What side effects are associated with this type of intervention?

Setrusumab, a monoclonal antibody targeting sclerostin, may cause side effects such as injection site reactions, hypersensitivity, and potential cardiovascular risks. Intravenous bisphosphonates, commonly used to treat OI, can lead to side effects including flu-like symptoms, nephrotoxicity, hypocalcemia, osteonecrosis of the jaw, and musculoskeletal pain. Both treatments aim to improve bone density and reduce fracture rates but come with their respective risk profiles.

What prior FDA approvals exist?

The FDA has approved bisphosphonates for the treatment of Osteogenesis Imperfecta, which work by inhibiting osteoclast-mediated bone resorption. Commonly used bisphosphonates include pamidronate and zoledronic acid. These drugs have been shown to increase bone density and reduce fracture rates in patients with OI. There are currently no FDA-approved monoclonal antibodies specifically targeting sclerostin for OI, but Setrusumab is being studied for its potential to promote bone growth by inhibiting sclerostin, a protein that negatively regulates bone formation.

What other types of research exist?

Promising novel alternatives to Setrusumab and Intravenous Bisphosphonates for Osteogenesis Imperfecta patients include Denosumab, which inhibits RANKL to reduce bone resorption, and Romosozumab, which targets sclerostin to promote bone growth. These options should be evaluated with a healthcare provider.

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Setrusumab vs Bisphosphonates for Osteogenesis Imperfecta (Cosmic Trial)

Phase 3

Waitlist Available

Research Sponsored by Ultragenyx Pharmaceutical Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial compares setrusumab, a new bone-strengthening drug, with standard treatments in children. It aims to see if setrusumab can better prevent bone fractures. Both treatments work by making bones stronger. Standard treatments have been used for a long time to increase bone mineral density and improve bone resistance, leading to a decrease in fracture rate.

Who is the study for?

This trial is for children with Osteogenesis Imperfecta (OI) types I, III, or IV who have had fractures in the past year and are currently on or have had bisphosphonate therapy. They must not have severe kidney issues, untreated hypocalcemia, a history of certain bone diseases other than OI, cardiovascular disease without clearance, or recent use of certain bone-impacting drugs.

What is being tested?

The study compares setrusumab to intravenous bisphosphonates in reducing fracture rates in pediatric patients with OI. It aims to see if setrusumab is more effective at preventing new fractures including specific spinal ones.

What are the potential side effects?

While the document doesn't specify side effects directly, common side effects for medications like setrusumab and bisphosphonates may include flu-like symptoms, joint pain, risk of unusual thigh bone fractures and potential jawbone problems.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: SetrusumabExperimental Treatment1 Intervention

Participants will receive Setrusumab during the active-controlled and extension period

Group II: Intravenous Bisphosphonates (IV-BP) -> SetrusumabActive Control2 Interventions

Participants 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

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Setrusumab, a monoclonal antibody targeting sclerostin, promotes bone growth by inhibiting a protein that normally suppresses bone formation. This is particularly beneficial for Osteogenesis Imperfecta (OI) patients, who have brittle bones due to defective collagen. Intravenous Bisphosphonates, on the other hand, inhibit osteoclast-mediated bone resorption, leading to increased bone density and reduced bone turnover. This helps to strengthen the bones and lower the risk of fractures in OI patients. Both treatments aim to enhance bone strength and reduce fracture rates, addressing the fundamental issue of bone fragility in OI.