Download Bando 2006. Risultati II fase di valutazione. Area 1. Farmaci orfani

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
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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