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RecruitingNCT07523581Updated Sep 30, 2026

EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety

A Phase 2 interventional study of Exaluren in Alport Syndrome, X-Linked and Alport Syndrome, Autosomal Recessive, sponsored by Eloxx Pharmaceuticals, Inc.. Recruiting at 11 sites in 2 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-09-30.

Sponsored by Eloxx Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Jun 2026; still recruiting 4 months later.
Updated Sep 30, 2026Eligibility revised1 site removedGo to Updates ↓
Phase
Phase 2
Study type
Interventional
Enrollment
24
Allocation
Randomized
Ages
12 Years and older
Sex
All
01

Study summary

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
02

Conditions studied

  • Alport Syndrome, X-Linked
  • Alport Syndrome, Autosomal Recessive

Browse trials for

Keywords

  • Nonsense Mutation
  • Translational read through
03

In context

Nephritis, Hereditary

38 studies on the registry are indexed under Nephritis, Hereditary; 14 are open to participants now.

This study's planned enrollment of 24 is below the median of 50 across 21 interventional studies indexed under Nephritis, Hereditary.

Browse Nephritis, Hereditary studies →

Lead sponsor

Eloxx Pharmaceuticals, Inc. is the lead sponsor of 9 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

The criteria were revised on the registry on Sep 30, 2026. See what changed

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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)
  • >40 ml/min/1.73 m2
  • Urinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 300 mg/g]
  • Stable regimen of ACEi/ARB for at least 12 weeks before Day 1

Exclusion criteria

Exclusion Criteria:

  • History of any organ transplantation
  • Liver disease characterized by cirrhosis or portal hypertension. Participants with (ALT), aspartate aminotransferase (AST), and/or a total 1.5 times the (ULN) will be excluded
  • History of dialysis
  • Acute kidney injury within 4 weeks before screening
  • Active dizziness
Terms in these criteria, in plain language(4)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Sequential assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
24 participants (estimated)

Study arms

  • Experimental
    Exaluren

    Drug: Exaluren

  • Placebo comparator
    Placebo

    Drug: Exaluren

Interventions

  • Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)

06

What researchers measure

Primary outcomes

  1. The change in the degree of podocyte foot process effacement

    Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)

    Time frame: Baseline to Week 16

  2. The change in the degree of podocyte foot process effacement

    Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)

    Time frame: Baseline to Week 32

Secondary outcomes

  1. The percentage change in Urine Protein Creatinine Ratio (UPCR)

    Calculated from geometric mean of 3 consecutive days of first void urine collection

    Time frame: Baseline to Week 16

  2. The percentage change in Urine Protein Creatinine Ratio (UPCR)

    Calculated from geometric mean of 3 consecutive days of first void urine collection

    Time frame: Baseline to Week 32

Other outcomes

  1. The percentage change in estimated Glomerular Filtration Rate (eGFR)

    Change in eGFR level

    Time frame: Baseline to Week 16 and Week 32

  2. The change in the degree of podocyte foot process effacement

    Measured by the change in Foot Process Width (FPW)

    Time frame: Baseline to Week 16 and Week 32

  3. The change in collagen expression

    Measured by structural changes

    Time frame: Baseline to Week 16 and Week 32

  4. Evaluate the safety and tolerability of Exaluren

    The incidence and characteristics of adverse events (AEs)

    Time frame: Screening to End of Study (Week 36)

07

Study locations

3 of 11 sites recruiting
08

Updates

1 registry update since Sep 25, 2026
Sites
1 site removed
Show 1 removed
  • Cleveland Clinic · Cleveland, United States
Sep 30, 2026
Eligibility
2 inclusion criteria edited
What changed in the criteria
Inclusion criteria
  • EditedeGFR>4540 ml/min/1.73 m2
  • EditedUrinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 500300 mg/g]
Sep 30, 2026
Show all 1 update
  1. Sep 30, 2026
    1 site removed
    Show 1 removed
    • Cleveland Clinic · Cleveland, United States
    Eligibility 2 inclusion criteria edited
    What changed in the criteria
    Inclusion criteria
    • EditedeGFR>4540 ml/min/1.73 m2
    • EditedUrinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 500300 mg/g]
    + 1 other change: date precision

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

09

Registry details

Key details

Study ID
NCT07523581
Lead sponsor
Eloxx Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Apr 13, 2026
Start date
Jun 30, 2026
Primary completion
Jun 30, 2027 (estimated)
Completion
Dec 31, 2027 (estimated)
Last update
Sep 30, 2026

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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No contact was published for this record. The registry link below has the sponsor’s details.

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