Trial · NCT03118570
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
NCT03118570, Phase 2, Completed, Ultragenyx Pharmaceutical Inc; results first posted 2022-03-02
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804. Trials, record NCT03118570. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03118570
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
- NCT number
- NCT03118570
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 112 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- Osteogenesis Imperfecta, Type I, Osteogenesis Imperfecta Type III, Osteogenesis Imperfecta Type IV
- Interventions
- setrusumab, Calcium, Vitamin D, zoledronic acid (optional)
- First posted
- 2017-04-18
- Last updated
- 2023-07-05
- Other registries' IDs
- eudract:2016-005096-27
- Results first posted
- 2022-03-02
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 | Baseline, Month 12 (end of treatment [EOT]) | Assessed by high resolution peripheral quantitative computed tomography (HRpQCT). HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presents the ratio of the means between the visit and Baseline from analysis of covariance (ANCOVA). |
| Primary | Change From Baseline in Radial Bone Strength (Failure Load) at Month 12 | Baseline, Month 12 (EOT) | Assessed by finite element analysis (FEA) of models generated from HRpQCT images of the distal radius. |
| Primary | Change From Baseline in Radial Bone Strength (Stiffness) at Month 12 | Baseline, Month 12 (EOT) | Assessed by FEA of models generated from HRpQCT images of the distal radius. |
| Secondary | Change From Baseline in Radial and Tibial Tr VBMD Over Time: Full Analysis Set | Baseline, Months 6, 12 (EOT), 18, 24 | Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA. |
| Secondary | Changes From Baseline in Radial and Tibial Tr VBMD at Months 6 and 12: Open-Label Arm | Baseline, Months 6, 12 (EOT) | Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA. |
| Secondary | Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) Over Time: Full Analysis Set | Baseline, Months 6, 12 (EOT), 18, 24 | Assessed by FEA of models generated from HRpQCT images of the distal radius. |
| Secondary | Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) at Months 6 and 12: Open-Label Arm | Baseline, Months 6, 12 (EOT) | Assessed by FEA of models generated from HRpQCT images of the distal radius. |
| Secondary | Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) Over Time: Full Analysis Set | Baseline, Months 6, 12 (EOT), 18, 24 | Assessed by FEA of models generated from HRpQCT images of the distal radius. |
| Secondary | Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) at Months 6 and 12: Open-Label Arm | Baseline, Months 6, 12 (EOT) | Assessed by FEA of models generated from HRpQCT images of the distal radius. |
| Secondary | Percentage of Participants With at Least 1 New Fracture (Peripheral, Vertebral, Long-Bone, Any) at Month 12 | Month 12 (EOT) | Fracture assessment, confirmed by central radiographic reading, was carried out for peripheral including all major long bones, minor bone (digits, ribs) and vertebral fractures. Fractures without clinical symptoms, detected only by means of radiographic investigations, were not included in the analysis. |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 | Baseline, Month 12 (end of treatment [EOT]) | Full Analysis Set: all participants who are randomized to one of the blinded treatment arms and took at least 1 dose of study treatment (per protocol almost all efficacy endpoints were to be analyzed only in the blinded treatment arms). Participants with an assessment at given time point. | ratio | NUMBER |
| PRIMARY | Change From Baseline in Radial Bone Strength (Failure Load) at Month 12 | Baseline, Month 12 (EOT) | Full Analysis Set: all participants who are randomized to one of the blinded treatment arms and took at least 1 dose of study treatment. Participants with an assessment at given time point. | newton (N) | LEAST_SQUARES_MEAN |
| PRIMARY | Change From Baseline in Radial Bone Strength (Stiffness) at Month 12 | Baseline, Month 12 (EOT) | Full Analysis Set: all participants who are randomized to one of the blinded treatment arms and took at least 1 dose of study treatment. Participants with an assessment at given time point. | N/mm | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Radial and Tibial Tr VBMD Over Time: Full Analysis Set | Baseline, Months 6, 12 (EOT), 18, 24 | Full Analysis Set: all participants who are randomized to one of the blinded treatment arms and took at least 1 dose of study treatment. Participants with an assessment at given time point. | ratio | NUMBER |
| SECONDARY | Changes From Baseline in Radial and Tibial Tr VBMD at Months 6 and 12: Open-Label Arm | Baseline, Months 6, 12 (EOT) | All participants in the open-label arm who took at least 1 dose of study treatment. Participants with an assessment at given time point. | ratio | NUMBER |
| SECONDARY | Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) Over Time: Full Analysis Set | Baseline, Months 6, 12 (EOT), 18, 24 | Full Analysis Set: all participants who are randomized to one of the blinded treatment arms and took at least 1 dose of study treatment. Participants with an assessment at given time point. | N | LEAST_SQUARES_MEAN |
| SECONDARY | Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) at Months 6 and 12: Open-Label Arm | Baseline, Months 6, 12 (EOT) | All participants in the open-label arm who took at least 1 dose of study treatment. Participants with an assessment at given time point. | N | LEAST_SQUARES_MEAN |
| SECONDARY | Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) Over Time: Full Analysis Set | Baseline, Months 6, 12 (EOT), 18, 24 | Full Analysis Set: all participants who are randomized to one of the blinded treatment arms and took at least 1 dose of study treatment. Participants with an assessment at given time point. | N/mm | LEAST_SQUARES_MEAN |
| SECONDARY | Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) at Months 6 and 12: Open-Label Arm | Baseline, Months 6, 12 (EOT) | All participants in the open-label arm who took at least 1 dose of study treatment. Participants with an assessment at given time point. | N/mm | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants With at Least 1 New Fracture (Peripheral, Vertebral, Long-Bone, Any) at Month 12 | Month 12 (EOT) | Full Analysis Set: all participants who are randomized to one of the blinded treatment arms and took at least 1 dose of study treatment. | percentage of participants | NUMBER |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group335
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 | Setrusumab 20 mg/kg (Blinded) | 1.004 | 95% Confidence Interval 0.987 to 1.021 | ratio | |
| Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 | Setrusumab 8 mg/kg (Blinded) | 0.993 | 95% Confidence Interval 0.975 to 1.012 | ratio | |
| Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 | Setrusumab 2 mg/kg (Blinded) | 0.992 | 95% Confidence Interval 0.974 to 1.011 | ratio | |
| Change From Baseline in Radial Bone Strength (Failure Load) at Month 12 | Setrusumab 20 mg/kg (Blinded) | 61.25 | Standard Error 21.669 | newton (N) | |
| Change From Baseline in Radial Bone Strength (Failure Load) at Month 12 | Setrusumab 8 mg/kg (Blinded) | 32.25 | Standard Error 24.342 | newton (N) | |
| Change From Baseline in Radial Bone Strength (Failure Load) at Month 12 | Setrusumab 2 mg/kg (Blinded) | 8.86 | Standard Error 23.200 | newton (N) | |
| Change From Baseline in Radial Bone Strength (Stiffness) at Month 12 | Setrusumab 20 mg/kg (Blinded) | 1638.70 | Standard Error 625.808 | N/mm | |
| Change From Baseline in Radial Bone Strength (Stiffness) at Month 12 | Setrusumab 8 mg/kg (Blinded) | 1422.00 | Standard Error 703.275 | N/mm | |
| Change From Baseline in Radial Bone Strength (Stiffness) at Month 12 | Setrusumab 2 mg/kg (Blinded) | 209.89 | Standard Error 671.777 | N/mm | |
| Change From Baseline in Radial and Tibial Tr VBMD Over Time: Full Analysis Set | Setrusumab 20 mg/kg (Blinded) | Radial: Month 6 | 1.007 | 95% Confidence Interval 0.993 to 1.021 | ratio |
1 to 10 of 335Source ClinicalTrials.gov
See all 335 results by groupParticipant flow20
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Setrusumab 20 mg/kg (Blinded) | 31 | |
| Overall Study | STARTED | Setrusumab 8 mg/kg (Blinded) | 29 | |
| Overall Study | STARTED | Setrusumab 2 mg/kg (Blinded) | 30 | |
| Overall Study | STARTED | Setrusumab 20 mg/kg (Open-Label) | 2 | |
| Overall Study | STARTED | Placebo | 20 | |
| Overall Study | Reassigned From Placebo to 20 mg/kg Open-Label Setrusumab | Setrusumab 20 mg/kg (Blinded) | 0 | |
| Overall Study | Reassigned From Placebo to 20 mg/kg Open-Label Setrusumab | Setrusumab 8 mg/kg (Blinded) | 0 | |
| Overall Study | Reassigned From Placebo to 20 mg/kg Open-Label Setrusumab | Setrusumab 2 mg/kg (Blinded) | 0 | |
| Overall Study | Reassigned From Placebo to 20 mg/kg Open-Label Setrusumab | Setrusumab 20 mg/kg (Open-Label) | 0 | |
| Overall Study | Reassigned From Placebo to 20 mg/kg Open-Label Setrusumab | Placebo | 19 |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 participant flowAdverse events630
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Setrusumab 20 mg/kg (Blinded) | 1 | 31 | |
| other | Anaemia | Blood and lymphatic system disorders | Setrusumab 8 mg/kg (Blinded) | 0 | 29 | |
| other | Anaemia | Blood and lymphatic system disorders | Setrusumab 2 mg/kg (Blinded) | 2 | 30 | |
| other | Anaemia | Blood and lymphatic system disorders | Setrusumab 20 mg/kg (Open-Label) | 2 | 21 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo | 0 | 20 | |
| other | Increased tendency to bruise | Blood and lymphatic system disorders | Setrusumab 20 mg/kg (Blinded) | 0 | 31 | |
| other | Increased tendency to bruise | Blood and lymphatic system disorders | Setrusumab 8 mg/kg (Blinded) | 0 | 29 | |
| other | Increased tendency to bruise | Blood and lymphatic system disorders | Setrusumab 2 mg/kg (Blinded) | 0 | 30 | |
| other | Increased tendency to bruise | Blood and lymphatic system disorders | Setrusumab 20 mg/kg (Open-Label) | 0 | 21 | |
| other | Increased tendency to bruise | Blood and lymphatic system disorders | Placebo | 1 | 20 |
1 to 10 of 630Source ClinicalTrials.gov
See all 630 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Setrusumab 20 mg/kg (Blinded) | 0 | 31 |
| Total, all-cause mortality | Setrusumab 8 mg/kg (Blinded) | 0 | 29 |
| Total, all-cause mortality | Setrusumab 2 mg/kg (Blinded) | 0 | 30 |
| Total, all-cause mortality | Setrusumab 20 mg/kg (Open-Label) | 0 | 21 |
| Total, all-cause mortality | Placebo | 0 | 20 |
| Total, other adverse events | Setrusumab 20 mg/kg (Blinded) | 29 | 31 |
| Total, other adverse events | Setrusumab 8 mg/kg (Blinded) | 28 | 29 |
| Total, other adverse events | Setrusumab 2 mg/kg (Blinded) | 27 | 30 |
| Total, other adverse events | Setrusumab 20 mg/kg (Open-Label) | 19 | 21 |
| Total, other adverse events | Placebo | 16 | 20 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39012717 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03118570
GET https://conductscience.org/api/trials/v1/NCT03118570/registered?page=2