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

Conditions

• Osteoporosis
• Thalassemia Major
• Thalassemia Intermedia

Interventions

DRUG

Neridronate

Neridronate is an aminobisphosfonate of third generation that has been proved in several clinical trials to have the ability to inhibit osteoclast-mediated bone resorption and it is generally well tollerated also at high doses. Thanks to these properties it has been used in the treatment of several bone disorders such as Paget's disease and Osteogenesis Imperfecta. Neridronate, given intravenously or orally (tablets), reduces both the levels of bone alcaline phosphatase and of the other markers of bone resorption activity. Recently it has been approved by the Italian Ministry of Health for the commercialization with the indication "Osteogenesis Imperfecta".

Sponsors & Collaborators

• Ospedale Maggiore Policlinico Mangiagalli e Regina Elena

  collaborator OTHER

• Ospedale "Perrino" Brindisi

  collaborator UNKNOWN

• Reggio Calabria

  collaborator OTHER

• Azienda Ospedaliera Villa Sofia

  collaborator UNKNOWN

• Azienda Ospedaliera V. Cervello

  collaborator OTHER

• Università di Ferrara

  collaborator UNKNOWN

• University of Campania Luigi Vanvitelli

  collaborator OTHER

• Ente Ospedaliero Ospedali Galliera

  lead OTHER

Principal Investigators

• Gian Luca Forni, MD · Centro della Microcitemia e delle Anemie Congenite E.O. Ospedali Galliera- Genova Italy

• Ernesto Palummeri, MD · S.C. Geriatria E.O. Ospedali Galliera - Genova Italy

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2004-01-31

Primary Completion

2010-05-31

Completion

2010-05-31

Countries

• Italy

Study Locations