Alport SyndromePhase 2NCT05448755
A Study of ELX-02 in Patients With Alport Syndrome
A Phase 2 study testing ELX-02 in people with Alport Syndrome, run by Eloxx Pharmaceuticals, Inc. at 4 sites in 2 countries.
Trial summary
This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation. In total, up to 8 participants, with a minimum of 3 adults, will be enrolled in the trial. Read more about this study
- Who is it for?
- Ages 6 to 30 years
- Conditions the study is for
- Alport Syndrome
- What is being tested?
- ELX-02
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 28 Nov 2022to 11 Sep 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 6 to 30 years
- All sexes
- No healthy volunteers
- Child, 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)
- The nonsense mutation should be UAG or UGA
- eGFR>60 ml/min/1.73 m2 (based on CKD-EPI for ages ≥18 and Schwartz formula for participants <18)
- Urinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 500 mg/g]
- Stable regimen of ACEi/ARB for at least 4 weeks before screening (unless there is a contraindication)
Who cannot join
- History of any organ transplantation
- Mutation consistent with autosomal dominant Alport Syndrome
- Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 3.0 times the upper limit of normal (ULN) will be excluded
- History of congestive heart failure diagnosed clinically or with documented left ventricular ejection fraction (LVEF) ≤ 40%
- History of dialysis
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Australia2 sites
Monash Medical Center
Clayton, Victoria, 3168
Royal Children's Hospital
Parkville, Victoria, 3051
United Kingdom2 sites
Royal Free Hospital
London, NW3 2QG
Great Ormond Street Hospital
London, WC1N 3JH
About this study
Official title: A Phase 2 Open Label Pilot Study to Evaluate the Safety and Efficacy of Subcutaneously Administered ELX-02 in Patients With Alport Syndrome With Col4A5 and Col4A3/4 Nonsense Mutation
This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation.
In total, up to 8 participants, with a minimum of 3 adults, 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 8 weeks (60 days)
- a safety/efficacy Follow-up Period of 12 weeks (90 days) after the last treatment The Treatment Period will be a treatment of ELX-02 0.75 mg/kg SC QD for 8 weeks.
- Conditions
- Alport Syndrome
- Being tested
- ELX-02
- Keywords
- Aminoglycoside, 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.
ELX-02Drug
ELX-02 is a small molecule, new chemical entity being developed for the treatment of genetic diseases caused by nonsense mutations. ELX-02 is a eukaryotic ribosomal selective glycoside (ERSG).
| Study group | Role | What they receive |
|---|---|---|
| Open label study drug treatment | Experimental | Not described Receives: Drug: ELX-02 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events Associated With Administration of 0.75 mg/kg of ELX-02 Once Daily
Treatment Emerging Adverse Events were defined as any adverse events with onset after the first administration of study medication through the end of the study, or any event that was present at baseline but worsened in intensity or was subsequently considered drug-related by the Investigator through the end of the study. Severe Treatment Emerging Adverse Event is a Treatment Emerging Adverse Events with CTCAE Grade 3 or above. Related Treatment Emerging Adverse Events is defined as a Treatment Emerging Adverse Events with a certain, probable/likely, or possible relationship to the study drug. Serious Adverse Events were Adverse Events resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening.
Measured: From the time of first dosing through the end of the follow-up period, a total of 5 months
Secondary outcomes (4)
Change From Baseline in Proteinuria
Change from baseline to End Of Treatment. Measurement of urine protein/ creatinine ratio (UPCR) in urine samples
Measured: From screening assessment to end of study treatment, total of 3 timepoints for results at Baseline, Week 4 and End of Treatment at Day 60.
Change From Baseline to End of Treatment in Collagen IV Expression (Combined Average of Alpha 3 and Alpha 4 Chains)
Collagen IV expression was assessed by measuring the combined average of alpha 3 and alpha 4 chains, using Immunofluorescence method. Results are reported as percentage change from baseline to End of Treatment. Increase in Collagen IV expression indicate kidney structural improvement.
Measured: Baseline to End of Treatment (at Day 60)
Change From Baseline to End of Treatment in Collagen IV Expression (Foot Process Width in Renal Biopsy)
Collagen IV expression was assessed by measuring podocyte foot process width in renal biopsy specimens, using Transmission Electron Microscopy (TEM) method. Results are reported as percentage change from baseline to End of Treatment.
Decrease values indicate kidney structural and morphological improvement.
Measured: Baseline to End of Treatment (at Day 60)
Change From Baseline to End of Treatment in Collagen IV Expression (Filtration Slit Density in Renal Biopsy)
Collagen IV expression was assessed by measuring filtration slit density in renal biopsy specimens, using 3D-Structured Illumination Microscopy method. Results are reported as change from baseline. Increase values indicate improvement and protein recovery.
Measured: Baseline to End of Treatment (at Day 60)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2022
- Study start28 Nov 2022
- Primary completion11 Sep 2023
- Study completion11 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Alport SyndromeStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eloxx Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05448755
- Sponsor's protocol ID
- EL-014
- Status last verified
- Feb 2026
- First submitted
- 29 Jun 2022
- Last updated
- 24 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Rheault MN. Treatment Approaches for Alport Syndrome. J Am Soc Nephrol. 2026 Jan 1;37(1):172-179. doi: 10.1681/ASN.0000000897. Epub 2025 Sep 12.
PubMed 40938675
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open Label Pilot Study to Evaluate the Safety and Efficacy of Subcutaneously Administered ELX-02 in Patients With Alport Syndrome With Col4A5 and Col4A3/4 Nonsense Mutation
- ClinicalTrials.gov ID
- NCT05448755
- Acronym / sponsor ID
- EL-014
- Conditions
- Alport Syndrome
- Keywords
- Aminoglycoside, Nonsense Mutation, Translational read through
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Eloxx Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Nov 2022 (actual)
- Primary completion
- 11 Sep 2023 (actual)
- Study completion
- 11 Sep 2023 (actual)
- First posted
- 7 Jul 2022 (actual)
- Results
- Posted 24 Feb 2026
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 Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation.
In total, up to 8 participants, with a minimum of 3 adults, 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 8 weeks (60 days)
- a safety/efficacy Follow-up Period of 12 weeks (90 days) after the last treatment The Treatment Period will be a treatment of ELX-02 0.75 mg/kg SC QD for 8 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open label study drug treatment | Experimental | Drug: ELX-02 |
Who may qualify
- Ages
- 6 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 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)
- The nonsense mutation should be UAG or UGA
- eGFR>60 ml/min/1.73 m2 (based on CKD-EPI for ages ≥18 and Schwartz formula for participants <18)
- Urinary protein based on two spot urine collections [urine protein/creatinine ratio (UPCR) ≥ 500 mg/g]
- Stable regimen of ACEi/ARB for at least 4 weeks before screening (unless there is a contraindication)
Key exclusion criteria (5 listed)
- History of any organ transplantation
- Mutation consistent with autosomal dominant Alport Syndrome
- Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 3.0 times the upper limit of normal (ULN) will be excluded
- History of congestive heart failure diagnosed clinically or with documented left ventricular ejection fraction (LVEF) ≤ 40%
- History of dialysis
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 |
|---|---|---|
| ELX-02ELX-02 is a small molecule, new chemical entity being developed for the treatment of genetic diseases caused by nonsense mutations. ELX-02 is a eukaryotic ribosomal selective glycoside (ERSG). | Drug | Open label study drug treatment |
Primary outcome
Number of Participants With Adverse Events Associated With Administration of 0.75 mg/kg of ELX-02 Once Daily
Time frame: From the time of first dosing through the end of the follow-up period, a total of 5 months
Treatment Emerging Adverse Events were defined as any adverse events with onset after the first administration of study medication through the end of the study, or any event that was present at baseline but worsened in intensity or was subsequently considered drug-related by the Investigator through the end of the study. Severe Treatment Emerging Adverse Event is a Treatment Emerging Adverse Events with CTCAE Grade 3 or above. Related Treatment Emerging Adverse Events is defined as a Treatment Emerging Adverse Events with a certain, probable/likely, or possible relationship to the study drug. Serious Adverse Events were Adverse Events resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening.
4 secondary outcomes
Change From Baseline in Proteinuria
Time frame: From screening assessment to end of study treatment, total of 3 timepoints for results at Baseline, Week 4 and End of Treatment at Day 60.
Change From Baseline to End of Treatment in Collagen IV Expression (Combined Average of Alpha 3 and Alpha 4 Chains)
Time frame: Baseline to End of Treatment (at Day 60)
Change From Baseline to End of Treatment in Collagen IV Expression (Foot Process Width in Renal Biopsy)
Time frame: Baseline to End of Treatment (at Day 60)
Change From Baseline to End of Treatment in Collagen IV Expression (Filtration Slit Density in Renal Biopsy)
Time frame: Baseline to End of Treatment (at Day 60)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40938675Rheault MN. Treatment Approaches for Alport Syndrome. J Am Soc Nephrol. 2026 Jan 1;37(1):172-179. doi: 10.1681/ASN.0000000897. Epub 2025 Sep 12.
- Eloxx Pharmaceuticals WebsiteLink provided by the sponsor.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 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 ELX-02 in people with Alport Syndrome, run by Eloxx Pharmaceuticals, Inc. at 4 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 5 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: finished, as of the sponsor's last update on 24 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Australia, 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. 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?
Yes. Results are posted in the registry (first posted 24 Feb 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events Associated With Administration of 0.75 mg/kg of ELX-02 Once Daily (time frame: From the time of first dosing through the end of the follow-up period, a total of 5 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05448755, which also lists 1 linked publication.
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 24 Feb 2026.