Osteogenesis ImperfectaPhase 3NCT06636071
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
A Phase 3 study testing setrusumab in people with Osteogenesis Imperfecta, run by Ultragenyx Pharmaceutical Inc at 3 sites in Japan.
Trial summary
The primary objective of the study is to evaluate the effect of setrusumab on reduction in fracture rate, including morphometric vertebral fractures. Read more about this study
- Who is it for?
- Ages 2 to 6 years
- Conditions the study is for
- Osteogenesis Imperfecta
- What is being tested?
- setrusumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 3 sitesJapan
- Study dates
- 25 Oct 2024to Jan 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 2 to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Clinical diagnosis of OI Type I, III, or IV confirmed by identification of genetic mutation in COL1A1 or COL1A2
- History of ≥ 1 fracture in the past 12 months, ≥ 2 fractures in the past 24 months, or ≥ 1 femur, tibia, or humerus fracture in the past 24 months
- Any prior exposure to, or currently receiving, IV-bisphosphonate therapy for treatment of OI
- Serum 25-hydroxyvitamin D level ≥ 20 ng/mL at the Screening visit. If 25- hydroxyvitamin D levels are below 20 ng/mL, the subject may be rescreened after a minimum of 14 days of vitamin D supplementation as directed by the Investigator
Who cannot join
- History of skeletal malignancies or bone metastases at any time
- History of neural foraminal stenosis (except if due to scoliosis)
- Clinical manifestations of Chiari malformation or basilar invagination. Presence of any other neurologic disease that has been clinically unstable within past 2 years requires review by the Medical Monitor.
- History of or current uncontrolled concomitant diseases that may impact bone metabolism, such as hypo/hyperparathyroidism, abnormal thyroid function, nephrotic syndrome, or Stage IV/V renal disease
- Any skeletal condition (other than OI) leading to bone deformity and/or increased risk of fractures, such as rickets, osteopetrosis, idiopathic juvenile osteoporosis, or skeletal dysplasia
- History of known cardiovascular disease such as coronary artery anomaly, Kawasaki disease, myocarditis, cardiomyopathy, myocardial infarction, stroke, or thromboembolic disease. Individuals with other congenital or acquired cardiovascular disease necessitating an echocardiogram require Medical Monitor review. Investigators should consider whether the potential benefits of treatment outweigh the potential risks in patients with cardiovascular risk factors such as confirmed arterial hypertension.
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- Hypocalcemia, defined as serum calcium levels below the age-adjusted normal limit reference ranges after a recommended ≥ 4 hour fast, at Screening
- Estimated glomerular filtration rate ≤ 35 mL/min/1.73 m2 at Screening
- Prior treatment with growth hormone, denosumab, anti-sclerostin antibody, or other anabolic or anti-resorptive medications impacting the bone (other than bisphosphonates) at any time
- History of external radiation therapy
- Known hypersensitivity to setrusumab or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results
- Use of any investigational product or investigational medical device within 4 weeks or 5 half-lives (whichever is longer) of investigational drug prior to Screening, or during the study (per discretion of the Investigator in consultation with the Medical Monitor)
- Concurrent participation in another clinical study without prior approval from the study Medical Monitor
- Pregnant or nursing
Trial locations and contacts
3 sites in 1 country.
Japan3 sites
Osaka Metropolitan University Hospital
Osaka
Osaka University Hospital
Osaka
Keio University Hospital
Tokyo
About this study
Official title: An Open-label Phase 3 Study of Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta Type I, III, or IV
The primary objective of the study is to evaluate the effect of setrusumab on reduction in fracture rate, including morphometric vertebral fractures.
Read the detailed description
This study will be conducted in Japan only and consists of a screening period, an open-label treatment period, and an open-label extension period. Participants will receive setrusumab for up to 24 months during the open-label Treatment Period followed by continued setrusumab treatment during the open-label Extension Period. All participants will receive setrusumab until it becomes commercially available in Japan, consent is withdrawn, or the study is otherwise discontinued.
- Conditions
- Osteogenesis Imperfecta
- Being tested
- setrusumab
- Medical subject headings
- Osteogenesis Imperfecta
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
setrusumabBiological
Also called BPS804, UX143
A fully human sclerostin neutralizing monoclonal antibody (mAb) administered once a month (QM) via intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| Setrusumab | Experimental | Not described Receives: Biological: setrusumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Rate of All Radiographically-Confirmed Fractures, Including Morphometric Vertebral Fractures During the Treatment Period
Measured: Up to Month 24
Secondary outcomes (8)
Annualized Rate of Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures, but Including Fractures of the Fingers, Toes, Face, and Skull During the Treatment Period
Measured: Up to Month 24
Annualized Rate of All Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures and Fractures of the Fingers, Toes, Face, and Skull During the Treatment Period
Measured: Up to Month 24
Change from Baseline in Dual-Energy X-Ray Absorptiometry (DXA) Bone Mineral Density (BMD) Z-Score at the Lumbar Spine During the Treatment Period
Measured: Baseline, Up to Month 24
Percent Change from Baseline in DXA BMD at the Lumbar Spine During the Treatment Period
Measured: Baseline, Up to Month 24
Proportion of Participants Experiencing New Radiographically-Confirmed Fractures, Including Morphometric Vertebral Fractures, at the Primary Analysis
Measured: Up to Month 24
Serum Setrusumab Concentration Over Time
Measured: Up to Month 24
Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESIs), Frequency, Severity and Relationship to Treatment
Measured: Up to Month 24
Incidence of Binding and Neutralizing Anti-Setrusumab Antibodies Over Time
Measured: Baseline, Up to Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2024
- Study start25 Oct 2024
- Primary completionJan 2027 (estimated)
- Study completionJan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Osteogenesis ImperfectaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ultragenyx Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06636071
- Sponsor's protocol ID
- UX143-CL307
- Status last verified
- Sep 2026
- First submitted
- 8 Oct 2024
- Last updated
- 28 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Phase 3 Study of Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta Type I, III, or IV
- ClinicalTrials.gov ID
- NCT06636071
- Acronym / sponsor ID
- UX143-CL307
- Conditions
- Osteogenesis Imperfecta
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 25 Oct 2024 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jan 2028 (estimated)
- First posted
- 10 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study is to evaluate the effect of setrusumab on reduction in fracture rate, including morphometric vertebral fractures.
Detailed description
This study will be conducted in Japan only and consists of a screening period, an open-label treatment period, and an open-label extension period. Participants will receive setrusumab for up to 24 months during the open-label Treatment Period followed by continued setrusumab treatment during the open-label Extension Period. All participants will receive setrusumab until it becomes commercially available in Japan, consent is withdrawn, or the study is otherwise discontinued.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Setrusumab | Experimental | Biological: setrusumab |
Who may qualify
- Ages
- 2 to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Clinical diagnosis of OI Type I, III, or IV confirmed by identification of genetic mutation in COL1A1 or COL1A2
- History of ≥ 1 fracture in the past 12 months, ≥ 2 fractures in the past 24 months, or ≥ 1 femur, tibia, or humerus fracture in the past 24 months
- Any prior exposure to, or currently receiving, IV-bisphosphonate therapy for treatment of OI
- Serum 25-hydroxyvitamin D level ≥ 20 ng/mL at the Screening visit. If 25- hydroxyvitamin D levels are below 20 ng/mL, the subject may be rescreened after a minimum of 14 days of vitamin D supplementation as directed by the Investigator
Key exclusion criteria (15 listed)
- History of skeletal malignancies or bone metastases at any time
- History of neural foraminal stenosis (except if due to scoliosis)
- Clinical manifestations of Chiari malformation or basilar invagination. Presence of any other neurologic disease that has been clinically unstable within past 2 years requires review by the Medical Monitor.
- History of or current uncontrolled concomitant diseases that may impact bone metabolism, such as hypo/hyperparathyroidism, abnormal thyroid function, nephrotic syndrome, or Stage IV/V renal disease
- Any skeletal condition (other than OI) leading to bone deformity and/or increased risk of fractures, such as rickets, osteopetrosis, idiopathic juvenile osteoporosis, or skeletal dysplasia
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: BPS804, UX143A fully human sclerostin neutralizing monoclonal antibody (mAb) administered once a month (QM) via intravenous (IV) infusion | Biological | Setrusumab |
Primary outcome
Annualized Rate of All Radiographically-Confirmed Fractures, Including Morphometric Vertebral Fractures During the Treatment Period
Time frame: Up to Month 24
8 secondary outcomes
Annualized Rate of Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures, but Including Fractures of the Fingers, Toes, Face, and Skull During the Treatment Period
Time frame: Up to Month 24
Annualized Rate of All Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures and Fractures of the Fingers, Toes, Face, and Skull During the Treatment Period
Time frame: Up to Month 24
Change from Baseline in Dual-Energy X-Ray Absorptiometry (DXA) Bone Mineral Density (BMD) Z-Score at the Lumbar Spine During the Treatment Period
Time frame: Baseline, Up to Month 24
Percent Change from Baseline in DXA BMD at the Lumbar Spine During the Treatment Period
Time frame: Baseline, Up to Month 24
Proportion of Participants Experiencing New Radiographically-Confirmed Fractures, Including Morphometric Vertebral Fractures, at the Primary Analysis
Time frame: Up to Month 24
Serum Setrusumab Concentration Over Time
Time frame: Up to Month 24
Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESIs), Frequency, Severity and Relationship to Treatment
Time frame: Up to Month 24
Incidence of Binding and Neutralizing Anti-Setrusumab Antibodies Over Time
Time frame: Baseline, Up to Month 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2026.
- Ultragenyx Osteogenesis Imperfecta (OI) Research Study OpportunityLink provided by the sponsor.
- Our Clinical Trial Transparency Commitment-UltragenyxLink provided by the sponsor.
- 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.
Ultragenyx Medical Director study director
Ultragenyx Pharmaceutical Inc
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing setrusumab in people with Osteogenesis Imperfecta, run by Ultragenyx Pharmaceutical Inc at 3 sites in Japan.
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 4 inclusion and 15 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: under way, not looking for participants, as of the sponsor's last update on 28 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Japan. 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Annualized Rate of All Radiographically-Confirmed Fractures, Including Morphometric Vertebral Fractures During the Treatment Period (time frame: Up to Month 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Sep 2026; AzTrials last refreshed its copy on 8 Nov 2026.