TerminatedPhase 2

Study of Lademirsen (SAR339375) in Patients With Alport Syndrome

Stopped: The results of the futility analysis led to the study termination. No unexpected safety findings were identified.

United States, Australia, China43 participantsStarted 2019-11-02

Plain-language 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.

Who can participate

Age range

18 Years – 55 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Clinical diagnosis (hematuria, family history, hearing loss, ocular change), AND
. Genetic confirmation of Alport Syndrome in the participant or the family member, OR
. Kidney biopsy showing glomerular basement membrane abnormalities (e.g., significant thinning, thickening, irregularity or lucencies) consistent with Alport Syndrome.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

Timeframe: DB: from 1st dose of IMP upto 1st dose of IMP in OLE for participant who entered OLE (Week 48); up to 7 days post last dose for participant not continuing to OLE (Week 49); OLE:1st dose of IMP (at Week 48) in OLE upto 10 weeks post last dose (Week 106)

DB Period: Annualized Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 48

Timeframe: Baseline, Week 48

Trial details

NCT IDNCT02855268

SponsorGenzyme, a Sanofi Company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-09-22

Results submitted2023-09-08

View on ClinicalTrials.gov More Alport Syndrome trials