Osteogenesis ImperfectaPhase 2NCT05312697
Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta
A Phase 2 study testing Setrusumab in people with Osteogenesis Imperfecta, run by Ultragenyx Pharmaceutical Inc at 1 site in United States.
Trial summary
The primary objective of the study is to evaluate bone mineral density (BMD) after 12 months of retreatment with monthly setrusumab in adults with osteogenesis imperfecta (OI). Read more about this study
- Who is it for?
- Ages 19 to 80 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
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Apr 2022to 7 Jul 2022
- 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 19 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males or females who participated in the Phase 2b ASTEROID study
- Females of childbearing potential must consent to use highly effective contraception during the Observation, Retreatment, and Extension Periods through 2 months after the last dose of setrusumab and agree not to become pregnant
Who cannot join
- Known hypersensitivity to setrusumab or its excipients that, in the judgment of the Investigator, places the participant 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
- Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
- Not willing or able to discontinue bisphosphonates 6 months prior to the Retreatment Screening Visit
- Once enrolled in UX143-CL203, individuals who meet any of the following exclusion criteria will not be eligible to participate in the Retreatment Period:
- Concurrent participation in any study that is examining the safety and efficacy of any investigational product or investigational medical device that alters bone health during the Retreatment and Extension Periods, per discretion of the Investigator in consultation with the Medical Monitor.
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- Calcium levels outside the normal range at the Retreatment Screening Visit. If the participant's calcium level is not within the normal range, decision to retest should be made in conjunction with the Medical Monitor
- Glomerular filtration rate (GFR) ≤ 29 mL/min at the Retreatment Screening Visit. If the participant's GFR is ≤ 29 mL/min, decision to retest should be made in conjunction with the Medical Monitor
- History of skeletal malignancies or bone metastases at any time
- History of neural foraminal stenosis (except if due to scoliosis)
- History of myocardial infarction, angina pectoris, ischemic stroke or transient ischemic attack (Investigators should consider whether the potential benefits of treatment outweigh the potential risks in patients with other cardiovascular risk factors such as hypertension, hyperlipidemia, familial hyperlipidemia, family history of premature ischemic cardiovascular disease, smoking, diabetes mellitus, and metabolic syndrome.)
- History of or concomitant uncontrolled diseases such as hypo-/hyperparathyroidism, hypo-/hyperthyroidism, Paget's disease, abnormal thyroid function or thyroid disease or other endocrine disorders or conditions that could affect bone metabolism eg, Stage IV/V renal disease
- A history of rickets or osteomalacia or any skeletal condition (other than OI) leading to long-bone deformities and/or increased risk of fractures
- Documented alcohol and/or drug abuse within 12 months prior to dosing or evidence of such abuse as indicated by the laboratory results during the screening/baseline assessments
- Documented history of significant psychiatric or medical disorder that would prevent the participant complying with the requirements of the protocol or would make it unsafe for the participant to participate in the study as judged by the investigator
- Current/previously reported allergy to the study drug or any of its excipients or the class of drug under investigation
- History of external radiation
Trial locations and contacts
1 site in 1 country.
United States1 site
New Mexico Clinical Research & Osteoporosis Center
Albuquerque, New Mexico, 87106
About this study
Official title: A Phase 2b, Multicenter, Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta
The primary objective of the study is to evaluate bone mineral density (BMD) after 12 months of retreatment with monthly setrusumab in adults with osteogenesis imperfecta (OI).
Read the detailed description
UX143-CL203 is a long-term extension study in adults with OI who participated in the ASTEROID study (also referred to as MBPS205) [NCT03118570]. The UX143-CL203 study comprises Observation, Retreatment, and Extension Periods. There is no intervention during the Observation Period. During the single-arm Retreatment Period, participants receive open-label setrusumab once a month (QM) for 12 months. Following the Retreatment Period, participants enter an Extension Period in which they will continue treatment with open-label setrusumab at different dosing frequencies.
- 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 UX143, BPS804
A fully human anti-sclerostin monoclonal antibody (mAb)
| Study group | Role | What they receive |
|---|---|---|
| Setrusumab QM -->Setrusumab QM | Experimental | During Retreatment Period: Setrusumab will be administered via intravenous (IV) infusion once a month (QM) for 12 months.
During Extension Period: Setrusumab will be administered via IV infusion QM for at least 12 months or until commercially available. Receives: Biological: Setrusumab |
| Setrusumab QM --> Setrusumab TBD | Experimental | During Retreatment Period: Setrusumab will be administered via IV infusion QM for 12 months.
During Extension Period: Setrusumab will be administered via IV infusion at a dose frequency to be determined (TBD) for at least 12 months or until commercially available. Receives: Biological: Setrusumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Change From Retreatment Baseline in Lumbar Spine Bone Mineral Density (BMD) Measured by Dual-Energy X-Ray Absorptiometry (DXA) After 12 Months of Setrusumab
Measured: Retreatment Baseline, Month 12
Secondary outcomes (2)
Percentage Change From Retreatment Baseline in Total Hip BMD Measured by DXA at Month 12 of the Retreatment Period
Measured: Retreatment Baseline, Month 12
Annualized New Fracture Rate as Confirmed by Radiograph During the Retreatment Period
Measured: Up to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2022
- Study start28 Apr 2022
- Primary completion7 Jul 2022
- Study completion7 Jul 2022
Why it stopped: Sponsor decision not related to safety concerns
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.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 2 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, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05312697
- Sponsor's protocol ID
- UX143-CL203
- Status last verified
- Jul 2025
- First submitted
- 28 Mar 2022
- Last updated
- 23 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- 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 23 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b, Multicenter, Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta
- ClinicalTrials.gov ID
- NCT05312697
- Acronym / sponsor ID
- UX143-CL203
- Conditions
- Osteogenesis Imperfecta
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision not related to safety concerns
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Collaborators
- Mereo BioPharma
- Study start
- 28 Apr 2022 (actual)
- Primary completion
- 7 Jul 2022 (actual)
- Study completion
- 7 Jul 2022 (actual)
- First posted
- 5 Apr 2022 (actual)
- Results
- Posted 23 Jul 2025
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 bone mineral density (BMD) after 12 months of retreatment with monthly setrusumab in adults with osteogenesis imperfecta (OI).
Detailed description
UX143-CL203 is a long-term extension study in adults with OI who participated in the ASTEROID study (also referred to as MBPS205) [NCT03118570]. The UX143-CL203 study comprises Observation, Retreatment, and Extension Periods. There is no intervention during the Observation Period. During the single-arm Retreatment Period, participants receive open-label setrusumab once a month (QM) for 12 months. Following the Retreatment Period, participants enter an Extension Period in which they will continue treatment with open-label setrusumab at different dosing frequencies.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Setrusumab QM -->Setrusumab QM | Experimental | Biological: SetrusumabDuring Retreatment Period: Setrusumab will be administered via intravenous (IV) infusion once a month (QM) for 12 months. During Extension Period: Setrusumab will be administered via IV infusion QM for at least 12 months or until commercially available. |
| Setrusumab QM --> Setrusumab TBD | Experimental | Biological: SetrusumabDuring Retreatment Period: Setrusumab will be administered via IV infusion QM for 12 months. During Extension Period: Setrusumab will be administered via IV infusion at a dose frequency to be determined (TBD) for at least 12 months or until commercially available. |
Who may qualify
- Ages
- 19 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Males or females who participated in the Phase 2b ASTEROID study
- Females of childbearing potential must consent to use highly effective contraception during the Observation, Retreatment, and Extension Periods through 2 months after the last dose of setrusumab and agree not to become pregnant
Key exclusion criteria (17 listed)
- Known hypersensitivity to setrusumab or its excipients that, in the judgment of the Investigator, places the participant 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
- Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
- Not willing or able to discontinue bisphosphonates 6 months prior to the Retreatment Screening Visit
- Once enrolled in UX143-CL203, individuals who meet any of the following exclusion criteria will not be eligible to participate in the Retreatment Period:
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: UX143, BPS804A fully human anti-sclerostin monoclonal antibody (mAb) | Biological | Setrusumab QM --> Setrusumab TBD, Setrusumab QM -->Setrusumab QM |
Primary outcome
Percentage Change From Retreatment Baseline in Lumbar Spine Bone Mineral Density (BMD) Measured by Dual-Energy X-Ray Absorptiometry (DXA) After 12 Months of Setrusumab
Time frame: Retreatment Baseline, Month 12
2 secondary outcomes
Percentage Change From Retreatment Baseline in Total Hip BMD Measured by DXA at Month 12 of the Retreatment Period
Time frame: Retreatment Baseline, Month 12
Annualized New Fracture Rate as Confirmed by Radiograph During the Retreatment Period
Time frame: Up to Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Ultragenyx Patient Advocacy/OI Disease InformationLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Osteogenesis Imperfecta
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Cardiopulmonary Outcomes in Osteogenesis Imperfecta: BBD7708
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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
Ultragenyx Pharmaceutical Inc
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Setrusumab in people with Osteogenesis Imperfecta, run by Ultragenyx Pharmaceutical Inc at 1 site in United States.
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 2 inclusion and 17 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: stopped early, as of the sponsor's last update on 23 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 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 23 Jul 2025). 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?
Percentage Change From Retreatment Baseline in Lumbar Spine Bone Mineral Density (BMD) Measured by Dual-Energy X-Ray Absorptiometry (DXA) After 12 Months of Setrusumab (time frame: Retreatment Baseline, Month 12). 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 23 Jul 2025.