EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
Eloxx Pharmaceuticals, Inc. · NCT07523581
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 12+ · All sexes
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 2
- Target enrollment
- 24
- US sites recruiting
- 1
- Study start
- June 30, 2026
- Record last updated
- September 30, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study is about
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
Being studied: Exaluren
Conditions: Alport Syndrome, X-Linked; Alport Syndrome, Autosomal Recessive
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full 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>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
- 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
Recruiting US locations
Denver Nephrologists PC, Colorado Kidney Care PC
Denver, CO 80220
Site contact: Laura Kooienga · 303-364-4775 · lkooienga@cokidneycare.com