Phase 2 N=3 Treatment

A Study of ELX-02 in Patients With Alport Syndrome

Alport Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT05448755 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Feb 2026

Primary outcome: Primary: Number of Participants With Adverse Events Associated With Administration of 0.75 mg/kg of ELX-02 Once Daily — 3 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: ELX-02 (Drug)
Age: Pediatric, Adult · 6+ yrs
Sex: All
Sponsor: Eloxx Pharmaceuticals, Inc.
Primary completion: Sep 2023

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Adverse Events Associated With Administration of 0.75 mg/kg of ELX-02 Once Daily	3	—
SECONDARY Change From Baseline in Proteinuria	1299.5; 1646.3; 1645.3; 1943.3; 706.8; 3522.2	—
SECONDARY 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	—
SECONDARY Change From Baseline to End of Treatment in Collagen IV Expression (Foot Process Width in Renal Biopsy)	-18.7; 6.8; -45	—
SECONDARY Change From Baseline to End of Treatment in Collagen IV Expression (Filtration Slit Density in Renal Biopsy)	0.496; 0.207; 0.931	—

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. 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.

Eligibility Criteria

Inclusion Criteria

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)

Exclusion Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT05448755). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.