Studi Scientifici in corso e protocolli di ricerca aperti

Acronym

Sponsor

Title

Status

P.I. Prof. Bisogno, Co-Investigator Dott. Opocher

BERNIE
BO20924

Roche

Open-label, multi-center, randomized, phase II study evaluating the addition of Bevacizumab to chemotherapy in childhood and adolescent patients presenting with metastatic rhabdomyosarcoma and non-rabdomyosarcoma soft tissue sarcoma.

Ongoing

P.I. Prof. Bisogno, Co-Investigator Dott. Opocher

HERBY
BO25041

Roche

A phase II open-label, randomized, multi-centre comparative study of Bevacizumab-based therapy in paediatric patients with newly diagnosed supratentorial high-grade glioma.

Ongoing

P.I. Prof. Bisogno

ABRAXANE
ABI 007-PST001

Celgene

A phase I/II, multicenter, open label, dose-finding study to assess the safety, tolerability and preliminary efficacy of weekly Nab-paclitaxel in pediatric patients with recurrent or refractory solid tumors.

Ongoing

P.I. Prof. Bisogno

FOSAPREPITANT
MK0517-029

Merck

A Phase IIb, Partially-Blinded, Randomized, Active Comparator- Controlled Study to Evaluate the Pharmacokinetics/Pharmacodynamics, Safety, and Tolerability of Fosaprepitant in Pediatric Patients for the Prevention of Chemotherapy-Induced Nausea and Vomiting (CINV) Associated with Emetogenic Chemotherapy.
EudraCT 2012-002340-24

Ongoing

P.I. Prof. Bisogno

MOZAIC FASE 2
MOZ15609/DFI12860

Genzyme

A Phase 1/2 Combined Dose Ranging and Randomised, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilisation of Haematopoietic Stem Cells into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilisation Regimens Alone in Paediatric Patients with Solid Tumours Eligible for Autologous Transplants.

1st phase
concluded in 2013. 2nd phase ongoing

PI. Prof. Bisogno

ATEZOLIZUMAB
GO29664

Genentech/ Roche

An early phase multicenter open-label study of the safety and pharmacokinetics of anti-pd-L1 antiboby in pediatric and young adult patients with previously treated solid tumors.

Opened in 2016

P.I. Prof. Bisogno

MK 0869-208
APREPITANT

Merck

A Phase III, Randomized, Double-Blind, Active Comparator-Controlled Clinical Trial, Conducted Under In-House Blinding Conditions, to Examine the Efficacy and Safety of Aprepitant for the Prevention of Chemotherapy-Induced Nausea and Vomiting (CINV) Associated with Emetogenic Chemotherapy in Pediatric Patients.

Closed in 2013

P.I. Prof. Bisogno

MEPACT
C23003

Millennium / Takeda

Phase IV Surveillance study of patients with newly diagnosed high-grade, resectable non metastatic osteosarcoma to investigate the short-term safety profile of mifamurtide (MEPACT) as part of a combined chemotherapy treatment regime for this condition.

Closed in 2014

P.I. Prof. Bisogno

GIST
A6181196

Pfizer

A phase I/II of Sunitinib in young patients with advanced gastrointestinal stromal tumor.
EudraCT: 2011-002008-33

Closed in 2016

P.I. Prof. Bisogno

TADO
H7T-MC-TADO

Eli Lilly

A Phase 3, Double-Blind, Randomized, Efficacy and Safety Comparison of Prasugrel and Placebo in Pediatric Patients with Sickle Cell Disease.
NCT01794000

Closed in 2016

P.I. Prof. Bisogno, Co-Investigator Dott. Opocher

IPILIMUMAB
CA184-178

Bristol-Myers Squibb

A Phase 2 Study of Ipilimumab in Children and Adolescents (12 to < 18 years) with Previously Treated or Untreated, Unresectable Stage III or Stage IV Malignant Melanoma. 2012-002249-39.

Closed in 2016

P.I. Prof. Bisogno

AFATINIB

Boehringer Ingelheim / ITCC

Phase I open label, dose escalation basket trial to determine the MTD of afatinib in children aged 2-18 years with relapsed/recurrent neuroectodermal tumours and rhabdomyosarcoma.

Opening in 2016

P.I. Prof. Bisogno

GO29665

Genentech/ Roche

A phase I/II, multicenter, dose-escalation study of the safety and pharmacokinetics of cobimetinib in pediatric and young adult patients with previously treated solid tumors.

opening in 2016

P.I.Dott.ssa Putti

A multi-center, open label, non controlled phase II study to evaluate efficacy and safety of oral nilotinib in pediatric patients with newly diagnosed Ph+ chronic myelogenous leukemia (CML) in chronic phase (CP) or with Ph+ CML in CP or accelerated phase (AP) restistant or intolerant to either imatinib or dasatinib.

P.I. Dott.ssa Putti

NI-0501-04

Novimmune

A Pilot, Open-label, Single Arm, Multicentre Study to Explore Safety, Tolerability, Pharmacokinetics and Efficacy of Intravenous Multiple Administrations of NI-0501, an Anti-interferon Gamma (Anti-IFNγ) Monoclonal Antibody, in Paediatric Patients with Primary Haemophagocytic Lymphohistiocytosis which has Reactivated.
EudraCT#2012-003632-23

Ongoing

P.I. Dott.ssa Putti

NI-0501-05

Novimmune

A multicentre study for the long-term follow-up of HLH patients who received treatment with NI-0501, an anti-interferon gamma monoclonal antibody PI Cristina de Min (CH).
EudraCT#2012-005753-23

Ongoing

P.I. Dr.ssa Pillon

Pfizer

A Phase 1/2 Study of Brentuximab Vedotin (SGN-35) in Pediatric Patients With Relapsed or Refractory Systemic Anaplastic Large-Cell Lymphoma or Hodgkin Lymphoma

Ongoing

P.I. Prof.ssa Messina

Amgen

A Single-Arm Multicenter Phase II Study preceded by Dose Evaluation to Investigate the Efficacy, Safety, and Tolerability of the BiTE® Antibody Blinatumomab (MT103) in Pediatric and Adolescent Patients with Relapsed/Refractory B-Precursor Acute Lymphoblastic Leukemia (ALL).

Closed on 2015

P.I. Dr.ssa Calore

VOD-2013-03-REG

Gentium

A Multicentre, multinational, prospective observational registry to collect safety and outcome data in patients diagnosed with severe hepatic VOD following hematopietic stem cell transplantation (HSCT) and treatment with Defitelio or supportive care (control group). DF Studio EBMT/Gentium

Ongoing

P.I. Dott.ssa Putti

CAMN 107A2120

Covance

Clinical Trial Protocol CAMN 107A2120. A multi-center, open-label, pharmacokinetic study of oral nilotinib in pediatric patients with Gleevec® (imatinib)-resistant/intolerant Ph+ CML chronic phase (CP) or accelerated phase (AP) or with refractory/relapsed Ph+ ALL

P.I. Dott.ssa Putti

CAMN 107A2203

Covance

Clinical Trial Protocol CAMN 107A2203. A multi-center, open-label, non controlled phase 2 study to evaluate efficacy and safety of Nilotinib in pediatric patients with newly diagnosed Ph+chronic myelogenous leukemia (CML) in chronic phase (CP) or with Ph+ CML in CP or accelerated phase (AP) resistant or intolerant to either Imatinib or dasatinib. (EUDRACT n.2013-000200-41) accelerated phase (AP) or with refractory/relapsed Ph+ ALL

Ongoing

Co-Investigator Dott. Massano

MPDL3280A

(anti-PD-L1 - EudraCT n: 2014-004697-41).

P.I. Dott.ssa Putti

Essential thrombocytemia (ET) in pediatric age: European Registry (Elliot: SPD422-404) and study of rare genetic mutations.

Investigator Dott.ssa Putti

BAY 59-7939/14372 Rivaroxaban Pediatric Study: to evaluate safety, tolerability and pharmakokinetics of rivaroxaban, an oral anti-thrombotic drug in children with venous thromboembolic events.

P.I. Dott.ssa Putti

Genzyme Europe 2009

The aim of the study is to characterize the risk/benefit profile of Evoltra during routine clinical use for the treatment of paediatric patients (≤ 21 years at initial diagnosis) with acute lymphoblastic leukaemia. The study has started in 2009; it has been recently amended with the inclusion of patients treated with clofarabine within any combination regimen and also patients affected by acute myeloid leukaemia
The Evoltra® European Registry Programme : Paediatric ALL (No CLO08708)

Ongoing

AddMedica

ESCORT_HU: European Sickle Cell Disease Cohort – Hydroxyurea, NCT02516579, 2009-2018.

Ongoing

P.I. Prof. Basso

UVADEX

Therakos

Single-arm study to assess the efficacy of UVADEX® (methoxsalen) sterile solution in conjunction with the Therakos® Cellex® Photopheresis System in Pediatric Patients With Steroid-Refractory or Steroid-Dependent Acute Graft Versus Host Disease (aGvHD).

Ongoing

AMGEN

Amgen

Studio Adattativo di Fase 3 Controllato, in aperto Randomizzato per valutare l’efficacia. La Sicurezza e la tollerabilitá dell’anticorpo BiTE Blinatumomab come Terapia di Consolidamento rispetto alla chemioterapia di consolidamento convenzionale in soggetti pediatrici con leucemia linfoblastica acuta (LLA) da precursori delle cellule B in prima recidiva ad alto rischio.

Ongoing

RIALTO

Amgen

Studio multicentrico, in aperto, ad accesso allargato con Blinatumomab per il trattamento di soggetti pediatrici e adolescenti affetti da leucemia linfoblastica acuta (LLA) da precursori delle cellule B recidiva e/o refrattaria (studio RIALTO)

Ongoing

AZA-JMML-001

Gelgene

Studio multicentrico in aperto di fase II per valutare la farmacocinetica , la farmacodinamica , la sicurezza e l’attivitá di Azacitidina e per confrontare Azacitidina con i controlli storici in soggetti pediatrici con sindrome mielodisplastica avanzata o leucemia mielomonocitica giovanile di nuova diagnosi non ancora sottoposti a trapianto di cellule staminali ematopoietiche.

Ongoing

OPTO-01-2015

AIL TREVISO

Sindrome autoimmune linfoproliferativa (ALPS) e malattie ALPS correlate creazione di una rete nazionale.
(Samital - EudraCT n: 2015-000386-31)

Ongoing

Acronym

Sponsor

Title

Status

Prof. Giuseppe Basso

Studio osservazionale: trapianto aploidentico di cellule staminali ematopoietiche da donatore familiare HLA-parzialmente compatibile dopo selezione negativa di cellule TcR αβ+ e CD19+ in pazienti pediatrici affetti da patologie non maligne.

Ongoing

InTeRALL SR 2010

Ospedale Pediatrico Bambin Gesù, Roma

Studio Internazionale per il trattamento del rischio medio nella popolazione infantile con leucemia linfoblastica acuta (LLA) recidiva. InTeRALL SR 2010

Ongoing

Implicazioni psicoaffettive nel trapianto di cellule staminali emopoietiche.

Ongoing

Studio prospettico randomizzato sul ruolo della foto chemioterapia extracorporea precondizionamento versus condizionamento standard nella prevenzione della malattia del trapianto contro l’ospite.

Ongoing

TCSE-HC

Trapianto di cellule staminali ematopoietiche da donatore compatibile dopo condizionamento ad intensitá ridotta nella malattia di Hodgkin ad altissimo rischio. TCSE HD

Ongoing

CILI

Studio multicentrico di fase II per valutare l’attivitá e la tossicitá della Citarabina liposomiale nel trattamento di bambini e adolescenti con leucemia acuta linfoblastica e localizzazione meningea resistente o in ricaduta dopo trattamento sistemico e intrarachideo. CILI

Ongoing

AIEOP LAM 2013/01

Trattamento della leucemia acuta mieloide secondo il protocollo AIEOP LAM 2013/01

Ongoing

P.I. Dott.ssa Elisabetta Viscardi

PNET 5

An International Prospective Study On Clinically Standard-Risk Medulloblastoma In Children Older Than 3 To 5 Years With Low-Risk Biological Profile (PNET 5 Mb - LR) Or Average-Risk Biological Profile (PNET 5 Mb – SR).

Ongoing

NB-AR-01

Primo studio cooperativo europeo per il Neuroblastoma ad alto rischio. NB-AR-01.

Ongoing

P.I. Prof. Bisogno

VIT
ITCC- 025

Centre Oscar Lambret-Lille

International randomized phase II study of the combination of vincristine, and irinotecan with or without temozolomide in patients with refractory or relapsed rhabdomyosarcoma.

1st phase concluded. 2nd phase opening in 2017

P.I. Prof. Bisogno

BIOTECH

Regione Veneto Ricerca Sanitaria Finalizzata

Novel biomarkers for the early diagnosis of hepatic toxicity from chemotherapy.

Ongoing

P.I. Prof. Bisogno

Novel Biomarker in RMS

AIRC

Novel strategies in metastasis research and biomarker discovery in rhabdomyosarcoma.

Ongoing

P.I. Prof. Bisogno

SAMITAL

Ministero della Salute
Ricerca Finalizzata 2010

Role of SAMITAL in prevention of CT/RT induced oral mucositis in cancer patients. A Phase 2 Randomized Controlled trial in adults and a feasibility study in paediatric patients pROSAM.

Ongoing

P.I. Prof. Bisogno

EPOC-MS-001-Doxo

Comunitá Europea, FP-7HEALTH-2007-4.2-1

Phase II pharmacokinetic study to assess the age dependency in the clearance of doxorubicin in paediatric patients with solid tumours and leukaemia.

Closed in 2013

P.I. Prof. Bisogno

TOTEM 2

Institute Gustave Roussy ITCC

Phase II study of temozolamide in combination with Topotecan in patients with resistant/relapsed neuroblastoma and other solid tumors.

Closed in 2015

P.I. Prof. Bisogno

Studio internazionale controllato, randomizzato per il trattamento chemioterapico di pazienti con sarcoma di Ewing in ricaduta o resistenti (studio rEECur).

Ongoing

P.I. Prof. Bisogno, Co-PI Dott. Opocher

VINILO

ITCC

Vinblastine and nilotinib (VINILO) in patients with relapsed or refractory low grade glioma.

Opening in 2017

PI Prof. Bisogno

AIEOP-RTB012

Ospedale pediatrico Bambino Gesù, Roma

Protocollo Diagnostico e Terapeutico per il Retinoblastoma alla Diagnosi. Clinical trial 2012

Ongoing

P.I. Dott.ssa Todesco

Italian Association of Pediatric Oncology and Hematology Study for the treatment of Langerhans Cell Histiocytosis.

Ongoing

P.I. Dott.ssa Todesco

Studio clinico internazionale HLH 94, HLH 2004 per la cura delle HLH.

Closed in 2015

P.I. Dott.ssa Todesco

LCH-IV: protocollo internazionale collaborativo per il trattamento delle Istiocitosi a Cellule di Langerhans.

Ongoing

Co-Investigator Dott.ssa Todesco

Local principal investigator of the Italian Association of Pediatric Oncology and Hematology Study for the treatment of Hodgkin's Lymphoma pediatric patients.

Closed in 2015

P.I. Dott.ssa Pillon

Euro PHL-LP1

AIEOP

First International Inter-Group Study for Nodular Lymphocyte predominant Hodgkin's Lymphoma in Children and Adolescents, Euro-PHL-LP1.

Ongoing

P.I. Dott.ssa Pillon

Inter B-NHL Ritux 2010

Institute Gustave Roussy

Intergroup trial for children or adolescents
with b-cell nhl or b-al: evaluation
of rituximab efficacy and safety in high risk patients.

Ongoing

P.I. Dott.ssa Pillon

LBL 2014

Oncoematologia Pediatrica di padova

LBL 2014 - International cooperation of the European Inter-group Co-operation on Childhood Non-Hodgkin-Lymphoma (eicnhl) and the International BFM (I-BFM) Groups for the prospective evaluation of molecular prognostic factors for lymphoblastic lymphoma in children and adolescents.

Ongoing

P.I. Dott.ssa Pillon

Screening Fanconi

AIRFA

Screening di pazienti affetti da Anemia di Fanconi in Italia per le lesioni di carcinoma di cellule squamoso nella cavitá orale.

Ongoing

Co-Investigator Dott. Opocher

SIOP-LGG2004

Collaborative research activities within the European SIOP-LGG2004 group with a particular competence and experience in functional and visual outcome of children affected by optic pathways glioma, with or without NF1.

Ongoing

P.I. Prof. Bisogno Co-Investigator Dott.ssa Affinita

Studio internazionale controllato, randomizzato per il trattamento chemioterapico di pazienti con sarcoma di Ewing in ricaduta o resistenti (studio rEECur).

Ongoing

Co-Investigator Dott.ssa E. Calore

Current treatment of HCV infection after HSCT. EBMT/IDWP non interventional prospective study.

Ongoing

Co-Investigator Dott.ssa E. Calore

Gentium

A retrospective data collection (RDC) study regarding patients enrolled in Italian sites during the previous interventional trial: “Prospective study of the incidence and outcome of veno-occlusive disease (VOD) with the prophylactic use of Defibrotide (DF, Gentium, Italy) in pediatric stem cell transplantation - Eudract 2004-000592-33”. Protocollo DF VOD-2012-02-RDC.

Closed in 2013

Co-Investigator Dott.ssa E. Calore

A retrospective and prospective observational study on incidence, treatment and outcome of invasive Aspergillosis and other mould infections among the AIEOP centres (Italian Association of Pediatric Hematology).

Closed 2013

Co-Investigator Dott.ssa E. Calore

Gram negative bacteremia in HSCT recipients. EBMT/IDWP non-interventional prospective study.

Closed in 2015

Co-Investigator Dott.ssa E. Calore

Mesenchymal stromal cells (MSC) for the treatment of severe (grade II-IV) steroid-resistant graft versus host disease (GVHD): a phase I trial.

Closed in 2012

Co-Investigator Dott.ssa E. Calore

Impact of pre-existing invasive aspergillosis on alllogeneic stem cell transplantation for treatment of acute leukaemia and myelodisplastic syndrome (IPAT study EBMT/IDWP non interventional prospective study).

Ongoing

Co-Investigator Dott.ssa E. Calore

Pneumocystis Jirovecii Pneumonia (PcP) after allogeneic HSCT. EBMT/IDWP non-interventional prospective study.

Ongoing

Co-Investigator Dott.ssa E. Calore

Studio prospettico sulla malattia veno-occlusiva epatica in pazienti pediatrici sottoposti a trapianto di cellule staminali emopoietiche.

Submitted to the Ethics Committee

Co-Investigator Dott.ssa E. Calore

Protocol DF-2012-01 TUP: Guidance for the therapeutic use of Defibrotide for the prophylaxis of hepatic veno-occlusive disease (VOD) pursuant to the Ministerial Decree of 8 May 2003.

Closed in 2014

Co-Investigator Dott.ssa R. Colombatti

AIEOP

Monitoraggio della Terapia con Ferro Orale nella Anemia Sideropenica Infantile – Monitoring of Therapy with Oral Iron in Sideropenic Anemia of Infancy (2015-)

Ongoing

Co-Investigator Dott.ssa R. Colombatti

AIEOP

Multicenter study on the Treatment with Erythropoietin in Spherocytosis.

Ongoing

Co- P.I. Prof. Bisogno

AIEOP

Idiopathic Trhombocytopenia and Autoimmunity.

Ongoing