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Bisphosphonate

Phase 3

Osteogenesis Imperfecta | Small molecule | Other |Ultragenyx Pharmaceutical Inc.|Last Updated: Feb 25, 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
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment69
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05768854Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis ImperfectaPHASE3 ACTIVE NOT_RECRUITING 69Jun 14, 2023Apr 1, 2027Feb 25, 202620 United States, Brazil +5
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Study Endpoints
Primary Endpoints
Annualized Rate of All Radiographically-Confirmed Fractures, Including Morphometric Vertebral Fractures, at the Primary Analysis
Up to 24 Months
Secondary Endpoints
Annualized Rate of Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures, but Including Fractures of the Fingers, Toes, Face and Skull at the Primary Analysis
Up to 24 Months
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 24 Months
Change from Baseline in Dual-energy X-ray Absorptiometry (DXA) Bone Mineral Density (BMD) Z-score at the Lumbar Spine at the Primary Analysis
Up to 24 Months
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Intravenous Bisphosphonates (IV-BP) -> SetrusumabACTIVE_COMPARATORParticipants on IV-BP will continue their existing dose/regimen per investigator discretion; for participants not on IV-BP, the dose/regimen will be determined by the investigator. After the active-controlled period, participants will receive Setrusumab during the extension period
SetrusumabEXPERIMENTALParticipants will receive Setrusumab during the active-controlled and extension period
Interventions
NameTypeDescription
BisphosphonateDRUGAdministered per investigator discretion via intravenous (IV) infusion
SetrusumabBIOLOGICALA fully human sclerostin neutralizing monoclonal antibody (mAb) administered once a month (QM) via intravenous (IV) infusion
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Eligibility Criteria
Age Range2 Years — 6 Years
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: * Male or female 2 to \< 7 years of age at time of informed consent * Clinical diagnosis of OI Types 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 fem...

Countries:United StatesBrazilCanadaFranceItalyNetherlandsPoland
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05768854primaryCompletionDate: changed
LOWMay 24, 2026NCT05768854studyFirstPostDate: changed