NCT02855268

Study of Lademirsen (SAR339375) in Patients With Alport Syndrome

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Efficacy, Pharmacodynamics, and Pharmacokinetics of Lademirsen (SAR339375) for Subcutaneous Injection Administered Every Week in Patients With Alport Syndrome

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 43 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

Primary Objectives: * To assess the efficacy of lademirsen (SAR339375) in reducing the decline in renal function. * To assess the safety and tolerability of lademirsen (SAR339375) in participants with Alport syndrome. Secondary Objectives: * To assess plasma pharmacokinetic (PK) parameters of the parent compound and its active major metabolite. * To assess the potential formation of anti-drug antibodies (ADAs) following administration of lademirsen (SAR339375). * To assess the pharmacodynamic effect of lademirsen (SAR339375) on miR-21 and on changes in renal injury and function biomarkers.

Detailed description

The planned length of participation in the study for each participant was up to approximately 110 weeks (from screening through completion of follow-up). This included: * Screening/baseline period of up to 4 weeks * Double-blind, placebo-controlled treatment period of 48 weeks * Open-label extension treatment period of 48 weeks (all participant to enter a 48-week open label extension period and receive active treatment with lademirsen \[SAR339375\]). * Post-treatment follow-up period of 10 weeks.

Conditions

• Alport Syndrome

Interventions

Type	Name	Description
DRUG	lademirsen (SAR339375)	Pharmaceutical form: Solution for injection Route of administration: Subcutaneous injection
DRUG	Placebo	Pharmaceutical form: Solution for injection Route of administration: Subcutaneous injection

Timeline

• Start date: 2019-11-02
• Primary completion: 2022-09-22
• Completion: 2022-09-22
• First posted: 2016-08-04
• Last updated: 2025-09-11
• Results posted: 2023-10-23

Locations

23 sites across 7 countries: United States, Australia, China, France, Germany, Spain, United Kingdom

Regulatory

• FDA-regulated drug study