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.
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
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
16
Powder for solution for infusion; IV infusion
Solution for infusion; IV infusion
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
...and 3 more locations
Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs)
Time frame: From baseline to Week 24
Assessment of PK parameters: area under the curve (AUC)
Time frame: From baseline to Week 24
Assessment of PK parameters: maximum serum concentration observed (Cmax)
Time frame: From baseline to Week 24
Assessment of PK parameters: time to reach maximum concentration observed (tmax)
Time frame: From baseline to Week 24
Titer of anti-SAR439459 antibodies (if detected)
Time frame: From baseline to Week 24
Percent change from baseline in bone mineral density (BMD)
Time frame: From baseline to Week 24
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