A Study of ELX-02 in Patients With Alport Syndrome

NCT05448755 | Completed Phase 2 Results

• 1 other identifier: EL-014
• Study Type: interventional
• Target: 3
• Locations: 2 countries
• Sites: 4

Brief 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.

Conditions

Alport Syndrome

Keywords

Aminoglycoside; Nonsense Mutation; Translational read through

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (4)

• Change From Baseline in Proteinuria

  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)

  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)

  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)

  Baseline to End of Treatment (at Day 60)

Study Arms (1)

Open label study drug treatment

EXPERIMENTAL

Drug: ELX-02

Interventions

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).

Open label study drug treatment

Eligibility Criteria

• Age: 6 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Eloxx Pharmaceuticals, Inc.: lead

Study Sites (4)

Monash Medical Center

Clayton, Victoria, 3168, Australia

Location

Royal Children's Hospital

Parkville, Victoria, 3051, Australia

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 40938675 DERIVED

Related Links

• Eloxx Pharmaceuticals Website

MeSH Terms

Conditions

Nephritis, Hereditary

Interventions

ELX-02

Condition Hierarchy (Ancestors)

Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Nephritis; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Head of Clinical Operations
• Organization: Eloxx Pharmaceuticals

lior.horonchik@eloxxpharma.com

0584003313

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2022
• First Posted: July 7, 2022
• Study Start: November 28, 2022
• Primary Completion: September 11, 2023
• Study Completion: September 11, 2023
• Last Updated: February 24, 2026
• Results First Posted: February 24, 2026

Record last verified: 2026-02

Locations

GB (2); AU (2)