Efficacy and Safety of Neridronate (Nerixia®)to Treat Osteoporosis in Patients With TM and TI
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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. |
| 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 |
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| 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 | |
| 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:
|
Beta-Thalassemia Osteoporosis Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia |
Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Bone Diseases, Metabolic Bone Diseases Musculoskeletal Diseases |
ClinicalTrials.gov processed this record on May 22, 2013