Evaluation of Efficacy of Zoledronic Acid in Patients With Haemoglobin Syndromes (Thalassemia and Sicle Cell Anaemia) and Risk of Skeletal Events

NCT00346242 | Completed Phase 4

• 1 other identifier: CZOL446EGR05
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

The evaluation of efficacy is performed by laboratory monitoring of bone density and resorption markers and clinical monitoring of bone density improvement. This is a prospective, randomized, parallel group, single blind study of one year treatment with zoledronic acid every 6 months as compared to one year treatment with zoledronic acid every 3 months and to placebo every 3 months in patients with hemoglobin syndromes and risk of skeletal complications.

Conditions

Thalassemia

Keywords

thalassemia; zoledronic acid; skeletal events

Outcome Measures

Primary Outcomes (3)

• Bone density by means of bone densitometry with dual energy x-ray absorptiometry (DEXA) at three body sites (lumbar region, femoral head, wrist) prior to treatment initiation the start and at the end of treatment

• N-telopeptide of type I collagen (NTx) levels, TRACP-5b, with ELISA, prior to the start of treatment initiation and every 3 or 6 months

• Serum Bone alkaline phosphatase, Osteocalcin, OPG and S-RANKL with ELISA, prior to the start of treatment initiation, and every 3 or 6 months

Secondary Outcomes (1)

• Biochemistry (Ca, P, urea/creatinine, SGOT/SGPT, ALT, γGT), endocrine and cardiac test prior to the start of treatment initiation and at 6 and 12 month.

Interventions

Zoledronic Acid DRUG

Eligibility Criteria

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

You may qualify if:

• Patients with thalassaemia or sickle cell anaemia and bone density, z-scores \< -1.5, in at least one of the examined sites
• Normal renal function. Urea and creatinine should not exceed \> 2-fold the upper limit of normal (\< 2 x ULN).

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis Investigative Site

Athens, Greece

Location

Related Publications (1)

• Voskaridou E, Christoulas D, Xirakia C, Varvagiannis K, Boutsikas G, Bilalis A, Kastritis E, Papatheodorou A, Terpos E. Serum Dickkopf-1 is increased and correlates with reduced bone mineral density in patients with thalassemia-induced osteoporosis. Reduction post-zoledronic acid administration. Haematologica. 2009 May;94(5):725-8. doi: 10.3324/haema-tol.2008.000893.

  PMID: 19407319 DERIVED

MeSH Terms

Conditions

Thalassemia

Interventions

Zoledronic Acid

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Diphosphonates; Organophosphonates; Organophosphorus Compounds; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Novartis

  Novartis

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: SINGLE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 27, 2006
• First Posted: June 29, 2006
• Study Start: March 1, 2004
• Primary Completion: March 1, 2006
• Last Updated: May 1, 2012

Record last verified: 2012-04

Locations

GR (1)