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

PHASE1RECRUITING

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.

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Study details:

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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

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.

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.

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2022-08-25

Primary completion: 2025-06-30

Study completion finish: 2025-06-30

Study type

TREATMENT

Phase

PHASE1

Trial ID

NCT05231668

Intervention or treatment

DRUG: SAR439459

DRUG: Placebo

Conditions

• Osteogenesis Imperfecta

Image related to Osteogenesis Imperfecta

Condition: Osteogenesis Imperfecta
DRUG: SAR439459 and other drugs
Westmead, New South Wales, Australia and more
Sponsor: Sanofi

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Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

Westmead Hospital_Site Number :0360003

Research sites nearby

Select from list below to view details:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: SAR439459 Participants will receive a single dose of SAR439459	DRUG: SAR439459 Powder for solution for infusion; IV infusion
PLACEBO_COMPARATOR: Placebo Participants will receive a single dose of placebo	DRUG: Placebo Solution for infusion; IV infusion

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs)	Not Specified	From baseline to Week 24

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Assessment of PK parameters: area under the curve (AUC)	Not Specified	From baseline to Week 24
Assessment of PK parameters: maximum serum concentration observed (Cmax)	Not Specified	From baseline to Week 24
Assessment of PK parameters: time to reach maximum concentration observed (tmax)	Not Specified	From baseline to Week 24
Titer of anti-SAR439459 antibodies (if detected)	Not Specified	From baseline to Week 24
Percent change from baseline in bone mineral density (BMD)	Not Specified	From baseline to Week 24

Frequently Asked Questions

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References

Clinical Trials Gov: Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI)