AzTrials
AzTrials

Bone SarcomaPhase 2REGOMAINNCT04698785

Efficacy of Regorafenib Combined With Best Supportive Care as Maintenance Treatment in High Grade Bone Sarcomas Patients

A Phase 2 study of Bone Sarcoma and Osteosarcoma, run by Centre Leon Berard at 16 sites in France.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This is a multicenter phase II study concerning patients with high-grade bone sarcoma (HGBS) without complete remission after standard treatment at diagnosis or at relapse. Patients will be treated with regorafenib + best supportive care (BSC) for a maximum of 12 months as maintenance therapy after standard line… Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Bone Sarcoma, Osteosarcoma
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
36 participants
Where
16 sitesFrance
Study dates
21 Jul 2021to 21 Jul 2026
Phase
Phase 2
Are there results?
Not yet: the study is under way

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 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • I1. Age ≥ 12 years at the day of consenting to the study;
  • I2. Patients must have histologically confirmed high-grade sarcomas of bone primary localisation, including but not limited to: Osteosarcomas, Ewing sarcomas, Chondrosarcomas, Undifferenciated Pleomorphic Sarcomas (UPS), Leiomyosarcomas (LMS) and Angiosarcomas
  • I3. Evaluable residual disease not amenable to resection after multimodal treatment principles either at diagnosis (after standard multimodal treatment based on the histological subtype) or at relapse (chemotherapy)
  • I4. Non progressive disease (defined by the investigator according to the RECIST version 1.1 Appendix 1) at study entry;
  • I5. Interval between the date of last anticancer treatment (chemotherapy or surgery) and the start date of regorafenib: at least 4 weeks but no longer than 2 months;
  • I6. Life expectancy of greater than 6 months;
Show all 19Show fewer
  • I7. Eastern Cooperative Oncology Group (ECOG) performance status < 2 (Karnofsky ≥ 70%) (Appendix 2);
  • I8. Adequate bone marrow and organ function defined by the following laboratory results:
  • Bone marrow: i. Absolute neutrophil count ≥ 1.5 Giga/l ii. Platelets ≥ 100 Giga/l iii. Haemoglobin≥ 9 g/dl
  • Hepatic function: i. Aspartate aminotransferase (AST) and Alanine aminotransferase (ALT) ≤ 2.5 x Upper Limit of Normal (ULN) (≤ 5.0 × ULN for patients with liver involvement of their cancer) ii. Bilirubin ≤1.5 X ULN iii. Alkaline phosphatase ≤ 2.5 x ULN (≤ 5 x ULN in patient with liver involvement of their cancer). If Alkaline phosphatase > 2.5 ULN, hepatic isoenzymes 5-nucleotidase or gamma-glutamyltransferase (GGT) tests must be performed; hepatic isoenzymes 5-nucleotidase must be within the normal range and/or GGT < 1.5 x ULN.
  • Renal function: i. Serum creatinine ≤ 1.5 x ULN ii. Glomerular Filtration Rate (GFR) ≥ 30 ml/min/1.73m2 according to the Modified Diet in Renal Disease (MDRD) abbreviated formula iii. Spot urine must not show ≥ 1 "+" protein in urine or the patient will require a repeat urine analysis. If repeat urinalysis shows 1 "+" protein or more, a 24-hour urine collection will be required and must show total protein excretion < 1000 mg/24 hours
  • Coagulation: International Normalized Ratio (INR)/Partial Thromboplastin Time (PTT) ≤1.5 x ULN; Patients who are therapeutically treated with an agent such as warfarin or heparin will be allowed to participate provided that no prior evidence of underlying abnormality in coagulation parameters exists. Close monitoring of at least weekly evaluations will be performed until INR/PTT is stable based on a measurement that is pre-dose as defined by the local standard of care;
  • Pancreatic function: Lipase ≤ 1.5 x ULN
  • I9. Recovery to anticancer-treatment related 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);
  • I10. Women of childbearing potential and male patients must agree to use adequate contraception (including at least the use of condoms) for the duration of treatment and for 7 months (210 days) in women of childbearing potential or 4 months (120 days) in men sexually active with women of childbearing potential after the last dose of regorafenib
  • I11. Patients, and their parents when applicable, must sign and date an informed consent document indicating that they have been informed of all the pertinent aspects of the trial prior to enrolment;
  • I12. Patients must be willing and able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures;
  • I13. Patients affiliated to the Social Security System
  • I14. Body Surface Area (BSA) ≥ 1.30m² at the time of consenting to the study

Who cannot join

  • E1. Prior treatment with any VEGFR inhibitor (thus, any prior exposure to regorafenib, sunitinib, sorafenib, pazopanib, bevacizumab, or other VEGFR inhibitor);
  • E2. All soft tissue sarcomas (including but not limited to soft tissue osteosarcoma), and chordomas;
  • E3. Prior history of 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 defined by:
  • Left ventricular ejection fraction (LVEF) < 50%,
  • Congestive heart failure ≥ New York Heart Association (NYHA) class 2,
Show all 34Show fewer
  • 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;
  • Uncontrolled hypertension (systolic blood pressure > 150 mm Hg or diastolic pressure > 90 mm Hg despite optimal treatment);
  • 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;
  • E5. Major surgical procedure, open biopsy or significant traumatic injury within 28 days before the first study drug administration;
  • E6. Ongoing infection > Grade 2 according to NCI-CTCAE v5;
  • E7. Known history of human immunodeficiency virus infection;
  • Nota Bene: Subjects with diagnosed human immunodeficiency virus (HIV) are eligible to participate in the study if they meet the following criteria :
  • No history of acquired immunodeficiency syndrome (AIDS)-defining opportunistic infection within the past 12 months prior to enrolment;
  • No history of AIDS-defining cancers (e.g. Kaposi's sarcoma, aggressive B-cell lymphoma and invasive cervical cancer);
  • Subjects should be on established anti-retroviral therapy for at least 4 weeks and have an HIV viral load of < 400 copies/mL prior to enrolment;
  • E8. Active or chronic hepatitis B or C requiring treatment with antiviral therapy; Nota Bene: Subjects with a history of hepatitis B or C who have normal alanine aminotransferase (ALT) and are hepatitis B surface antigen negative and/or have undetectable HCV RNA are eligible
  • E9. Dehydration according to NCI-CTCAE v5 Grade >1;
  • E10. Difficulties to swallow oral medication and/or any mal-absorption condition and/or any Gastrointestinal (GI) disease that may significantly alter the absorption of regorafenib (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhoea, malabsorption syndrome, or small bowel resection);
  • E11. Patients with seizure disorder requiring medication;
  • E12. Concurrent enrolment in another clinical trial in which investigational therapies are administered;
  • E13. Known hypersensitivity to the active substance or to any of the excipients;
  • E14. Pregnant women, women who are likely to become pregnant or are breast-feeding. Women of childbearing potential must have a negative serum β-Human Chorionic Gonadotropin (HCG) pregnancy test within 7 days prior randomization;
  • E15. Patients with any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial;
  • E16. Patients with history of non-compliance to medical regimens or unwilling or unable to comply with the protocol;
  • E17. Interstitial lung disease with ongoing signs and symptoms at the time of informed consent;
  • E18. Non-healing wound, non-healing ulcer, or non-healing bone fracture;
  • E19. Patients with evidence or history of any bleeding diathesis, irrespective of severity;
  • E20. Any haemorrhage or bleeding event ≥ CTCAE v5 Grade 3 within 4 weeks prior to the first study drug administration;
  • E21. Clinically significant unrelated systemic illness (e.g., serious infection or significant cardiac, pulmonary, hepatic, or other organ dysfunction) that would compromise the patient's ability to tolerate study treatment or would likely interfere with study procedures or results;
  • E22. Patients using prohibited concomitant and/or concurrent medications (see section "Prohibited concomitant/concurrent treatments);
  • E23. Patients under tutorship or curatorship.

Trial locations and contacts

16 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Julien GAUTIER contact

+33(0)426556829

julien.gautier@lyon.unicancer.fr

16 sites in 1 country

France16 sites, 16 recruiting

  • Chu Besancon

    Besançon, 25030

    Loïc CHAIGNEAU, +33(0)3 81 66 81 66, lchaigneau@chu-besancon.fr

    Not yet recruiting

  • Institut Bergonie

    Bordeaux, 33076

    Maud TOULMONDE, + 33 (0)5 56 33 34 48, m.toulmonde@bordeaux.unicancer.fr

    Recruiting

  • Centre Georges Francois Leclerc

    Dijon, 21079

    Alice HERVIEUX, +33 (0)3 80 73 75 06, ahervieu@cgfl.fr

    Not yet recruiting

  • Centre Oscar Lambret

    Lille, 59020

    Cyril LERVAT, +33 (0)3 20 29 59 59, c-lervat@o-lambret.fr

    Not yet recruiting

  • Centre Leon Berard

    Lyon, 69373

    Mehdi BRAHMI, +33(0)4 78 78 59 73, mehdi.brahmi@lyon.unicancer.fr

    Recruiting

  • Hopital de La Timone

    Marseille, 13385

    +33(0)4 91 38 57 08, florence.duffaud@ap-hm.fr

    Recruiting

  • Icm Val D'Aurelle

    Montpellier, 34298

    Nelly FIRMIN, +33(0)4 67 61 45 65, nelly.firmin@icm.unicancer.fr

    Not yet recruiting

  • Hotel Dieu Nantes

    Nantes, 44093

    Morgane CLEIREC, +33 (0)2 40 08 36 10, morgane.cleirec@chu-nantes.fr

    Not yet recruiting

  • Institut Curie

    Paris, 75005

    Sophie PIPERNO-NEUMANN, +33 (0)1 44 32 46 72, sophie.piperno-neumann@curie.fr

    Recruiting

  • Hôpital COCHIN

    Paris, 75014

    Pascaline BOUDOU-ROUQUETTE, +33(0)1 58 41 14 39, pascaline.boudou@aphp.fr

    Recruiting

  • Ico Rene Gauducheau

    Saint-Herblain, 44805

    Emmanuelle BOMPAS, +33(0)2 40 67 99 39, emmanuelle.bompas@ico.unicancer.fr

    Recruiting

  • Chu Saint-Etienne

    Saint-Priest-en-Jarez, 42270

    Faustine TARDY, +33(0)4 77 82 91 26, faustine.tardy@chu-st-etienne.fr

    Not yet recruiting

  • CHRU Hôpital Hautepierre

    Strasbourg, 67098

    Natacha ENTZ WERLE, +33(0)3 88 12 83 89, natacha.entz-werle@chru-strasbourg.fr

    Recruiting

  • ICANS

    Strasbourg, 67200

    Justine GANTZER, +33(0)3 68 76 66 66, j.gantzer@icans.eu

    Not yet recruiting

  • Iuct Oncopole

    Toulouse, 31059

    Thibaud VALENTIN, +33(0)5 31 15 51 70, valentin.thibaud@iuct-oncopole.fr

    Recruiting

  • Institut Gustave Roussy

    Villejuif, 94805

    Nathalie GASPAR, +33(0)1 42 11 43 16, nathalie.gaspar@gustaveroussy.fr

    Not yet recruiting

Other trials in France

About this study

Official title: A Multicentre Phase II Study of Efficacy of Regorafenib as Maintenance Treatment in Patients With High Grade Bone Sarcomas at Diagnosis or Relapse and Without Complete Remission After Standard Treatment

This is a multicenter phase II study concerning patients with high-grade bone sarcoma (HGBS) without complete remission after standard treatment at diagnosis or at relapse.

Patients will be treated with regorafenib + best supportive care (BSC) for a maximum of 12 months as maintenance therapy after standard line therapy completion.

Progression free rate (PFR) data will be collected and analysed for all included patients to evaluate if regorafenib + BSC can be considered as an interesting treatment for further investigations in this indication.

Read the detailed description

This is a multicenter phase II study.

Patients with evaluable unresectable residual disease will be accrued after they completed standard of care, consisting of:

  • At diagnosis: standard multimodal treatment based on histological subtype
  • At relapse: chemotherapy

Patients who meet the eligibility criteria will receive regorafenib + best supportive care (BSC) as maintenance treatment for a maximum 12 months period.

After their eligibility has been confirmed, patients will receive regorafenib until disease progression, or for a maximum of 12 months, or unacceptable toxicity or willingness to stop, whichever occurs first.

After the completion of the maintenance therapy (12 months) patients will be followed-up until the first radiological disease progression, unless a premature disease progression occurred. All patients will be followed-up until the data cut-off (12 months after the last inclusion).

The vital status will be updated once for all patients at 24 months after the last inclusion, based on patient's medical file.

Being tested
Treatment by regorafenib and best supportive care
Keywords
Bone sarcoma, Osteosarcoma, Maintenance therapy, Regorafenib, Progression-free rate, Objective response rate, Disease control rate, Overall survival, Quality of life, Tolerance, Tyrosine inhibitor kinase, Multitarget inhibitor, High-grade, Progression-Free survival
Medical subject headings
Bone Neoplasms, Osteosarcoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Treatment by regorafenib and best supportive careDrug

    Also called Experimental arm

    Treatment for 13 cycles (12 months) maximum.

    During each cycle :

    • patients ≥ 16 years old and patients <16 old with Body Surface Area (BSA) ≥ 1,70 m2 will take 3 tablets, once a day, corresponding to a total of 120 mg Regorafenib, during 21 days, followed by 7 days without treatment.
    • patients < 16 years old with a 1,30 m2 ≤ BSA ≥ 1,70 m2 will take 2 tablets, once a day, corresponding to a total of 80 mg Regorafenib, during 21 days, followed by 7 days without treatment
Study groups in NCT04698785
Study groupRoleWhat they receive
Regorafenib and best supportive careExperimentalTreatment will be divided in 28 days cycles, including a 21-day period of treatment by regorafenib and best supportive care followed by a 7-day period of rest. In case of toxicity, dose can be reduced or treatment interrupted according to Specific Product Characteristics (SPC). Patients can receive up to a maximum of 13 cycles (maximum treatment period : 12 Months).
Receives: Drug: Treatment by regorafenib and best supportive care

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Progression-Free Rate at 4 months (4m-PFR)

    The Progression-Free Rate at 4 months (4m-PFR) will be defined as the proportion of patients with a complete response, a partial response or stable disease, confirmed on tumour assessments according to the RECIST V1.1 at 4 months (16 weeks) after the date of regorafenib + BSC treatment initiation.

    Patients with a non-progressive disease at 16 ± 2 weeks after the date of regorafenib + BSC treatment initiation will be considered as "success" patients.

    Patients with a progressive disease and patients who died from any cause at 16 ± 2 weeks after the date of regorafenib + BSC treatment initiation will be considered as "failure" patients.

    At the time of analysis, if at least 18 "successes" are observed among the 36 analyzed patients, the regorafenib + BSC treatment will be considered as interesting for further investigations in this indication.

    Measured: Up to 16 weeks after the date of regorafenib + BSC treatment initiation

Secondary outcomes (7)

  1. Progression-Free Survival (PFS)

    PFS will be described from date of regorafenib + BSC treatment initiation to the date first documented disease progression according to RECIST V1.1 or the date of death due to any cause.

    Patients alive without progression at the time of data cut-off will be censored at the time of latest tumour evaluation.

    Measured: Up to 3 years

  2. Objective Response Rate (ORR)

    The Objective Response Rate (ORR) will be defined as the proportion of patients with a best overall response of Complete Response (CR) or Partial Response (PR) (RECIST V1.1)

    Measured: Up to 3 years

  3. Disease Control Rate (DCR) at 2 months

    The Disease Control Rate (DCR) will be defined as the proportion of patients with a best overall response of CR, PR or Stable Disease (SD) (RECIST V1.1) 2 months after randomization

    Measured: 2 months

  4. Disease Control Rate (DCR) at 4 months

    The Disease Control Rate (DCR) will be defined as the proportion of patients with a best overall response of CR, PR or Stable Disease (SD) (RECIST V1.1) 4 months after randomization

    Measured: 4 months

  5. Overall Survival (OS)

    The Overall Survival (OS) will be defined as the time from date of regorafenib +BSC treatment initiation to the date of death, from any cause. Patient not known to have died at the time of analysis will be censored based on the last recorded date on which the patient was known to be alive.

    Measured: Up to 3 years

  6. Quality of Life (QoL)

    The patient's Quality of Life (QoL) will be assessed using the EORTC Quality Of Life Questionnaire (QLQ)-C30 file. Scores will be calculated at each time point according to the scoring manuals. Descriptive statistics will be used to evaluate baseline scores and evolution of scores from baseline to each time point. Data will be compared between arms using the Student's t-test. The QoL data will also be presented graphically if deemed relevant.

    Measured: Up to 1 year

  7. Tolerance profile of treatment

    The safety will be described mainly on the frequency of adverse events (AE) coded using the common toxicity criteria (NCI-CTCAE v5.0) grade. Descriptive statistics will be provided for characterizing and assessing patient tolerance to treatment. AE will be coded according to the MedDRA®.

    Measured: Up to 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Jan 2021
  2. Study start21 Jul 2021
  3. Primary completion21 Jul 2026 (estimated)
  4. Study completion21 Jul 2026 (estimated)

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
36 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04698785
Sponsor's protocol ID
ET19000144 (REGOMAIN)
Acronym
REGOMAIN
Status last verified
Feb 2026
First submitted
5 Jan 2021
Last updated
17 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study of Bone Sarcoma and Osteosarcoma, run by Centre Leon Berard at 16 sites in France.

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 19 inclusion and 34 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: looking for participants now, as of the sponsor's last update on 17 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in France. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Julien GAUTIER (+33(0)426556829, julien.gautier@lyon.unicancer.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression-Free Rate at 4 months (4m-PFR) (time frame: Up to 16 weeks after the date of regorafenib + BSC treatment initiation). 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 NCT04698785.

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 17 Feb 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in