Study of Lademirsen (SAR339375) in Patients With Alport Syndrome
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.
Timeline
- Start
- 2019-11-02
- Primary completion
- 2022-09-22
- Completion
- 2022-09-22
Outcome
Missed primary endpoint
Stopped: “The results of the futility analysis led to the study termination. No unexpected safety findings were identified.”
registry DB Period: Placebo vs DB Period: Lademirsen; p = 0.9472; Least square mean difference -0.22 (95% CI -6.92 to 6.48) NCT02855268 ↗
paper There were no significant differences between lademirsen-treated and placebo-treated participants in eGFR at any timepoint. PMID 38829703 ↗
Publications
- Gale DP, Gross O, Wang F, Esteban de la Rosa RJ, Hall M, Sayer JA, Appel G, Hariri A, Liu S, Maski M, Shen Y, Zhang Q, Iqbal S, Kowthalam MU, Lin J, Ding J; HERA Clinical Trial Group. A Randomized Controlled Clinical Trial Testing Effects of Lademirsen on Kidney Function Decline in Adults with Alport Syndrome. Clin J Am Soc Nephrol. 2024 Aug 1;19(8):995-1004. doi: 10.2215/CJN.0000000000000458. Epub 2024 Jun 3.
- Kashtan CE, Gross O. Clinical practice recommendations for the diagnosis and management of Alport syndrome in children, adolescents, and young adults-an update for 2020. Pediatr Nephrol. 2021 Mar;36(3):711-719. doi: 10.1007/s00467-020-04819-6. Epub 2020 Nov 6.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | lademirsen | Oligonucleotide (other) | 110 mg | Subcutaneous |