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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:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open label study drug treatment | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ELX-02 | Drug | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | From the time of first dosing through the end of the follow-up period, a total of 5 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Proteinuria | Change from baseline to End Of Treatment. Measurement of urine protein/ creatinine ratio (UPCR) in urine samples | 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) |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Monash Medical Center | Clayton | Victoria | 3168 | Australia | ||
| Royal Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40938675 | Derived | 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. |
| Label | URL |
|---|---|
| Eloxx Pharmaceuticals Website | View source |
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The study consisted of 3 periods for each subject; a screening period of 6 weeks, a treatment period of 8 weeks and a safety follow up period of 12 weeks after the last treatment.
A total of 3 subjects (two 12 years old and one 18 years old) were enrolled in this study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Open Label Study Drug Treatment | 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). |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX-02: All Patients | Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Count of Participants | Participants | From the time of first dosing through the end of the follow-up period, a total of 5 months |
|
From baseline to End of Study (at Day 150, last follow-up visit)
Safety population consisted of all treated patients who received at least one dose of study medication. No deaths or serious adverse events occurred, other adverse events that occurred during the study are reported in the tables below.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Open Label Study Drug Treatment | 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). |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Head of Clinical Operations | Eloxx Pharmaceuticals | 0584003313 | lior.horonchik@eloxxpharma.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 21, 2023 | Dec 9, 2025 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D009394 | Nephritis, Hereditary |
| ID | Term |
|---|---|
| D014564 | Urogenital Abnormalities |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
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| ID | Term |
|---|---|
| C000709849 | ELX-02 |
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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. |
| 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. | 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. | Baseline to End of Treatment (at Day 60) |
| Parkville |
| Victoria |
| 3051 |
| Australia |
| Royal Free Hospital | London | NW3 2QG | United Kingdom |
| Great Ormond Street Hospital | London | WC1N 3JH | United Kingdom |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Study Treatment: ELX-02 0.75 mg/kg |
Patients received 0.75 mg/kg SC daily for 60 days |
|
|
| Secondary | Change From Baseline in Proteinuria | Change from baseline to End Of Treatment. Measurement of urine protein/ creatinine ratio (UPCR) in urine samples | Measurements in 3 enrolled patients. Individual data is repeated due to study sample size. | Posted | Geometric Mean | Standard Deviation | mg/g | 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. |
|
|
|
| Secondary | 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. | Analysis performed with immunofluorescence and light microscopy | Posted | Number | percentage of change from baseline | Baseline to End of Treatment (at Day 60) |
|
|
|
| Secondary | 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. | Analysis performed with transmission electron microscopy | Posted | Number | percentage of change from baseline | Baseline to End of Treatment (at Day 60) |
|
|
|
| Secondary | 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. | Analysis performed with transmission electron microscopy | Posted | Number | Change from baseline in µm-1 | Baseline to End of Treatment (at Day 60) |
|
|
|
| 0 |
| 3 |
| 0 |
| 3 |
| 3 |
| 3 |
| postural dizziness | Nervous system disorders | Systematic Assessment |
|
| lethargy | Nervous system disorders | Systematic Assessment |
|
| Migraine | Nervous system disorders | Systematic Assessment |
|
| Injection site erythema | General disorders | Systematic Assessment |
|
| Injection site pruritus | General disorders | Systematic Assessment |
|
| Chills | General disorders | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
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| D009393 | Nephritis |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052801 | Male Urogenital Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003095 | Collagen Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
|
| End of Treatment |
|