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AzTrials

Pediatric CancerPhase 1/2ESMARTNCT02813135

European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors

A Phase 1/2 study testing Ribociclib, Topotecan and Temozolomide in people with Pediatric Cancer, run by Gustave Roussy, Cancer Campus, Grand Paris at 23 sites in 6 countries.

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 proof-of-concept platform trial is designed to cover the targeting of several survival pathways in oncogenesis that are currently not adequately employed for pediatric patients in Europe (Geoerger 2017; Geoerger 2019). The aims of the trial are: 1. Read more about this study

Who is it for?
Ages Up to 18 years
Conditions the study is for
Pediatric Cancer
What is being tested?
Ribociclib, Topotecan and Temozolomide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
472 participants
Where
23 sitesin 6 countries
Study dates
3 Aug 2016to Feb 2031
Phase
Phase 1/2
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 Up to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Patients must be diagnosed with a haematologic or solid tumor malignancy that has progressed despite standard therapy, or for which no effective standard therapy exists.
  • Age < 18 years at inclusion; patients 18 years and older may be included after discussion with the sponsor if they have a pediatric recurrent/refractory malignancy.
  • Patient must have had advanced molecular profiling (i.e. WES/WGS +/- RNAseq) of their recurrent or refractory tumor i.e. at the time of disease progression/relapse; exceptionally patients with advanced molecular profiling at diagnosis may be allowed.
  • Evaluable or measurable disease as defined by standard imaging criteria for the patient's tumor type (RECIST v1.1, RANO criteria for patients with HGG, INRC criteria for patients with NB, Leukemia criteria, etc.).
  • Patients with relapsed or refractory leukemia are eligible for this study.
  • Performance status: Karnofsky performance status (for patients >12 years of age) or Lansky Play score (for patients ≤12 years of age) ≥ 70%. Patients who are unable to walk because of paralysis or stable neurological disability, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
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  • Life expectancy ≥ 3 months
  • Adequate organ function:
  • Hematologic criteria (Leukemia patients are excluded from hematological criteria):
  • Peripheral absolute neutrophil count (ANC) ≥ 1000/μL(unsupported)
  • Platelet count ≥ 100,000/μL (unsupported)
  • Hemoglobin ≥ 8.0 g/dL (transfusion is allowed)
  • Cardiac function:
  • Shortening fraction (SF) >29% (>35% for children < 3 years) and left ventricular ejection fraction (LVEF) ≥50% at baseline, as determined by echocardiography (mandatory only for patients who have received cardiotoxic therapy).
  • Absence of QTc prolongation (QTc > 450 msec on baseline ECG, using the Fridericia correction [QTcF formula]) or other clinically significant ventricular or atrial arrhythmia.
  • Renal and hepatic function:
  • Serum creatinine ≤ 1.5 x upper limit of normal (ULN) for age
  • Total bilirubin ≤ 1.5 x ULN
  • Alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase (SGPT) ≤ 2,5 x ULN; aspartate aminotransferase (AST)/serum glutamic oxaloacetic transaminase/SGOT ≤ 2,5 x ULN except in patients with documented tumor involvement of the liver who must have AST/SGOT and ALT/SGPT ≤ 5 x ULN.
  • Able to comply with scheduled follow-up and with management of toxicity.
  • Females of childbearing potential must have a negative serum or urine pregnancy test within 72 hours prior to initiation of treatment. Sexually active women of childbearing potential must agree to use acceptable and appropriate contraception during the study and for at least 6 months after the last study treatment administration. Sexually active males patients must agree to use condom during the study and for at least 6 months (7 months for arm J) after the last study treatment administration. Acceptable contraception are defined in CTFG Guidelines "Recommendations related to contraception and pregnancy testing in clinical trials"
  • For all oral medications patients must be able to comfortably swallow capsules (except for those for which an oral solution is available); nasogastric or gastrostomy feeding tube administration is allowed only if indicated.
  • Written informed consent from parents/legal representative, patient, and age-appropriate assent before any study-specific screening procedures are conducted according to local, regional or national guidelines.
  • Patient affiliated to a social security regimen or beneficiary of the same according to local requirements.

Who cannot join

  • Patients with symptomatic central nervous system (CNS) metastases who are neurologically unstable or require increasing doses of corticosteroids or local CNS-directed therapy to control their CNS disease. Patients on stable doses of corticosteroids for at least 7 days prior to receiving study drug may be included.
  • Impairment of gastrointestinal (GI) function or GI disease that may significantly alter drug absorption of oral drugs (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, or malabsorption syndrome).
  • Clinically significant, uncontrolled heart disease (including history of any cardiac arrhythmias, e.g., ventricular, supraventricular, nodal arrhythmias, or conduction abnormality, unstable ischemia,congestive heart failure within 12 months of screening)
  • Active viral hepatitis or known human immunodeficiency virus (HIV) infection or any other uncontrolled infection.
  • Presence of any ≥ CTCAE grade 2 treatment-related toxicity with the exception of alopecia, ototoxicity or peripheral neuropathy.
  • Systemic anticancer therapy within 21 days of the first study dose or 5 times its half-life, whichever is less.
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  • Previous myeloablative therapy with autologous hematopoietic stem cell rescue within 8 weeks of the first study drug dose
  • Allogeneic stem cell transplant within 3 months prior to the first study drug dose. Patients receiving any agent to treat or prevent graft-versus host disease (GVHD) post bone marrow transplant are not eligible for this trial.
  • Radiotherapy (non-palliative) within 21 days prior to the first dose of drug (or within 6 weeks for therapeutic doses of MIBG or craniospinal irradiation).
  • Major surgery within 21 days of the first dose. Gastrostomy, ventriculo-peritoneal shunt, endoscopic ventriculostomy, tumor biopsy and insertion of central venous access devices are not considered major surgery, but for these procedures, a 48 hour interval must be maintained before the first dose of the investigational drug is administered.
  • Currently taking medications with a known risk of prolonging the QT interval or inducing Torsades de Pointes (Refer to Appendix 8).
  • Currently taking medications that are mainly metabolized by CYP3A4/5, CYP2C8, CYP2C9, CYP2C19, CYP2D6 or the drug transporters Pgp (MDR1), BCRP, OATP1B1, OATP1B3, OCT1 and OCT2 and have a low therapeutic index that cannot be discontinued at least 7 days or 5 x reported elimination half-life prior to start of treatment with any of the investigational drugs and for the duration of the study (Refer to Appendix 9).
  • Known hypersensitivity to any study drug or component of the formulation.
  • Pregnant or nursing (lactating) females.
  • Vaccinated with live, attenuated vaccines within 4 weeks of the first dose of study drug.

Trial locations and contacts

23 sites in 6 countries. Contact the study team or a site near you to ask about taking part.

Birgit Geoerger, MD contact

+33 (0)1 42 11 46 61

birgit.geoerger@gustaveroussy.fr

Estelle Jullemier, MS contact

+33 (0)1 42 11 55 51

Estelle.JULLEMIER@gustaveroussy.fr

23 sites in 6 countries

France10 sites, 10 recruiting

  • CHU Angers

    Angers, 49933

    Claire Brisset

    Recruiting

  • CHU Pellegrin

    Bordeaux, 33076

    Stephane Ducassou

    Recruiting

  • Centre Oscar Lambret

    Lille, 59020

    Sandra Raimbault

    Recruiting

  • Centre Léon Bérard

    Lyon, 69373

    Nadège Corradini

    Recruiting

  • Hôpital de La Timone

    Marseille, 13385

    Nicolas André

    Recruiting

  • CHU Nantes

    Nantes, 44093

    Morgane Cleirec

    Recruiting

  • Institut Curie

    Paris, 75005

    Isabelle Aerts

    Recruiting

  • Hôpital Armand Trousseau

    Paris, 75012

    Arnaud Petit

    Recruiting

  • Hôpital de Hautepierre

    Strasbourg, 67098

    Natacha Entz-Werle

    Recruiting

  • Gustave Roussy

    Villejuif, Val de Marne, 94805

    Pablo Berlanga, MD

    Recruiting

Other trials in France

United Kingdom5 sites, 5 recruiting

  • Birmingham Children's Hospital

    Birmingham, B4 6NH

    Gerard Millen

    Recruiting

  • Great Ormond Street Hospital

    London, WC1N 3JH

    Giuseppe Barone

    Recruiting

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL

    Guy Makin

    Recruiting

  • Royal Victoria Infirmary

    Newcastle, NE1 4LP

    Quentin Campbell-Hewson

    Recruiting

  • Pediatric and Adolescent Oncology The Royal Marsden Hospital

    Sutton

    Lynley Marshall, MD, +44 208 661 3678, Lynley.Marshall@icr.ac.uk

    Recruiting

Other trials in United Kingdom

Italy3 sites, 3 recruiting

  • Istituto Giannina Gaslini

    Genova, 16147

    Teresa Battaglia

    Recruiting

  • Fondazione IRCCS Istituto Nazionale dei Tumori

    Milan

    Michela Casanova, MD, +39 02 23 90 25 94, Michela.Casanova@istitutotumori.mi.it

    Recruiting

  • Ospedale Infantile Regina Margherita

    Torino, 10126

    Franca Fagioli

    Recruiting

Other trials in Italy

Spain3 sites, 3 recruiting

  • Vall d'Hebron

    Barcelona, 08035

    Raquel Hladun

    Recruiting

  • Hospital del Nino Jesus

    Madrid, 28009

    Alba Rubio

    Recruiting

  • Hospital Universitario La Fe

    Valencia, 46026

    Adela Cañete

    Recruiting

Other trials in Spain

Denmark1 site, 1 recruiting

  • Rigshospitalet

    Copenhagen, 2100

    Karsten Nyson, MD, +45 35 45 08 09, karsten.nysom@regionh.dk

    Recruiting

Other trials in Denmark

Netherlands1 site

  • Prinses Maxima Centrum

    Utrecht, 3584 EA

    Active, not recruiting

Other trials in Netherlands

About this study

This proof-of-concept platform trial is designed to cover the targeting of several survival pathways in oncogenesis that are currently not adequately employed for pediatric patients in Europe (Geoerger 2017; Geoerger 2019).

The aims of the trial are:

  1. To determine the recommended phase II dose (RP2D) of a specific anticancer agent and/or a relevant combination in a pediatric population, to document its tolerability and
  2. To explore first signals of activity in a molecularly enriched study population.
Read the detailed description

The first molecular profiling protocols have been launched in Europe (MOSCATO-01 (Geoerger 2014), MAPPYACTS, INFORM, iTHER, SM-PAEDS, etc.) determining multiple actionable alterations in pediatric recurrent cancers. Increasingly, stratified approaches are being implemented to enrich clinical trials of molecularly targeted agents and possibly improve outcomes in specific populations i.e. a molecularly enriched/predictive biomarker-driven approach. The diversity and heterogeneity of the detected molecular alterations and the low number of pediatric patients mandate an adapted, innovative trial design for the attributed treatment options in order to satisfy the current unmet medical need.

This basket trial is designed to cover the targeting of several survival pathways in oncogenesis that are currently not adequately employed for pediatric patients in Europe.

Being tested
Ribociclib, Topotecan, Temozolomide, Everolimus, Adavosertib
Keywords
Children, Adolescents, Young adults, Recurrent malignancies, Refractory malignancies
Medical subject headings
Neoplasms

What participants receive

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

  • RibociclibDrug

    Also called Kisqali, LEE011

  • TopotecanDrug

    Also called Hycamtin

  • TemozolomideDrug

    Also called Temodar

  • EverolimusDrug

    Also called Afinitor, votubia

  • AdavosertibDrug

    Also called AZD1775

  • CarboplatinDrug

    Also called Paraplatin

  • OlaparibDrug

    Also called Lynparza

  • IrinotecanDrug

    Also called Camptosar, CPT-11

  • VistusertibDrug

    Also called AZD2014

  • NivolumabDrug

    Also called Opdivo

  • CyclophosphamideDrug

    Also called Cytoxan

  • SelumetinibDrug

    Also called Koselugo

  • EnasidenibDrug

    Also called Idhifa

  • LirilumabDrug

    Also called BMS-986015

  • FadraciclibDrug

    Also called CYC065

  • CytarabineDrug

    Also called Arabinosylcytosine, Cytosar-U

  • DexamethasoneDrug

    Also called Decadron, Dexasone, Diodex, Hexadrol

  • CeralasertibDrug

    Also called AZD6738

  • FutibatinibDrug

    Also called Tas-120

  • CapmatinibDrug

    Also called Tabrecta, INC280

  • AvelumabDrug

    Also called BAVENCIO

  • PeposertibDrug

  • CapivasertibDrug

    Also called TRUQAP, AZD5363

  • VinorelbineDrug

    Also called Navelbine

Study groups in NCT02813135
Study groupRoleWhat they receive
Arm A. Ribociclib + Topotecan and TemozolomideExperimentalTopotecan iv QD and temozolomide capsules orally QD Days 1 to 5; Ribociclib capsules or oral solution orally QD from Day 6 to 20 of a 28 day cycle.
Receives: Drug: Ribociclib; Drug: Topotecan; Drug: Temozolomide
Arm B. Ribociclib + EverolimusExperimentalRibociclib capsules or oral solution orally QD for 21 days of each 28 day cycle; Everolimus orodispersible tablets orally QD for 28 days.
Receives: Drug: Ribociclib; Drug: Everolimus
ARM C. Adavosertib + CarboplatinExperimentalAdavosertib capsules orally BID 3 days on / 4 days off in week 1; Carboplatin iv QD AUC 5 on Day 1 of a 21 day cycle.
Receives: Drug: Adavosertib; Drug: Carboplatin
Arm D. Olaparib + IrinotecanExperimentalOlaparib tablets orally BID on Day 1-10 of a 21 day cycle Irinotecan iv QD on Day 4-8 of a 21 day cycle.
Receives: Drug: Olaparib; Drug: Irinotecan
Arm E. Vistusertib single agentExperimentalVistusertib tablets orally BID 2 days on/5 days off per week of a 28 day cycle.
Receives: Drug: Vistusertib
Arm F. Vistusertib + Topotecan and TemozolomideExperimentalTopotecan iv QD and temozolomide capsules orally QD Days 1 to 5; Vistusertib tablets orally BID 3 days on/4 days off per week of a 28 day cycle.
Receives: Drug: Topotecan; Drug: Temozolomide; Drug: Vistusertib
Arm G. Nivolumab + Cyclophosphamide +/- RadiotherapyExperimentalNivolumab iv QD every 2 weeks of a 28 day cycle (Days 1 and 15); Cyclophosphamide tablets or oral solution orally BID, 1 week on/1 week off; Palliative irradiation/radiofrequency/cryotherapy starting 2 weeks after the first nivolumab injection.
Receives: Drug: Nivolumab; Drug: Cyclophosphamide
Arm H. Selumetinib + VistusertibExperimentalSelumetinib capsules twice daily on a continous administration. Vistusertib orally twice daily on an intermittent schedule : 2 days on / 5 days off per week of a 28 day cycle.
Receives: Drug: Vistusertib; Drug: Selumetinib
Arm I. EnasidenibExperimentalEnasidenib tablets or sprinkle solution orally on a continuous dosing once daily (QD) per 28 day cycle.
Receives: Drug: Enasidenib
Arm J. Lirilumab + NivolumabExperimentalNivolumab iv QD on Day 1 and 15 of a 28 day cycle; Lirilumab iv QD on Day 1 of a 28 day cycle
Receives: Drug: Nivolumab; Drug: Lirilumab
Arm K. Fadraciclib (CYC065) + TemozolomideExperimentalFadraciclib iv QD on Day 1 (+/- 15) of a 28 day cycle Temozolomide capsules orally QD on Day 1-5 of a 28 day cycle
Receives: Drug: Temozolomide; Drug: Fadraciclib
Arm L. Fadraciclib (CYC065) + CytarabineExperimentalFadraciclib iv QD on Day 1 (+/- 15) of a 28 day cycle Cytarabine iv or sc on Day 2-5 and Day 8-11 of a 28 day cycle
Receives: Drug: Fadraciclib; Drug: Cytarabine
Arm M. Ribociclib + Everolimus +/- DexamethasoneExperimentalRibociclib capsules or tablets orally QD on Day 1-21 of a 28 day cycle. Everolimus dispersible tablets orally QD on a continuous dosing per 28 day cycle. For patients with leukemia and lymphoma: Dexamethasone orally or iv on Day 1-7 of each 28 day cycle. For patients with ALL, AML and Non-Hodgkin Lymphoma (NHL), Intrathecal chemotherapy will be administered additionally as per standard practice depending on CNS status.
Receives: Drug: Ribociclib; Drug: Everolimus; Drug: Dexamethasone
Arm N. Ceralasertib (AZD6738) + OlaparibExperimentalOlaparib tablets orally BID per 28 days Ceralasertib tablets QD or BID per 28 day cycle
Receives: Drug: Olaparib; Drug: Ceralasertib
Arm O. Futibatinib (TAS-120)ExperimentalFutibatinib tablets orally on a continuous dosing QD per 28 day cycle
Receives: Drug: Futibatinib
Arm P. Capmatinib (INC280) + EverolimusExperimentalCapmatinib tablets orally on a continuous dosing BID per 28 day cycle. Everolimus dispersible tablets orally QD on a continuous dosing per 28 day cycle.
Receives: Drug: Everolimus; Drug: Capmatinib
Arm Q. Peposertib + Avelumab and Metronomic TemozolomideExperimentalPeposertib tablets orally on a continuous dosing BID per 28 day cycle Avelumab IV QD on Day 1 and Day 15 of a 28 day cycle Temozolomide capsules orally QD 5 days/week of a 28 day cycle
Receives: Drug: Temozolomide; Drug: Avelumab; Drug: Peposertib
Arm R. Capivasertib + metronomic VinorelbineExperimentalCapivasertib tablets orally BID on Day 1-4/week (4 days on/3 days off, every week) of a 28 day cycle. Vinorelbine soft capsules orally once daily on Day 1, 3 and 5/week of a 28 day cycle.
Receives: Drug: Capivasertib; Drug: Vinorelbine

What is being measured

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

  1. Recommended phase II dose (RP2D)

    Defined as the adult recommended dose (adjusted for weight or BSA) if toxicity and/or PK profiling are similar in children and in adults, or a higher dose, providing it is below or equal to the maximum tolerated dose (MTD)

    Measured: During the first cycle

  2. Maximum Tolerated Dose (MTD)

    The MTD will be defined as the dose associated with or closest to 25% of Dose Limiting Toxicities (DLTs)

    Measured: During the first cycle

  3. Objective Response Rate (ORR)

    Defined as the proportion of participants who achieved a confirmed complete response (CR) or partial response (PR) assessed by investigators.

    In patients with leukemia, ORR is defined as the percentage of patients attaining CR, CRi or CRp.

    Measured: During treatment period

Secondary outcomes (3)

  1. Pharmacokinetics (PK)

    To characterize single or multiple-dose PK of the agent(s)

    Measured: Depending on the treatment arm

  2. Progression Free Survival (PFS)

    Defined as the time from treatment initiation until the date of first documented progression (clinically or radiologically) or death from any cause. Patients alive and free of progression at the cut-off date will be censored at the last assessment date.

    Measured: From treatment initiation until the date of first documented progression or death

  3. Evaluation of duration of response (DoR)

    Defined as the time period between the first documented response (complete response (CR) or partial response (PR)) and the time of progression, according to RECIST v1.1, RANO criteria for patients with HGG, INRC criteria for patients with NB, etc.

    Measured: Between the first document response and the time of first documented progression

Timeline

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

  1. First posted24 Jun 2016 (estimated)
  2. Study start3 Aug 2016
  3. Primary completionFeb 2031 (estimated)
  4. Study completionFeb 2031 (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 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
472 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02813135
Sponsor's protocol ID
2016-000133-40
Other IDs
2016/2396, 2024-514791-40-00, 2016
Acronym
ESMART
Status last verified
Jul 2026
First submitted
16 Jun 2016
Last updated
10 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Lee WG, Kim ES. Precision Oncology in Pediatric Cancer Surgery. Hematol Oncol Clin North Am. 2026 Oct;40(5):895-931. doi: 10.1016/j.hoc.2026.05.022.

    PubMed 42686334

  • Rubio-San-Simon A, Marshall LV, Karamouza E, Andre N, Abbou S, Rubino J, Nebchi S, Aerts I, Thebaud E, Brisset C, Ducassou S, Le Teuff G, Geoerger B. Phase I/II Study of the CDK2/9 Inhibitor Fadraciclib in Combination with Chemotherapy in Children with Advanced Malignancies: Arm K of the AcSe-ESMART Trial. Target Oncol. 2026 Mar;21(2):199-211. doi: 10.1007/s11523-026-01208-1. Epub 2026 Mar 16.

    PubMed 41838385

  • Berlanga P, Pierron G, Lacroix L, Chicard M, Adam de Beaumais T, Marchais A, Harttrampf AC, Iddir Y, Larive A, Soriano Fernandez A, Hezam I, Chevassus C, Bernard V, Cotteret S, Scoazec JY, Gauthier A, Abbou S, Corradini N, Andre N, Aerts I, Thebaud E, Casanova M, Owens C, Hladun-Alvaro R, Michiels S, Delattre O, Vassal G, Schleiermacher G, Geoerger B. The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies. Cancer Discov. 2022 May 2;12(5):1266-1281. doi: 10.1158/2159-8290.CD-21-1136.

    PubMed 35292802

  • Morscher RJ, Brard C, Berlanga P, Marshall LV, Andre N, Rubino J, Aerts I, De Carli E, Corradini N, Nebchi S, Paoletti X, Mortimer P, Lacroix L, Pierron G, Schleiermacher G, Vassal G, Geoerger B. First-in-child phase I/II study of the dual mTORC1/2 inhibitor vistusertib (AZD2014) as monotherapy and in combination with topotecan-temozolomide in children with advanced malignancies: arms E and F of the AcSe-ESMART trial. Eur J Cancer. 2021 Nov;157:268-277. doi: 10.1016/j.ejca.2021.08.010. Epub 2021 Sep 17.

    PubMed 34543871

  • Bautista F, Paoletti X, Rubino J, Brard C, Rezai K, Nebchi S, Andre N, Aerts I, De Carli E, van Eijkelenburg N, Thebaud E, Corradini N, Defachelles AS, Ducassou S, Morscher RJ, Vassal G, Geoerger B. Phase I or II Study of Ribociclib in Combination With Topotecan-Temozolomide or Everolimus in Children With Advanced Malignancies: Arms A and B of the AcSe-ESMART Trial. J Clin Oncol. 2021 Nov 10;39(32):3546-3560. doi: 10.1200/JCO.21.01152. Epub 2021 Aug 4.

    PubMed 34347542

  • Pasqualini C, Rubino J, Brard C, Cassard L, Andre N, Rondof W, Scoazec JY, Marchais A, Nebchi S, Boselli L, Grivel J, Aerts I, Thebaud E, Paoletti X, Minard-Colin V, Vassal G, Geoerger B. Phase II and biomarker study of programmed cell death protein 1 inhibitor nivolumab and metronomic cyclophosphamide in paediatric relapsed/refractory solid tumours: Arm G of AcSe-ESMART, a trial of the European Innovative Therapies for Children With Cancer Consortium. Eur J Cancer. 2021 Jun;150:53-62. doi: 10.1016/j.ejca.2021.03.032. Epub 2021 Apr 20.

    PubMed 33892407

  • Rossoni C, Bardet A, Geoerger B, Paoletti X. Sequential or combined designs for Phase I/II clinical trials? A simulation study. Clin Trials. 2019 Dec;16(6):635-644. doi: 10.1177/1740774519872702. Epub 2019 Sep 20.

    PubMed 31538815

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Ribociclib, Topotecan and Temozolomide in people with Pediatric Cancer, run by Gustave Roussy, Cancer Campus, Grand Paris at 23 sites in 6 countries.

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 24 inclusion and 15 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 10 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 23 sites in France, United Kingdom, Italy, Spain, Denmark and 1 more country. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 Birgit Geoerger, MD (+33 (0)1 42 11 46 61, birgit.geoerger@gustaveroussy.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Recommended phase II dose (RP2D) (time frame: During the first cycle). The study lists 3 primary outcomes in all. 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 NCT02813135, which also lists 7 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 10 Jul 2026.

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