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Bando 2006. Risultati II fase di valutazione. Area 1. Farmaci orfani per malattie rare o farmaci per sottogruppi di pazienti non responder Tematica 1: Valutazione del profilo beneficio-rischio dei farmaci orfani per malattie rare, approvati o designati dall’EMEA. Codice FARM6LW94J FARM6223SS FARM6JBLZ2 FARM65BEM9 FARM6LXS93 FARM6J8WS4 FARM6NJ3TZ FARM6FYZB3 FARM6CC747 Titolo Punteggio finale Insufficiente Treatment of patients with early stages of the Mycosis Fungoides with monoclonal antibodies directed against CD4 Randomized study comparing intravenous Busulfan (I.V. Bu; Busilvex®) plus 3,9 Cyclophosphamide (BuCy2) versus intravenous Busulfan plus Fludarabine (BuFlu) as conditioning regimens prior to allogeneic hematopoietic stem cell transplantation in patients with Acute Myeloid Leukemia, Chronic Myeloid Leukemia and Myelodysplastic Syndrome Multicenter randomized study to assess the efficacy and the safety of two 3,3 therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase Efficacy of adjuvant mitotane treatment in prolonging disease-free survival in 3,9 patients with adrenocortical carcinoma submitted to radical resection Efficacy and evaluation of stiripentol as add-on treatment in patients with Insufficiente severe myoclonic epilepsy in infancy (SMEI): double blind, multicenter, randomized, controlled vs placebo study. Evaluation of the efficacy of enzyme replacement therapy in pediatric patients 3,7 affected by mucopolysaccharidosis type II (Hunter syndrome) Double blind randomized controlled study on the benefit-risk profile of the 3,2 administration of Sildenafil (Viagra®) in the treatment of at term or late preterm newborn infants with severe respiratory disorders and pulmonary hypertension Multicentric comparative randomized double blind cross over study with 4 tacrolimus (FK506)0.1% eyedrops and cyclosporine 1% eyedrops in children with severe active vernal keratoconjunctivitis, lasting 7 weeks, followed by an open trial lasting 24 months to evaluate long term efficacy and safety of the treatment Evaluation of enzyme replacement therapy with Naglazyme (Aryplase) on joint 3 disease in mucopolysaccharidosis type VI patients. Tematica 2: Valutazione del profilo beneficio-rischio nei trattamenti off-label nelle malattie rare. Codice Titolo FARM6PMFJM Multicenter phase III randomized study of cisplatin and etoposide with or without bevacizumab as first-line treatment in extensive stage (ED) small cell lung cancer (SCLC) Multicenter, randomised, double masked, controlled clinical trial on the safety and efficacy of Cyclosporine A eye drop treatment on patients with ocular cicatricial pemphigoid. Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich’s ataxia (a pilot study) Study of the efficacy and tolerability of nilotinib and dasatinib in patients with systemic mastocytosis Phase III study with adjuvant radiotherapy plus or without concurrent chemotherapy with paclitaxel in patients with intermediate-high risk endometrial carcinoma Arterial Hypertension After Successful Aortic Decoarctation: Atenolol vs Enalapril Comparison of Efficacy and Tolerability in Pediatric Age. Intensity of CYTOreductive therapy to prevent cardiovascular events in patients with Polycythemia vera (PV) – CYTO-PV Phase II monitored clinical trial for evaluation of treatment of patients with Thymic Epithelial Tumours (TET) or Histiocitosi X (LCH) with Imatinib Mesylate. Evaluation of the benefit/cost/safety profile of low-dose anti-CD20 monoclonal antibody (rituximab) treatment for refractory mixed cryoglobulinemia A multicenter, randomized, open, prospective study to evaluate the efficacy of pharmacotherapy with somatostatin analogues, dopamine-agonists and a combination of these compounds in elderly patients with non functioning pituitary tumors Double-blind, placebo-controlled, pilot study on the efficacy of tadalafil in the treatment of systemic sclerosis A multicentric randomized trial in adult patients with acute myelogenous leukemia (AML), to compare 1) a standard-dose versus high-dose remission induction regimen, and 2) an autologous blood stem cell transplantation versus an autologous blood stem cell-supported multicycle high-dose chemotherapy program,within a risk-oriented postremission strategy reserving allogeneic stem cell transplantation for high-risk cases Double-blind placebo-controlled pilot study on the efficacy of lithium therapy in amyotrophic lateral sclerosis patients. Effects of tetrahydrobiopterin (BH4) on flow-mediated dilation in CADASIL patients: a randomised controlled trial Low-dose Oral Imatinib in the Treatment of Scleroderma Pulmonary Involvement: a Phase II pilot study. Therapeutic efficacy of butyrate in pediatric patients with congenital chloride diarrhea Multicenter, randomized, double masked, controlled study on efficacy and safety of bicalutamide treatment in patients affected by familiar polycystic ovary syndrome (PCOS) A phase 2 study of efficacy/safety of everolimus in subjects with idiopathic myelofibrosis (IMF) Evaluation of the tolerability and efficacy of erythropoietin (epo) treatment in spinal shock: comparative study vs methylprednisolone (MP) Use of dexamethasone to treat neurological symptoms in children with ataxiatelangiectasia Hydroxychloroquine as Steroid-Sparing Agent in pulmonary Sarcoidosis (HySSAS). A multicenter, prospectic, controlled, randomized trial. Multicenter, randomized, double blind, comparative clinical trial on the use of imatinib in association with angiotensin converting enzyme inhibitors and corticosteroids in the treatment of IGA nephropathy FARM6AS8AW FARM6H95MJ FARM6A2JE4 FARM6X5T4M FARM6HCEM5 FARM6YNXAN FARM6HJ7CA FARM6KMZFY FARM6ERPC4 FARM6CA84K FARM6YMY2N FARM6FWLBT FARM659PTX FARM69JAJ9 FARM6FJ728 FARM6XAXBR FARM6AKR9W FARM6Y35XM FARM6P7WR9 FARM693KLZ FARM6ZMESX Punteggio finale 3,2 4 3,9 Insufficiente Insufficiente 3,5 3,1 3 4 Insufficiente Insufficiente 3,6 Insufficiente 3,9 3,6 4,1 3,6 4,1 4 Insufficiente 4,3 3,3 Tematica 3: Valutazione del profilo beneficio-rischio dei farmaci in pazienti non-responder nei confronti delle terapie standard. Codice FARM64J2EB Titolo Randomized controlled double-blind vs. placebo multicentre study on the safety and effectiveness of thalidomide in the treatment of refractory Crohn’s disease and ulcerative colitis in children and adolescents FARM6FWBTR Mycophenolate Mofetil in myasthenic patients non responder to standard immunosuppressive treatment with prednisone and azathioprine. FARM6XNY7N Multicenter, randomised, controlled, double-masked study on the efficacy of systemic cyclosporine treatment versus long-term steroid eye drop treatment in high-risk patients for corneal rejection (no-responder to standard treatment) FARM6FLXW3 Efficacy and tolerability of Linezolid in the treatment of tuberculosis multi-drug resistant to first line antimicobacterial compounds FARM6TR9T8 Randomised, open-label clinical trial on the efficacy of colistin plus rifampicin treatment versus colistin alone for severe infections by multidrug-resistant Acinetobacter Baumannii. FARM6ZRWW5 A multicentric randomized study comparing two antiretroviral treatment strategies in HIV-infected individuals with multi-drug failure, extensive viral resistance and limited or no therapeutical options (Comparing Suppressive vs non-suppressive strategies in Multi-failed Advanced HIV patients – COSMA Study). FARM68KYHX EMMA-CAV Study (Everolimus or Mycophenolate Mofetil vs. Azathioprine in Cardiac Allograft Vasculopathy study) A multicenter, prospective, randomized, open-label study to compare the efficacy and safety of Everolimus and Mycophenolate Mofetil (in association with Cyclosporine) in patients with chronic rejection/cardiac allograft vasculopathy not responsive to traditional immunosoppression (Cyclosporine, Azathioprine, Steroids) Punteggio finale 3 Insufficiente Insufficiente Insufficiente Insufficiente Insufficiente Insufficiente