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

NCT01140321 | Completed Phase 2

• 1 other identifier: 60/2003/Nerixia
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 8

Brief 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

Keywords

Thalassemia; Neridronate; Osteopenia; Osteoporosis

Study Arms (2)

Neridronato

EXPERIMENTAL

Thalassemia Major or Severe Thalassemia Intermedia

Drug: Neridronate

Placebo

NO INTERVENTION

Thalassemia Major or Severe Thalassemia Intermedia

Interventions

Neridronate DRUG

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".

Neridronato

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Entravenous administration of bisphosphonates within the past 2 years
• Administration of di bisphosphonates per os, unless wash out as it follows:
• 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.

Sponsors & Collaborators

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

Study Sites (8)

Divisione di Ematologia, Ospedale "Perrino"

Brindisi, Italy

Location

Dipartimento di Medicina Clinica e Sperimentale, Sezione di Pediatria, Università di Ferrara

Ferrara, Italy

Location

Centro della Microcitemia e delle Anemie Congenite - Ematologia E.O. Ospedali Galliera

Genova, 16128, Italy

Location

SC Geriatria E.O. Ospedali Galliera

Genova, 16128, Italy

Location

Centro Anemie Congenite, Ospedale Maggiore Policlinico, IRCCS, University of Milan

Milan, 20100, Italy

Location

U.O. "Ematologia II con Talassemia" A.O. " V. Cervello

Palermo, Italy

Location

U.O. Pediatria II, A.O. "Villa Sofia"

Palermo, Italy

Location

Centro Microcitemia, A.O. B.M.M.

Reggio Calabria, Italy

Location

Related Publications (1)

• 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.

  PMID: 22571408 DERIVED

MeSH Terms

Conditions

Osteoporosis; beta-Thalassemia; Thalassemia; Bone Diseases, Metabolic

Interventions

6-amino-1-hydroxyhexane-1,1-diphosphonate

Condition Hierarchy (Ancestors)

Bone Diseases; Musculoskeletal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Gian Luca Forni, MD

  Centro della Microcitemia e delle Anemie Congenite E.O. Ospedali Galliera- Genova Italy

  PRINCIPAL INVESTIGATOR

• Ernesto Palummeri, MD

  S.C. Geriatria E.O. Ospedali Galliera - Genova Italy

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: June 7, 2010
• First Posted: June 9, 2010
• Study Start: January 1, 2004
• Primary Completion: May 1, 2010
• Study Completion: May 1, 2010
• Last Updated: February 5, 2020

Record last verified: 2020-02

Locations

IT (8)