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

Phase 4

Completed

• Conditions: Thalassemia

• Registration Number: NCT00346242
• Lead Sponsor: Novartis Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2004-03
• Primary Completion: 2006-03
• Study First Submitted: 2006-06-27
• Study First Submitted QC: 2006-06-27
• Study First Posted: 2006-06-29
• Status Verified: 2012-04
• Last Update Submitted: 2012-04-29
• Last Update Posted: 2012-05-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

• 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).

Exclusion Criteria

Other protocol-defined inclusion / exclusion criteria may apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇬🇷 Athens, Greece