Trial Outcomes & Findings for A Study of ELX-02 in Patients With Alport Syndrome (NCT NCT05448755)
NCT ID: NCT05448755
Last Updated: 2026-02-24
Results Overview
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.
COMPLETED
PHASE2
3 participants
From the time of first dosing through the end of the follow-up period, a total of 5 months
2026-02-24
Participant Flow
A total of 3 subjects (two 12 years old and one 18 years old) were enrolled in this study.
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.
Participant milestones
| Measure |
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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|---|---|
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Overall Study
STARTED
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3
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Overall Study
COMPLETED
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3
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of ELX-02 in Patients With Alport Syndrome
Baseline characteristics by cohort
| Measure |
ELX-02: All Patients
n=3 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
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|---|---|
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Age, Customized
Between 12 and 18
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2 Participants
n=58 Participants
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Age, Customized
Between 18 and 30
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1 Participants
n=58 Participants
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Sex: Female, Male
Female
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1 Participants
n=58 Participants
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Sex: Female, Male
Male
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2 Participants
n=58 Participants
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Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=58 Participants
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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3 Participants
n=58 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=58 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=58 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=58 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=58 Participants
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Race (NIH/OMB)
Black or African American
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0 Participants
n=58 Participants
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Race (NIH/OMB)
White
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3 Participants
n=58 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=58 Participants
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Race (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=58 Participants
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Region of Enrollment
United Kingdom
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3 participants
n=58 Participants
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PRIMARY outcome
Timeframe: From the time of first dosing through the end of the follow-up period, a total of 5 monthsTreatment 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.
Outcome measures
| Measure |
ELX-02 Patient 1
n=3 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
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ELX-02 Patient 2
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
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ELX-02: Patient 3
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
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|---|---|---|---|
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Number of Participants With Adverse Events Associated With Administration of 0.75 mg/kg of ELX-02 Once Daily
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3 Participants
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—
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—
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SECONDARY outcome
Timeframe: 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.Population: Measurements in 3 enrolled patients. Individual data is repeated due to study sample size.
Change from baseline to End Of Treatment. Measurement of urine protein/ creatinine ratio (UPCR) in urine samples
Outcome measures
| Measure |
ELX-02 Patient 1
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
|
ELX-02 Patient 2
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
|
ELX-02: Patient 3
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
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|---|---|---|---|
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Change From Baseline in Proteinuria
Week 4
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1943.3 mg/g
Standard Deviation 1.13
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706.8 mg/g
Standard Deviation 1.25
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3522.2 mg/g
Standard Deviation 1.14
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Change From Baseline in Proteinuria
End of Treatment
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2465.7 mg/g
Standard Deviation 1.7
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1028.1 mg/g
Standard Deviation 1.65
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2634.7 mg/g
Standard Deviation 1.2
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Change From Baseline in Proteinuria
Baseline
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1299.5 mg/g
Standard Deviation 1.37
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1646.3 mg/g
Standard Deviation 1.24
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1645.3 mg/g
Standard Deviation 1.26
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SECONDARY outcome
Timeframe: Baseline to End of Treatment (at Day 60)Population: Analysis performed with immunofluorescence and light microscopy
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.
Outcome measures
| Measure |
ELX-02 Patient 1
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
|
ELX-02 Patient 2
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
|
ELX-02: Patient 3
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
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|---|---|---|---|
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Change From Baseline to End of Treatment in Collagen IV Expression (Combined Average of Alpha 3 and Alpha 4 Chains)
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38.5 percentage of change from baseline
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8.3 percentage of change from baseline
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170.8 percentage of change from baseline
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SECONDARY outcome
Timeframe: Baseline to End of Treatment (at Day 60)Population: Analysis performed with transmission electron microscopy
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.
Outcome measures
| Measure |
ELX-02 Patient 1
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
|
ELX-02 Patient 2
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
|
ELX-02: Patient 3
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
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|---|---|---|---|
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Change From Baseline to End of Treatment in Collagen IV Expression (Foot Process Width in Renal Biopsy)
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-18.7 percentage of change from baseline
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6.8 percentage of change from baseline
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-45 percentage of change from baseline
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SECONDARY outcome
Timeframe: Baseline to End of Treatment (at Day 60)Population: Analysis performed with transmission electron microscopy
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.
Outcome measures
| Measure |
ELX-02 Patient 1
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
|
ELX-02 Patient 2
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
|
ELX-02: Patient 3
n=1 Participants
Patients received ELX-02 0.75 milligram per kilograms (mg/kg) subcutaneously (SC) daily for 60 days
|
|---|---|---|---|
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Change From Baseline to End of Treatment in Collagen IV Expression (Filtration Slit Density in Renal Biopsy)
|
0.496 Change from baseline in µm-1
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0.207 Change from baseline in µm-1
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0.931 Change from baseline in µm-1
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Adverse Events
Open Label Study Drug Treatment
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Open Label Study Drug Treatment
n=3 participants at risk
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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|---|---|
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Nervous system disorders
Headache
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66.7%
2/3 • Number of events 2 • 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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Nervous system disorders
postural dizziness
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33.3%
1/3 • Number of events 1 • 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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Nervous system disorders
lethargy
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33.3%
1/3 • Number of events 1 • 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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Nervous system disorders
Migraine
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33.3%
1/3 • Number of events 1 • 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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General disorders
Injection site erythema
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66.7%
2/3 • Number of events 2 • 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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General disorders
Injection site pruritus
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33.3%
1/3 • Number of events 1 • 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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General disorders
Chills
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33.3%
1/3 • Number of events 1 • 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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Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
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66.7%
2/3 • Number of events 4 • 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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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place