EXACT Study
A Phase 2b clinical study of exaluren in people with Alport syndrome caused by nonsense mutations in COL4A3, COL4A4, or COL4A5.
The EXACT Study is evaluating exaluren, an investigational study medication. Exaluren has not been approved for treatment anywhere in the world. The study is designed to learn more about how well exaluren may work and how safe it is.
ClinicalTrials.gov Identifier: NCT07523581 · Sponsor: Eloxx Pharmaceuticals Inc.
What is the EXACT Study?
Alport syndrome is a rare genetic condition that occurs due to defects in specific genes (COL4A3, COL4A4 and COL4A5) that help in producing an important protein called collagen IV. Patients suffer a loss in kidney function, often leading to kidney failure, and can have loss of hearing. Specific defects known as nonsense mutations prevent the body from making the full-length Collagen IV protein. Alport syndrome patients with nonsense mutations (NMAS) can have an even earlier onset of kidney failure.
Nonsense mutations can interrupt the instructions the body uses to make full-length Collagen IV, a protein important for kidney filtering structures. Exaluren is designed to help cells read through certain nonsense mutations so they may produce full-length protein.
The EXACT Study is randomized, double-blind, placebo-controlled, and delayed-start. This means participants are assigned by chance to a study group, placebo is used so the study can be evaluated fairly, and participants who receive placebo in the first part of the study may receive exaluren later in the study.
The full study design is described on ClinicalTrials.gov.
Who may be able to participate?
- Diagnosed with Alport syndrome
- Age 12 years and older
- Confirmed nonsense mutation in COL4A3, COL4A4, or COL4A5
- Meet other study criteria
Only the study team can determine whether someone is eligible to participate.
Other criteria may include kidney function, urine protein levels, current medications, medical history, safety assessments, and other requirements described in the full study record.
The full eligibility criteria are available on ClinicalTrials.gov and will be reviewed by the study team for each person being evaluated for the study.
What Participation May Involve
Taking part in a clinical research study is voluntary. The study team will explain the study visits, procedures, and what to expect before participation begins.
Participation may last up to about 42 weeks and may involve approximately 11 study-center visits. The study includes a screening period, a 32-week treatment period divided into two 16-week parts, and a safety follow-up period.
Screening period
Assessments to determine whether the study may be a suitable option.
Treatment period
Participants receive study medication or placebo in the first part; all participants receive study medication in the second part.
Safety follow-up period
Safety and wellbeing assessments after the last dose of study treatment.
Taking part in a clinical research study is voluntary. A person can choose not to take part or can leave the study at any time without this affecting their medical care. For adolescents, permission from a parent or legal guardian is required, and the adolescent’s own agreement is also important.
Current official study information
Study locations, site status, and contact information may change over time. For the current official study status, locations, eligibility criteria, contact information, and detailed study description, please visit the ClinicalTrials.gov study record.
ClinicalTrials.gov is the source for current official study information. This website is for background information only and does not independently maintain site status.
View current study information on ClinicalTrials.gov →Learning more about the EXACT Study
If you or a family member has Alport syndrome caused by a nonsense mutation, consider discussing the EXACT Study with your nephrologist or a genetic kidney disease specialist.
Taking part in a clinical research study is voluntary. You may want to review the official study record and discuss any questions with your doctor, family, and the study team before making a decision.
Study design and referral
Healthcare professionals can review the full study design, eligibility criteria, study locations, and contact information through the ClinicalTrials.gov record.
Healthcare professionals with investigator or site-related questions may contact Eloxx. Detailed study and eligibility information is available via the study record.
Important information
Exaluren is investigational and has not been approved for treatment anywhere in the world. This page is for informational purposes only and is not medical advice. Eligibility and participation are determined by the study team based on the full study criteria and informed consent process.
Study status, locations, contacts, and eligibility criteria may change. ClinicalTrials.gov should be used as the source for current official study information.
Learn about nonsense mutation Alport syndrome
Understand the genetic basis of the disease, its progression, and why addressing the nonsense mutation is central to Eloxx’s approach.
Alport syndrome program →