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

Conditions

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

Eligibility Criteria

Sex

ALL

Age

12 Years - N/A

Healthy Volunteers

Not accepted

Interventions

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

Study Details

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

Key Dates

First listed

Apr 13, 2026

Start date

Jun 30, 2026

Status verified

Jul 2026

Primary completion

Jun 30, 2027

Completion

Dec 31, 2027

Study Design

Enrollment

24 participants (estimated)

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Exaluren
• Placebo Comparator: Placebo

Primary Outcome Measure

The change in the degree of podocyte foot process effacement [ Time Frame: Baseline to Week 16 ]

Locations (8)

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