Recent Updates
Recently added Catalysts

Setrusumab

Phase 3

Osteogenesis Imperfecta | Monoclonal antibody | Rare Disease |Ultragenyx Pharmaceutical Inc.|Last Updated: Jun 15, 2026

Target and mechanism

Molecular targetSOST
Target classInhibitor
ModalityMonoclonal antibody

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment189

FDA Designations

BREAKTHROUGH_THERAPY

Clinical trial landscape

Setrusumab · 3 trials · 4 indications

Phase 3 1Phase 2 2
NCT06636071Setrusumab in Pediatric Japanese Subjects With Osteogenesis ImperfectaOsteogenesis Imperfecta
ACTIVE NOT_RECRUITING6 Analytics
PHASE3ACTIVE NOT_RECRUITING
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
Osteogenesis ImperfectaUnlock trial analytics

Study Endpoints

Primary Endpoints

Annualized Rate of All Radiographically-Confirmed Fractures, Including Morphometric Vertebral Fractures During the Treatment Period
Up to Month 24
Phase 2: Percent Change in Serum Amino-terminal Propeptide of Type 1 Procollagen (P1NP) from Baseline at Month 1
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
Up to Month 24
Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12
Baseline, Month 12 (end of treatment [EOT])

Assessed by high resolution peripheral quantitative computed tomography (HRpQCT). HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presents the ratio of the means between the visit and Baseline from analysis of covariance (ANCOVA).

Change From Baseline in Radial Bone Strength (Failure Load) at Month 12
Baseline, Month 12 (EOT)

Assessed by finite element analysis (FEA) of models generated from HRpQCT images of the distal radius.

Change From Baseline in Radial Bone Strength (Stiffness) at Month 12
Baseline, Month 12 (EOT)

Assessed by FEA of models generated from HRpQCT images of the distal radius.

Secondary Endpoints

Annualized Rate of Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures, but Including Fractures of the Fingers, Toes, Face, and Skull During the Treatment Period
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
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
Baseline, Up to Month 24
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SetrusumabEXPERIMENTAL -
Low Dose Setrusumab -> Open-Label (OL) Setrusumab Selected DoseEXPERIMENTALSingle-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 DoseEXPERIMENTALSingle-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 DoseEXPERIMENTALDouble-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 DosePLACEBO_COMPARATORDouble-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
Setrusumab 20 mg/kg (Blinded)EXPERIMENTALSetrusumab 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)EXPERIMENTALSetrusumab 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)EXPERIMENTALSetrusumab 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)EXPERIMENTALSetrusumab 20 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.
PlaceboPLACEBO_COMPARATORPlacebo IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Interventions

NameTypeDescription
setrusumabBIOLOGICALA fully human sclerostin neutralizing monoclonal antibody (mAb) administered once a month (QM) via intravenous (IV) infusion
PlaceboOTHERA 5% dextrose/glucose solution administered QM via IV infusion
CalciumDIETARY_SUPPLEMENTtablets
Vitamin DDIETARY_SUPPLEMENTcapsules
zoledronic acid (optional)DRUGFollowing 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.
Unlock Study Design Details

Eligibility Criteria

Age Range2 Years to 6 Years
SexALL
Healthy VolunteersNo
Study Sites3

Key Inclusion Criteria: * 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 ...

Countries:JapanUnited StatesAustraliaCanadaFranceGermanyItalyNetherlandsPolandPortugalTurkey (Türkiye)United KingdomDenmark
Unlock Eligibility Criteria

Recent Changes (Last 90 Days)

LOWJun 16, 2026NCT06636071lastUpdatePostDate: changed
LOWJun 16, 2026NCT06636071lastUpdatePostDate: changed
LOWJun 16, 2026NCT06636071lastUpdatePostDate: changed
LOWJun 16, 2026NCT06636071lastUpdatePostDate: changed

Frequently asked questions about Setrusumab

What is Setrusumab used for?

Setrusumab is an investigational monoclonal antibody being developed for the treatment of Osteogenesis Imperfecta, a rare genetic bone disorder. It is being studied in patients with Type I, III, and IV Osteogenesis Imperfecta, as well as in pediatric and adult populations. The drug is currently in Phase 3 clinical development.

What does Setrusumab target?

Setrusumab is a monoclonal antibody that targets sclerostin, a protein that inhibits bone formation. By inhibiting sclerostin, Setrusumab aims to increase bone formation and improve bone density in patients with Osteogenesis Imperfecta. This mechanism is being evaluated in clinical trials for its potential to reduce fractures and improve bone health.

Who is developing Setrusumab?

Setrusumab is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on rare diseases. Ultragenyx is listed on the NASDAQ under the ticker symbol RARE. The company is conducting clinical trials to evaluate the safety and efficacy of Setrusumab in patients with Osteogenesis Imperfecta.

What phase is Setrusumab in?

Setrusumab is currently in Phase 3 clinical development. It has received Breakthrough Therapy designation from the FDA for the treatment of Osteogenesis Imperfecta. The drug is being studied in multiple trials, including a Phase 3 trial in pediatric Japanese subjects and a Phase 2 trial in patients with Osteogenesis Imperfecta.

What clinical trials is Setrusumab in?

Setrusumab is being evaluated in several clinical trials. NCT05125809 is a Phase 2 study comparing Setrusumab to placebo in patients with Osteogenesis Imperfecta. NCT06636071 is a Phase 3 trial in pediatric Japanese subjects with Osteogenesis Imperfecta. NCT03118570 is a completed Phase 2 study in adults with Type I, III, or IV Osteogenesis Imperfecta.

Is Setrusumab the same as BPS804?

Yes, Setrusumab is also known as BPS804. In clinical trials, the drug has been referred to by both names. For example, NCT03118570, a completed study in adults with Osteogenesis Imperfecta, was titled 'A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804.'