Setrusumab for Osteogenesis Imperfecta
(Orbit Trial)

Recruiting in Palo Alto (17 mi)

+69 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Ultragenyx Pharmaceutical Inc

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Research Team

Ultragenyx Medical Director

Principal Investigator

Ultragenyx Pharmaceutical Inc

Eligibility Criteria

This trial is for people with Osteogenesis Imperfecta (OI) Types I, III, or IV who've had fractures in the past year. They must not be on bisphosphonate therapy and agree to use effective contraception. Excluded are those with certain skeletal conditions, unstable neurologic diseases, severe kidney issues, recent drug abuse, hypersensitivity to setrusumab or its components.

Inclusion Criteria

I have Osteogenesis Imperfecta Type I, III, or IV confirmed by a genetic test.

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

I agree not to take bisphosphonate therapy during the study.

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Exclusion Criteria

Concurrent participation in another clinical study without prior approval from the Investigator in consultation with the Medical Monitor

I have received specific prior treatments.

I have symptoms of Chiari malformation or basilar invagination, and no unstable neurological conditions in the past 2 years.

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Treatment Details

Interventions

Placebo (Other)
Setrusumab (Monoclonal Antibodies)

Trial OverviewThe study tests Setrusumab against a placebo to find the best dose and see if it lowers fracture rates in OI patients. Participants will either receive Setrusumab or a placebo without knowing which one they're getting.

Participant Groups

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Setrusumab Selected Dose -> OL Setrusumab Selected DoseExperimental Treatment1 Intervention

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

Group II: Low Dose Setrusumab -> Open-Label (OL) Setrusumab Selected DoseExperimental Treatment1 Intervention

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

Group III: High Dose Setrusumab -> OL Setrusumab Selected DoseExperimental Treatment1 Intervention

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

Group IV: Placebo -> OL Setrusumab Selected DosePlacebo Group2 Interventions

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