A Phase 1b, Single Ascending Dose, Randomized, Double-blind Study to Evaluate the Safety, Tolerability, and Activity of SAR439459 in Adults With Osteogenesis Imperfecta

  • STATUS
    Recruiting
  • End date
    Nov 21, 2023
  • participants needed
    24
  • sponsor
    Sanofi
Updated on 19 September 2022

Summary

SAR439459 is a human anti-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.

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 safety and PD for 24 weeks
  • Final study visit at Week 24

Details
Condition Osteogenesis Imperfecta
Treatment Placebo, SAR439459
Clinical Study IdentifierNCT05231668
SponsorSanofi
Last Modified on19 September 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Participants who are clinically categorized as Type I or IV osteogenesis imperfecta with a previously documented pathogenic genetic variant in COL1A1 or 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
History of moderate (25-40°) to severe (>40°) scoliosis assessed as Cobb angle
Postmenopausal women
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 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
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