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Bisphosphonate

Phase 3

Osteogenesis Imperfecta | Small molecule | Rare Disease |Ultragenyx Pharmaceutical Inc.|Last Updated: Feb 25, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLEDDMC
Total Trials1
Total Enrollment69

FDA Designations

No designations recorded

Clinical trial landscape

Bisphosphonate · 1 trial · 1 indication

Phase 3 1
NCT05768854Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis ImperfectaOsteogenesis Imperfecta
ACTIVE NOT_RECRUITING69 Analytics
PHASE3ACTIVE NOT_RECRUITING
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
Osteogenesis ImperfectaUnlock trial analytics

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 to 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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Frequently asked questions about Bisphosphonate

What is Bisphosphonate used for in Osteogenesis Imperfecta?

Bisphosphonate is a small molecule being studied for the treatment of Osteogenesis Imperfecta, a rare genetic bone disorder. It is currently in Phase 3 clinical development as an active-controlled therapy, meaning it is being compared against another treatment in a randomized trial.

Who makes Bisphosphonate?

Bisphosphonate is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol RARE. The company is conducting a Phase 3 clinical trial to evaluate the drug in pediatric patients with Osteogenesis Imperfecta.

What phase is Bisphosphonate in?

Bisphosphonate is in Phase 3 clinical development for Osteogenesis Imperfecta. It is an investigational drug, not yet approved by regulatory authorities. The ongoing Phase 3 trial is active but not recruiting participants, with an enrollment target of 69 patients.

What clinical trials is Bisphosphonate in?

Bisphosphonate is being evaluated in a Phase 3 clinical trial registered as NCT05768854, titled 'Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta.' The trial is active but not recruiting, enrolling 69 participants across the United States, Brazil, Canada, France, Italy, Netherlands, and Poland.

Is Bisphosphonate the same as Setrusumab?

No, Bisphosphonate is not the same as Setrusumab. In the clinical trial NCT05768854, Setrusumab is the investigational drug being compared against bisphosphonates, which are the standard active control. Bisphosphonate is a class of drugs used to treat bone disorders, while Setrusumab is a separate investigational therapy.

What is the design of the Bisphosphonate Phase 3 trial?

The Phase 3 trial for Bisphosphonate, NCT05768854, is a randomized, active-controlled study in pediatric patients aged 2 years and older with Osteogenesis Imperfecta. It is not double-blinded and does not use a biomarker-selected patient population. The trial is enrolling 69 participants across multiple countries.