Efficacy and Safety of Neridronate (Nerixia®)to Treat Osteoporosis in Patients With TM and TI

Summary

An Italian Multicentric randomized, open-label therapeutic trial evaluating the efficacy and safety of Neridronate in the treatment of Osteoporosis in patients with Thalassemia Major and Severe Thalassemia Intermedia. Efficacy and safety of the drug will be evaluated measuring at every visit this parameters: * haematological: Haemochrome * blood chemistry: creatinine, BUN, AST, ALT, Ca, P, proteins electrophoresis, total proteins. The prevalence of ectopic calcification and pseudoxantoma elasticum (PXE)-like syndrome and their follow-up will be evaluated at the beginning of the study vs 24 months through physical examination, abdominal echography and fundus oculi examination. During the trial other known risks factors for osteoporosis will be recorded, including prevalence and incidence of bone fractures and, if executed, Polimorphisms COLIA1. At the beginning of the study and at months 12 and 24 morphometry DXA will be performed to evaluate of the presence of bone deformities. Furthermore data regarding QOL and symptom pain will be evaluated trough administration of scale SF-36. At 12 months an intratrial analisis will be performed on efficacy and safety parameters in order to introduce possible amendments to the study design and to decide the prosecution of the trial During the trial all adverse events will be recorded

Timeline

Start

2004-01

Primary completion

2010-05

Completion

2010-05

Publications

• Forni GL, Perrotta S, Giusti A, Quarta G, Pitrolo L, Cappellini MD, D'Ascola DG, Borgna Pignatti C, Rigano P, Filosa A, Iolascon G, Nobili B, Baldini M, Rosa A, Pinto V, Palummeri E. Neridronate improves bone mineral density and reduces back pain in beta-thalassaemia patients with osteoporosis: results from a phase 2, randomized, parallel-arm, open-label study. Br J Haematol. 2012 Jul;158(2):274-282. doi: 10.1111/j.1365-2141.2012.09152.x. Epub 2012 May 10.

Drugs

Indications