Study of Lademirsen (SAR339375) in Patients With Alport Syndrome

  • STATUS
    Recruiting
  • participants needed
    45
  • sponsor
    Genzyme, a Sanofi Company
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Updated on 19 February 2024
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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 subjects with Alport syndrome

Secondary Objectives:

  • To assess plasma pharmacokinetic (PK) parameters of the parent compound and its metabolites
  • 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.

Description

The planned length of participation in the study for each subject is up to approximately 110 weeks (from screening through completion of follow-up). This includes:

  • 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 subject to enter a 48-week open label extension period and receive active treatment with lademirsen (SAR339375))
  • Post-treatment follow-up period of 10 weeks

Details
Condition Alport's Syndrome
Age 18year - 55year
Treatment Placebo, Lademirsen (SAR339375)
Clinical Study IdentifierNCT02855268
SponsorGenzyme, a Sanofi Company
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

Inclusion Criteria

Male or female
Confirmed diagnosis of Alport syndrome
Clinical diagnosis (hematuria, family history, hearing loss, ocular change), AND
Genetic confirmation of Alport Syndrome in the subject or the family member, OR
Kidney biopsy showing glomerular basement membrane abnormalities (eg, 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 (patients must meet 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 should be 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 subjects of childbearing potential and sexually mature male subjects must agree 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, subjects prescribed benzodiazepines, cannabinoids, or opiates with positive results on a drug screen may be 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 willing 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)
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 subject's study compliance; confound the study results; impact subject 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 subject'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 is 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 (eg, other oligonucleotide products)
Any other condition or circumstance that, in the opinion of the Investigator, may make the subject unlikely to complete the study or comply with study procedures and requirements, or may pose a risk to the subject's safety and well-being
The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial
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