NCT07523581 EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
| NCT ID | NCT07523581 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Eloxx Pharmaceuticals, Inc. |
| Condition | Alport Syndrome, X-Linked |
| Study Type | INTERVENTIONAL |
| Enrollment | 24 participants |
| Start Date | 2026-06-30 |
| Primary Completion | 2027-06-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 24 participants in total. It began in 2026-06-30 with a primary completion date of 2027-06-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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
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\>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
Frequently Asked Questions
Who can join the NCT07523581 clinical trial?
This trial is open to participants of all sexes, aged 12 Years or older, studying Alport Syndrome, X-Linked. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07523581 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT07523581 currently recruiting?
Yes, NCT07523581 is actively recruiting participants. Visit ClinicalTrials.gov or contact Eloxx Pharmaceuticals, Inc. to inquire about joining.
Where is the NCT07523581 trial being conducted?
This trial is being conducted at Los Angeles, United States, Stanford, United States, Denver, United States, Minneapolis, United States and 7 additional locations.
Who is sponsoring the NCT07523581 clinical trial?
NCT07523581 is sponsored by Eloxx Pharmaceuticals, Inc.. The trial plans to enroll 24 participants.