EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Exaluren.
- Who it may be relevant to
- Registry conditions: Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Randomized, Double-Blind, Placebo-Controlled, Delayed-Start Study to Evaluate the Efficacy and Safety of Exaluren in Alport Syndrome Patients With Nonsense Mutations in COL4A3/4/5 Genes
Overview
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations in COL4A3/4/5 genes. Targeted 24 patients aged 12 and older will be enrolled in the trial. The study will be comprised of the following periods for each participant: * a Screening period of up to 6 weeks (42 days) * a total Treatment Period of exaluren 0.75 mg/kg or placebo administered daily subcutaneously for 32 weeks: Part 1: patients are randomized to either exaluren or placebo for 16 weeks. Part 2: all patients across both randomized arms receive exaluren for 16 additional weeks. * a safety/efficacy Follow-up Period of 4 weeks after the last treatment
Interventions
- Drug Exaluren
Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)
Primary outcome measures
- The change in the degree of podocyte foot process effacement [Time frame: Baseline to Week 16]
- The change in the degree of podocyte foot process effacement [Time frame: Baseline to Week 32]
Secondary outcome measures (2)
- The percentage change in Urine Protein Creatinine Ratio (UPCR) [Time frame: Baseline to Week 16]
- The percentage change in Urine Protein Creatinine Ratio (UPCR) [Time frame: Baseline to Week 32]
Eligibility criteria
Inclusion criteria
- A confirmed diagnosis of X-linked or autosomal recessive Alport Syndrome with a documented nonsense mutation of COL4A5 in a male or nonsense mutation of COL4A3 or COL4A4 (male or female)
- eGFR>45 ml/min/1.73 m2
- Urinary protein based on two spot urine collections \[urine protein/creatinine ratio (UPCR) ≥ 500 mg/g\]
- Stable regimen of ACEi/ARB for at least 12 weeks before Day 1
Exclusion criteria
- History of any organ transplantation
- Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 1.5 times the upper limit of normal (ULN) will be excluded
- History of dialysis
- Acute kidney injury within 4 weeks before screening
- Active dizziness
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Sequential
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 8 centers
- University of California, Los Angeles — Los Angeles
- Stanford University — Stanford
- Denver Nephrologists PC, Colorado Kidney Care PC — Denver
- University of Minnesota — Minneapolis
- Hackensack University Medical Center — Hackensack
- Cleveland Clinic — Cleveland
- University of Pennsylvania — Philadelphia
- Renal Associates, P.A. — San Antonio
United Kingdom · 4 centers
- Alder Hey Children's NHS Foundation Trust — Liverpool
- Royal Free Hospital — London
- Great Ormond Street Hospital — London
- Royal Manchester Children's Hospital — Manchester
Identifiers
NCT: NCT07523581 · EL-017