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

This study has been completed.
Sponsor:
Collaborators:
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
Ospedale "Perrino" Brindisi
A.O. B.M.M., Reggio Calabria
Azienda Ospedaliera Villa Sofia
Azienda Ospedaliera V. Cervello
Università di Ferrara
Second University of Naples
Information provided by:
Ente Ospedaliero Ospedali Galliera
ClinicalTrials.gov Identifier:
NCT01140321
First received: June 7, 2010
Last updated: June 8, 2010
Last verified: June 2010
  Purpose

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


Condition Intervention Phase
Osteoporosis
Thalassemia Major
Thalassemia Intermedia
Drug: Neridronate
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Randomized, Open-label Therapeutic Trial Evaluating the Efficacy and Safety of Neridronate (Nerixia®) in the Treatment of Osteoporosis in Patients With Thalassemia Major and Severe Thalassemia Intermedia.

Resource links provided by NLM:


Further study details as provided by Ente Ospedaliero Ospedali Galliera:

Enrollment: 120
Study Start Date: January 2004
Study Completion Date: May 2010
Primary Completion Date: May 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Neridronato
Thalassemia Major or Severe Thalassemia Intermedia
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".
No Intervention: Placebo
Thalassemia Major or Severe Thalassemia Intermedia

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01140321

Locations
Italy
Divisione di Ematologia, Ospedale "Perrino"
Brindisi, Italy
Dipartimento di Medicina Clinica e Sperimentale, Sezione di Pediatria, Università di Ferrara
Ferrara, Italy
Centro della Microcitemia e delle Anemie Congenite - Ematologia E.O. Ospedali Galliera
Genova, Italy, 16128
SC Geriatria E.O. Ospedali Galliera
Genova, Italy, 16128
Centro Anemie Congenite, Ospedale Maggiore Policlinico, IRCCS, University of Milan
Milano,, Italy, 20100
U.O. Pediatria II, A.O. "Villa Sofia"
Palermo, Italy
U.O. "Ematologia II con Talassemia" A.O. " V. Cervello
Palermo, Italy
Centro Microcitemia, A.O. B.M.M.
Reggio Calabria, Italy
Sponsors and Collaborators
Ente Ospedaliero Ospedali Galliera
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
Ospedale "Perrino" Brindisi
A.O. B.M.M., Reggio Calabria
Azienda Ospedaliera Villa Sofia
Azienda Ospedaliera V. Cervello
Università di Ferrara
Second University of Naples
Investigators
Principal Investigator: 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
  More Information

No publications provided by Ente Ospedaliero Ospedali Galliera

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Dr. Gian Luca Forni and Prof. Ernesto Palummeri, Centro della Microcitemia e delle Anemia Congenite - Ematologia and S.C. Geriatria
ClinicalTrials.gov Identifier: NCT01140321     History of Changes
Other Study ID Numbers: 60/2003/Nerixia
Study First Received: June 7, 2010
Last Updated: June 8, 2010
Health Authority: Italy: The Italian Medicines Agency

Keywords provided by Ente Ospedaliero Ospedali Galliera:
Thalassemia
Neridronate
Osteopenia
Osteoporosis

Additional relevant MeSH terms:
Hematologic Diseases
Genetic Diseases, Inborn
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Beta-Thalassemia
Osteoporosis
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hemoglobinopathies

ClinicalTrials.gov processed this record on July 23, 2014