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. Author manuscript; available in PMC: 2013 Jan 25.
Published in final edited form as: Lancet. 2011 Mar 28;377(9773):1276–1287. doi: 10.1016/S0140-6736(10)62349-5

Table 3. Study programs involving investigational osteoporosis drugs.

Trial n Primary endpoint Main results Ref.
DENOSUMAB
Phase 1
Single-dose placebo-controlled study in postmenopausal women 49 Biochemical markers of bone resorption after 6 months vs. placebo Urinary NTX decreased by 81% (3 mg/kg denosumab) vs. −10% with placebo (p<0.001) Bekker50
Phase 2
Efficacy and safety in postmenopausal women with low bone mineral density 412 % change from baseline BMD at the lumbar spine after 12 months vs. placebo Lumbar spine BMD +3.0% to +6.7% vs. −0.6% with placebo (p<0.001) McClung51
Phase 3
Treatment of postmenopausal osteoporosis (FREEDOM) 7,868 Reduction of new vertebral fractures after 36 months vs. placebo Vertebral fractures decreased by 68% (RR: 0.32, 95%CI 0.26–0.41); hip fractures decreased by 40% (RR: 0.60, 95%CI 0.37–0.97) Cummings55
Prevention of postmenopausal osteoporosis 332 % change from baseline BMD at the lumbar spine after 24 months vs. placebo Lumbar spine BMD +6.5% vs. −0.6% with placebo (p<0.0001) Bone57
Comparison with alendronate in postmenopausal Women with low bone mineral density (DECIDE) 1,189 % change from baseline BMD at the total hip after 12 months vs. alendronate Total hip BMD +3.5% vs. +2.6% with alendronate (p<0.0001) Brown56
Treatment of bone loss in men on androgen-deprivation therapy for non-metastatic prostate cancer (HALT) 1,468 % change from baseline BMD at the lumbar spine after 24 months Lumbar spine BMD +5.6% vs. −1.0 with placebo (p<0.001). After 36 months, vertebral fractures decreased by 62% (RR: 0.38; 95%CI 0.19–0.78) Smith61
Treatment of bone loss in women on aromatase inhibitors for non-metastatic breast cancer 252 % change from baseline BMD at the lumbar spine after 12 months Lumbar spine BMD +5.5% vs. placebo (p<0.0001) Ellis60
ODANACATIB
Phase 1
Multiple oral doses in healthy adults 62 Safety and tolerability No increase in adverse events; serum CTX decreased by 62% (50 or 100 mg per week) BSAP and osteocalcin remained unaffected Stoch64
Once-weekly doses in healthy adult females 78
Phase 2
Treatment of postmenopausal osteoporosis 399 % change from baseline BMD at the lumbar spine after 24 months Lumbar spine BMD +5.5% vs. −0.2% with placebo Bone65
Phase 3
Treatment of postmenopausal osteoporosis 16,716 Vertebral, hip, and clinical non-clinical fractures after 36 months Expected to be completed in July 2012 NCT00529373
ONO-5334
Postmenopausal women with low BMD 265 % change from baseline BMD at the lumbar spine after 12 months Completed in October 2009, results pending NCT 00532337
SARACATINIB
Phase 1
Multiple oral doses on bone turnover in healthy men 59 Effect on bone turnover of multiple daily oral dosing for up to 24 days Serum CTX −88% (95%CI 84–91%) vs. +17% with placebo (p<0.001); PINP +13 vs. +17% with placebo (p=NS) Hannon67
MK-5442 (Calcilytic agent)
Phase 2
Dose-ranging study in postmenopausal osteoporosis 384 % change from baseline BMD at the lumbar spine after 12 months Expected to be completed in February 2012 NCT00960934
Postmenopausal osteoporosis previously treated with alendronate 480 % change from baseline BMD at the lumbar spine vs. alendronate after 12 months Expected to be completed in August 2012 NCT00996801
AMG 785 (Antibody against sclerostin)
Phase 1
Healthy men and postmenopausal women 74 Safety No increase in adverse events; after 21 days 3 mg/kg increased PINP, osteocalcin and BSAP by 60 to 100% NCT01059435
Phase 2
Postmenopausal women with low BMD (vs. alendronate and teriparatide) 419 % change from baseline BMD at the lumber spine after 12 months Expected to be completed in August 2012 NCT00896532
BHQ 880 (Antibody against dickkopf-1)
Phase 1/2
Combination with zoledronic acid in relapsed or refractory myeloma patients 267 Time to skeletal-related event; SRE, changes in bone resorption and formation markers after 9 months Expected to be completed in November 2010 NCT00741377

Abbreviations: BSAP, bone-specific alkaline phosphatase; CTX, C-terminal telopeptide of type I collagen; NTX, N-terminal telopeptide of type I collagen; PINP, serum procollagen propeptide of type I collagen.