Overview of study design
Design aspect . | Description . |
|---|---|
| General design | Double-blind, 2-arm; alendronate versus placebo |
| Duration | Up to 24 months |
| Study interval | 6 months |
| Treatment | Alendronate: 40 mg once per day versus placebo |
| Vitamin D: 400 U once per day | |
| Calcium carbonate (Tums): 1.5 g once per day | |
| Key inclusion criteria | Gaucher disease, on ERT more than 24 months |
| Older than 18 years and less than 50 years of age | |
| Lumbar spine Z score less than — 1 | |
| Key exclusion criteria | Pregnancy |
| Prior use of antiresorptive agents | |
| Active gastric or duodenal ulcer disease | |
| Endpoints | |
| Primary | Changes in lumber spine BMD in g/cm2 |
| Reduction in focal changes in long-bone x-rays | |
| Secondary | Reduction of biochemical indices of bone turnover |
Design aspect . | Description . |
|---|---|
| General design | Double-blind, 2-arm; alendronate versus placebo |
| Duration | Up to 24 months |
| Study interval | 6 months |
| Treatment | Alendronate: 40 mg once per day versus placebo |
| Vitamin D: 400 U once per day | |
| Calcium carbonate (Tums): 1.5 g once per day | |
| Key inclusion criteria | Gaucher disease, on ERT more than 24 months |
| Older than 18 years and less than 50 years of age | |
| Lumbar spine Z score less than — 1 | |
| Key exclusion criteria | Pregnancy |
| Prior use of antiresorptive agents | |
| Active gastric or duodenal ulcer disease | |
| Endpoints | |
| Primary | Changes in lumber spine BMD in g/cm2 |
| Reduction in focal changes in long-bone x-rays | |
| Secondary | Reduction of biochemical indices of bone turnover |