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Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI) | Clinical Trials | Clareo Health

TERMINATEDPHASE1

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

NCT05231668•Sanofi

View on ClinicalTrials.gov

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

Eligibility

Age

18 - 65 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Participants who are clinically categorized as Type I or IV osteogenesis imperfecta with a previously documented pathogenic genetic variant in human collagen type 1 alpha 1 gene (COL1A1) or human collagen type 1 alpha 2 gene (COL1A2).
•Participants who have experienced at least 1 bone fracture in the past 10 years OR 2 or more (≥2) fractures since the age of 18.
•Body weight ≥30.0 kg.
•Contraception for sexually active male participants or female patient; not pregnant or breastfeeding; no sperm donating for male participant.
•Signed written informed assent/consent.

Exclusion Criteria

•Previously installed rods or metal hardware that would prevent bone mineral density evaluation of the lumbar spine (note: only two of the L1-L4 vertebrae are necessary for evaluation).
•History of moderate (25-40°) to severe (\>40°) scoliosis assessed as Cobb angle (unless scoliosis does not impact assessment of bone mineral density in the lumbar vertebrae in the opinion of the investigator).
•Postmenopausal women who:
•* Are within 5 years of the onset of menopause (for example less than 5 years from their last menstruation or post-hysterectomy), however if the person has been on hormone replacement therapy for more than 1 year prior to enrollment, then they are eligible regardless of time from onset of menopause. The person must be willing to continue hormone replacement therapy throughout the study duration. OR
•* Were previously on hormone replacement therapy but have stopped within the past 5 years.
•History of treatment with denosumab, anti-sclerostin antibody, parathyroid hormone, bisphosphonates, or any other experimental therapy for OI within 6 months prior to any study baseline assessment.
•Known bleeding disorder.
•History of significant bleeding event that required hospitalization, surgery, or a blood transfusion that was possibly associated with increased bleeding tendency.
•Any major surgery within the last 28 days prior to investigational medicinal product (IMP) administration.
•Elective surgery or invasive procedure anticipated within 6 months after the IMP administration.
+6 more criteria

Locations (13)

UCLA Health_Site Number: 8400006

Los Angeles, California, United States

Yale University - Site Number:8400007

New Haven, Connecticut, United States

Indiana University School of Medicine_Site Number: 8400002

Indianapolis, Indiana, United States

Kennedy Krieger Institute_Site number 8400004

Baltimore, Maryland, United States

Cincinnati Children's Hospital Medical Center Site Number : 8400010

Cincinnati, Ohio, United States

Vanderbilt University Site Number : 8400011

Nashville, Tennessee, United States

Baylor College of Medicine - Site Number:8400003

Houston, Texas, United States

Westmead Hospital_Site Number :0360003

Westmead, New South Wales, Australia

Department of Medicine/ School of Clinical Sciences at Monash Health Monash University_246 Clayton Road_Site Number :0360002

Clayton, Victoria, Australia

Bone Research and Education Centre_Site Number :1240003

Oakville, Ontario, Canada

Key Dates

Start Date

August 25, 2022

Primary Completion Date

November 12, 2024

Completion Date

November 12, 2024

Last Updated

September 11, 2025

Enrollment

ACTUAL participants

Conditions

Osteogenesis Imperfecta

Interventions

SAR439459

DRUG

Placebo

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 11, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Effects of Bisphosphonates on OI-Related Hearing Loss

Cardiopulmonary Outcomes in Osteogenesis Imperfecta: BBD7708

Boost Brittle Bones Before Birth

Evaluation and Intervention for the Effects of Osteogenesis Imperfecta

Mesenchymal Cell Therapy in Osteogenesis Imperfecta (OI)

Treatment of Osteogenesis Imperfecta With Parathyroid Hormone and Zoledronic Acid