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Ewing SarcomasPhase 2REGOBONENCT02389244

A Phase II Study Evaluating Efficacy and Safety of Regorafenib in Patients With Metastatic Bone Sarcomas

A Phase 2 study testing Regorafenib in people with Ewing Sarcomas and Chondrosarcomas, run by UNICANCER at 19 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

INDICATION: Metastatic bone sarcomas: conventional high grade osteosarcoma, Ewing sarcoma of bone, intermediate or high-grade chondrosarcomas and chordomas and either bone or soft tissue metastatic CIC-rearranged sarcomas Read more about this study

Who is it for?
Ages 10 years and older
Conditions the study is for
Ewing Sarcomas, Chondrosarcomas, Osteosarcomas
What is being tested?
Regorafenib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
163 participants
Where
19 sitesFrance
Study dates
Sep 2014to 3 Sep 2025
Phase
Phase 2
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 10 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patients must have histologically confirmed diagnosis of bone sarcoma (osteosarcoma, Ewing sarcoma of bone, chondrosarcoma or chordoma);
  • Patients with confirmed disease progression at study entry;
  • Metastatic disease not amenable to surgical resection or radiation with curative intent;
  • Patients must have measurable disease;
  • Prior treatment :
  • at least one, but no more than two prior chemotherapy regimen for metastatic disease for osteosarcoma, chondrosarcoma and Ewing sarcoma; neo-adjuvant /maintenance therapy are not counted towards this requirement. Chordoma not pretreated or with 1 or 2 prior (combination) chemotherapy regimen or with one or two prior molecularly targeted therapy, but no more than 2 prior lines of treatment (whatever the indication) can be included. At least 4 weeks since last chemotherapy (6 weeks in case of nitrosoureas and mitomycin C), immunotherapy or any other pharmacological treatment and/or radiotherapy;
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  • Age ≥10 years for osteosarcomas, Ewing sarcomas and chondrosarcomas (for chordomas, patients must be ≥18 years);
  • Body Surface Area ≥1.30 m²;
  • Life expectancy of greater than 3 months;
  • Eastern Cooperative Oncology Group (ECOG) performance status <2 (Karnofsky ≥60%) for adults patients;
  • Karnofsky scale ≥ 60% for children aged >12 years old / Lansky scale ≥60% for children aged ≤12 years old;
  • Patients must have adequate bone marrow, renal, and hepatic function, as evidenced by the following within 7 days of study treatment initiation: normal organ function as defined below:
  • Absolute neutrophil count ≥1.5 Giga/L
  • Platelets ≥100 Giga/L
  • Hemoglobin ≥9 g/dL
  • Serum creatinin ≤1.5 x upper limit of normal (ULN)
  • Glomerular filtration rate (GFR) ≥30 ml/min/1.73 m² according to the modified Diet in Renal Disease (MDRD) abbreviated formula
  • Aspartate transaminase (AST) and alanine transaminase (ALT) ≤2.5 x ULN
  • Bilirubin ≤1.5 X ULN
  • 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-glutamyl transferase (GGT) tests must be performed; hepatic isoenzymes 5-nucleotidase must be within the normal range and/or GGT <1.5 x ULN;
  • lipase ≤1.5 x ULN;
  • Spot urine must not show 1+ or more 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
  • International Normalized Ratio(INR)/ Partial Thromboplastin Time (PTT) ≤1.5 x ULN;
  • Recovery to National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) v4.0 Grade 0 or 1 level or recovery to baseline preceding the prior treatment from any previous drug/procedure related toxicity (except alopecia, anemia, and hypothyroidism);
  • Women of childbearing potential and male patients must agree to use adequate contraception for the duration of study participation and up to 3 months following completion of therapy;
  • Women of childbearing potential must have a negative serum β-HCG pregnancy test within 7 days prior randomization and/or urine pregnancy test within 48 hours before the first administration of the study treatment;
  • Signed informed consent form by adult patients and/or patients parents/legal representatives (if age <18 years) and age appropriate assent form by the patients' parents/legal representatives obtained before any study specific procedure is conducted;
  • Patients must be willing and able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures;
  • Patients or parents/legal representatives affiliated to the Social Security System.

Who cannot join

  • Prior treatment with any VEGFR inhibitor;
  • Soft tissue sarcoma;
  • Other cancer (different histology) within 5 years prior to randomization;
  • Major surgical procedure, open biopsy, significant trauma, within the last 28 days before randomization;
  • Cardiovascular dysfunction:
  • Left ventricular ejection fraction (LVEF) <50%
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  • Congestive heart failure (New York Heart Association [NYHA]) ≥2
  • Myocardial infarction <6 months before study
  • Cardiac arrhythmias requiring therapy
  • Uncontrolled hypertension
  • Unstable angina or new-onset angina
  • 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 randomization;
  • Severe hepatic impairment (Child-Pugh C);
  • Ongoing infection > Grade 2 according to NCI-CTCAE v4.0;
  • Known history of human immunodeficiency virus (HIV) infection;
  • Active hepatitis B or C or chronic hepatitis B or C requiring treatment with antiviral therapy;
  • Difficulties with swallowing study tablets;
  • Prior anticancer therapy, including radiotherapy, endocrine therapy, immunotherapy, chemotherapy (CT) within the last 4 weeks (6 weeks for nitrosoureas and mitomycin C), or other investigational agents ; Concomitant antalgic palliative radiotherapy allowed;
  • Concurrent enrolment in another clinical trial in which investigational therapies are administered;
  • Known hypersensitivity to the active substance or to any of the excipients;
  • Pregnant women, women who are likely to become pregnant or are breast-feeding;
  • For adult patients, individual deprived of liberty or placed under the authority of a tutor;
  • 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;
  • Patients with history of non compliance to medical regimens or unwilling or unable to comply with the protocol;
  • Interstitial lung disease with ongoing signs and symptoms at the time of informed consent;
  • Non-healing wound, non-healing ulcer, or non-healing bone fracture;
  • Patients with evidence or history of any bleeding diathesis, irrespective of severity;
  • Any hemorrhage or bleeding event ≥ CTCAE Grade 3 within 4 weeks prior to the start of study medication;
  • Use of biological response modifiers, such as granulocyte colony stimulating factor (G-CSF), within 3 weeks of study entry.

Trial locations and contacts

19 sites in 1 country.

19 sites in 1 country

France19 sites

  • Hopital Jean Monjoz

    Besançon, 25030

  • Institut Bergonie

    Bordeaux, 33076

  • Centre Francois Baclesse

    Caen, 14176

  • Centre Georges Francois Leclerc

    Dijon, 21079

  • Centre Oscar Lambret

    Lille, 59020

  • Centre Léon Berard

    Lyon, 69373

  • Institut Paoli Calmettes

    Marseille, 13273

  • La Timone University Hospital

    Marseille, 13385

  • ICM Val d'Aurelle

    Montpellier, 34298

  • Centre Antoine Lacassagne

    Nice, 06189

  • Hôpital Cochin

    Paris, 75014

  • Institut Curie

    Paris

  • Centre Eugene Marquis

    Rennes, 35042

  • Institut de cancerologie de l'ouest site Rene Gauducheau

    Saint-Herblain, 44805

  • Institut de Cancérologie Lucien Neuwirth (ICLN)

    Saint-Priest-en-Jarez, 42270

  • Institut Claudius Regaud

    Toulouse, 31052

  • CHU Bretonneau

    Tours, 37000

  • Institut de cancerologie de lorraine alexis Vautrin

    Vandœuvre-lès-Nancy, 54519

  • Gustave Roussy

    Villejuif, 94800

Other trials in France

About this study

Official title: A Randomized Phase II, Placebo-controlled, Multicenter Study Evaluating Efficacy and Safety of Regorafenib in Patients With Metastatic Bone Sarcomas

INDICATION:

Metastatic bone sarcomas: conventional high grade osteosarcoma, Ewing sarcoma of bone, intermediate or high-grade chondrosarcomas and chordomas and either bone or soft tissue metastatic CIC-rearranged sarcomas

Read the detailed description

METHODOLOGY:

Randomized, placebo-controlled, multicentric, phase II study -This is a double-blind placebo-controlled trial, with 5 cohorts: cohort A: Osteosarcoma, cohort B: Ewing sarcoma, cohort C: Chondrosarcoma, cohort D : chondroma, cohort E: CIC-rearranged sarcoma. Cohort A, B and C will involve a total of 36 patients (24 Regorafenib + 12 placebo), cohort D a total of 24 evaluable patients (16 Regorafenib + 8 placebo) and cohort E will involve a total of 27 evaluable patients (18 Regorafenib + 9 placebo).

159 patients who meet the eligibility criteria will be randomly assigned in a 2:1 ratio to the following treatment groups :

The Arm A:

Regorafenib (160 mg/d) once daily for the 3 weeks on / 1 week off plus Best Supportive Care (BSC) until progression (according to RECIST 1.1), intolerance or withdrawal of consent .

Patients receiving regorafenib who experience disease progression and for whom in the investigator opinion, treatment with regorafenib is providing clinical benefit, may continue the treatment following consultation with the study coordinator and the sponsor.

The Arm B:

Placebo plus BSC until progression (according to RECIST V1.1) intolerance or withdrawal of consent. Patients who have received placebo will receive open-label regorafenib after objective tumor progression.

Patients will be stratified at randomization according to histology .

Being tested
Regorafenib, Placebo
Medical subject headings
Chondrosarcoma, Osteosarcoma, Chondroma

What participants receive

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

  • RegorafenibDrug

    Also called Stivarga

    For adults patients and children with BSA ≥1.70 m² : 4 tablets once daily until progression or unacceptable toxicity For children with BSA ≥1.30 and ≤1.69 m² : 3 tablets once daily until progression or unacceptable toxicity

  • PlaceboDrug

    Also called Placebo tablet

    For adults patients and children with BSA ≥1.70 m² : 4 tablets once daily and switch to regorafenib after confirmed progression For children with BSA ≥1.30 and ≤1.69 m² : 3 tablets once daily and switch to regorafenib after confirmed progression

Study groups in NCT02389244
Study groupRoleWhat they receive
RegorafenibExperimentalFor adult patients (≥18 years old) : 160 mg/d once daily for the 3 weeks on / 1 week off plus Best Supportive Care (BSC) until progression (according to RECIST 1.1), intolerance or withdrawal of consent . For children Age ≥10 years to <18 years old and BSA ≥1.30 m², regorafenib (82 mg/m²) once daily for the 3 weeks on/1 week off (without exceeding 160 mg/day) plus Best Supportive care (BSC) until progression (according to RECIST 1.1), intolerance or withdrawal of consent.
Receives: Drug: Regorafenib
placeboPlacebo comparatorPlacebo plus BCS until progression (according to RECIST V1.1) intolerance or withdrawal of consent. Patients who have received placebo will receive open-label regorafenib after objective tumor progression.
Receives: Drug: Placebo

What is being measured

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

  1. Non-progression rate

    Proportion of patients without disease progression at the defined timepoint after central radiological review (using RECIST 1.1)

    Measured: 8 weeks for cohorts A, B and E, 12 weeks for cohort C, 6 months for cohort D

Secondary outcomes (11)

  1. Progression Free Survival

    from the date of randomization until the date of radiological progression or death whatever the cause (if death occurs before progression)

    Measured: expected average duration of 3 months

  2. Objective response rate

    complete response (CR) or partial response (PR) according to RECIST 2009, version 1.1, for all cohorts, and CHOI criteria for chordoma

    Measured: 6 months

  3. Disease control rate at 6 months

    from the date of randomization until the date of death due to any cause

    Measured: 6 months

  4. Overall survival

    from the date of randomization until the date of death due to any cause

    Measured: 2 years

  5. Duration of response

    objective response of CR or PR, whichever is noted earlier, to first disease progression or death before progression

    Measured: expected average duration of 6 months

  6. Progression-free rate at 3 and 6 months

    the proportion of patients without progression at 3 and 6 months post randomization

    Measured: at 3 and 6 months

  7. Time to progression

    from date of randomization until the date of first observation of progression

    Measured: from date of randomization until the date of first observation of progression (up to 6 months)

  8. Growth Modulation Index defined as ratio of time to progressive disease (PD) under regorafenib to time to progression (TTP) under previous treatment

    ratio of time to PD under regorafenib to TTP under previous treatment

    Measured: expected average duration of 3 months

  9. Toxicity according to NCI-CTCAE V4-0

    according to NCI-CTCAE V4-0 (National Cancer Institut Common Terminology Criteria for Adverse Events)

    Measured: expected average duration of 6 months

  10. Pain assessment using Visual analog scale (VAS), DN4 scale (Neuropathic Pain Diagnostic Questionnaire) and NPSI scale (Neuropathic Pain Symptom Inventory)

    for chordomas cohort only

    Measured: expected average duration of 6 months

  11. PFS

    Progression Free Survival according to Choi criteria for Chordoma

    Measured: from date of randomization until the date of first observation of progression (up to 6 months)

Timeline

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

  1. First posted17 Mar 2015 (estimated)
  2. Study startSep 2014
  3. Primary completion25 Oct 2024
  4. Study completion3 Sep 2025

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
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
163 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02389244
Sponsor's protocol ID
UC-0150/1309
Other IDs
2013-003910-42
Acronym
REGOBONE
Status last verified
Sep 2025
First submitted
9 Feb 2015
Last updated
21 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: no individual participant data is shared

Publications

  • Duffaud F, Italiano A, Bompas E, Rios M, Penel N, Mir O, Piperno-Neumann S, Chevreau C, Delcambre C, Bertucci F, Boudou-Rouquette P, Cancel M, Perrin C, Saada-Bouzid E, Monard L, Schiffler C, Chaigneau L, Hervieu A, Collard O, Bouvier C, Vidal V, Chabaud S, Blay JY; French Sarcoma Group. Efficacy and safety of regorafenib in patients with metastatic or locally advanced chondrosarcoma: Results of a non-comparative, randomised, double-blind, placebo controlled, multicentre phase II study. Eur J Cancer. 2021 Jun;150:108-118. doi: 10.1016/j.ejca.2021.03.039. Epub 2021 Apr 22.

    PubMed 33895682

  • Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14.

    PubMed 31401903

  • Duffaud F, Mir O, Boudou-Rouquette P, Piperno-Neumann S, Penel N, Bompas E, Delcambre C, Kalbacher E, Italiano A, Collard O, Chevreau C, Saada E, Isambert N, Delaye J, Schiffler C, Bouvier C, Vidal V, Chabaud S, Blay JY; French Sarcoma Group. Efficacy and safety of regorafenib in adult patients with metastatic osteosarcoma: a non-comparative, randomised, double-blind, placebo-controlled, phase 2 study. Lancet Oncol. 2019 Jan;20(1):120-133. doi: 10.1016/S1470-2045(18)30742-3. Epub 2018 Nov 23.

    PubMed 30477937

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Regorafenib in people with Ewing Sarcomas and Chondrosarcomas, run by UNICANCER at 19 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 29 inclusion and 29 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 21 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 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. Some participants in this study may receive a placebo. 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?

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?

Non-progression rate (time frame: 8 weeks for cohorts A, B and E, 12 weeks for cohort C, 6 months for cohort D). 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 NCT02389244, which also lists 3 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 21 Jul 2026.

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