SAR439459 for Osteogenesis Imperfecta

Recruiting at 20 trial locations

Overseen By Trial Transparency email recommended (Toll free for US & Canada)

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Sanofi

Must not be taking: Anticoagulants, Antiplatelets, Bisphosphonates, others

Disqualifiers: Scoliosis, Bleeding disorders, Heart failure, others

Compensation: Varies

Number of Visits: 1

Duration: 1 day

16 Participants Needed

Trial Summary

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop taking your current medications. However, if you are on certain treatments like denosumab, anti-sclerostin antibody, parathyroid hormone, bisphosphonates, or any experimental therapy for osteogenesis imperfecta, you must not have used them within 6 months before the study. Also, you should not be on therapeutic doses of anticoagulants or antiplatelet agents within 7 days before the study.

What data supports the effectiveness of the drug SAR439459 for treating osteogenesis imperfecta?

There is no direct data on SAR439459 for osteogenesis imperfecta, but research on similar treatments targeting TGF-β (a protein involved in bone growth) shows potential benefits. A study on fresolimumab, a TGF-β inhibitor, indicated it could increase bone density in some patients with osteogenesis imperfecta. ^{1 2 3 4 5}

How is the drug SAR439459 different from other treatments for osteogenesis imperfecta?

SAR439459, also known as amcenestrant, is unique because it targets TGF-β signaling, a key pathway involved in the development of osteogenesis imperfecta, whereas other treatments like bisphosphonates focus on increasing bone density without addressing this specific pathway. ^{2 4 5 6 7}

What is the purpose of this trial?

This trial tests SAR439459, a medicine that blocks certain proteins, in adults with Osteogenesis Imperfecta. The goal is to see if it can safely improve bone strength and density over several months.

Research Team

Clinical Sciences & Operations

Principal Investigator

Sanofi

Eligibility Criteria

Adults with Osteogenesis Imperfecta (OI) types I or IV, who've had at least one bone fracture in the past decade or two since turning 18. They must weigh over 30 kg and not be pregnant, breastfeeding, or donating sperm. Participants need a documented genetic variant in COL1A1/COL1A2 and must consent to the study.

Inclusion Criteria

I have Type I or IV osteogenesis imperfecta with a known genetic cause.

You have given your written permission to participate in the study.

I am using birth control, not pregnant, not breastfeeding, and if male, not donating sperm.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

Up to 5 weeks

Treatment

Participants receive a single IV dose of SAR439459 or placebo

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety, pharmacokinetics, pharmacodynamics, and bone mineral density over 24 weeks

24 weeks

Multiple visits (in-person)

Treatment Details

Interventions

• SAR439459

Trial Overview The trial is testing SAR439459, an antibody targeting TGFβ, given once via IV to see if it's safe and how it affects people with OI. It includes checking for side effects, how the body processes the drug (PK), its impact on bones (PD), bone density through DXA scans, and blood biomarkers over six months.

Participant Groups

Group I: SAR439459 Experimental Treatment 1 Intervention

Participants will receive a single dose of SAR439459

Group II: Placebo Placebo Group 1 Intervention

Participants will receive a single dose of placebo

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

References

1. Denmark pubmed.ncbi.nlm.nih.gov

Health-related quality of life in adults with osteogenesis imperfecta. [2022]

2. Australia pubmed.ncbi.nlm.nih.gov

Oral Bisphosphonate Therapy for Osteogenesis Imperfecta: A Systematic Review and Meta-Analysis of Six Randomized Placebo-Controlled Trials. [2021]

3. England pubmed.ncbi.nlm.nih.gov

A Multicenter Observational Cohort Study to Evaluate the Effects of Bisphosphonate Exposure on Bone Mineral Density and Other Health Outcomes in Osteogenesis Imperfecta. [2020]

4. United States pubmed.ncbi.nlm.nih.gov

Effects of oral alendronate on BMD in adult patients with osteogenesis imperfecta: a 3-year randomized placebo-controlled trial. [2016]

5. United States pubmed.ncbi.nlm.nih.gov

Targeting TGF-β for treatment of osteogenesis imperfecta. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Sclerostin-Antibody Treatment Decreases Fracture Rates in Axial Skeleton and Improves the Skeletal Phenotype in Growing oim/oim Mice. [2021]

7. England pubmed.ncbi.nlm.nih.gov

Beneficial effect of long term intravenous bisphosphonate treatment of osteogenesis imperfecta. [2019]