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Experimental kidney drug for alport syndrome falls short in early trial

NCT ID NCT02855268

Disease control Sponsor: Genzyme, a Sanofi Company Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This phase 2 trial tested a weekly injection of lademirsen in 43 adults with Alport syndrome, a genetic condition that damages kidneys. The goal was to see if the drug could slow the decline in kidney function. The study was terminated early, so the results are limited and it's unclear if lademirsen is safe or effective.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
lademirsen (a drug injection)
What this could lead to
If it worked, lademirsen could slow kidney function loss in people with Alport syndrome, delaying the need for dialysis or transplant.
What could go wrong
The trial was terminated early with only 43 participants, so we don't have enough data to know if it's safe or effective. Early-phase results often don't lead to approved treatments.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

43 people

The number who actually took part.

Started

Nov 2019

Finished

Sep 2022

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 55 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion criteria: * Male or female. * Confirmed diagnosis of Alport syndrome 1. Clinical diagnosis (hematuria, family history, hearing loss, ocular change), AND 2. Genetic confirmation of Alport Syndrome in the participant or the family member, OR 3. Kidney biopsy showing glomerular basement membrane abnormalities (e.g., significant thinning, thickening, irregularity or lucencies) consistent with Alport Syndrome. * Age 18-55 years old. * eGFR \> 35 ml/min/1.73m\^2 and \<90 mL/min/1.73m\^2 (based on CKD-EPI) at screening. * Renal Function Criteria (participants must have met at least one of the following CRITERIA A, B or C): * A) Decline in eGFR of \>=4 mL/min/1.73 m\^2/year (eGFR slope \<= -4) based on a linear regression slope analysis of \>=4 eGFR measurements within 3 years prior to the study and with a minimum of 2-year time span (the last, of the screening measurement, and first eGFR measurements should be separated by at least 2 years). eGFR was calculated by using either the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation. * B) proteinuria (UPCR) \>2000 mg/g (UACR\>1000 mg/g). * C) Age and sex adjusted eGFR (based on CKD-Epi; male 18-23 eGFR\<90 mL/min/1.73m\^2 * ACE inhibitor and/or ARB, the dosing regimen should be stable for at least 30 days prior to screening. * Sexually active female participants of childbearing potential and sexually mature male participants must have agreed to practice true abstinence in line with their preferred and usual lifestyle or to use two acceptable effective methods of contraception for the entire duration of the study and for at least 6 weeks after last dose. * Negative drug screen for opiates, cocaine, heroin, phencyclidine, amphetamines (including ecstasy), barbiturates, benzodiazepines, and cannabinoids. At the Investigator's discretion, participants prescribed benzodiazepines, cannabinoids, or opiates with positive results on a drug screen were allowed. * Negative screening results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, and human immunodeficiency virus (HIV) antibody. * Normal biological tests. * Able to understand all study procedures in the informed consent form (ICF) and to comply with all aspects of the protocol. Exclusion criteria: * Causes of chronic kidney disease aside from Alport syndrome (including but not limited to other heritable disorders leading to chronic kidney disease, diabetic nephropathy, hypertensive nephropathy, lupus nephritis, IgA nephropathy). * End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transplantation. * Any clinically significant illness within 30 days before screening or surgical or medical condition (other than Alport syndrome) that could interfere with the participant's study compliance; confound the study results; impact participant safety; or significantly alter the absorption, distribution, metabolism, or excretion of drugs. * Weight \> 110 kg. * Any history of active malignancy within the last 1 year (history of localized basal cell or squamous cell carcinoma and cervical carcinoma in situ that has been excised/appropriately treated or a fully excised malignant lesion with a low probability of recurrence will not be considered exclusionary). * Prior treatment with Bardoxolone within 90 days prior to screening. * History or presence of alcoholism or drug abuse within 2 years before screening or other concurrent social conditions that would potentially interfere with the participant's study compliance, at the discretion of the Investigator. * Participation in a recent investigational study and receipt of an investigational drug or investigational use of a licensed drug within 30 days or 5 half-lives, whichever was longer, prior to screening. * History or presence of hypersensitivity or idiosyncratic, allergic, or other clinically significant reaction to the study drug (including placebo), inactive ingredients, or related compounds (e.g., other oligonucleotide products). * Any other condition or circumstance that, in the opinion of the Investigator, may make the participant unlikely to complete the study or comply with study procedures and requirements, or may pose a risk to the participant's safety and well-being. The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

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Conditions

The condition(s) this trial relates to.

Alport syndrome hereditary nephritis kidney disorder nephritis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Alport syndrome

Contacts and locations

Locations

  • Columbia University Medical Center_Investigational Site Number :8400004

    New York, New York, 10032, United States

  • Investigational Site Number :0360001

    Parkville, Victoria, 3050, Australia

  • Investigational Site Number :0360002

    Nedlands, Western Australia, 6009, Australia

  • Investigational Site Number :0360003

    Herston, Queensland, 4029, Australia

  • Investigational Site Number :1560001

    Beijing, 100034, China

  • Investigational Site Number :1560002

    Beijing, 100730, China

  • Investigational Site Number :1560004

    Guangzhou, 510080, China

  • Investigational Site Number :2500001

    Paris, 75015, France

  • Investigational Site Number :2500002

    Toulouse, 31403, France

  • Investigational Site Number :7240001

    Barcelona, Barcelona [Barcelona], 08025, Spain

  • Investigational Site Number :7240002

    Madrid / Madrid, Madrid, Comunidad de, 28040, Spain

  • Investigational Site Number :7240003

    Granada, 18014, Spain

  • Investigational Site Number :7240004

    Barcelona, Barcelona [Barcelona], 08035, Spain

  • Investigational Site Number :7240005

    Córdoba, Andalusia, 14004, Spain

  • Investigational Site Number :8260001

    London, London, City of, NW3 2QG, United Kingdom

  • Investigational Site Number :8260002

    Nottingham, Nottinghamshire, NG5 1PB, United Kingdom

  • Investigational Site Number :8260003

    Newcastle upon Tyne, NE7 7DN, United Kingdom

  • Investigational Site Number :8400002

    Los Angeles, California, 90024, United States

  • The Cleveland Clinic Foundation_Investigational Site Number :8400001

    Cleveland, Ohio, 44195, United States

  • Uniklinik Köln, Innere Medizin II - Nephrologie, Rheumatologie, Diabetologie und Allgemeine Innere Medizin_Investigational Site Number :2760001

    Cologne, 50937, Germany

  • University of Minnesota Childrens' Hospital_Investigational Site Number :8400003

    Minneapolis, Minnesota, 55454, United States

  • University of Utah_Investigational Site Number :8400005

    Salt Lake City, Utah, 84132, United States

  • Universitätsmedizin Göttingen, Klinik für Nephrologie und Rheumatologie_Investigational Site Number :2760002

    Göttingen, 37075, Germany

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