Osteogenesis Imperfecta, Type IPhase 2AsteroidNCT03118570
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
A Phase 2 study testing setrusumab, Calcium and Vitamin D in people with Osteogenesis Imperfecta, Type I and Osteogenesis Imperfecta Type III, run by Ultragenyx Pharmaceutical Inc at 25 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Osteogenesis Imperfecta, Type I, Osteogenesis Imperfecta Type III, Osteogenesis Imperfecta Type IV
- What is being tested?
- setrusumab, Calcium and Vitamin D
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 112 participants
- Where
- 25 sitesin 5 countries
- Study dates
- 11 Sep 2017to 12 Nov 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
25 sites in 5 countries.
25 sites in 5 countries
United States12 sites
Mereo Investigator Site
Albuquerque, New Mexico, 87106
Mereo Investigator Site
Baltimore, Maryland, 21287
Mereo Investigator Site
Birmingham, Alabama, 35294
Mereo Investigator Site
Boston, Massachusetts, 012115
Mereo Investigator Site
Cincinnati, Ohio, 45229
Mereo Investigator Site
Houston, Texas, 77030
Mereo Investigator Site
Jacksonville, Florida, 32207
Mereo Investigator Site
Nashville, Tennessee, 37232
Mereo Investigator Site
Pittsburgh, Pennsylvania, 15225
Mereo Investigator Site
Portland, Oregon, 97239
Mereo Investigator Site
Saint Paul, Minnesota, 55101
Mereo Investigator Site
St Louis, Missouri, 63110
United Kingdom5 sites
Mereo Investigator Site
Bristol
Mereo Investigator Site
Cambridge, Cambridgeshire
Mereo Investigator Site
London
Mereo Investigator Site
Newcastle upon Tyne, Newcastle
Mereo Investigator Site
Oxford, Oxfordshire
Canada3 sites
Mereo Investigator Site
Montreal, Quebec
Mereo Investigator Site
Québec, Quebec
Mereo Investigator Site
Toronto, Ontario
France3 sites
Mereo Investigator Site
Lyon
Mereo Investigator Site
Paris, Paris Cedex 14
Mereo Investigator Site
Paris
About this study
Official title: A Phase 2b, Multicentre, Multinational, Double-blind, Dose-finding Study, Incorporating an Open Label Substudy, in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With Setrusumab (BPS804)
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.
Read the detailed description
This study was previously posted by Mereo Biopharma and was transferred to Ultragenyx in February 2021.
- Conditions
- Osteogenesis Imperfecta, Type I, Osteogenesis Imperfecta Type III, Osteogenesis Imperfecta Type IV
- Being tested
- setrusumab, Calcium, Vitamin D, zoledronic acid (optional)
- Keywords
- Osteogenesis Imperfecta, Brittle Bone Disease
- Medical subject headings
- Osteogenesis Imperfecta, Osteogenesis imperfecta, type 3
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
setrusumabDrug
Also called BPS804
Intravenous infusion
CalciumDietary supplement
tablets
Vitamin DDietary supplement
capsules
zoledronic acid (optional)Drug
Following completion of the study treatment (Month 12) participants can receive an optional single dose of zoledronic acid. Participants can receive an optional further dose of zoledronic acid at Month 18 at the discretion of their treating physician.
| Study group | Role | What they receive |
|---|---|---|
| Setrusumab 20 mg/kg (Blinded) | Experimental | Setrusumab 20 mg/kg intravenous (IV) infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. Receives: Drug: setrusumab; Dietary Supplement: Calcium; Dietary Supplement: Vitamin D; Drug: zoledronic acid (optional) |
| Setrusumab 8 mg/kg (Blinded) | Experimental | Setrusumab 8 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. Receives: Drug: setrusumab; Dietary Supplement: Calcium; Dietary Supplement: Vitamin D; Drug: zoledronic acid (optional) |
| Setrusumab 2 mg/kg (Blinded) | Experimental | Setrusumab 2 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. Receives: Drug: setrusumab; Dietary Supplement: Calcium; Dietary Supplement: Vitamin D; Drug: zoledronic acid (optional) |
| Setrusumab 20 mg/kg (Open-Label) | Experimental | Setrusumab 20 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. Receives: Drug: setrusumab; Dietary Supplement: Calcium; Dietary Supplement: Vitamin D; Drug: zoledronic acid (optional) |
| Placebo | Placebo comparator | Placebo IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. Receives: Dietary Supplement: Calcium; Dietary Supplement: Vitamin D |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12
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).
Measured: Baseline, Month 12 (end of treatment [EOT])
Change From Baseline in Radial Bone Strength (Failure Load) at Month 12
Assessed by finite element analysis (FEA) of models generated from HRpQCT images of the distal radius.
Measured: Baseline, Month 12 (EOT)
Change From Baseline in Radial Bone Strength (Stiffness) at Month 12
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Measured: Baseline, Month 12 (EOT)
Secondary outcomes (25)
Change From Baseline in Radial and Tibial Tr VBMD Over Time: Full Analysis Set
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.
Measured: Baseline, Months 6, 12 (EOT), 18, 24
Changes From Baseline in Radial and Tibial Tr VBMD at Months 6 and 12: Open-Label Arm
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.
Measured: Baseline, Months 6, 12 (EOT)
Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) Over Time: Full Analysis Set
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Measured: Baseline, Months 6, 12 (EOT), 18, 24
Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) at Months 6 and 12: Open-Label Arm
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Measured: Baseline, Months 6, 12 (EOT)
Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) Over Time: Full Analysis Set
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Measured: Baseline, Months 6, 12 (EOT), 18, 24
Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) at Months 6 and 12: Open-Label Arm
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Measured: Baseline, Months 6, 12 (EOT)
Percentage of Participants With at Least 1 New Fracture (Peripheral, Vertebral, Long-Bone, Any) at Month 12
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.
Measured: Month 12 (EOT)
Change From Baseline in Lumbar, Total Body, and Femoral Neck Bone Mineral Density (BMD) T-score at Month 6
BMD was evaluated by dual-energy x-ray absorptiometry (DXA). T-Score was calculated based on actual measured bone density value. T-scores are standardized scores that reflect the standard deviations (SDs) above/below the normal mean for young adults. A score of 50 indicates the population mean with a standard deviation of 10. A positive change in DXA T-score indicates an improvement in BMD.
Measured: Baseline, Month 6
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 6
BMD was evaluated by DXA.
Measured: Baseline, Month 6
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD T-score at Month 12
BMD was evaluated by DXA. T-Score was calculated based on actual measured bone density value. T-scores are standardized scores that reflect the standard deviations (SDs) above/below the normal mean for young adults. A score of 50 indicates the population mean with a standard deviation of 10. A positive change in DXA T-score indicates an improvement in BMD.
Measured: Baseline, Month 12 (EOT)
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 12
BMD was evaluated by DXA.
Measured: Baseline, Month 12 (EOT)
Change From Baseline in Total vBMD (Radial and Tibial) Over Time
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.
Measured: Baseline, Months 6, 12 (EOT), 18, and 24
Change From Baseline in Cortical vBMD (Radial and Tibial) Over Time
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.
Measured: Baseline, Months 6, 12 (EOT), 18, and 24
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
Measured: Baseline, Month 6, Month 12 (EOT)
Change From Baseline in Lean and Fat Body Mass From Whole Body at Months 6 and 12
Lean and fat body mass was evaluated using whole body DXA (including the head).
Measured: Baseline, Months 6, 12 (EOT)
Change From Baseline in Amino-Terminal Propeptide of Type 1 Procollagen (P1NP) up to Month 12
Measured: Baseline, Months 1, 3, 6, 9, 12 (EOT)
Change From Baseline in Carboxy-Terminal Telo-Peptide [CTX-1] up to Month 12
Measured: Baseline, Months 1, 3, 6, 9, 12 (EOT)
Change From Baseline in Short Form 12 Health Survey (SF-12) Physical Component Summary Score at Months 6 and 12
The SF-12 is a generic, 12-item survey that measures 8 domains of health: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems, and mental health. It yields scale scores for each of these 8 domains and 2 summary measures of physical and mental health: The Physical Component Summary and the Mental Component Summary. The total score for the Physical Component Summary ranges from 0 to 100, where higher scores reflect better physical functioning.
Measured: Baseline, Months 6, 12 (EOT)
Change From Baseline in SF-12 Mental Component Summary Score at Months 6 and 12
The SF-12 is a generic, 12-item survey that measures 8 domains of health: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems, and mental health. It yields scale scores for each of these 8 domains and 2 summary measures of physical and mental health: The Physical Component Summary and the Mental Component Summary. The total score for the Mental Component Summary ranges from 0 to 100, where higher scores reflect better mental health functioning.
Measured: Baseline, Months 6, 12 (EOT)
Change From Baseline in Index (Utility) Score on EuroQol 5-Dimension 5-Level Descriptive System (EQ-5D-5L) Score at Months 6 and 12
The EQ-5D-5L is a standardised measure of health status comprised of a descriptive system of 5 health-related quality of life states (i.e., mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) and a Visual Analogue Scale (VAS) of overall health. Each dimension is rated on a 5-point response scale indicating severity of problems, where 1 is "no problems" and 5 is "extreme problems". The 5 questions are scored and together contribute to the EQ-5D index (utility) score between 0 and 1 (1 being perfect health).
Measured: Baseline, Months 6 and 12 (EOT)
Change From Baseline in Osteogenesis Imperfecta Specific Quality of Life Questionnaire for Adults (OIQoL-A) Total Score at Months 6 and 12
The OIQoL-A measures 5 areas of quality of life related to OI (Physical Function, Pain, Hearing Loss, Taking Care/Concerns, Social and Family Life and Activities). The total score is calculated on a 0-100 scale, where higher scores indicate a greater (negative) impact on quality of life.
Measured: Baseline, Months 6, 12 (EOT)
Change From Baseline in OIQoL-A Pain Subscale Score at Months 6 and 12
The OIQoL-A measures 5 areas of quality of life related to OI (Physical Function, Pain, Hearing Loss, Taking Care/Concerns, Social and Family Life and Activities). The Pain subscale ranges from 0 to 10, with higher value representing worse pain.
Measured: Baseline, Months 6, 12 (EOT)
Change From Baseline in OIQoL-A Activity Subscale Score at Months 6 and 12
The OIQoL-A measures 5 areas of quality of life related to OI (Physical Function, Pain, Hearing Loss, Taking Care/Concerns, Social and Family Life and Activities). The Activities subscale ranges from 0 to 100, with higher value representing increased difficulty.
Measured: Baseline, Months 6, 12 (EOT)
Percentage of Participants Who Were Positive for Anti-Setrusumab Antibodies at Any Time During the Study up to Month 14
Serum samples were screened for antibodies binding to setrusumab using a validated assay method by or under the supervision of the sponsor.
Measured: up to Month 14
Percentage of Participants With Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation or Death
An AE is any untoward medical occurrence, which does not necessarily have a causal relationship with treatment. A serious AE (SAE) is defined as any untoward medical occurrence that, at any dose: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent disability/incapacity; is a congenital anomaly/birth defect; is another important medical event. The intensity for each AE was graded as mild, moderate or severe, according to the investigator's judgement. An event was considered related to study drug if there were a "reasonable possibility" of a relationship, according to the investigator's clinical judgment. A TEAE was defined as an event occurring or worsening on or after the first dose of study medication.
Measured: Non-serious AEs: up to Month 14; Serious AEs: up to Month 24. (Average duration of exposure to placebo was 5 months and for setrusumab was 11 month plus follow-up to 24 months.)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2017
- Study start11 Sep 2017
- Primary completion1 Oct 2019
- Study completion12 Nov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Osteogenesis Imperfecta, Type IStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double-blind, Dose-finding Study, incorporating an open-label substudy
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 112 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Mereo BioPharma
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Mereo BioPharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03118570
- Sponsor's protocol ID
- MBPS205
- Other IDs
- 2016-005096-27, 2016
- Acronym
- Asteroid
- Status last verified
- Feb 2022
- First submitted
- 3 Apr 2017
- Last updated
- 5 Jul 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Glorieux FH, Langdahl B, Chapurlat R, De Beur SJ, Sutton VR, Poole KES, Dahir KM, Orwoll ES, Willie BM, Mikolajewicz N, Zimmermann E, Hosseinitabatabaei S, Ominsky MS, Saville C, Clancy J, MacKinnon A, Mistry A, Javaid MK. Setrusumab for the treatment of osteogenesis imperfecta: 12-month results from the phase 2b asteroid study. J Bone Miner Res. 2024 Sep 2;39(9):1215-1228. doi: 10.1093/jbmr/zjae112.
PubMed 39012717
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b, Multicentre, Multinational, Double-blind, Dose-finding Study, Incorporating an Open Label Substudy, in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With Setrusumab (BPS804)
- ClinicalTrials.gov ID
- NCT03118570
- Acronym / sponsor ID
- Asteroid / MBPS205
- Conditions
- Osteogenesis Imperfecta, Type I, Osteogenesis Imperfecta Type III, Osteogenesis Imperfecta Type IV
- Keywords
- Osteogenesis Imperfecta, Brittle Bone Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 112 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Collaborators
- Mereo BioPharma
- Study start
- 11 Sep 2017 (actual)
- Primary completion
- 1 Oct 2019 (actual)
- Study completion
- 12 Nov 2020 (actual)
- First posted
- 18 Apr 2017 (actual)
- Results
- Posted 2 Mar 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study was previously posted by Mereo Biopharma and was transferred to Ultragenyx in February 2021.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
- Model description
- Double-blind, Dose-finding Study, incorporating an open-label substudy
| Arm | Type | Receives |
|---|---|---|
| Setrusumab 20 mg/kg (Blinded) | Experimental | Drug: setrusumab, Dietary Supplement: Calcium, Dietary Supplement: Vitamin D, Drug: zoledronic acid (optional)Setrusumab 20 mg/kg intravenous (IV) infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. |
| Setrusumab 8 mg/kg (Blinded) | Experimental | Drug: setrusumab, Dietary Supplement: Calcium, Dietary Supplement: Vitamin D, Drug: zoledronic acid (optional)Setrusumab 8 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. |
| Setrusumab 2 mg/kg (Blinded) | Experimental | Drug: setrusumab, Dietary Supplement: Calcium, Dietary Supplement: Vitamin D, Drug: zoledronic acid (optional)Setrusumab 2 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. |
| Setrusumab 20 mg/kg (Open-Label) | Experimental | Drug: setrusumab, Dietary Supplement: Calcium, Dietary Supplement: Vitamin D, Drug: zoledronic acid (optional)Setrusumab 20 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. |
| Placebo | Placebo comparator | Dietary Supplement: Calcium, Dietary Supplement: Vitamin DPlacebo IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| setrusumabAlso: BPS804Intravenous infusion | Drug | Setrusumab 2 mg/kg (Blinded), Setrusumab 20 mg/kg (Blinded), Setrusumab 20 mg/kg (Open-Label), Setrusumab 8 mg/kg (Blinded) |
| Calciumtablets | Dietary supplement | Placebo, Setrusumab 2 mg/kg (Blinded), Setrusumab 20 mg/kg (Blinded), Setrusumab 20 mg/kg (Open-Label), Setrusumab 8 mg/kg (Blinded) |
| Vitamin Dcapsules | Dietary supplement | Placebo, Setrusumab 2 mg/kg (Blinded), Setrusumab 20 mg/kg (Blinded), Setrusumab 20 mg/kg (Open-Label), Setrusumab 8 mg/kg (Blinded) |
| zoledronic acid (optional)Following completion of the study treatment (Month 12) participants can receive an optional single dose of zoledronic acid. Participants can receive an optional further dose of zoledronic acid at Month 18 at the discretion of their treating physician. | Drug | Setrusumab 2 mg/kg (Blinded), Setrusumab 20 mg/kg (Blinded), Setrusumab 20 mg/kg (Open-Label), Setrusumab 8 mg/kg (Blinded) |
Primary outcome
Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12
Time frame: 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).
Change From Baseline in Radial Bone Strength (Failure Load) at Month 12
Time frame: Baseline, Month 12 (EOT)
Assessed by finite element analysis (FEA) of models generated from HRpQCT images of the distal radius.
Change From Baseline in Radial Bone Strength (Stiffness) at Month 12
Time frame: Baseline, Month 12 (EOT)
Assessed by FEA of models generated from HRpQCT images of the distal radius.
25 secondary outcomes
Change From Baseline in Radial and Tibial Tr VBMD Over Time: Full Analysis Set
Time frame: Baseline, Months 6, 12 (EOT), 18, 24
Changes From Baseline in Radial and Tibial Tr VBMD at Months 6 and 12: Open-Label Arm
Time frame: Baseline, Months 6, 12 (EOT)
Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) Over Time: Full Analysis Set
Time frame: Baseline, Months 6, 12 (EOT), 18, 24
Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) at Months 6 and 12: Open-Label Arm
Time frame: Baseline, Months 6, 12 (EOT)
Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) Over Time: Full Analysis Set
Time frame: Baseline, Months 6, 12 (EOT), 18, 24
Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) at Months 6 and 12: Open-Label Arm
Time frame: Baseline, Months 6, 12 (EOT)
Percentage of Participants With at Least 1 New Fracture (Peripheral, Vertebral, Long-Bone, Any) at Month 12
Time frame: Month 12 (EOT)
Change From Baseline in Lumbar, Total Body, and Femoral Neck Bone Mineral Density (BMD) T-score at Month 6
Time frame: Baseline, Month 6
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 6
Time frame: Baseline, Month 6
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD T-score at Month 12
Time frame: Baseline, Month 12 (EOT)
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 12
Time frame: Baseline, Month 12 (EOT)
Change From Baseline in Total vBMD (Radial and Tibial) Over Time
Time frame: Baseline, Months 6, 12 (EOT), 18, and 24
Change From Baseline in Cortical vBMD (Radial and Tibial) Over Time
Time frame: Baseline, Months 6, 12 (EOT), 18, and 24
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
Time frame: Baseline, Month 6, Month 12 (EOT)
Change From Baseline in Lean and Fat Body Mass From Whole Body at Months 6 and 12
Time frame: Baseline, Months 6, 12 (EOT)
Change From Baseline in Amino-Terminal Propeptide of Type 1 Procollagen (P1NP) up to Month 12
Time frame: Baseline, Months 1, 3, 6, 9, 12 (EOT)
Change From Baseline in Carboxy-Terminal Telo-Peptide [CTX-1] up to Month 12
Time frame: Baseline, Months 1, 3, 6, 9, 12 (EOT)
Change From Baseline in Short Form 12 Health Survey (SF-12) Physical Component Summary Score at Months 6 and 12
Time frame: Baseline, Months 6, 12 (EOT)
Change From Baseline in SF-12 Mental Component Summary Score at Months 6 and 12
Time frame: Baseline, Months 6, 12 (EOT)
Change From Baseline in Index (Utility) Score on EuroQol 5-Dimension 5-Level Descriptive System (EQ-5D-5L) Score at Months 6 and 12
Time frame: Baseline, Months 6 and 12 (EOT)
Change From Baseline in Osteogenesis Imperfecta Specific Quality of Life Questionnaire for Adults (OIQoL-A) Total Score at Months 6 and 12
Time frame: Baseline, Months 6, 12 (EOT)
Change From Baseline in OIQoL-A Pain Subscale Score at Months 6 and 12
Time frame: Baseline, Months 6, 12 (EOT)
Change From Baseline in OIQoL-A Activity Subscale Score at Months 6 and 12
Time frame: Baseline, Months 6, 12 (EOT)
Percentage of Participants Who Were Positive for Anti-Setrusumab Antibodies at Any Time During the Study up to Month 14
Time frame: up to Month 14
Percentage of Participants With Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation or Death
Time frame: Non-serious AEs: up to Month 14; Serious AEs: up to Month 24. (Average duration of exposure to placebo was 5 months and for setrusumab was 11 month plus follow-up to 24 months.)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jul 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39012717Glorieux FH, Langdahl B, Chapurlat R, De Beur SJ, Sutton VR, Poole KES, Dahir KM, Orwoll ES, Willie BM, Mikolajewicz N, Zimmermann E, Hosseinitabatabaei S, Ominsky MS, Saville C, Clancy J, MacKinnon A, Mistry A, Javaid MK. Setrusumab for the treatment of osteogenesis imperfecta: 12-month results from the phase 2b asteroid study. J Bone Miner Res. 2024 Sep 2;39(9):1215-1228. doi: 10.1093/jbmr/zjae112.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Mereo BioPharma
25 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing setrusumab, Calcium and Vitamin D in people with Osteogenesis Imperfecta, Type I and Osteogenesis Imperfecta Type III, run by Ultragenyx Pharmaceutical Inc at 25 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, United Kingdom, Canada, France, Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Mar 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 (time frame: Baseline, Month 12 (end of treatment [EOT])). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03118570, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jul 2023.