Status:
TERMINATED
Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI)
Lead Sponsor:
Sanofi
Conditions:
Osteogenesis Imperfecta
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
Brief 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 adul...
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 ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
August 25 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 12 2024
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT05231668
Start Date
August 25 2022
End Date
November 12 2024
Last Update
September 11 2025
Active Locations (13)
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1
UCLA Health_Site Number: 8400006
Los Angeles, California, United States, 90095
2
Yale University - Site Number:8400007
New Haven, Connecticut, United States, 06510
3
Indiana University School of Medicine_Site Number: 8400002
Indianapolis, Indiana, United States, 46202
4
Kennedy Krieger Institute_Site number 8400004
Baltimore, Maryland, United States, 21205