Phase 3
N=143
Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children
Osteogenesis Imperfecta
Bottom Line
View on ClinicalTrials.gov: NCT00106028 ↗Enrolled (actual)
143
Serious AEs
17.2%
Results posted
Jun 2010
Primary outcome: Primary: Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 12, ITT Population — 7.592; 16.159 Percent Change — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- risedronate sodium (Actonel) (Drug); Placebo (Drug)
- Age
- Pediatric · 4+ yrs
- Sex
- All
- Sponsor
- Warner Chilcott
- Primary completion
- Apr 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 12, ITT Population |
7.592; 16.159 | <0.0001 sig |
| SECONDARY Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 24, ITT Population |
21.316; 25.754 | 0.0625 |
| SECONDARY Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 36, ITT Population |
33.216; 34.753 | 0.6103 |
| SECONDARY Percent Change From Baseline in Total Body BMD at Month 12, ITT Population |
4.252; 5.806 | 0.0808 |
| SECONDARY Percent Change From Baseline in Total Body BMD at Month 24, ITT Population |
9.716; 10.214 | 0.6466 |
| SECONDARY Percent Change From Baseline in Total Body BMD at Month 36, ITT Population |
13.540; 13.076 | 0.7391 |
| SECONDARY Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 12, ITT Population |
17.885; 28.218 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 24, ITT Population |
42.367; 48.407 | 01789 |
| SECONDARY Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 36, ITT Population |
68.054; 68.333 | 0.9646 |
| SECONDARY Percent Change From Baseline in Total Body BMC at Month 12, ITT Population |
16.483; 21.977 | 0.0030 sig |
| SECONDARY Percent Change From Baseline in Total Body BMC at Month 24, ITT Population |
36.465; 37.938 | 0.6106 |
| SECONDARY Percent Change From Baseline in Total Body BMC at Month 36, ITT Population |
56.211; 56.526 | 0.9372 |
| SECONDARY Lumbar Spine Z-score - Percent Change From Baseline to Month 12, ITT Population |
-6.028; 25.648 | <0.0001 sig |
| SECONDARY Lumbar Spine Z-score - Percent Change From Baseline to Month 24, ITT Population |
15.608; 29.637 | 0.0793 |
| SECONDARY Lumbar Spine Z-score - Percent Change From Baseline to Month 36, ITT Population |
19.325; 25.640 | 0.4575 |
| SECONDARY Total Body Z-score- Percent Change From Baseline to Month 12, ITT Population |
-20.661; 16.933 | 0.0172 sig |
| SECONDARY Total Body Z-score- Percent Change From Baseline to Month 24, ITT Population |
8.371; 7.879 | 0.9786 |
| SECONDARY Total Body Z-score- Percent Change From Baseline to Month 36, ITT Population |
7.146; -1.494 | 0.5971 |
| SECONDARY Percent Change From Baseline in Lumbar Spine Bone Area at Month 12, ITT Population |
8.803; 9.817 | 0.4154 |
| SECONDARY Percent Change From Baseline in Lumbar Spine Bone Area at Month 24, ITT Population |
16.381; 17.266 | 0.6404 |
| SECONDARY Percent Change From Baseline in Lumbar Spine Bone Area at Month 36, ITT Population |
24.952; 23.292 | 0.4978 |
| SECONDARY Percent Change From Baseline in Total Body Bone Area Month 12, ITT Population |
11.405; 14.939 | 0.0270 sig |
| SECONDARY Percent Change From Baseline in Total Body Bone Area Month 24, ITT Population |
24.051; 25.116 | 0.5925 |
| SECONDARY Percent Change From Baseline in Total Body Bone Area Month 36, ITT Population |
37.109; 38.303 | 0.6840 |
| SECONDARY New Morphometric Vertebral Fracture at Month 12, ITT Population |
8; 28; 40; 60 | 0.0680 |
| SECONDARY New Morphometric Vertebral Fracture at Month 36, ITT Population |
14; 20; 31; 62 | 0.4121 |
| SECONDARY Categorization by Number of New Morphometric Vertebral Fracture at Month 12, ITT |
40; 60; 3; 17; 2; 8 | 0.3658 |
| SECONDARY Categorization by Number of New Morphometric Vertebral Fracture at Month 36, ITT |
31; 62; 6; 10; 5; 7 | 0.3408 |
| SECONDARY Incidence New Vertebral Fractures by SQ (Semi-Quantitative) Score, Patients Aged 4-9 Years, Month 12, ITT Population |
7; 19; 19; 32; 7; 17 | — |
| SECONDARY Incidence New Vertebral Fractures by SQ Score, Patients Aged 10-15 Years, Month 12, ITT Population |
1; 10; 1; 13; 1; 10 | — |
| SECONDARY Probability of Fracture in 12 Months (Kaplan-Meier Cumulative Incidence), ITT Population |
24; 29; 0.5043; 0.314; 0; 0 | 0.0253 sig |
| SECONDARY Number of Clinical Fractures, Month 12, ITT Population |
38; 42; 24; 29; 0; 0 | 0.0416 sig |
| SECONDARY Serum BAP - Percent Change From Baseline to Month 12, ITT Population |
6.783; -4.895 | 0.0318 sig |
| SECONDARY Serum BAP - Percent Change From Baseline to Month 24, ITT Population |
-11.196; -11.128 | 0.9907 |
| SECONDARY Serum BAP - Percent Change From Baseline to Month 36, ITT Population |
-19.884; -24.570 | 0.3826 |
| SECONDARY Urine NTX/Cr - Percent Change From Baseline at Month 12, ITT Population |
-14.556; -41.185 | <0.0001 sig |
| SECONDARY Urine NTX/Cr - Percent Change From Baseline at Month 24, ITT Population |
-40.358; -31.318 | 0.3075 |
| SECONDARY Urine NTX/Cr - Percent Change From Baseline at Month 36, ITT Population |
-47.570; -52.609 | 0.3998 |
| SECONDARY Wong-Baker FACES Pain Rating Scale - Change From Baseline to Month 12, ITT Population |
-0.056; -0.409 | 0.1592 |
| SECONDARY Bone Age (Years), Change From Baseline to Month 12, ITT Population |
0.956; 1.083 | 0.2917 |
| SECONDARY Bone Age (Years), Change From Baseline to Month 24, ITT Population |
2.147; 2.155 | 0.9622 |
| SECONDARY Bone Age (Years), Change From Baseline to Month 36, ITT Population |
3.087; 3.096 | 0.9596 |
| SECONDARY Annualized Growth Velocity - Change From Baseline to Month 12, ITT Population |
0.895; 1.002 | 0.3400 |
| SECONDARY Annualized Growth Velocity - Change From Baseline to Month 36, ITT Population |
0.982; 1.011 | 0.6218 |
Summary
Children with Osteogenesis Imperfecta (OI) have bone pain, low bone mass and fractures. There are no approved drugs for the treatment of OI in children, even though some intravenous (IV) bisphosphonates are used off-label in some countries. In a single dose, pharmacokinetic study, data showed that risedronate was well tolerated in 28 children with OI. This three year study will test the safety and efficacy of risedronate in the treatment of children with OI. For the first year, patients will be randomized to the risedronate and placebo groups in a 2:1 ratio. For the second and third years of the study, all patients will receive risedronate.
Eligibility Criteria
Inclusion Criteria
- OI diagnosis
- increased risk of fracture: either has a history of at least 1 radiographically confirmed, non-traumatic or low impact fracture plus low bone mineral density (BMD) or has very low BMD with or without a history of fractures.
Exclusion Criteria
- Any bisphosphonate use within one year of enrollment
Data sourced from ClinicalTrials.gov (NCT00106028). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.