MedPath

Study evaluating the efficacy and safety of regorafenib as maintenance therapy after first-line treatment in patients with bone sarcomas

Phase 1
Conditions
Patients with bone sarcomas other than Ewing sarcoma or chondrosarcoma or chordoma, who have no residual disease after neoadjuvant chemotherapy, surgery and/or adjuvant chemotherapy.
Therapeutic area: Diseases [C] - Cancer [C04]
Registration Number
EUCTR2018-004045-16-FR
Lead Sponsor
Centre Léon Bérard
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
168
Inclusion Criteria

I1. Age = 16 years at the day of consenting to the study;
I2. Patients must have histologically confirmed diagnosis of primary bone sarcoma of one of the following histotypes:
• Osteosarcomas (conventional-intramedullary/central high grade, small cell, telangiectatic or high-grade surface osteosarcomas);
• Bone sarcomas other than Ewing sarcoma, chondrosarcoma and chordoma.
I3. Availability of archival Formalin-Fixed Paraffin Embedded (FFPE) block.
I4. Prior treatment for localized or metastatic disease for bone sarcoma must have been completed. It should include:
• Neoadjuvant chemotherapy with documented assessment of histological response,
• Local procedure: Surgery (or radiotherapy if tumour is unresectable)
• Adjuvant chemotherapy (Nota Bene: patients with histotype different from osteosarcoma may not have received adjuvant treatment).
For OS, (excepted head and neck localisations), neoadjuvant and/or adjuvant chemotherapy should include methotrexate-based regimen for patients < 18 years old or anthracycline and cisplatin-based regimen for patients = 18 years old.
For head and neck OS, neoadjuvant and adjuvant chemotherapy should include adriamycin, cisplatin or ifosfamide-based regimen.
For non-OS, neoadjuvant and/or adjuvant chemotherapy should include adriamycine and/or cisplatine-based regimen.
I5. Recovery to NCI-CTCAE v5 Grade 0 or 1 level or recovery to baseline preceding the prior treatment from any previous drug/procedure related toxicity (except alopecia, anaemia, and hypothyroidism);
I6. Interval between the last chemotherapy administration and the date of randomisation: at least 4 weeks but no longer than 2 months;
...See the protocol
Are the trial subjects under 18? yes
Number of subjects for this age range: 68
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

Exclusion Criteria

E1. Prior treatment with any VEGFR inhibitor (thus, any prior exposure to sunitinib, sorafenib, pazopanib, bevacizumab, or other VEGFR inhibitor);
E2. All soft tissue sarcomas (including but not limited to soft tissue osteosarcoma and Ewing soft tissue sarcoma), and Ewing sarcoma, chondrosarcoma and chordoma;
E3. Prior history of other malignancies other than study disease (except for basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix) within 3 years prior to randomization;
E4. Cardiovascular dysfunction:
• Left ventricular ejection fraction (LVEF) < 50%,
• Congestive heart failure = New York Heart Association (NYHA) class 2,
• Myocardial infarction < 6 months prior to first study drug administration,
• Cardiac arrhythmias requiring therapy (beta blockers or digoxin are permitted),
• Unstable (angina symptoms at rest) or new-onset angina within the last 3 months prior to first study drug administration;
E5. Uncontrolled hypertension (systolic blood pressure > 150mmHg or diastolic pressure > 90 mmHg despite optimal treatment);
E6. Arterial or venous thrombotic or embolic events such as cerebrovascular accident (including transient ischemic attacks), deep vein thrombosis, or pulmonary embolism within the last 6 months before the first study drug administration;
...See the protocol

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: The primary objective is to compare the antitumor efficacy of regorafenib versus placebo as maintenance treatment in patients with bone sarcomas. ;Secondary Objective: • The Time to Treatment Failure (TTF),<br>• The Overall Survival,<br>• The Quality of Life (EORTC QLQ-C30),<br>• The tolerance profile (NCI-CTC AE version 5),<br>• Compliance to study treatment<br>;Primary end point(s): the Relapse-Free Survival (RFS).;Timepoint(s) of evaluation of this end point: assessment according to the RECISTv1.1.
Secondary Outcome Measures
NameTimeMethod
Secondary end point(s): • the Time to Treatment Failure (TTF),<br>• the Overall Survival,<br>• the Quality of Life (EORTC QLQ-C30),<br>• the tolerance profile (NCI-CTC AE version 5).<br>;Timepoint(s) of evaluation of this end point: • Throughout the study<br>• Each tumor assessment<br>
© Copyright 2025. All Rights Reserved by MedPath