Alport Syndrome, X-LinkedPhase 2NCT07523581
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
A Phase 2 study testing Exaluren in people with Alport Syndrome, X-Linked and Alport Syndrome, Autosomal Recessive, run by Eloxx Pharmaceuticals, Inc. at 11 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
- What is being tested?
- Exaluren
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 30 Jun 2026to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
11 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
11 sites in 2 countries
United States7 sites, 7 recruiting
- Recruiting
Denver Nephrologists PC, Colorado Kidney Care PC
Denver, Colorado, 80220
Laura Kooienga, 303-364-4775, lkooienga@cokidneycare.com
- Not yet recruiting
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Kenneth Lieberman, 551-996-8228, kenneth.lieberman@hmhn.org
- Not yet recruiting
University of California, Los Angeles
Los Angeles, California, 90095
Anjay Rastogi, 310-794-5023, COREKidneyResearch@mednet.ucla.edu
- Not yet recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
Michelle Rheault, 612-626-2922, rheau002@umn.edu
- Not yet recruiting
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Gaia Coppock, 215-662-2638, gaia.coppock@pennmedicine.upenn.edu
- Not yet recruiting
Renal Associates, P.A.
San Antonio, Texas, 78109
Pablo Pergola, 210-223-4444, ppergola@raparesearch.com
- Not yet recruiting
Stanford University
Stanford, California, 94305
Richard Lafayette, 650-498-6063, czar@stanford.edu
United Kingdom4 sites, 4 recruiting
- Not yet recruiting
Alder Hey Children's NHS Foundation Trust
Liverpool, L12 2AP
Caroline Jones, 0151 252 5570, Caroline.Jones@alderhey.nhs.uk
- Recruiting
Royal Free Hospital
London, NW3 2QG
Daniel Gale, MD, +442074726491, d.gale@ucl.ac.uk
- Recruiting
Great Ormond Street Hospital
London, WC1N 3JH
Zainab Arslan, MD, 0207 405 9200 ext. 7867, Zainab.Arslan@gosh.nhs.uk
- Not yet recruiting
Royal Manchester Children's Hospital
Manchester, M13 9WL
Rachel Lennon, MD, 0161 701 1676, rachel.lennon@mft.nhs.uk
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Delayed-Start Study to Evaluate the Efficacy and Safety of Exaluren in Alport Syndrome Patients With Nonsense Mutations in COL4A3/4/5 Genes
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.
Read more
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 tested
- Exaluren
- Keywords
- Nonsense Mutation, Translational read through
- Medical subject headings
- Nephritis, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExalurenDrug
Exaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG)
| Study group | Role | What they receive |
|---|---|---|
| Exaluren | Experimental | Not described Receives: Drug: Exaluren |
| Placebo | Placebo comparator | Not described Receives: Drug: Exaluren |
What is being measured
The primary outcome is the main question the study is designed to answer.
The change in the degree of podocyte foot process effacement
Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
Measured: Baseline to Week 16
The change in the degree of podocyte foot process effacement
Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
Measured: Baseline to Week 32
Secondary outcomes (2)
The percentage change in Urine Protein Creatinine Ratio (UPCR)
Calculated from geometric mean of 3 consecutive days of first void urine collection
Measured: Baseline to Week 16
The percentage change in Urine Protein Creatinine Ratio (UPCR)
Calculated from geometric mean of 3 consecutive days of first void urine collection
Measured: Baseline to Week 32
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2026
- Study start30 Jun 2026
- Primary completion30 Jun 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alport Syndrome, X-LinkedStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eloxx Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07523581
- Sponsor's protocol ID
- EL-017
- Status last verified
- Jul 2026
- First submitted
- 5 Apr 2026
- Last updated
- 30 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Delayed-Start Study to Evaluate the Efficacy and Safety of Exaluren in Alport Syndrome Patients With Nonsense Mutations in COL4A3/4/5 Genes
- ClinicalTrials.gov ID
- NCT07523581
- Acronym / sponsor ID
- EL-017
- Keywords
- Nonsense Mutation, Translational read through
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Eloxx Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Jun 2026 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 13 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exaluren | Experimental | Drug: Exaluren |
| Placebo | Placebo comparator | Drug: Exaluren |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ExalurenExaluren is a synthetic Eukaryotic Ribosome Selective Glycoside (ERSG) | Drug | Exaluren, Placebo |
Primary outcome
The change in the degree of podocyte foot process effacement
Time frame: Baseline to Week 16
Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
The change in the degree of podocyte foot process effacement
Time frame: Baseline to Week 32
Measured in kidney biopsies by the change in the Filtration Slit Density (FSD)
2 secondary outcomes
The percentage change in Urine Protein Creatinine Ratio (UPCR)
Time frame: Baseline to Week 16
The percentage change in Urine Protein Creatinine Ratio (UPCR)
Time frame: Baseline to Week 32
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
University of California, Los Angeles
Los Angeles, United States
Denver Nephrologists PC, Colorado Kidney Care PC
Denver, United States
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Exaluren in people with Alport Syndrome, X-Linked and Alport Syndrome, Autosomal Recessive, run by Eloxx Pharmaceuticals, Inc. at 11 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change in the degree of podocyte foot process effacement (time frame: Baseline to Week 16). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Sep 2026.