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

• Status: Completed
• Trial end date: 2010-05-01
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ente Ospedaliero Ospedali Galliera
• Collaborators: Azienda Ospedaliera V. Cervello; Azienda Ospedaliera Villa Sofia; Ospedale "Perrino" Brindisi; Ospedale Maggiore Policlinico Mangiagalli e Regina Elena; Reggio Calabria; Second University of Naples; Università di Ferrara; University of Campania "Luigi Vanvitelli"

Criteria

Inclusion Criteria:

• Patients presenting BMD Z score <-2 at the level of the femoral neck or of the lumbar column Regular transfusional regimen in order to mantain pre-transfusional Hb values >9 g/dl.

Written informed consent by the patient

Exclusion Criteria:

Entravenous administration of bisphosphonates within the past 2 years

Administration of di bisphosphonates per os, unless wash out as it follows:

1 year if >8 weeks <48 weeks 6 months if > 2 weeks and <8 weeks Hypoparathyroidism Thalassemia Intermedia if not regularly transfused Pregnancy and breast feeding Impaired renal function (creat. > 1.5 mg/dl) Neoplastic disease Patients with mean levels of alanine aminotransferase ALT > 300 U/l and patients with variations of AST or AST of 300% within the year before randomization. (At least 4 misurations over 12 months) Systemic cardiovascular, renal, hepatic disease etc. which would prevent the patient from undergoing study treatment Known hypersensibility to bisphosphonates. History of non compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative.