Multi-centre, oral single dose experimental and modelling study to evaluate the pharmacokinetics of deferiprone in patients aged from 1 month to less than 6 years of age affected by transfusion-dependent haemoglobinopathies.
The study was concluded on February 2014, providing scientific evidence that the dosage of deferiprone used in adults can provide sufficient exposure to ensure efficacy also in small children and allowing children aged under 6 years of age to start to be recruited in DEEP-2 Safety/Efficacy Study. The study has enrolled a total of 23 patients, of which 18 were considered as evaluable.
Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate the efficacy and safety of deferiprone compared to deferasirox in paediatric patients aged from 1 month to less than 18 years affected by transfusion-dependent haemoglobinopathies. The study was aimed at comparing for the first time the efficacy and safety of deferiprone (DFP) vs deferasirox (DFX) in paediatric patients affected by hereditary haemoglobinopaties requiring chronic transfusions and chelation.
The trial has involved 344 paediatric patients starting from 1 month of age in EU and non-EU recruiting sites.
Multi-centre randomized clinical trial to compare the safety and efficacy of Deferasirox and Deferiprone administrated in sequential mode versus Deferasirox or Deferiprone in monotherapy in patients affected by Thalassemia Major.
The study intends to provide a prospective validation and clinical evaluation of safety and efficacy of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection.
Prospective multicentre randomised, placebo-controlled double-blind trial to verify the effects of the administration of probiotics on intestinal microflora in paediatric patients with Cystic Fibrosis.
Strategy for Maintenance of HIVsuppression with once daiLy Integraseinhibitor + darunavir/ritonavir in childrEn. A Phase 2/3 multicentre, open-label, randomised study evaluating safety and antiviral effect of elvitegravir (EVG) administered with darunavir/ritonavir (DRV/r) compared to current standard antiretroviral therapy in HIV-1 infected, virologically suppressed paediatric participants. The study is expected to enrol 300 HIV-1 infected children aged 6 to < 18 years from Western and Eastern Europe, South Africa, Thailand, Uganda and Latin America.
Efficacy, pharmacokinetics and safety of Meropenem in subjects below 90 days of age (inclusive) with clinical or confirmed late-onset sepsis: a European multicentre randomised phase III trial. The study ended in December 2014 with the enrolment of 272 patients.
Pharmacokinetics and safety of meropenem in infants below 90 days of age (inclusive) with probable and confirmed meningitis: a European multicenter phase I-II trial. The study ended in December 2014 with the enrolment of 51 patients.
Multi-centre randomised open label phase IIb study to compare the efficacy, safety and pharmacokinetics (PK) of an optimised dosing to a standard dosing regimen of vancomycin in neonates and infants aged less than 90 days with late onset bacterial sepsis known or suspected to be caused by Gram-positive microorganisms. The study is expected to start on 1st June 2016 and to enrol 300 patients.
A 36-months multi-centre randomized interventional pragmatic trial to evaluate the effectiveness and safety of glatiramer-acetate (GA) compared to interferon-beta (IFN-beta) in paediatric patients affected by multiple sclerosis.
A randomized phase II/III trial of doxycycline (versus standard supportive therapy) aimed at establishing whether the addition of the antibiotic doxycycline to anti-plasma cell therapy can reduce early mortality in newly-diagnosed patients with cardiac AL amyloidosis.
Osteo-muscular system and connective tissue diseases
Prospective, monocentric, post-CE mark study to assess efficacy and safety of Hymovis® SPORT (32 mg/4 ml) intra-articular injection in in the management of knee pain and function in active patient affected by knee osteoarthritis due to overuse.
Observational study based on the collection of at least 1000 cases of juvenile idiopathic arthritis (JIA) and aimed to review the International League Against Rheumatism (ILAR) Classification criteria for JIA.
The primary clinical objective of this study is to establish the Optimal Biologic Dose (OBD) of losartan in 30 children aged 8-17 years with OI. Moreover, it aims to determine the changes in proxy efficacy outcomes of bone (mass, architecture and strength) and muscle (strength), and determine changes in quality of life using a validated disease-specific tool.
Innovative Devices for the prevention of recurrent VAginitis.
DIVA project intends to develop an innovative treatment specific for recurrent vaginitis with the aim to reduce the use of antibiotic therapy. py. This objective is acquired through the development of:
1. A food enriched with probiotic lactobacilli with antimicrobial activity and dietary fibres;
2. Device / cosmetic with bacteriostatic and fungicidal action derived from plant products;
3. Sanitary towels functionalized with bacteria and fungicidal extracts derived from plant products;
The treatment foresees both the intake of a food based on specific lactobacilli for the modulation of the intestinal microbiota and the use of a product (cosmetic or device medical) for vaginal use based on substances with bacteriostatic and fungicidal action.
Randomized, double-blind, double-dummy, active controlled, multicentre, non-inferiority phase III study to compare the pharmacokinetic, efficacy and safety of gabapentin liquid formulation to tramadol in children from 3 months to less than 18 years of age experiencing moderate to severe chronic neuropathic or mixed. The protocol involves the enrolment of 94 patients to test the efficacy and safety of gabapentin monotherapy in the treatment of chronic pain with a neuropathic component.
GABA-1 is part of a project called GAPP – GAbapentin in Paediatric Pain.
Randomized, double-blind, placebo controlled, multi-centre, superiority phase II study to evaluate the safety, pharmacokinetic, efficacy of gabapentin liquid formulation as add-on to morphine in children from 3 months to less than 18 years of age experiencing severe chronic neuropathic or mixed pain. 66 patients will be enrolled to test the efficacy and safety of gabapentin in combination with morphine in the treatment of severe chronic pain with a neuropathic component.
GABA-2 is part of a project called GAPP – GAbapentin in Paediatric Pain.
A double blind, randomised, multicentre, active controlled, parallel-group, phase III trial to evaluate the efficacy, safety and pharmacokinetics of intravenous clonidine (hydrochloride) compared to midazolam for sedation in children from birth to less than 18 years of age. The study is aimed at comparing a new paediatric formulation of clonidine with midazolam. It is ongoing and involves 5 EU countries.Pain.
A prospective, double blind, randomised, 24 month study to assess the effect of prebiotic Galacto-oligosaccharide/polydextrose (GOS/PDX) supplemented formula compared with standard formula in preventing and modifying the history of allergy and acute infections in infants at risk of atopy.