NCT05231668

Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI)

A Phase 1b, Single Ascending Dose, Randomized, Double-blind Study to Evaluate the Safety, Tolerability, and Activity of SAR439459 in Adults With Osteogenesis Imperfecta

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

SAR439459 is a human anti-Transforming growth factor β (TGFβ) monoclonal antibody. This phase 1 clinical study investigates the safety, tolerability, and activity of a single dose of SAR439459 in adult participants with OI. Participants will receive a single IV dose of SAR439459 with safety, pharmacokinetic (PK), and pharmacodynamic (PD) assessments over 24 weeks. There will be up to 3 dose cohorts. In addition to safety, tolerability, and PK assessments, bone mineral density (BMD) will be evaluated by dual-energy Xray absorptimetry (DXA) scan and a series of blood biomarkers will be monitored to document pharmacodynamic effects of the single dose of SAR439459.

Detailed description

The duration of the study for all participants will be approximately 29 weeks: * Up to 5 weeks from initiation of screening to dose administration * Treatment on Day 1 * Follow-up and observation of safety and PD for 24 weeks * Final study visit at Week 24

Conditions

• Osteogenesis Imperfecta

Interventions

Type	Name	Description
DRUG	SAR439459	Powder for solution for infusion; IV infusion
DRUG	Placebo	Solution for infusion; IV infusion

Timeline

• Start date: 2022-08-25
• Primary completion: 2024-11-12
• Completion: 2024-11-12
• First posted: 2022-02-09
• Last updated: 2025-09-11

Locations

13 sites across 4 countries: United States, Australia, Canada, France

Regulatory

• FDA-regulated drug study