Ignite Creation Date:
2025-12-24 @ 5:34 PM
Ignite Modification Date:
2026-01-01 @ 6:10 PM
Study NCT ID:
NCT02855268
Status:
TERMINATED
Last Update Posted:
2025-09-11
First Post:
2016-07-28
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Lademirsen (SAR339375) in Patients With Alport Syndrome
Sponsor:
Genzyme, a Sanofi Company
Organization:
Study Overview
Official Title:
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
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
The results of the futility analysis led to the study termination. No unexpected safety findings were identified.
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HERA
Brief 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.
* 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.
* 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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2019-004394-10 | EUDRACT_NUMBER | None | View |