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

Stopped early · Phase 1 · Has a placebo group

Conditions studied: Osteogenesis Imperfecta

In brief

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.

Key facts

Study ID

NCT05231668

Run by

Sanofi

People needed

16

Starts

2022-08-25

Expected to finish

2024-11-12

Last updated by the study team

2025-09-11

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.
• Therapeutic doses of anticoagulants or antiplatelet agents (eg, 1 mg/kg bid of enoxaparin, 300 mg of aspirin daily, and 75 mg of clopidogrel daily or equivalent) within 7 days prior to the IMP administration.
• Any known central nervous system (CNS) or intraocular lesion that has a risk of bleeding.
• Prior history of skin cancers including melanoma, squamous cell carcinoma, or basal cell carcinoma.
• Clinically significant cardiac valvular disorder or symptomatic heart failure.
• Vitamin D (25-hydoxyvitamin D) <15 ng/dL; rescreening will be allowed after supplementation.
• The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Where it is running

• 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
• Toronto general Hospital_Site Number :1240002 — Toronto, Canada
• Hopital Edouard Herriot _Site Number :2500002 — Lyon, France
• Hopital Lariboisiere_Site Number :2500001 — Paris, France

Full record on ClinicalTrials.gov

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