NCT06067425

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia

A Phase 2, Open-label, Multi-center, 2-stage Sequential Cohort, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Subcutaneous SAR442501 in Pediatric Participants With Achondroplasia

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 0 Days – 12 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.

Detailed description

Up to approximately 275 weeks: 3 weeks Screening + 52 weeks primary treatment period + up to approximately 216 weeks extended treatment period+ 4 weeks follow-up.

Conditions

• Osteochondrodysplasia

Interventions

Type	Name	Description
DRUG	SAR442501	Solution for injection; Subcutaneous injection

Timeline

• Start date: 2023-10-10
• Primary completion: 2025-02-12
• Completion: 2025-02-12
• First posted: 2023-10-04
• Last updated: 2025-02-25

Locations

9 sites across 5 countries: Australia, China, Italy, South Korea, Spain

Regulatory

• FDA-regulated drug study