Exaluren for Alport Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called exaluren for individuals with Alport Syndrome, a genetic condition affecting kidney function. The trial aims to determine if exaluren is safe and effective, particularly for those with specific genetic mutations in the COL4A3, COL4A4, or COL4A5 genes. Participants will receive either exaluren or a placebo (a harmless pill or injection) for 16 weeks, followed by exaluren for everyone for another 16 weeks. Eligible participants must have a confirmed diagnosis of Alport Syndrome with these mutations and be on a stable medication regimen. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.
Do I have to stop taking my current medications for the trial?
The trial requires that you have been on a stable regimen of ACE inhibitors or ARBs (medications for blood pressure and kidney protection) for at least 12 weeks before starting the trial. Other medications are not specified, so it's best to discuss with the trial team.
Is there any evidence suggesting that exaluren is likely to be safe for humans?
Research has shown that exaluren, also known as ELX-02, is under study for its safety in treating Alport Syndrome, a genetic condition affecting the kidneys. Although detailed information from completed trials is limited, exaluren is currently in Phase 2 testing, indicating it has already been tested in humans.
Phase 2 studies aim to gather more information about a treatment's safety and side effects. Earlier tests demonstrated that exaluren was generally well-tolerated by patients with Alport Syndrome, suggesting it may not cause serious side effects for most individuals. However, specific details about side effects in Alport Syndrome are still under review.
Prospective trial participants should discuss potential risks and benefits with their healthcare provider. This conversation can help clarify what has been observed so far and its implications for individual patients.12345Why do researchers think this study treatment might be promising for Alport Syndrome?
Exaluren is unique because it targets the genetic root of Alport Syndrome, a condition where the kidneys, ears, and eyes can be affected due to mutations in certain collagen genes. Most current treatments focus on managing symptoms, like using ACE inhibitors to protect the kidneys, but Exaluren works by addressing the underlying genetic cause. Researchers are excited because Exaluren has the potential to modify the disease itself, rather than just alleviating symptoms, offering hope for a more comprehensive approach to treatment.
What evidence suggests that exaluren might be an effective treatment for Alport Syndrome?
Research has shown that exaluren, also known as ELX-02, may help treat Alport Syndrome caused by specific genetic changes called nonsense mutations. These mutations result in a faulty protein that harms kidney function. In early studies, exaluren enabled cells to bypass these mutations, potentially allowing the production of normal proteins. Other studies have found that exaluren can reach high levels in the body, which is crucial for its effectiveness. Although still under investigation, this suggests that exaluren could help slow kidney damage in people with Alport Syndrome. Participants in this trial will receive either exaluren or a placebo to further evaluate its effectiveness.12356
Are You a Good Fit for This Trial?
This trial is for people aged 12 and older with Alport Syndrome caused by specific genetic mutations (nonsense mutations in COL4A3, COL4A4, or COL4A5), who have stable kidney function and are on certain blood pressure medicines. People with recent serious kidney issues, organ transplants, liver disease, or active dizziness cannot join.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive exaluren or placebo daily for 32 weeks. Part 1: Randomized to exaluren or placebo for 16 weeks. Part 2: All receive exaluren for 16 weeks.
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Exaluren
Trial Overview
The study tests exaluren versus a placebo in patients with Alport Syndrome. For the first 16 weeks, participants get either exaluren or placebo by daily injection; then all receive exaluren for another 16 weeks. The study is randomized and double-blind.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Placebo Group
Find a Clinic Near You
Who Is Running the Clinical Trial?
Eloxx Pharmaceuticals, Inc.
Lead Sponsor
Citations
A Blinded Study in Patients With Alport Syndrome to ...
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with ...
2.
trial.medpath.com
trial.medpath.com/clinical-trial/2e4acb085a607935/nct07523581-exaluren-alport-syndrome-col4a3-4-5-nonsense-mutationsA Blinded Study in Patients With Alport Syndrome to Evaluate ...
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients ...
Clinical Trials – Active Studies
The main purpose of the ASSESS study is to look into whether the investigational drug can reduce the decline in kidney function in patients with Alport syndrome ...
exaluren (ELX-02) News
2) ELX-02 shows nonsense mutation readthrough across a range of nonsense mutations in Alport Syndrome. 3) PBPK modeling shows high levels of ELX-02 exposures ...
A comprehensive review of Alport syndrome - PMC - NIH
Although no specific treatment exists, early diagnosis improves outcomes, emphasizing the importance of genetic testing for prognosis and familial screening.
Pivotal trial of ELX-02 for the treatment of Alport syndrome with ...
This pivotal study will evaluate ELX-02 for the treatment of Alport syndrome with nonsense mutations.
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