Phase 2
N=112
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
Osteogenesis Imperfecta, Type I · Osteogenesis Imperfecta Type III · Osteogenesis Imperfecta Type IV
Bottom Line
View on ClinicalTrials.gov: NCT03118570 ↗Enrolled (actual)
112
Serious AEs
19.1%
Results posted
Mar 2022
Primary outcome: Primary: Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 — 1.004; 0.993; 0.992 ratio — p=0.644
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- setrusumab (Drug); Calcium (Dietary_supplement); Vitamin D (Dietary_supplement); zoledronic acid (optional) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ultragenyx Pharmaceutical Inc
- Primary completion
- Oct 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 |
1.004; 0.993; 0.992 | 0.644 |
| PRIMARY Change From Baseline in Radial Bone Strength (Failure Load) at Month 12 |
61.25; 32.25; 8.86 | 0.006 sig |
| PRIMARY Change From Baseline in Radial Bone Strength (Stiffness) at Month 12 |
1638.70; 1422.00; 209.89 | 0.011 sig |
| SECONDARY Change From Baseline in Radial and Tibial Tr VBMD Over Time: Full Analysis Set |
1.007; 1.000; 0.998; 1.004; 0.993; 0.992 | 0.329 |
| SECONDARY Changes From Baseline in Radial and Tibial Tr VBMD at Months 6 and 12: Open-Label Arm |
0.994; 1.011; 1.013; 1.035 | 0.615 |
| SECONDARY Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) Over Time: Full Analysis Set |
31.22; 39.95; -3.28; 61.25; 32.25; 8.86 | 0.064 |
| SECONDARY Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) at Months 6 and 12: Open-Label Arm |
110.16; 88.32; 69.78; 112.92 | < 0.001 sig |
| SECONDARY Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) Over Time: Full Analysis Set |
795.67; 1048.61; 109.65; 1638.70; 1422.00; 209.89 | 0.060 |
| SECONDARY Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) at Months 6 and 12: Open-Label Arm |
5056.51; 4992.82; 4225.92; 5827.81 | < 0.001 sig |
| SECONDARY Percentage of Participants With at Least 1 New Fracture (Peripheral, Vertebral, Long-Bone, Any) at Month 12 |
6.5; 17.2; 13.3; 0; 0; 0 | — |
| SECONDARY Change From Baseline in Lumbar, Total Body, and Femoral Neck Bone Mineral Density (BMD) T-score at Month 6 |
0.273; 0.338; 0.110; 0.072; 0.071; 0.122 | < 0.001 sig |
| SECONDARY Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 6 |
4.06; 4.70; 1.58; 0.77; 0.83; 1.21 | < 0.001 sig |
| SECONDARY Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD T-score at Month 12 |
0.587; 0.486; 0.174; 0.181; 0.199; 0.108 | < 0.001 sig |
| SECONDARY Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 12 |
8.55; 6.79; 2.50; 1.98; 2.03; 1.06 | < 0.001 sig |
| SECONDARY Change From Baseline in Total vBMD (Radial and Tibial) Over Time |
1.011; 0.995; 1.000; 1.017; 1.008; 0.999 | 0.065 |
| SECONDARY Change From Baseline in Cortical vBMD (Radial and Tibial) Over Time |
1.004; 1.002; 0.998; 1.005; 1.003; 1.001 | 0.285 |
| SECONDARY Number of Participants With Clinically Significant Changes From Baseline in Body Height, Weight and Body Mass Index (BMI) at 6 and 12 Months: Full Analysis Set |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Change From Baseline in Lean and Fat Body Mass From Whole Body at Months 6 and 12 |
519.152; -410.664; 168.206; 867.668; -225.474; 184.164 | 0.045 sig |
| SECONDARY Change From Baseline in Amino-Terminal Propeptide of Type 1 Procollagen (P1NP) up to Month 12 |
24.349; 14.288; 0.060; 17.903; 6.735; 0.640 | < 0.001 sig |
| SECONDARY Change From Baseline in Carboxy-Terminal Telo-Peptide [CTX-1] up to Month 12 |
-0.077; -0.047; -0.041; -0.044; -0.029; -0.043 | < 0.001 sig |
| SECONDARY Change From Baseline in Short Form 12 Health Survey (SF-12) Physical Component Summary Score at Months 6 and 12 |
0.672; -0.463; 1.342; -1.178; -0.994; 2.171 | 0.588 |
| SECONDARY Change From Baseline in SF-12 Mental Component Summary Score at Months 6 and 12 |
0.966; -0.492; -1.133; 2.807; -1.473; -1.664 | 0.527 |
| SECONDARY Change From Baseline in Index (Utility) Score on EuroQol 5-Dimension 5-Level Descriptive System (EQ-5D-5L) Score at Months 6 and 12 |
0.0627; 0.0362; -0.0383; 0.0424; 0.0252; 0.0214 | 0.092 |
| SECONDARY Change From Baseline in Osteogenesis Imperfecta Specific Quality of Life Questionnaire for Adults (OIQoL-A) Total Score at Months 6 and 12 |
-3.584; -1.848; -0.649; -1.668; -0.587; -3.846 | 0.150 |
| SECONDARY Change From Baseline in OIQoL-A Pain Subscale Score at Months 6 and 12 |
-3.990; -3.906; -6.003; -3.655; -4.968; -7.178 | 0.278 |
| SECONDARY Change From Baseline in OIQoL-A Activity Subscale Score at Months 6 and 12 |
-5.980; -2.722; 1.907; -0.964; 4.489; -1.630 | 0.037 sig |
| SECONDARY Percentage of Participants Who Were Positive for Anti-Setrusumab Antibodies at Any Time During the Study up to Month 14 |
16.1; 17.2; 16.7; 9.5; 15.0; 16.1 | — |
| SECONDARY Percentage of Participants With Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation or Death |
100.0; 96.6; 90.0; 100.0; 90.0; 100.0 | — |
Summary
The purpose of this study is to select a suitable dose of BPS804 by measuring the strength/quality of bone using a special type of CT scanner. Participants will be treated for 12 months and followed up for a further 12 months.
Eligibility Criteria
Inclusion Criteria
- Patients with a clinical diagnosis of OI Type I, III or IV with a confirmed defect in the COL1A1/COL1A2 genes, as confirmed by genetic testing
- One or more fractures in the past 5 years
- Capable of giving signed consent
Exclusion Criteria
- History of skeletal malignancies or other bone diseases (other than OI)
- History of neural foraminal stenosis (except if due to scoliosis)
- History of myocardial infarction, angina pectoris, ischaemic stroke or transient ischaemic attack
- History of endocrine or thyroid/parathyroid conditions that could affect bone metabolism
- Treatment with bisphosphonates within 3 months of randomisation
- Treatment with teraparatide, denosumab or other anabolic/anti-reabsorptive medications within 6 months of randomisation
Data sourced from ClinicalTrials.gov (NCT03118570). 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.