Osteogenesis ImperfectaPhase 2/3OrbitNCT05125809
Setrusumab vs Placebo for Osteogenesis Imperfecta
A Phase 2/3 study testing Setrusumab in people with Osteogenesis Imperfecta, run by Ultragenyx Pharmaceutical Inc at 43 sites in 11 countries.
Trial summary
The primary objectives of the study are to identify a setrusumab dosing strategy in participants with OI and to evaluate the effect of setrusumab vs placebo on reduction in fracture rate. Read more about this study
- Who is it for?
- Ages 5 to 25 years
- Conditions the study is for
- Osteogenesis Imperfecta
- What is being tested?
- Setrusumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 183 participants
- Where
- 43 sitesin 11 countries
- Study dates
- 21 Feb 2022to Apr 2027
- Phase
- Phase 2/3
- 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 5 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of OI Type I, III, or IV as confirmed by identification of pathogenic or likely pathogenic genetic variants in COL1A1 or COL1A2. If a variant of uncertain significance is identified, then clinical presence of the expected phenotype can be used to confirm the diagnosis
- ≥ 1 fracture in the past 12 months, ≥ 2 fractures in the past 24 months or ≥ 1 tibia, femur or humerus fracture in the past 24 months
- Serum 25-hydroxyvitamin D ≥ 20 ng/mL at the Screening Visit. If 25-hydroxyvitamin D levels are below 20 ng/mL, 25-hydroxyvitamin D testing can repeated after a minimum of 14 days of vitamin D supplementation as directed by the treating physician
- Willing to not receive bisphosphonate therapy during the study
- From the period following informed consent to 60 days after the last dose of the study drug, females of childbearing potential and fertile males must consent to use highly effective contraception. If female, agree not to become pregnant. If male, agree not to father a child or donate sperm
- Willing and able to provide informed consent for subjects greater than or equal to 18 years of age, or provide assent (if possible) and have a legally authorized representative provide informed consent, after the nature of the study has been explained and prior to any research-related procedures
- Willing to provide access to medical records for the collection of radiographic data, fracture data, growth data, and disease history
- Must, in the opinion of the Investigator, be willing and able to complete all aspects of the study, adhere to the study visit schedule, and comply with the assessments
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 unstable within past 2 years requires review by the Medical Monitor
- History of or uncontrolled concomitant diseases such as hypo/hyperparathyroidism, Paget's disease, abnormal thyroid function, thyroid disease or other endocrine disorders or conditions that could affect bone metabolism such as Stage IV/V renal disease
- Rickets or any skeletal condition (other than OI) leading to long-bone deformities and/or increased risk of fractures
- History of stroke, myocardial infarction, transient ischemic attack or angina.
Show all 20Show fewer
- Hypocalcemia, defined as serum calcium levels below the age-adjusted normal limits after a ≥ 4 hour fast
- Estimated glomerular filtration rate ≤ 29 mL/min/1.73 m2
- Prior treatment with the following:
- Teriparatide, growth hormone, bone anabolic, or anti-resorptive medications (other than bisphosphonates) within 6 months of the first dose with study drug (Month 0)
- Denosumab within 24 months of Screening
- Romosozumab at any time
- 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 assessments
- 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
- Known hypersensitivity to setrusumab or excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
- History of external radiation therapy
- Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
- Use of any investigational product or investigational medical device within 4 weeks or 5 half-lives of investigational drug (whichever is longer) 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 Investigator in consultation with the Medical Monitor
- For Phase 2 Only: A history of bone surgery within the previous 6 months prior to Screening or planned bone surgery for the first 3 months of the study
Trial locations and contacts
43 sites in 11 countries.
43 sites in 11 countries
United States22 sites
New Mexico Clinical Research & Osteoporosis Center, Inc.
Albuquerque, New Mexico, 87106
Children's Hospital Colorado
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Atrium Health Levine Children's Hospital
Charlotte, North Carolina, 28203
Shriners Hospitals for Children - Chicago
Chicago, Illinois, 60707
Nationwide Children's Hospital- Ohio State University College of Medicine
Columbus, Ohio, 43205
Cook Children's Medical Center
Fort Worth, Texas, 76104
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Baylor College of Medicine
Houston, Texas, 77030
Indiana University Hospital
Indianapolis, Indiana, 46202
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital Los Angeles
Los Angeles, California, 90027
UW Health University Hospital
Madison, Wisconsin, 53792
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Yale New Haven Hospital
New Haven, Connecticut, 06510
Children's Hospital and Medical Center
Omaha, Nebraska, 68114
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Shriners Hospital for Children - Northern California
Sacramento, California, 95817
Washington University School of Medicine
St Louis, Missouri, 63110
University of South Florida
Tampa, Florida, 33612
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Nemours/ Alfred i. duPoint Hospital for Children
Wilmington, Delaware, 19803
Canada4 sites
London Health Sciences Center
London, Ontario
McGill University Health Centre
Montreal, H4A 3J1
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
University of Toronto- The Hospital for Sick Children (SickKids)
Toronto
Germany3 sites
University of Cologne
Cologne
Otto von Guericke University Magdeburg
Magdeburg, 39106
Musculoskeletal Center Würzburg
Würzburg, 97074
Italy3 sites
Istituto Ortopedico Rizzoli
Bologna
Azienda Ospedaliera Universitaria Policlinico Umberto I
Rome, 00161
Universita Degli Studi Di Verona
Verona
Australia2 sites
Royal Children's Hospital
Melbourne
Queensland Paediatric Endocrinology
South Brisbane, Queensland, QLD 4101
Portugal2 sites
Hospital de Santa Maria
Lisbon, 1649-028
Centro Hospitalar do Porto
Porto, 4099-001
Turkey (Türkiye)2 sites
Gazi University
Ankara
Marmara University
Istanbul
United Kingdom2 sites
Royal Manchester Childrens Hospital
Manchester
Sheffield Children's NHS Foundation Trust
Sheffield, S10 2TH
Poland1 site
Uniwersytet Medyczny w Lodzi - Klinika Endokrynologii i Chorob Metabolicznych
Lodz
About this study
Official title: An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-Controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects With Osteogenesis Imperfecta
The primary objectives of the study are to identify a setrusumab dosing strategy in participants with OI and to evaluate the effect of setrusumab vs placebo on reduction in fracture rate.
Read the detailed description
Participants in Phase 2 will be randomized 1:1 to receive low dose or high dose setrusumab. Phase 2 participants will continue receiving their assigned dose of setrusumab until all Phase 2 participants have completed the Month 6 study visit. After this point, Phase 2 participants will begin receiving the selected dosing strategy in the Phase 2 open-label Treatment Extension Period. Phase 3 participants will be randomized 2:1 to receive setrusumab or placebo during the double-blind treatment period. Phase 3 participants will transition to the open-label Treatment Extension Period after the end of the double-blind period. Participants in the Phase 2 and Phase 3 treatment extension periods will receive open-label setrusumab treatment for at least 12 months, and have the option to remain in the open-label treatment period until setrusumab is commercially available in their region. An optional substudy will be conducted in approximately 10 participants (≥ 8 years) consisting of a bone biopsy to investigate the impact of setrusumab on bone histomorphology.
- Conditions
- Osteogenesis Imperfecta
- Being tested
- Setrusumab, Placebo
- 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
PlaceboOther
A 5% dextrose/glucose solution administered QM via IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Low Dose Setrusumab -> Open-Label (OL) Setrusumab Selected Dose | Experimental | Single-blind setrusumab low dose during Phase 2 followed by open-label setrusumab selected dose
During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician Receives: Biological: Setrusumab |
| High Dose Setrusumab -> OL Setrusumab Selected Dose | Experimental | Single-blind setrusumab high dose during Phase 2 followed by open-label setrusumab
During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician Receives: Biological: Setrusumab |
| Setrusumab Selected Dose -> OL Setrusumab Selected Dose | Experimental | Double-blind setrusumab selected dose during Phase 3 followed by open-label setrusumab
During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician Receives: Biological: Setrusumab |
| Placebo -> OL Setrusumab Selected Dose | Placebo comparator | Double-blind placebo during Phase 3 followed by open-label setrusumab
During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician Receives: Biological: Setrusumab; Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 2: Percent Change in Serum Amino-terminal Propeptide of Type 1 Procollagen (P1NP) from Baseline at Month 1
Measured: Baseline, Month 1
Phase 3: Annualized Rate of All Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures and Fractures of the Fingers, Toes, Face, and Skull at the Primary Analysis
Measured: Up to Month 24
Secondary outcomes (18)
Phase 2: Serum Setrusumab Concentration
Measured: From Predose up to Month 24
Phase 2: Baseline-Corrected Area Under the Effect Curve (AUEC) for Serum P1NP Over a 1 and 2-Month Period
Measured: Baseline, Up to Month 2
Phase 2: Percent Change from Baseline Over Time in Bone Turnover Marker: P1NP
Measured: Baseline, Up to Month 24
Phase 2: Percent Change from Baseline Over Time in Bone Turnover Marker: Osteocalcin (OCN)
Measured: Baseline, Up to Month 24
Phase 2: Change from Baseline in Dual Energy X-ray (DXA) Lumbar Spine Bone Mineral Density (BMD) Z-score Over Time
Measured: Baseline, Up to Month 24
Phase 2: Percent Change from Baseline in DXA Lumbar Spine BMD Over Time
Measured: Baseline, Up to Month 24
Phase 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Measured: Up to Month 24
Phase 2: Number of Participants With Anti-Setrusumab Binding and Neutralizing Antibodies
Measured: Up to Month 24
Phase 3: Annualized Rate of All Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures, but Including Fractures of the Fingers, Toes, Face and Skull at the Primary Analysis
Measured: Up to Month 24
Phase 3: Annualized Rate of All Radiographically-Confirmed Fractures at the Primary Analysis
Measured: Up to Month 24
Phase 3: Change from Baseline in DXA Lumbar Spine BMD Z-score at the Primary Analysis
Measured: Baseline, up to Month 24
Phase 3: Change from Baseline in Pediatric Orthopedic Society of North America Pediatric Outcomes Data Collection Instrument (POSNA-PODCI) Sports/Physical Functioning Subscale Score for Pediatric Participants at the Primary Analysis
Measured: Baseline, up to Month 24
Phase 3: Proportion of Participants Experiencing New Radiographically Confirmed Fractures, Excluding Morphometric Vertebral Fractures and Fractures of the Fingers, Toes, Face, and Skull at the Primary Analysis
Measured: Up to Month 24
Phase 3: Change from Baseline in POSNA-PODCI Pain/Comfort Subscale Score for Pediatric Participants at the Primary Analysis
Measured: Baseline, Up to Month 24
Phase 3: Change from Baseline in 36-item Short Form Health Survey (SF-36) Physical Functioning Domain Scale for Adult Participants at the Primary Analysis
Measured: Baseline, Up to Month 24
Phase 3: Change from Baseline in SF-36 Bodily Pain (BP) Domain Scale for Adult Participants at the Primary Analysis
Measured: Baseline, Up to Month 24
Phase 3: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Measured: Up to Month 24
Phase 3: Number of Participants With Anti-Setrusumab Binding and Neutralizing Antibodies
Measured: 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 posted18 Nov 2021
- Study start21 Feb 2022
- Primary completion20 Oct 2025
- Study completionApr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 183 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Mereo BioPharma
- Responsible party
- Sponsor
- Investigators
- Ultragenyx Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05125809
- Sponsor's protocol ID
- UX143-CL301
- Other IDs
- 2021-006597-23, 2024-510919-29-00
- Acronym
- Orbit
- Status last verified
- Oct 2026
- First submitted
- 8 Nov 2021
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Bartosik M, Windels O, von Brackel FN, Amling M, Oheim R. Romosozumab in postmenopausal women with classical Osteogenesis imperfecta. Arch Osteoporos. 2026 Jul 18;21(1):100. doi: 10.1007/s11657-026-01744-3.
PubMed 42470570
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-Controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects With Osteogenesis Imperfecta
- ClinicalTrials.gov ID
- NCT05125809
- Acronym / sponsor ID
- Orbit / UX143-CL301
- Conditions
- Osteogenesis Imperfecta
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 183 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Collaborators
- Mereo BioPharma
- Study start
- 21 Feb 2022 (actual)
- Primary completion
- 20 Oct 2025 (actual)
- Study completion
- Apr 2027 (estimated)
- First posted
- 18 Nov 2021 (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 objectives of the study are to identify a setrusumab dosing strategy in participants with OI and to evaluate the effect of setrusumab vs placebo on reduction in fracture rate.
Detailed description
Participants in Phase 2 will be randomized 1:1 to receive low dose or high dose setrusumab. Phase 2 participants will continue receiving their assigned dose of setrusumab until all Phase 2 participants have completed the Month 6 study visit. After this point, Phase 2 participants will begin receiving the selected dosing strategy in the Phase 2 open-label Treatment Extension Period. Phase 3 participants will be randomized 2:1 to receive setrusumab or placebo during the double-blind treatment period. Phase 3 participants will transition to the open-label Treatment Extension Period after the end of the double-blind period. Participants in the Phase 2 and Phase 3 treatment extension periods will receive open-label setrusumab treatment for at least 12 months, and have the option to remain in the open-label treatment period until setrusumab is commercially available in their region. An optional substudy will be conducted in approximately 10 participants (≥ 8 years) consisting of a bone biopsy to investigate the impact of setrusumab on bone histomorphology.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Low Dose Setrusumab -> Open-Label (OL) Setrusumab Selected Dose | Experimental | Biological: SetrusumabSingle-blind setrusumab low dose during Phase 2 followed by open-label setrusumab selected dose During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician |
| High Dose Setrusumab -> OL Setrusumab Selected Dose | Experimental | Biological: SetrusumabSingle-blind setrusumab high dose during Phase 2 followed by open-label setrusumab During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician |
| Setrusumab Selected Dose -> OL Setrusumab Selected Dose | Experimental | Biological: SetrusumabDouble-blind setrusumab selected dose during Phase 3 followed by open-label setrusumab During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician |
| Placebo -> OL Setrusumab Selected Dose | Placebo comparator | Biological: Setrusumab, Other: PlaceboDouble-blind placebo during Phase 3 followed by open-label setrusumab During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician |
Who may qualify
- Ages
- 5 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Diagnosis of OI Type I, III, or IV as confirmed by identification of pathogenic or likely pathogenic genetic variants in COL1A1 or COL1A2. If a variant of uncertain significance is identified, then clinical presence of the expected phenotype can be used to confirm the diagnosis
- ≥ 1 fracture in the past 12 months, ≥ 2 fractures in the past 24 months or ≥ 1 tibia, femur or humerus fracture in the past 24 months
- Serum 25-hydroxyvitamin D ≥ 20 ng/mL at the Screening Visit. If 25-hydroxyvitamin D levels are below 20 ng/mL, 25-hydroxyvitamin D testing can repeated after a minimum of 14 days of vitamin D supplementation as directed by the treating physician
- Willing to not receive bisphosphonate therapy during the study
- From the period following informed consent to 60 days after the last dose of the study drug, females of childbearing potential and fertile males must consent to use highly effective contraception. If female, agree not to become pregnant. If male, agree not to father a child or donate sperm
Key exclusion criteria (20 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 unstable within past 2 years requires review by the Medical Monitor
- History of or uncontrolled concomitant diseases such as hypo/hyperparathyroidism, Paget's disease, abnormal thyroid function, thyroid disease or other endocrine disorders or conditions that could affect bone metabolism such as Stage IV/V renal disease
- Rickets or any skeletal condition (other than OI) leading to long-bone deformities and/or increased risk of fractures
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 | High Dose Setrusumab -> OL Setrusumab Selected Dose, Low Dose Setrusumab -> Open-Label (OL) Setrusumab Selected Dose, Placebo -> OL Setrusumab Selected Dose, Setrusumab Selected Dose -> OL Setrusumab Selected Dose |
| PlaceboA 5% dextrose/glucose solution administered QM via IV infusion | Other | Placebo -> OL Setrusumab Selected Dose |
Primary outcome
Phase 2: Percent Change in Serum Amino-terminal Propeptide of Type 1 Procollagen (P1NP) from Baseline at Month 1
Time frame: Baseline, Month 1
Phase 3: Annualized Rate of All Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures and Fractures of the Fingers, Toes, Face, and Skull at the Primary Analysis
Time frame: Up to Month 24
18 secondary outcomes
Phase 2: Serum Setrusumab Concentration
Time frame: From Predose up to Month 24
Phase 2: Baseline-Corrected Area Under the Effect Curve (AUEC) for Serum P1NP Over a 1 and 2-Month Period
Time frame: Baseline, Up to Month 2
Phase 2: Percent Change from Baseline Over Time in Bone Turnover Marker: P1NP
Time frame: Baseline, Up to Month 24
Phase 2: Percent Change from Baseline Over Time in Bone Turnover Marker: Osteocalcin (OCN)
Time frame: Baseline, Up to Month 24
Phase 2: Change from Baseline in Dual Energy X-ray (DXA) Lumbar Spine Bone Mineral Density (BMD) Z-score Over Time
Time frame: Baseline, Up to Month 24
Phase 2: Percent Change from Baseline in DXA Lumbar Spine BMD Over Time
Time frame: Baseline, Up to Month 24
Phase 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Time frame: Up to Month 24
Phase 2: Number of Participants With Anti-Setrusumab Binding and Neutralizing Antibodies
Time frame: Up to Month 24
Phase 3: Annualized Rate of All Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures, but Including Fractures of the Fingers, Toes, Face and Skull at the Primary Analysis
Time frame: Up to Month 24
Phase 3: Annualized Rate of All Radiographically-Confirmed Fractures at the Primary Analysis
Time frame: Up to Month 24
Phase 3: Change from Baseline in DXA Lumbar Spine BMD Z-score at the Primary Analysis
Time frame: Baseline, up to Month 24
Phase 3: Change from Baseline in Pediatric Orthopedic Society of North America Pediatric Outcomes Data Collection Instrument (POSNA-PODCI) Sports/Physical Functioning Subscale Score for Pediatric Participants at the Primary Analysis
Time frame: Baseline, up to Month 24
Phase 3: Proportion of Participants Experiencing New Radiographically Confirmed Fractures, Excluding Morphometric Vertebral Fractures and Fractures of the Fingers, Toes, Face, and Skull at the Primary Analysis
Time frame: Up to Month 24
Phase 3: Change from Baseline in POSNA-PODCI Pain/Comfort Subscale Score for Pediatric Participants at the Primary Analysis
Time frame: Baseline, Up to Month 24
Phase 3: Change from Baseline in 36-item Short Form Health Survey (SF-36) Physical Functioning Domain Scale for Adult Participants at the Primary Analysis
Time frame: Baseline, Up to Month 24
Phase 3: Change from Baseline in SF-36 Bodily Pain (BP) Domain Scale for Adult Participants at the Primary Analysis
Time frame: Baseline, Up to Month 24
Phase 3: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Time frame: Up to Month 24
Phase 3: Number of Participants With Anti-Setrusumab Binding and Neutralizing Antibodies
Time frame: 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 20 Oct 2026.
- Derived from this study: PubMed 42470570Bartosik M, Windels O, von Brackel FN, Amling M, Oheim R. Romosozumab in postmenopausal women with classical Osteogenesis imperfecta. Arch Osteoporos. 2026 Jul 18;21(1):100. doi: 10.1007/s11657-026-01744-3.
- Ultragenyx Osteogenesis Imperfecta (OI) Research StudyLink provided by the sponsor.
- Ultragenyx Transparency CommitmentLink 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
43 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Setrusumab in people with Osteogenesis Imperfecta, run by Ultragenyx Pharmaceutical Inc at 43 sites in 11 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 8 inclusion and 20 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 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 43 sites in United States, Canada, Germany, Italy, Australia and 6 more countries. 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 combined 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.
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?
Phase 2: Percent Change in Serum Amino-terminal Propeptide of Type 1 Procollagen (P1NP) from Baseline at Month 1 (time frame: Baseline, Month 1). The study lists 2 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 NCT05125809, 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 20 Oct 2026; AzTrials last refreshed its copy on 30 Oct 2026.