reduced by limitations to movement or play caused by motor paresis or paralysis due to disease. Any neurological deficits in subjects must have been stable for a minimum of 1 week prior to first dose of study treatment. - Adequate renal, hepatic and hematologic function Exclusion Criteria: - Prior treatment with regorafenib. Subjects permanently withdrawn from study participation will not be allowed to re-enter the study. - Other anticancer treatment, including any investigational new drugs within 28 days or 5 drug half-lives (if drug half-life in subjects is known), whichever is shorter (or within 6 weeks for mitomycin C), before start of study treatment - Subjects with uncontrolled baseline hypertension higher than Grade 1 NCI-CTCAE v.4.0 (recurrent or persistent [≥24 hours] blood pressure [BP] >ULN: BP > the 95th percentile for age, height, and gender measured) - 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

Additional Information

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.
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Bayer.