A Phase 2 interventional study of ELX-02 in Alport Syndrome, sponsored by Eloxx Pharmaceuticals, Inc.. Completed at 4 sites in 2 countries. Open to participants aged 6 Years to 30 Years. Per ClinicalTrials.gov, last updated 2026-02-24.
Sponsored by Eloxx Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment
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:
38 studies on the registry are indexed under Nephritis, Hereditary; 14 are open to participants now.
This study's enrollment of 3 is below the median of 50 across 21 interventional studies indexed under Nephritis, Hereditary.
Browse Nephritis, Hereditary studies →Eloxx Pharmaceuticals, Inc. is the lead sponsor of 9 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: ELX-02
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).
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.
Time frame: From the time of first dosing through the end of the follow-up period, a total of 5 months
Change From Baseline in Proteinuria
Change from baseline to End Of Treatment. Measurement of urine protein/ creatinine ratio (UPCR) in urine samples
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)
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.
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)
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.
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)
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.
Time frame: Baseline to End of Treatment (at Day 60)
A total of 3 subjects (two 12 years old and one 18 years old) were enrolled in this study.
| Milestone | Open Label Study Drug Treatment |
|---|---|
| Started | 3 |
| Completed | 3 |
| Not completed | 0 |
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.
| Participants | Study Treatment: ELX-02 0.75 mg/kg |
|---|---|
| Number of Participants With Adverse Events Associated With Administration of 0.75 mg/kg of ELX-02 Once Daily | 3 |
Change from baseline to End Of Treatment. Measurement of urine protein/ creatinine ratio (UPCR) in urine samples
| mg/g | ELX-02 Patient 1 | ELX-02 Patient 2 | ELX-02: Patient 3 |
|---|---|---|---|
| Baseline | 1299.5 ± 1.37 | 1646.3 ± 1.24 | 1645.3 ± 1.26 |
| Week 4 | 1943.3 ± 1.13 | 706.8 ± 1.25 | 3522.2 ± 1.14 |
| End of Treatment | 2465.7 ± 1.7 | 1028.1 ± 1.65 | 2634.7 ± 1.2 |
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.
| percentage of change from baseline | ELX-02 Patient 1 | ELX-02 Patient 2 | ELX-02: Patient 3 |
|---|---|---|---|
| Change From Baseline to End of Treatment in Collagen IV Expression (Combined Average of Alpha 3 and Alpha 4 Chains) | 38.5 | 8.3 | 170.8 |
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.
| percentage of change from baseline | ELX-02 Patient 1 | ELX-02 Patient 2 | ELX-02: Patient 3 |
|---|---|---|---|
| Change From Baseline to End of Treatment in Collagen IV Expression (Foot Process Width in Renal Biopsy) | -18.7 | 6.8 | -45 |
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.
| Change from baseline in µm-1 | ELX-02 Patient 1 | ELX-02 Patient 2 | ELX-02: Patient 3 |
|---|---|---|---|
| Change From Baseline to End of Treatment in Collagen IV Expression (Filtration Slit Density in Renal Biopsy) | 0.496 | 0.207 | 0.931 |
Collected over From baseline to End of Study (at Day 150, last follow-up visit). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Open Label Study Drug Treatment | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Event | Open Label Study Drug Treatment |
|---|---|
| HeadacheNervous system disorders | 2/3 |
| Injection site erythemaGeneral disorders | 2/3 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 2/3 |
| postural dizzinessNervous system disorders | 1/3 |
| lethargyNervous system disorders | 1/3 |
| MigraineNervous system disorders | 1/3 |
| Injection site pruritusGeneral disorders | 1/3 |
| ChillsGeneral disorders | 1/3 |
| Age, Customized(Participants) | ELX-02: All Patients |
|---|---|
| Between 12 and 18 | 2 |
| Between 18 and 30 | 1 |
| Sex: Female, Male(Participants) | ELX-02: All Patients |
|---|---|
| Female | 1 |
| Male | 2 |
| Ethnicity (NIH/OMB)(Participants) | ELX-02: All Patients |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 3 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | ELX-02: All Patients |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 3 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | ELX-02: All Patients |
|---|---|
| United Kingdom | 3 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Nov 2022 to primary completion in Sep 2023, 9 months.
Results were first posted in Feb 2026, 2 years 5 months after primary completion.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: ELX-02
Eloxx Pharmaceuticals, Inc.