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Setrusumab

Phase 3

Osteogenesis Imperfecta | Monoclonal antibody | Other |Ultragenyx Pharmaceutical Inc.|Last Updated: Mar 13, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment189
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06636071Setrusumab in Pediatric Japanese Subjects With Osteogenesis ImperfectaPHASE3 ACTIVE NOT_RECRUITING 6Oct 25, 2024Jan 1, 2028Mar 13, 20263 Japan
NCT05125809Setrusumab vs Placebo for Osteogenesis ImperfectaPHASE2 ACTIVE NOT_RECRUITING 183Feb 21, 2022Apr 1, 2027Feb 25, 202644 United States, Australia +9
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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
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
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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
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
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Eligibility Criteria
Age Range2 Years — 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 Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05125809primaryCompletionDate: changed
LOWMay 26, 2026NCT06636071primaryCompletionDate: changed
LOWMay 24, 2026NCT05125809studyFirstPostDate: changed
LOWMay 24, 2026NCT06636071studyFirstPostDate: changed