Thalassemia
Conditions
Keywords
thalassemia, zoledronic acid, skeletal events
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.
Interventions
DRUGZoledronic Acid
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
SINGLE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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 /
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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. | — |
Countries
Greece
Outcome results
None listed